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Martin, Paul Langlie

Overview:

For most of my career in Pediatric Hematology/Oncology I have focused on the use of stem cell transplant for the treatment of pediatric leukemias (ALL, AML, CML and JMML) and other non-malignant blood disorders, such as thalassemia, hemaphagocytic disorders, Wiskott-Aldrich, aplastic anemia, Diamond-Blackfan Anemia, as well as inherited metabolic diseases. In addition to focusing on determining the best use of stem cell transplants for these disorders, I have also been involved in clinical research investigating the prevention and treatment of transplant related morbidity, particularly veno-occlusive disease of the liver, infections and diffuse alveolar hemorrhage. As study chair for the Children's Oncology Group protocol 9904, I was involved in the development, implementation and analysis of a large, international frontline study of childhood acute lymphoblastic leukemia. Results from this study show that a significant number of children with certain favorable cytogenetic abnormalities in their leukemic cells and who have a rapid response to their initial chemotherapy can expect to have a >95% chance of cure when treated with relatively low intensity chemotherapy.

Positions:

Professor of Pediatrics

Pediatrics, Blood and Marrow Transplantation
School of Medicine

Member of the Duke Cancer Institute

Duke Cancer Institute
School of Medicine

Chief, Division of Pediatric Blood and Marrow Transplantation

Pediatrics, Blood and Marrow Transplantation
School of Medicine

Education:

M.D. 1987

M.D. — Washington University

Ph.D. 1987

Ph.D. — Washington University

Grants:

Defibrotide Phase III Comparing Defibrotide vs Best Supportive Care

Administered By
Pediatrics, Blood and Marrow Transplantation
AwardedBy
Jazz Pharmaceuticals
Role
Principal Investigator
Start Date
March 22, 2017
End Date
December 31, 2021

CART19 CTL019B2205J

Administered By
Pediatrics, Blood and Marrow Transplantation
AwardedBy
Novartis Pharmaceuticals Corporation
Role
Principal Investigator
Start Date
April 01, 2015
End Date
March 31, 2019

Transfusion Medicine and Hematology

Administered By
Medicine, Hematology
AwardedBy
National Institutes of Health
Role
Participating Faculty Member
Start Date
July 01, 1975
End Date
June 30, 2016

A Phase 2 Multicenter Single arm Study of Moxetumomab Pasudotox in Pediatric Subjects with Relapsed or Refractory pALL

Administered By
Pediatrics, Blood and Marrow Transplantation
AwardedBy
MedImmune, Inc.
Role
Principal Investigator
Start Date
May 01, 2014
End Date
April 30, 2016

Pediatric Oncology Group (POG)

Administered By
Pediatrics, Blood and Marrow Transplantation
AwardedBy
National Institutes of Health
Role
Investigator
Start Date
January 01, 1983
End Date
December 31, 2002
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Publications:

Hepatitis C Treatment in Chronic Kidney Disease Patients: The Kidney Disease Improving Global Outcomes Perspective.

Hepatitis C virus (HCV) infection is a very common infection found among hemodialysis (HD) and kidney transplant patients. It is associated with substantial morbidity and mortality. Direct-acting antiviral agents (DAAs) have much better efficacy (sustained viral response (SVR)) and tolerance than interferon-based regimens. Very recent studies extend this breakthrough finding to chronic kidney disease (CKD) populations.CKD patients with an estimated glomerular filtration rate (eGFR) >30 ml/min/1.73 m2 can be treated with any licensed DAA regimen. In CKD stages 4-5 (mostly HD), the combination of grazoprevir (100 mg) and elbasvir (50 mg), a once-daily oral regimen active against genotypes 1 and 4, induced in a very recent RCT an SVR rate >95%, with tolerance similar to that of placebo. Case series suggest that other DAA regimens are also very effective and well tolerated in HD patients. In kidney transplant recipients, 2 case series have reported 100% SVR with good tolerance of sofosbuvir-based regimens. Importantly, there is a risk of drug-drug interaction of several DAAs including calcineurin inhibitors. Finally, the availability of HCV+ grafts may markedly shorten the waiting time for transplantation. Key Messages: (1) In patients with an eGFR >30, all licensed DAAs regimens can be used. (2) Cure of HCV appears at hand in CKD stages 4-5, including dialysis patients, and in kidney transplant recipients. (3) The choice of DAA regimen in CKD should be based on HCV genotype, viral load, eGFR, concomitant medications, transplant candidacy and comorbidities. (4) The timing of treatment in potential kidney transplantation candidates (before versus after transplantation) should be decided in collaboration with the transplant center. Video Journal Club 'Cappuccino with Claudio Ronco' at http://www.karger.com/?doi=452730.

Authors
Jadoul, M; Martin, P
MLA Citation
Jadoul, M, and Martin, P. "Hepatitis C Treatment in Chronic Kidney Disease Patients: The Kidney Disease Improving Global Outcomes Perspective." Blood purification 43.1-3 (January 24, 2017): 206-209.
PMID
28114144
Source
epmc
Published In
Blood purification
Volume
43
Issue
1-3
Publish Date
2017
Start Page
206
End Page
209
DOI
10.1159/000452730

Bone marrow transplantation for CVID-like humoral immune deficiency associated with red cell aplasia.

Patients with common variable immunodeficiency (CVID) have a higher incidence of autoimmune disease, which may mark the disease onset; however, anemia secondary to pure red cell aplasia is an uncommon presenting feature. Here, we describe a case of CVID-like humoral immune deficiency in a child who initially presented with red cell aplasia and ultimately developed progressive bone marrow failure. Although bone marrow transplantation (BMT) has been associated with high mortality in CVID, our patient was successfully treated with a matched sibling BMT and engrafted with >98% donor chimerism and the development of normal antibody titers to diphtheria and tetanus toxoids.

Authors
Sayour, EJ; Mousallem, T; Van Mater, D; Wang, E; Martin, P; Buckley, RH; Barfield, RC
MLA Citation
Sayour, EJ, Mousallem, T, Van Mater, D, Wang, E, Martin, P, Buckley, RH, and Barfield, RC. "Bone marrow transplantation for CVID-like humoral immune deficiency associated with red cell aplasia." Pediatric blood & cancer 63.10 (October 2016): 1856-1859.
PMID
27273469
Source
epmc
Published In
Pediatric Blood & Cancer
Volume
63
Issue
10
Publish Date
2016
Start Page
1856
End Page
1859
DOI
10.1002/pbc.26092

Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning.

Infants and young children who undergo allogeneic cord blood transplantation (CBT) are at increased risk for late effects because of exposure of developing organs to chemotherapy and radiation therapy typically used in transplant conditioning regimens. Busulfan (Bu)-based myeloablative regimens were developed to eliminate radiation exposure in these young children with the hope that late effects would be minimized. We now describe the late effects in 102 consecutive patients surviving a minimum of 5 years (median follow-up, 12.9 years) post-CBT. Patients were conditioned with high-dose chemotherapy using Bu-containing regimens. No patient received total body irradiation. The median age at transplant was 1 year (range, .1 to 2). Diagnoses included inherited metabolic diseases (59.8%), leukemia (17.6%), congenital immune deficiency (20.2%), bone marrow failure/myelodysplastic syndrome (3.9%), and hemoglobinopathy (2%). Among patients surviving 5 years, the overall survival rate at 10 years post-CBT was 93% (95% CI, 84.9 to 96.8). Virtually all patients (98%) experienced at least 1 significant late effect. Most (83.3%) experienced 2 or more late effects, and more than half of the patients (64.7%) experienced 3 or more late effects. The most commonly observed late effects included dental problems (92.2%), short stature (55.9%), cognitive deficits (53.6%), pulmonary dysfunction (18.6%), and abnormal pubertal development (27.9%). This is the first report of late effects of Bu-based conditioning in a cohort of very young patients at the time of transplant. These results will inform clinical care guidelines for long-term follow-up and add to the growing information regarding outcomes of hematopoietic stem cell transplantation.

Authors
Allewelt, H; El-Khorazaty, J; Mendizabal, A; Taskindoust, M; Martin, PL; Prasad, V; Page, K; Sanders, J; Kurtzberg, J
MLA Citation
Allewelt, H, El-Khorazaty, J, Mendizabal, A, Taskindoust, M, Martin, PL, Prasad, V, Page, K, Sanders, J, and Kurtzberg, J. "Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning." Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 22.9 (September 2016): 1627-1635.
PMID
27264632
Source
epmc
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
9
Publish Date
2016
Start Page
1627
End Page
1635
DOI
10.1016/j.bbmt.2016.05.024

Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning

Authors
Allewelt, H; El-Khorazaty, J; Mendizabal, A; Taskindoust, M; Martin, PL; Prasad, V; Page, K; Sanders, J; Kurtzberg, J
MLA Citation
Allewelt, H, El-Khorazaty, J, Mendizabal, A, Taskindoust, M, Martin, PL, Prasad, V, Page, K, Sanders, J, and Kurtzberg, J. "Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 22.9 (September 2016): 1627-1635.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
9
Publish Date
2016
Start Page
1627
End Page
1635
DOI
10.1016/j.bbmt.2016.05.024

Clinical and Genetic Risk Factors for Acute Pancreatitis in Patients With Acute Lymphoblastic Leukemia.

Acute pancreatitis is one of the common causes of asparaginase intolerance. The mechanism is unknown, and genetic predisposition to asparaginase-induced pancreatitis has not been previously identified.To determine clinical risk factors for asparaginase-induced pancreatitis, we studied a cohort of 5,185 children and young adults with acute lymphoblastic leukemia, including 117 (2.3%) who were diagnosed with at least one episode of acute pancreatitis during therapy. A genome-wide association study was performed in the cohort and in an independent case-control group of 213 patients to identify genetic risk factors.Risk factors associated with pancreatitis included genetically defined Native American ancestry (P < .001), older age (P < .001), and higher cumulative dose of asparaginase (P < .001). No common variants reached genome-wide significance in the genome-wide association study, but a rare nonsense variant rs199695765 in CPA2, encoding carboxypeptidase A2, was highly associated with pancreatitis (hazard ratio, 587; 95% CI, 66.8 to 5166; P = 9.0 × 10(-9)). A gene-level analysis showed an excess of additional CPA2 variants in patients who did versus those who did not develop pancreatitis (P = .001). Sixteen CPA2 single-nucleotide polymorphisms were associated (P < .05) with pancreatitis, and 13 of 24 patients who carried at least one of these variants developed pancreatitis. Biologic functions that were overrepresented by common variants modestly associated with pancreatitis included purine metabolism and cytoskeleton regulation.Older age, higher exposure to asparaginase, and higher Native American ancestry were independent risk factors for pancreatitis in patients with acute lymphoblastic leukemia. Those who inherit a nonsense rare variant in the CPA2 gene had a markedly increased risk of asparaginase-induced pancreatitis.

Authors
Liu, C; Yang, W; Devidas, M; Cheng, C; Pei, D; Smith, C; Carroll, WL; Raetz, EA; Bowman, WP; Larsen, EC; Maloney, KW; Martin, PL; Mattano, LA; Winick, NJ; Mardis, ER; Fulton, RS; Bhojwani, D; Howard, SC; Jeha, S; Pui, C-H; Hunger, SP; Evans, WE; Loh, ML; Relling, MV
MLA Citation
Liu, C, Yang, W, Devidas, M, Cheng, C, Pei, D, Smith, C, Carroll, WL, Raetz, EA, Bowman, WP, Larsen, EC, Maloney, KW, Martin, PL, Mattano, LA, Winick, NJ, Mardis, ER, Fulton, RS, Bhojwani, D, Howard, SC, Jeha, S, Pui, C-H, Hunger, SP, Evans, WE, Loh, ML, and Relling, MV. "Clinical and Genetic Risk Factors for Acute Pancreatitis in Patients With Acute Lymphoblastic Leukemia." Journal of clinical oncology : official journal of the American Society of Clinical Oncology 34.18 (June 2016): 2133-2140.
PMID
27114598
Source
epmc
Published In
Journal of Clinical Oncology
Volume
34
Issue
18
Publish Date
2016
Start Page
2133
End Page
2140
DOI
10.1200/jco.2015.64.5812

Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure.

Hepatic veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of hematopoietic stem cell transplantation (HSCT). Untreated hepatic VOD/SOS with multi-organ failure (MOF) is associated with >80% mortality. Defibrotide has shown promising efficacy treating hepatic VOD/SOS with MOF in phase 2 studies. This phase 3 study investigated safety and efficacy of defibrotide in patients with established hepatic VOD/SOS and advanced MOF. Patients (n = 102) given defibrotide 25 mg/kg per day were compared with 32 historical controls identified out of 6867 medical charts of HSCT patients by blinded independent reviewers. Baseline characteristics between groups were well balanced. The primary endpoint was survival at day +100 post-HSCT; observed rates equaled 38.2% in the defibrotide group and 25% in the controls (23% estimated difference; 95.1% confidence interval [CI], 5.2-40.8;P= .0109, using a propensity-adjusted analysis). Observed day +100 complete response (CR) rates equaled 25.5% for defibrotide and 12.5% for controls (19% difference using similar methodology; 95.1% CI, 3.5-34.6;P= .0160). Defibrotide was generally well tolerated with manageable toxicity. Related adverse events (AEs) included hemorrhage or hypotension; incidence of common hemorrhagic AEs (including pulmonary alveolar [11.8% and 15.6%] and gastrointestinal bleeding [7.8% and 9.4%]) was similar between the defibrotide and control groups, respectively. Defibrotide was associated with significant improvement in day +100 survival and CR rate. The historical-control methodology offers a novel, meaningful approach for phase 3 evaluation of orphan diseases associated with high mortality. This trial was registered at www.clinicaltrials.gov as #.

Authors
Richardson, PG; Riches, ML; Kernan, NA; Brochstein, JA; Mineishi, S; Termuhlen, AM; Arai, S; Grupp, SA; Guinan, EC; Martin, PL; Steinbach, G; Krishnan, A; Nemecek, ER; Giralt, S; Rodriguez, T; Duerst, R; Doyle, J; Antin, JH; Smith, A; Lehmann, L; Champlin, R; Gillio, A; Bajwa, R; D'Agostino, RB; Massaro, J; Warren, D; Miloslavsky, M; Hume, RL; Iacobelli, M; Nejadnik, B; Hannah, AL; Soiffer, RJ
MLA Citation
Richardson, PG, Riches, ML, Kernan, NA, Brochstein, JA, Mineishi, S, Termuhlen, AM, Arai, S, Grupp, SA, Guinan, EC, Martin, PL, Steinbach, G, Krishnan, A, Nemecek, ER, Giralt, S, Rodriguez, T, Duerst, R, Doyle, J, Antin, JH, Smith, A, Lehmann, L, Champlin, R, Gillio, A, Bajwa, R, D'Agostino, RB, Massaro, J, Warren, D, Miloslavsky, M, Hume, RL, Iacobelli, M, Nejadnik, B, Hannah, AL, and Soiffer, RJ. "Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure." Blood 127.13 (March 2016): 1656-1665.
PMID
26825712
Source
epmc
Published In
Blood
Volume
127
Issue
13
Publish Date
2016
Start Page
1656
End Page
1665
DOI
10.1182/blood-2015-10-676924

Unrelated Umbilical Cord Blood Transplantation for Pediatric Hemophagocytic Lymphohistiocytosis

Authors
Patel, S; Allewelt, HB; Martin, PL; Page, K; Driscoll, TA; Prasad, VK; Kurtzberg, J; Parikh, S
MLA Citation
Patel, S, Allewelt, HB, Martin, PL, Page, K, Driscoll, TA, Prasad, VK, Kurtzberg, J, and Parikh, S. "Unrelated Umbilical Cord Blood Transplantation for Pediatric Hemophagocytic Lymphohistiocytosis." March 2016.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
3
Publish Date
2016
Start Page
S257
End Page
S257

Umbilical Cord Blood Transplantation for Cartilage Hair Hypoplasia: A Single Center Experience Demonstrates Excellent Outcomes Using Myeloablative Preparative Regimens

Authors
Allewelt, HB; Patel, S; Prasad, VK; Kurtzberg, J; Driscoll, TA; Martin, PL; Page, K; Parikh, S
MLA Citation
Allewelt, HB, Patel, S, Prasad, VK, Kurtzberg, J, Driscoll, TA, Martin, PL, Page, K, and Parikh, S. "Umbilical Cord Blood Transplantation for Cartilage Hair Hypoplasia: A Single Center Experience Demonstrates Excellent Outcomes Using Myeloablative Preparative Regimens." March 2016.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
3
Publish Date
2016
Start Page
S235
End Page
S235

Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial

Authors
Richardson, PG; Kernan, NA; Brochstein, JA; Mineishi, S; Arai, S; Grupp, SA; Guinan, E; Martin, PL; Steinbach, G; Krishnan, A; Nemecek, ER; Duerst, RE; Antin, JH; Lehmann, L; Gillio, AP; Bajwa, R; Miloslavsky, M; Hume, R; Iacobelli, M; Nejadnik, B; Hannah, AL; Soiffer, RJ
MLA Citation
Richardson, PG, Kernan, NA, Brochstein, JA, Mineishi, S, Arai, S, Grupp, SA, Guinan, E, Martin, PL, Steinbach, G, Krishnan, A, Nemecek, ER, Duerst, RE, Antin, JH, Lehmann, L, Gillio, AP, Bajwa, R, Miloslavsky, M, Hume, R, Iacobelli, M, Nejadnik, B, Hannah, AL, and Soiffer, RJ. "Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial." March 2016.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
3
Publish Date
2016
Start Page
S25
End Page
S25

Pooled Dose Response Analysis of Defibrotide in > 1600 Patients for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome

Authors
Martin, PL; Steinbach, G; Chen, AR; Krishnan, A; Kernan, NA; Avigan, DE; Spitzer, TR; Richardson, PG
MLA Citation
Martin, PL, Steinbach, G, Chen, AR, Krishnan, A, Kernan, NA, Avigan, DE, Spitzer, TR, and Richardson, PG. "Pooled Dose Response Analysis of Defibrotide in > 1600 Patients for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome." March 2016.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
22
Issue
3
Publish Date
2016
Start Page
S337
End Page
S338

Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial

Authors
Richardson, PG; Riches, M; Kernan, NA; Brochstein, JA; Mineishi, S; Termuhlen, AM; Arai, S; Grupp, SA; Guinan, EC; Martin, PL; Steinbach, G; Krishnan, A; Nemecek, ER; Giralt, SA; Rodriguez, TE; Duerst, R; Doyle, JJ; Antin, JH; Smith, AR; Lehmann, LE; Champlin, RE; Gillio, A; Bajwa, R; D'Agostino, RB; Massaro, J; Miloslavsky, M; Hume, RL; Iacobelli, M; Nejadnik, B; Hannah, AL; Soiffer, RJ
MLA Citation
Richardson, PG, Riches, M, Kernan, NA, Brochstein, JA, Mineishi, S, Termuhlen, AM, Arai, S, Grupp, SA, Guinan, EC, Martin, PL, Steinbach, G, Krishnan, A, Nemecek, ER, Giralt, SA, Rodriguez, TE, Duerst, R, Doyle, JJ, Antin, JH, Smith, AR, Lehmann, LE, Champlin, RE, Gillio, A, Bajwa, R, D'Agostino, RB, Massaro, J, Miloslavsky, M, Hume, RL, Iacobelli, M, Nejadnik, B, Hannah, AL, and Soiffer, RJ. "Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial." December 3, 2015.
Source
wos-lite
Published In
Blood
Volume
126
Issue
23
Publish Date
2015

Germline Genetic Variation in ETV6 and Predisposition to Childhood Acute Lymphoblastic Leukemia

Authors
Moriyama, T; Metzger, M; Wu, G; Nishii, R; Qian, M; Devidas, M; Yang, W; Quinn, E; Gastier-Foster, J; Raetz, E; Larsen, EC; Martin, PL; Bowman, WP; Winick, NJ; Komada, Y; Mardis, ER; Fulton, R; Pui, C-H; Evans, WE; Zhang, J; Hunger, SP; Relling, MV; Nichols, KE; Loh, M; Yang, JJ
MLA Citation
Moriyama, T, Metzger, M, Wu, G, Nishii, R, Qian, M, Devidas, M, Yang, W, Quinn, E, Gastier-Foster, J, Raetz, E, Larsen, EC, Martin, PL, Bowman, WP, Winick, NJ, Komada, Y, Mardis, ER, Fulton, R, Pui, C-H, Evans, WE, Zhang, J, Hunger, SP, Relling, MV, Nichols, KE, Loh, M, and Yang, JJ. "Germline Genetic Variation in ETV6 and Predisposition to Childhood Acute Lymphoblastic Leukemia." December 3, 2015.
Source
wos-lite
Published In
Blood
Volume
126
Issue
23
Publish Date
2015

Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience.

X-linked hyper-IgM syndrome (X-HIGM) due to mutations in the gene encoding CD40 ligand results in failure of Ig class switching and an increased propensity for recurrent sinopulmonary and other infections, and thus decreased life expectancy. Allogeneic hematopoietic stem cell transplantation (HSCT) is curative, but long-term follow-up data are limited.We conducted a retrospective analysis of seven patients who have undergone allogeneic HSCT for HIGM syndrome at Duke University Medical Center.Median age at transplant was 5.2 years (range 0.7-19.3). None of the patients had active hepatic or pulmonary disease immediately prior to transplant, but all had a history of serious infections. Five patients received myeloablative conditioning, and two patients received reduced intensity conditioning. Graft sources included bone marrow, peripheral blood, and unrelated umbilical cord blood. Post-transplantation complications included veno-occlusive disease, hemorrhagic cystitis, adenoviremia, and cryptosporidium recurrence in one patient each. Two patients developed acute GVHD grades II-IV that resolved promptly with treatment and none developed extensive chronic GVHD. All patients are intravenous IgG-independent and 6/7 have normal antibody titers. Immunoglobulin (Ig) A levels normalized in all but one patient and T and B cell numbers and function are otherwise normal in all. All patients are alive at a median follow-up of 9.7 (range 9.7-16.1) years post-transplantation with predominantly donor chimerism and no recurrent infections.Allogeneic HSCT results in excellent survival and sustained immune reconstitution in patients with CD40 ligand deficiency using both myeloablative and reduced intensity conditioning approaches and various graft sources, including bone marrow, peripheral blood, and umbilical cord blood.

Authors
Allewelt, H; Martin, PL; Szabolcs, P; Chao, N; Buckley, R; Parikh, S
MLA Citation
Allewelt, H, Martin, PL, Szabolcs, P, Chao, N, Buckley, R, and Parikh, S. "Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience." Pediatric blood & cancer 62.12 (December 2015): 2216-2222.
PMID
26291959
Source
epmc
Published In
Pediatric Blood & Cancer
Volume
62
Issue
12
Publish Date
2015
Start Page
2216
End Page
2222
DOI
10.1002/pbc.25711

Germline genetic variation in ETV6 and risk of childhood acute lymphoblastic leukaemia: a systematic genetic study.

Hereditary predisposition is rarely suspected for childhood acute lymphoblastic leukaemia (ALL). Recent reports of germline ETV6 variations associated with substantial familial clustering of haematological malignancies indicated that this gene is a potentially important genetic determinant for ALL susceptibility. Our aims in this study were to comprehensively identify ALL predisposition variants in ETV6 and to determine the extent to which they contributed to the overall risk of childhood ALL.Whole-exome sequencing of an index family with several cases of ALL was done to identify causal variants for ALL predisposition. Targeted sequencing of ETV6 was done in children from the Children's Oncology Group and St Jude Children's Research Hospital front-line ALL trials. Patients were included in this study on the basis of their enrolment in these clinical trials and the availability of germline DNA. ETV6 variant genotypes were compared with non-ALL controls to define ALL-related germline risk variants. ETV6 variant function was characterised bioinformatically and correlated with clinical and demographic features in children with ALL.We identified a novel non-sense ETV6 variant (p.Arg359X) with a high penetrance in an index family. Subsequent targeted sequencing of ETV6 in 4405 childhood ALL cases identified 31 exonic variants (four non-sense, 21 missense, one splice site, and five frameshift variants) that were potentially related to ALL risk in 35 cases (1%). 15 (48%) of 31 ALL-related ETV6 variants clustered in the erythroblast transformation specific domain and were predicted to be highly deleterious. Children with ALL-related ETV6 variants were significantly older at leukaemia diagnosis than those without (10·2 years [IQR 5·3-13·8] vs 4·7 years [3·0-8·7]; p=0·017). The hyperdiploid leukaemia karyotype was highly over-represented in ALL cases harbouring germline ETV6 risk variants compared with the wild-type group (nine [64%] of 14 cases vs 538 [27%] of 2007 cases; p=0·0050).Our findings indicated germline ETV6 variations as the basis of a novel genetic syndrome associated with predisposition to childhood ALL. The development of recommendations for clinical interventions and surveillance for individuals harbouring ALL-related ETV6 variants are needed.US National Institutes of Health and American Lebanese Syrian Associated Charities.

Authors
Moriyama, T; Metzger, ML; Wu, G; Nishii, R; Qian, M; Devidas, M; Yang, W; Cheng, C; Cao, X; Quinn, E; Raimondi, S; Gastier-Foster, JM; Raetz, E; Larsen, E; Martin, PL; Bowman, WP; Winick, N; Komada, Y; Wang, S; Edmonson, M; Xu, H; Mardis, E; Fulton, R; Pui, C-H; Mullighan, C; Evans, WE; Zhang, J; Hunger, SP; Relling, MV; Nichols, KE; Loh, ML; Yang, JJ
MLA Citation
Moriyama, T, Metzger, ML, Wu, G, Nishii, R, Qian, M, Devidas, M, Yang, W, Cheng, C, Cao, X, Quinn, E, Raimondi, S, Gastier-Foster, JM, Raetz, E, Larsen, E, Martin, PL, Bowman, WP, Winick, N, Komada, Y, Wang, S, Edmonson, M, Xu, H, Mardis, E, Fulton, R, Pui, C-H, Mullighan, C, Evans, WE, Zhang, J, Hunger, SP, Relling, MV, Nichols, KE, Loh, ML, and Yang, JJ. "Germline genetic variation in ETV6 and risk of childhood acute lymphoblastic leukaemia: a systematic genetic study." The Lancet. Oncology 16.16 (December 2015): 1659-1666.
PMID
26522332
Source
epmc
Published In
The Lancet Oncology
Volume
16
Issue
16
Publish Date
2015
Start Page
1659
End Page
1666
DOI
10.1016/s1470-2045(15)00369-1

Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

The use of HSCT is the only potentially curative treatment for CAMT, but access is limited by the availability of suitable donors. We report five consecutive patients with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4). Median times to neutrophil (>500/μL) and platelet (≥20 000 and ≥50 000/μL) engraftment were 19, 57, and 70 days, respectively. Acute GvHD, grade II, developed in one patient, who subsequently developed limited chronic GvHD. At median follow-up of 14 yr, all patients are alive with sustained donor cell engraftment. To our knowledge, this is the largest single-center series of UCBT for patients with this disease and suggests that UCBT is a successful curative option for patients with CAMT.

Authors
Mahadeo, KM; Tewari, P; Parikh, SH; Driscoll, TA; Page, K; Martin, PL; Kurtzberg, J; Prasad, VK
MLA Citation
Mahadeo, KM, Tewari, P, Parikh, SH, Driscoll, TA, Page, K, Martin, PL, Kurtzberg, J, and Prasad, VK. "Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation." Pediatric transplantation 19.7 (November 2015): 753-757.
PMID
26369627
Source
epmc
Published In
Pediatric Transplantation
Volume
19
Issue
7
Publish Date
2015
Start Page
753
End Page
757
DOI
10.1111/petr.12577

Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements.

Mucopolysaccharidosis type I (MPS I; Hurler Syndrome) is a lysosomal storage disease caused by a deficiency of the enzyme α-L-iduronidase which affects multiple organs such as central nervous system (CNS), skeletal system, and physical appearance. Hematopoietic stem cell transplantation (HSCT) is recommended as a primary therapeutic option at an early stage of MPS I with a severe form to ameliorate CNS involvement; however, no description of pathological improvement in skeletal dysplasia has been investigated to date. We here report a 15-year-old male case with MPS I post-HSCT. This patient received successful HSCT at the age of 2 years and 1 month, followed for over 10 years. His activity of daily living including cognitive performance has been kept normal and the present height and weight are 162 cm and 55 kg. Bone deformity has been still developed, resulting in hemiepiphysiodesis of bilateral medial proximal tibia at 12 years of age and successive arthrodesis of thoraco-lumbar spine at 13 years of age; however, skeletal histopathology from surgical remnants showed substantial improvement in bone lesion with markedly reduced occurrence and cell size of vacuolated cells. After a series of surgical procedures, he became ambulant and independent in daily activity. The levels of GAGs in blood were substantially reduced. In conclusion, this long-term post-HSCT observation should shed light on a new aspect of therapeutic effect associated with skeletal pathology and GAG levels as a biomarker, indicating that HSCT is a primary choice at an early stage for not only CNS but skeletal system in combination of appropriate surgical procedures.

Authors
Yasuda, E; Mackenzie, W; Ruhnke, K; Shimada, T; Mason, RW; Zustin, J; Martin, PL; Thacker, M; Orii, T; Sai, Y; Tomatsu, S
MLA Citation
Yasuda, E, Mackenzie, W, Ruhnke, K, Shimada, T, Mason, RW, Zustin, J, Martin, PL, Thacker, M, Orii, T, Sai, Y, and Tomatsu, S. "Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements." Molecular genetics and metabolism reports 2 (March 2015): 65-76.
PMID
25709894
Source
epmc
Published In
Molecular Genetics and Metabolism Reports
Volume
2
Publish Date
2015
Start Page
65
End Page
76
DOI
10.1016/j.ymgmr.2014.12.006

Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma.

The outcome of children, adolescents and young adults (CAYA) with poor-risk recurrent/refractory lymphoma is dismal (⩽30%). To overcome this poor prognosis, we designed an approach to maximize an allogeneic graft vs lymphoma effect in the setting of low disease burden. We conducted a multi-center prospective study of myeloablative conditioning (MAC) and autologous stem cell transplantation (AutoSCT), followed by a reduced intensity conditioning (RIC) and allogeneic hematopoietic cell transplantation (AlloHCT) in CAYA, with poor-risk refractory or recurrent lymphoma. Conditioning for MAC AutoSCT consisted of carmustine/etoposide/cyclophosphamide, RIC consisted of busulfan/fludarabine. Thirty patients, 16 Hodgkin lymphoma (HL) and 14 non-Hodgkin lymphoma (NHL), with a median age of 16 years and median follow-up of 5years, were enrolled. Twenty-three patients completed both MAC AutoSCT and RIC AlloHCT. Allogeneic donor sources included unrelated cord blood (n=9), unrelated donor (n=8) and matched siblings (n=6). The incidence of transplant-related mortality following RIC AlloHCT was only 12%. In patients with HL and NHL, 10 year EFS was 59.8% and 70% (P=0.613), respectively. In summary, this approach is safe, and long-term EFS with this approach is encouraging considering the poor-risk patient characteristics and the use of unrelated donors for RIC AlloHCT in the majority of cases.

Authors
Satwani, P; Jin, Z; Martin, PL; Bhatia, M; Garvin, JH; George, D; Chaudhury, S; Talano, J; Morris, E; Harrison, L; Sosna, J; Peterson, M; Militano, O; Foley, S; Kurtzberg, J; Cairo, MS
MLA Citation
Satwani, P, Jin, Z, Martin, PL, Bhatia, M, Garvin, JH, George, D, Chaudhury, S, Talano, J, Morris, E, Harrison, L, Sosna, J, Peterson, M, Militano, O, Foley, S, Kurtzberg, J, and Cairo, MS. "Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma." February 2015.
PMID
24938649
Source
epmc
Published In
Leukemia
Volume
29
Issue
2
Publish Date
2015
Start Page
448
End Page
455
DOI
10.1038/leu.2014.194

Outcomes of Second Unrelated Donor Cord Blood Transplants (UCBT) Performed in Children with Graft Failure of Autologous Recovery Following the First UCBT

Authors
McFarren, A; Smith, PB; Page, K; Allewelt, HB; Parikh, S; Driscoll, TA; Martin, PL; Kurtzberg, J; Prasad, VK
MLA Citation
McFarren, A, Smith, PB, Page, K, Allewelt, HB, Parikh, S, Driscoll, TA, Martin, PL, Kurtzberg, J, and Prasad, VK. "Outcomes of Second Unrelated Donor Cord Blood Transplants (UCBT) Performed in Children with Graft Failure of Autologous Recovery Following the First UCBT." February 2015.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
21
Issue
2
Publish Date
2015
Start Page
S37
End Page
S38

Hyperthyroidism As a Complication Following Myloablative Therapy and Stem Cell Transplantation for Childhood Diseases

Authors
Ciocci, G; Stafford, L; Driscoll, TA; Martin, PL; Kurtzberg, J
MLA Citation
Ciocci, G, Stafford, L, Driscoll, TA, Martin, PL, and Kurtzberg, J. "Hyperthyroidism As a Complication Following Myloablative Therapy and Stem Cell Transplantation for Childhood Diseases." February 2015.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
21
Issue
2
Publish Date
2015
Start Page
S214
End Page
S215

Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma

Authors
Satwani, P; Jin, Z; Martin, PL; Bhatia, M; Garvin, JH; George, D; Chaudhury, S; Talano, J; Morris, E; Harrison, L; Sosna, J; Peterson, M; Militano, O; Foley, S; Kurtzberg, J; Cairo, MS
MLA Citation
Satwani, P, Jin, Z, Martin, PL, Bhatia, M, Garvin, JH, George, D, Chaudhury, S, Talano, J, Morris, E, Harrison, L, Sosna, J, Peterson, M, Militano, O, Foley, S, Kurtzberg, J, and Cairo, MS. "Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma." LEUKEMIA 29.2 (February 2015): 448-455.
Source
wos-lite
Published In
Leukemia
Volume
29
Issue
2
Publish Date
2015
Start Page
448
End Page
455
DOI
10.1038/leu.2014.194

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival

Authors
Richardson, PG; Kernan, NA; Grupp, SA; Martin, PL; Soiffer, RJ; Martin, R; Hannah, A; Villa, KF
MLA Citation
Richardson, PG, Kernan, NA, Grupp, SA, Martin, PL, Soiffer, RJ, Martin, R, Hannah, A, and Villa, KF. "Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival." February 2015.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
21
Issue
2
Publish Date
2015
Start Page
S110
End Page
S110

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival

Authors
Richardson, PG; Kernan, NA; Grupp, SA; Martin, PL; Soiffer, RJ; Martin, R; Hannah, AL; Villa, KF
MLA Citation
Richardson, PG, Kernan, NA, Grupp, SA, Martin, PL, Soiffer, RJ, Martin, R, Hannah, AL, and Villa, KF. "Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival." December 6, 2014.
Source
wos-lite
Published In
Blood
Volume
124
Issue
21
Publish Date
2014

Standardization of health care provider competencies for intrathecal access procedures.

This quality improvement (QI) project addresses a method for experienced health care providers to maintain skill-based competence for intrathecal access procedures.A prospective QI design using intrathecal access simulation to assess, educate, and evaluate skill competency. Simulation was used as a strategy to promote patient safety and standardize practice patterns. Pretest and posttest methodology using paired t tests were performed to assess anxiety, confidence, and knowledge.Fourteen pediatric providers participated in this QI project. There was a statistically significant improvement in confidence measuring intracranial pressure (ICP; t = -2.92, P = .013), performance-related overall anxiety (t = -2.132, P = .05) and administering intrathecal chemotherapy (t = -2.144, P = .053). Fifty percent of participants missed a medication error demonstrating confirmation bias.This simulation strategy resulted in improved confidence in measuring ICP, performance-related overall anxiety, and confidence in administering chemotherapy. Confirmation bias occurred during simulation testing for a medication error. We propose this method for maintaining clinical competencies in health care providers and introducing new skills to existing practices.

Authors
McLaughlin, CA; Hockenberry, MJ; Kurtzberg, J; Hueckel, R; Martin, PL; Docherty, SL
MLA Citation
McLaughlin, CA, Hockenberry, MJ, Kurtzberg, J, Hueckel, R, Martin, PL, and Docherty, SL. "Standardization of health care provider competencies for intrathecal access procedures." Journal of pediatric oncology nursing : official journal of the Association of Pediatric Oncology Nurses 31.6 (November 2014): 304-316.
PMID
25057001
Source
epmc
Published In
Journal of Pediatric Oncology Nursing (Elsevier)
Volume
31
Issue
6
Publish Date
2014
Start Page
304
End Page
316
DOI
10.1177/1043454214543019

A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases

Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemotherapy-naïve children with nonmalignant diseases (NMD). Twenty-two children with a median age of 2.8years, many with severe comorbidities and prior viral infections, were enrolled in a novel RIC protocol consisting of hydroxyurea, alemtuzumab, fludarabine, melphalan, and thiotepa followed by single UCBT. Patients underwent transplantation for inherited metabolic disorders (n=8), primary immunodeficiencies (n=9), hemoglobinopathies (n=4) and Diamond Blackfan anemia (n=1). Most umbilical cord blood (UCB) units were HLA-mismatched with median infused total nucleated cell dose of 7.9× 107/kg. No serious organ toxicities were attributable to the regimen. The cumulative incidence of neutrophil engraftment was 86.4% (95% confidence interval [CI], 65% to 100%) in a median of 20days, with the majority sustaining>95% donor chimerism at 1year. Cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV by day 180 was 27.3% (95% CI, 8.7% to 45.9%) and 13.6% (95 CI, 0% to 27.6%), respectively. Cumulative incidence of extensive chronic GVHD was 9.1% (95% CI, 0% to 20.8%). The primary causes of death were viral infections (n=3), acute GVHD (n=1) and transfusion reaction (n=1). One-year overall and event-free survivals were 77.3% (95% CI, 53.7% to 89.8%) and 68.2% (95% CI, 44.6% to 83.4%) with 31months median follow-up. This is the first RIC protocol demonstrating durable UCB engraftment in children with NMD. Future risk-based modifications of this regimen could decrease the incidence of viral infections. (www.clinicaltrials.gov/NCT00744692). © 2014 American Society for Blood and Marrow Transplantation.

Authors
Parikh, SH; Mendizabal, A; Benjamin, CL; Komanduri, KV; Antony, J; Petrovic, A; Hale, G; Driscoll, TA; Martin, PL; Page, KM; Flickinger, K; Moffet, J; Niedzwiecki, D; Kurtzberg, J; Szabolcs, P
MLA Citation
Parikh, SH, Mendizabal, A, Benjamin, CL, Komanduri, KV, Antony, J, Petrovic, A, Hale, G, Driscoll, TA, Martin, PL, Page, KM, Flickinger, K, Moffet, J, Niedzwiecki, D, Kurtzberg, J, and Szabolcs, P. "A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases." Biology of Blood and Marrow Transplantation 20.3 (March 1, 2014): 326-336.
Source
scopus
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
3
Publish Date
2014
Start Page
326
End Page
336
DOI
10.1016/j.bbmt.2013.11.021

A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.

Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemotherapy-naïve children with nonmalignant diseases (NMD). Twenty-two children with a median age of 2.8 years, many with severe comorbidities and prior viral infections, were enrolled in a novel RIC protocol consisting of hydroxyurea, alemtuzumab, fludarabine, melphalan, and thiotepa followed by single UCBT. Patients underwent transplantation for inherited metabolic disorders (n = 8), primary immunodeficiencies (n = 9), hemoglobinopathies (n = 4) and Diamond Blackfan anemia (n = 1). Most umbilical cord blood (UCB) units were HLA-mismatched with median infused total nucleated cell dose of 7.9 × 10(7)/kg. No serious organ toxicities were attributable to the regimen. The cumulative incidence of neutrophil engraftment was 86.4% (95% confidence interval [CI], 65% to 100%) in a median of 20 days, with the majority sustaining > 95% donor chimerism at 1 year. Cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV by day 180 was 27.3% (95% CI, 8.7% to 45.9%) and 13.6% (95 CI, 0% to 27.6%), respectively. Cumulative incidence of extensive chronic GVHD was 9.1% (95% CI, 0% to 20.8%). The primary causes of death were viral infections (n = 3), acute GVHD (n = 1) and transfusion reaction (n = 1). One-year overall and event-free survivals were 77.3% (95% CI, 53.7% to 89.8%) and 68.2% (95% CI, 44.6% to 83.4%) with 31 months median follow-up. This is the first RIC protocol demonstrating durable UCB engraftment in children with NMD. Future risk-based modifications of this regimen could decrease the incidence of viral infections. (www.clinicaltrials.gov/NCT00744692).

Authors
Parikh, SH; Mendizabal, A; Benjamin, CL; Komanduri, KV; Antony, J; Petrovic, A; Hale, G; Driscoll, TA; Martin, PL; Page, KM; Flickinger, K; Moffet, J; Niedzwiecki, D; Kurtzberg, J; Szabolcs, P
MLA Citation
Parikh, SH, Mendizabal, A, Benjamin, CL, Komanduri, KV, Antony, J, Petrovic, A, Hale, G, Driscoll, TA, Martin, PL, Page, KM, Flickinger, K, Moffet, J, Niedzwiecki, D, Kurtzberg, J, and Szabolcs, P. "A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases." Biol Blood Marrow Transplant 20.3 (March 2014): 326-336.
PMID
24296492
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
3
Publish Date
2014
Start Page
326
End Page
336
DOI
10.1016/j.bbmt.2013.11.021

Unrelated Umbilical Cord Blood Transplant for Diamond-Blackfan Anemia

Authors
McFarren, A; Page, K; Parikh, S; Martin, PL; Driscoll, TA; Kurtzberg, J; Prasad, VK
MLA Citation
McFarren, A, Page, K, Parikh, S, Martin, PL, Driscoll, TA, Kurtzberg, J, and Prasad, VK. "Unrelated Umbilical Cord Blood Transplant for Diamond-Blackfan Anemia." February 2014.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
2
Publish Date
2014
Start Page
S177
End Page
S177

Interplay of Recipient-Donor Matching for HLA, Race/Ethnicity and Gender on Long Term Outcomes in 365 Pediatric Recipients of Single 4/6 Matched Unrelated Cord Blood Transplantation (UCBT) after Myeloablative Therapy

Authors
Prasad, VK; Mendizabal, A; Page, K; Parikh, S; Wishnew, J; Driscoll, TA; Martin, PL; Kurtzberg, J
MLA Citation
Prasad, VK, Mendizabal, A, Page, K, Parikh, S, Wishnew, J, Driscoll, TA, Martin, PL, and Kurtzberg, J. "Interplay of Recipient-Donor Matching for HLA, Race/Ethnicity and Gender on Long Term Outcomes in 365 Pediatric Recipients of Single 4/6 Matched Unrelated Cord Blood Transplantation (UCBT) after Myeloablative Therapy." February 2014.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
2
Publish Date
2014
Start Page
S87
End Page
S88

Late Effects in Infants and Young Children Following Umbilical Cord Blood Transplant Using Busulfan-Based, Myeloablative Non-TBI Conditioning Regimens

Authors
Allewelt, HB; Martin, PL; Prasad, VK; Page, K; Kurtzberg, J
MLA Citation
Allewelt, HB, Martin, PL, Prasad, VK, Page, K, and Kurtzberg, J. "Late Effects in Infants and Young Children Following Umbilical Cord Blood Transplant Using Busulfan-Based, Myeloablative Non-TBI Conditioning Regimens." February 2014.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
2
Publish Date
2014
Start Page
S63
End Page
S63

A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases

Authors
Parikh, SH; Mendizabal, A; Benjamin, CL; Komanduri, KV; Antony, J; Petrovic, A; Hale, G; Driscoll, TA; Martin, PL; Page, KM; Flickinger, K; Moffet, J; Niedzwiecki, D; Kurtzberg, J; Szabolcs, P
MLA Citation
Parikh, SH, Mendizabal, A, Benjamin, CL, Komanduri, KV, Antony, J, Petrovic, A, Hale, G, Driscoll, TA, Martin, PL, Page, KM, Flickinger, K, Moffet, J, Niedzwiecki, D, Kurtzberg, J, and Szabolcs, P. "A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases." Biology of Blood and Marrow Transplantation 20.3 (2014): 326-336.
Source
scopus
Published In
Biology of Blood and Marrow Transplantation
Volume
20
Issue
3
Publish Date
2014
Start Page
326
End Page
336

Novel susceptibility variants at 10p12.31-12.2 for childhood acute lymphoblastic leukemia in ethnically diverse populations.

BACKGROUND: Acute lymphoblastic leukemia (ALL) is the most common cancer in children and the incidence of ALL varies by ethnicity. Although accumulating evidence indicates inherited predisposition to ALL, the genetic basis of ALL susceptibility in diverse ancestry has not been comprehensively examined. METHODS: We performed a multiethnic genome-wide association study in 1605 children with ALL and 6661 control subjects after adjusting for population structure, with validation in three replication series of 845 case subjects and 4316 control subjects. Association was tested by two-sided logistic regression. RESULTS: A novel ALL susceptibility locus at 10p12.31-12.2 (BMI1-PIP4K2A, rs7088318, P = 1.1 × 10(-11)) was identified in the genome-wide association study, with independent replication in European Americans, African Americans, and Hispanic Americans (P = .001, .009, and .04, respectively). Association was also validated at four known ALL susceptibility loci: ARID5B, IKZF1, CEBPE, and CDKN2A/2B. Associations at ARID5B, IKZF1, and BMI1-PIP4K2A variants were consistent across ethnicity, with multiple independent signals at IKZF1 and BMI1-PIP4K2A loci. The frequency of ARID5B and BMI1-PIP4K2A variants differed by ethnicity, in parallel with ethnic differences in ALL incidence. Suggestive evidence for modifying effects of age on genetic predisposition to ALL was also observed. ARID5B, IKZF1, CEBPE, and BMI1-PIP4K2A variants cumulatively conferred strong predisposition to ALL, with children carrying six to eight copies of risk alleles at a ninefold (95% confidence interval = 6.9 to 11.8) higher ALL risk relative to those carrying zero to one risk allele at these four single nucleotide polymorphisms. CONCLUSIONS: These findings indicate strong associations between inherited genetic variation and ALL susceptibility in children and shed new light on ALL molecular etiology in diverse ancestry.

Authors
Xu, H; Yang, W; Perez-Andreu, V; Devidas, M; Fan, Y; Cheng, C; Pei, D; Scheet, P; Burchard, EG; Eng, C; Huntsman, S; Torgerson, DG; Dean, M; Winick, NJ; Martin, PL; Camitta, BM; Bowman, WP; Willman, CL; Carroll, WL; Mullighan, CG; Bhojwani, D; Hunger, SP; Pui, C-H; Evans, WE; Relling, MV; Loh, ML; Yang, JJ
MLA Citation
Xu, H, Yang, W, Perez-Andreu, V, Devidas, M, Fan, Y, Cheng, C, Pei, D, Scheet, P, Burchard, EG, Eng, C, Huntsman, S, Torgerson, DG, Dean, M, Winick, NJ, Martin, PL, Camitta, BM, Bowman, WP, Willman, CL, Carroll, WL, Mullighan, CG, Bhojwani, D, Hunger, SP, Pui, C-H, Evans, WE, Relling, MV, Loh, ML, and Yang, JJ. "Novel susceptibility variants at 10p12.31-12.2 for childhood acute lymphoblastic leukemia in ethnically diverse populations." J Natl Cancer Inst 105.10 (May 15, 2013): 733-742.
PMID
23512250
Source
pubmed
Published In
Journal of the National Cancer Institute
Volume
105
Issue
10
Publish Date
2013
Start Page
733
End Page
742
DOI
10.1093/jnci/djt042

Genome-wide study of methotrexate clearance replicates SLCO1B1

Authors
Ramsey, LB; Panetta, JC; Smith, C; Yang, W; Fan, Y; Winick, NJ; Martin, PL; Cheng, C; Devidas, M; Pui, C-H; Evans, WE; Hunger, SP; Loh, M; Relling, MV
MLA Citation
Ramsey, LB, Panetta, JC, Smith, C, Yang, W, Fan, Y, Winick, NJ, Martin, PL, Cheng, C, Devidas, M, Pui, C-H, Evans, WE, Hunger, SP, Loh, M, and Relling, MV. "Genome-wide study of methotrexate clearance replicates SLCO1B1." BLOOD 121.6 (February 7, 2013): 898-904.
PMID
23233662
Source
wos-lite
Published In
Blood
Volume
121
Issue
6
Publish Date
2013
Start Page
898
End Page
904
DOI
10.1182/blood-2012-08-452839

Genome-Wide Association Study Identifies a Novel Susceptibility Locus At 10p12.31-12.2 for Childhood Acute Lymphoblastic Leukemia in Ethinically Diverse Populations

Authors
Yang, JJ; Xu, H; Yang, W; Perez-Andreu, V; Dmidas, M; Fan, Y; Cheng, C; Pei, D; Scheet, P; Burchard, EG; Eng, C; Huntsman, S; Torgerson, DG; Dean, M; Winick, N; Martin, PL; Camitta, B; Bowman, WP; Willman, CL; Carroll, WL; Mullighan, CG; Bhojwani, D; Hunger, SP; Pui, C-H; Evans, WE; Belling, MV; Loh, ML
MLA Citation
Yang, JJ, Xu, H, Yang, W, Perez-Andreu, V, Dmidas, M, Fan, Y, Cheng, C, Pei, D, Scheet, P, Burchard, EG, Eng, C, Huntsman, S, Torgerson, DG, Dean, M, Winick, N, Martin, PL, Camitta, B, Bowman, WP, Willman, CL, Carroll, WL, Mullighan, CG, Bhojwani, D, Hunger, SP, Pui, C-H, Evans, WE, Belling, MV, and Loh, ML. "Genome-Wide Association Study Identifies a Novel Susceptibility Locus At 10p12.31-12.2 for Childhood Acute Lymphoblastic Leukemia in Ethinically Diverse Populations." November 16, 2012.
Source
wos-lite
Published In
Blood
Volume
120
Issue
21
Publish Date
2012

Results of a Large Prospective Study On the Use of Defibrotide (DF) in the Treatment of Hepatic Veno-Occlusive Disease (VOD) in Hematopoietic Stem Cell Transplant (HSCT). Early Intervention Improves Outcome - Updated Results of a Treatment IND (T-IND) Expanded Access Protocol

Authors
Richardson, PG; Smith, A; Kernan, NA; Grupp, SA; Arai, S; Triplett, BM; Haut, PR; Chan, K; Symons, HJ; Horn, BN; III, GAP; Boyer, M; Krishnan, A; Lehmann, LE; Martin, PL; Mineishi, S; Shore, TB; Simms-Waldrip, T; Antin, JH; Hannah, AL; Hume, R; Tudone, E; Heringa, C; Soiffer, RJ; Grp, DS
MLA Citation
Richardson, PG, Smith, A, Kernan, NA, Grupp, SA, Arai, S, Triplett, BM, Haut, PR, Chan, K, Symons, HJ, Horn, BN, III, GAP, Boyer, M, Krishnan, A, Lehmann, LE, Martin, PL, Mineishi, S, Shore, TB, Simms-Waldrip, T, Antin, JH, Hannah, AL, Hume, R, Tudone, E, Heringa, C, Soiffer, RJ, and Grp, DS. "Results of a Large Prospective Study On the Use of Defibrotide (DF) in the Treatment of Hepatic Veno-Occlusive Disease (VOD) in Hematopoietic Stem Cell Transplant (HSCT). Early Intervention Improves Outcome - Updated Results of a Treatment IND (T-IND) Expanded Access Protocol." November 16, 2012.
Source
wos-lite
Published In
Blood
Volume
120
Issue
21
Publish Date
2012

Genome-Wide Association Study Identifies Germline Polymorphisms Associated with Relapse of Childhood Acute Lymphoblastic Leukemia

Authors
Yang, JJ; Cheng, C; Meenakshi, D; Cao, X; Campana, D; Yang, W; Fan, Y; Neale, GAM; Cox, N; Scheet, P; Borowitz, MJ; Winick, N; Martin, PL; Bowman, WP; Camitta, B; Reaman, G; Carroll, WL; Willman, CL; Hunger, SP; Evans, WE; Pui, C-H; Loh, ML; Relling, MV
MLA Citation
Yang, JJ, Cheng, C, Meenakshi, D, Cao, X, Campana, D, Yang, W, Fan, Y, Neale, GAM, Cox, N, Scheet, P, Borowitz, MJ, Winick, N, Martin, PL, Bowman, WP, Camitta, B, Reaman, G, Carroll, WL, Willman, CL, Hunger, SP, Evans, WE, Pui, C-H, Loh, ML, and Relling, MV. "Genome-Wide Association Study Identifies Germline Polymorphisms Associated with Relapse of Childhood Acute Lymphoblastic Leukemia." November 16, 2012.
Source
wos-lite
Published In
Blood
Volume
120
Issue
21
Publish Date
2012

A Genome-Wide Analysis of Variants Influencing Methotrexate Clearance Replicates SLCO1B1

Authors
Ramsey, LB; Panetta, JC; Smith, C; Yang, W; Fan, Y; Winick, N; Martin, PL; Cheng, C; Devidas, M; Pui, C-H; Evans, WE; Hunger, SP; Loh, ML; Relling, MV
MLA Citation
Ramsey, LB, Panetta, JC, Smith, C, Yang, W, Fan, Y, Winick, N, Martin, PL, Cheng, C, Devidas, M, Pui, C-H, Evans, WE, Hunger, SP, Loh, ML, and Relling, MV. "A Genome-Wide Analysis of Variants Influencing Methotrexate Clearance Replicates SLCO1B1." November 16, 2012.
Source
wos-lite
Published In
Blood
Volume
120
Issue
21
Publish Date
2012

Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high long-term donor chimerism and excellent survival in chronic granulomatous disease.

The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive children (median age, 59.5 months; range, 8-140 months) with severe chronic granulomatous disease (serious bacterial/fungal infections pretransplantation; median, 3; range, 2-9) received myeloablative hematopoietic stem cell transplantation using sibling bone marrow ([SibBM]; n = 5), unrelated cord blood (UCB; n = 6), and sibling cord blood (n = 1) at our center between 1997 and 2010. SibBM and sibling cord blood were HLA matched at 6/6, whereas UCB were 5/6 (n = 5) or 6/6 (n = 1). Recipients of SibBM were conditioned with busulfan and cyclophosphamide ± anti-thymocyte globulin (ATG), whereas 6 of 7 cord blood recipients received fludarabine/busulfan/cyclophosphamide/ATG. Seven patients received granulocyte-colony stimulating factor-mobilized granulocyte transfusions from directed donors. The first 2 UCB recipients had primary graft failure but successfully underwent retransplantation with UCB. Highest acute graft-versus-host disease was grade III (n = 1). Extensive chronic graft-vs-host disease developed in 3 patients. All patients are alive with median follow-up of 70.5 months (range, 12-167 months) with high donor chimerism (>98%, n = 10; 94%, n = 1; and 92%, n = 1). Myeloablative hematopoietic stem cell transplantation led to correction of neutrophil dysfunction, durable donor chimerism, excellent survival, good quality of life, and low incidence of graft-vs-host disease regardless of graft source.

Authors
Tewari, P; Martin, PL; Mendizabal, A; Parikh, SH; Page, KM; Driscoll, TA; Malech, HL; Kurtzberg, J; Prasad, VK
MLA Citation
Tewari, P, Martin, PL, Mendizabal, A, Parikh, SH, Page, KM, Driscoll, TA, Malech, HL, Kurtzberg, J, and Prasad, VK. "Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high long-term donor chimerism and excellent survival in chronic granulomatous disease." Biol Blood Marrow Transplant 18.9 (September 2012): 1368-1377.
PMID
22326631
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
18
Issue
9
Publish Date
2012
Start Page
1368
End Page
1377
DOI
10.1016/j.bbmt.2012.02.002

Therapeutic monitoring of voriconazole in children less than three years of age: a case report and summary of voriconazole concentrations for ten children.

Voriconazole is the treatment of choice for invasive aspergillosis and its use is increasing in pediatrics. Minimal pharmacokinetic data exist in young children. We report voriconazole concentrations for 10 children <3 years of age and pharmacokinetic parameters for 1 infant who had therapeutic drug monitoring performed. Trough concentrations were unpredictable based on dose, highlighting the need to follow values during therapy.

Authors
Doby, EH; Benjamin, DK; Blaschke, AJ; Ward, RM; Pavia, AT; Martin, PL; Driscoll, TA; Cohen-Wolkowiez, M; Moran, C
MLA Citation
Doby, EH, Benjamin, DK, Blaschke, AJ, Ward, RM, Pavia, AT, Martin, PL, Driscoll, TA, Cohen-Wolkowiez, M, and Moran, C. "Therapeutic monitoring of voriconazole in children less than three years of age: a case report and summary of voriconazole concentrations for ten children." Pediatr Infect Dis J 31.6 (June 2012): 632-635.
PMID
22301479
Source
pubmed
Published In
Pediatric Infectious Disease Journal
Volume
31
Issue
6
Publish Date
2012
Start Page
632
End Page
635
DOI
10.1097/INF.0b013e31824acc33

Implementation of condition help: family teaching and evaluation of family understanding.

Partnering with families to deliver safe care includes teaching how to activate the rapid response team (RRT) if their hospitalized child's condition worsens. Condition Help (Condition H) is how families call the RRT. Pediatric nurses used scripted Condition H teaching and follow-up surveys to evaluate family understanding about Condition H. Although there were only 2 Condition H calls during the study period, 53% to 90% of families received Condition H teaching, and family understanding was greater than 75%.

Authors
Hueckel, RM; Mericle, JM; Frush, K; Martin, PL; Champagne, MT
MLA Citation
Hueckel, RM, Mericle, JM, Frush, K, Martin, PL, and Champagne, MT. "Implementation of condition help: family teaching and evaluation of family understanding." J Nurs Care Qual 27.2 (April 2012): 176-181.
PMID
21989457
Source
pubmed
Published In
Journal of Nursing Care Quality
Volume
27
Issue
2
Publish Date
2012
Start Page
176
End Page
181
DOI
10.1097/NCQ.0b013e318235bdec

Genome-wide association study identifies germline polymorphisms associated with relapse of childhood acute lymphoblastic leukemia

With the use of risk-directed therapy for childhood acute lymphoblastic leukemia (ALL), outcome has improved dramatically in the past 40 years. However, a substantial portion of patients, many of whom have no known risk factors, experience relapse. Taking a genome-wide approach, in the present study, we evaluated the relationships between genotypes at 444 044 single nucleotide polymorphisms (SNPs) with the risk of relapse in 2535 children with newly diagnosed ALL after adjusting for genetic ancestry and treatment regimen. We identified 134 SNPs that were reproducibly associated with ALL relapse. Of 134 relapse SNPs, 133 remained prognostic after adjusting for all known relapse risk factors, including minimal residual disease, and 111 were significant even among patients who were negative for minimal residual disease after remission induction therapy. The C allele at rs7142143 in the PYGL gene was associated with 3.6-fold higher risk of relapse than the T allele (P = 6.7 × 10 -9). Fourteen of the 134 relapse SNPs, including variants in PDE4B and ABCB1, were also associated with antileukemic drug pharmacokinetics and/or pharmacodynamics. In the present study, we systematically identified host genetic variations related to treatment outcome of childhood ALL, most of which were prognostic independent of known risk factors for relapse, and some of which also influenced outcome by affecting host disposition of antileukemic drugs. © 2012 by The American Society of Hematology.

Authors
Yang, JJ; Cheng, C; Devidas, M; Cao, X; Campana, D; Yang, W; Fan, Y; Neale, G; Cox, N; Scheet, P; Borowitz, MJ; Winick, NJ; Martin, PL; Bowman, WP; Camitta, B; Reaman, GH; Carroll, WL; Willman, CL; Hunger, SP; Evans, WE; Pui, C-H; Loh, M; Relling, MV
MLA Citation
Yang, JJ, Cheng, C, Devidas, M, Cao, X, Campana, D, Yang, W, Fan, Y, Neale, G, Cox, N, Scheet, P, Borowitz, MJ, Winick, NJ, Martin, PL, Bowman, WP, Camitta, B, Reaman, GH, Carroll, WL, Willman, CL, Hunger, SP, Evans, WE, Pui, C-H, Loh, M, and Relling, MV. "Genome-wide association study identifies germline polymorphisms associated with relapse of childhood acute lymphoblastic leukemia." Blood 120.20 (2012): 4197-4204.
PMID
23007406
Source
scival
Published In
Blood
Volume
120
Issue
20
Publish Date
2012
Start Page
4197
End Page
4204
DOI
10.1182/blood-2012-07-440107

Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation

Hepatic veno-occlusive disease (VOD) is a serious complication of stem cell transplantation in children. VOD is characterized by rapid weight gain, hepatomegaly, hyperbilirubinemia and ascites. The pathogenesis of VOD is thought to involve chemotherapy and radiation-induced damage to the sinusoidal endothelium, resulting in endothelial injury, microthrombosis, subendothelial damage and cytokine activation. These processes lead to concomitant progressive hepatocellular dysfunction and subsequent fluid retention and renal impairment. Severe VOD is typically associated with multiorgan failure and high mortality. A number of possible strategies for the prevention and/or treatment of VOD in children have been investigated. The most promising agent to date is defibrotide, a novel polydeoxyribonucleotide with fibrinolytic properties but no major bleeding risk. Numerous studies, including Phase II/III trials, have shown clinical benefit in pediatric patients with the use of defibrotide treatment and prophylaxis. This review discusses VOD in children and focuses on therapeutic options, including defibrotide, in this patient population. © 2012 Expert Reviews Ltd.

Authors
Corbacioglu, S; Kernan, N; Lehmann, L; Brochstein, J; Revta, C; Grupp, S; Martin, P; Richardson, PG
MLA Citation
Corbacioglu, S, Kernan, N, Lehmann, L, Brochstein, J, Revta, C, Grupp, S, Martin, P, and Richardson, PG. "Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation." Expert Review of Hematology 5.3 (2012): 291-302.
PMID
22780209
Source
scival
Published In
Expert Review of Hematology
Volume
5
Issue
3
Publish Date
2012
Start Page
291
End Page
302
DOI
10.1586/ehm.12.18

ARID5B genetic polymorphisms contribute to racial disparities in the incidence and treatment outcome of childhood acute lymphoblastic leukemia

Purpose: Recent genome-wide screens have identified genetic variations in ARID5B associated with susceptibility to childhood acute lymphoblastic leukemia (ALL). We sought to determine the contribution of ARID5B single nucleotide polymorphisms (SNPs) to racial disparities in ALL susceptibility and treatment outcome. Patients and Methods: We compared the association between ARID5B SNP genotype and ALL susceptibility in whites (> 95% European genetic ancestry; 978 cases and 1,046 controls) versus in Hispanics (> 10% Native American ancestry; 330 cases and 541 controls). We determined the relationships between ARID5B SNP genotype and ALL relapse risk in 1,605 children treated on the Children's Oncology Group (COG) P9904/9905 clinical trials. Results: Among 49 ARID5B SNPs interrogated, 10 were significantly associated with ALL susceptibility in both whites and Hispanics (P < .05), with risk alleles consistently more frequent in Hispanics than in whites. rs10821936 exhibited the most significant association in both races (P = 8.4 × 10 -20in whites; P = 1 × 10 -6 in Hispanics), and genotype at this SNP was highly correlated with local Native American genetic ancestry (P = 1.8 × 10 -8). Multivariate analyses in Hispanics identified an additional SNP associated with ALL susceptibility independent of rs10821936. Eight ARID5B SNPs were associated with both ALL susceptibility and relapse hazard; the alleles related to higher ALL incidence were always linked to poorer treatment outcome and were more frequent in Hispanics. Conclusion: ARID5B polymorphisms are important determinants of childhood ALL susceptibility and treatment outcome, and they contribute to racial disparities in this disease. © 2012 by American Society of Clinical Oncology.

Authors
Xu, H; Cheng, C; Devidas, M; Pei, D; Fan, Y; Yang, W; Neale, G; Scheet, P; Burchard, EG; Torgerson, DG; Eng, C; Dean, M; Antillon, F; Winick, NJ; Martin, PL; Willman, CL; Camitta, BM; Reaman, GH; Carroll, WL; Loh, M; Evans, WE; Pui, C-H; Hunger, SP; Relling, MV; Yang, JJ
MLA Citation
Xu, H, Cheng, C, Devidas, M, Pei, D, Fan, Y, Yang, W, Neale, G, Scheet, P, Burchard, EG, Torgerson, DG, Eng, C, Dean, M, Antillon, F, Winick, NJ, Martin, PL, Willman, CL, Camitta, BM, Reaman, GH, Carroll, WL, Loh, M, Evans, WE, Pui, C-H, Hunger, SP, Relling, MV, and Yang, JJ. "ARID5B genetic polymorphisms contribute to racial disparities in the incidence and treatment outcome of childhood acute lymphoblastic leukemia." Journal of Clinical Oncology 30.7 (2012): 751-757.
PMID
22291082
Source
scival
Published In
Journal of Clinical Oncology
Volume
30
Issue
7
Publish Date
2012
Start Page
751
End Page
757
DOI
10.1200/JCO.2011.38.0345

Defibrotide (DF) in the Treatment of Hepatic Veno-Occlusive Disease (VOD) in Stem Cell Transplant (SCT) and Non-SCT Patients (Pts): Early Intervention Improves Outcome - Updated Results of a Treatment IND Expanded Access Protocol

Authors
Richardson, PG; Smith, AR; Grupp, SA; Kernan, NA; Arai, S; Haut, PR; Triplett, BM; III, GAP; Symons, HJ; Adams, RH; Horn, BN; Lucas, K; Martin, PL; Mineishi, S; Ball, ED; Boyer, M; Fort, J; Kirov, II; Lehmann, LE; Madigan, C; Maglio, ME; Massaro, J; D'Agostino, RB; Hannah, AL; Tudone, E; Hume, R; Iacobelli, M; Soiffer, RJ; Grp, DS
MLA Citation
Richardson, PG, Smith, AR, Grupp, SA, Kernan, NA, Arai, S, Haut, PR, Triplett, BM, III, GAP, Symons, HJ, Adams, RH, Horn, BN, Lucas, K, Martin, PL, Mineishi, S, Ball, ED, Boyer, M, Fort, J, Kirov, II, Lehmann, LE, Madigan, C, Maglio, ME, Massaro, J, D'Agostino, RB, Hannah, AL, Tudone, E, Hume, R, Iacobelli, M, Soiffer, RJ, and Grp, DS. "Defibrotide (DF) in the Treatment of Hepatic Veno-Occlusive Disease (VOD) in Stem Cell Transplant (SCT) and Non-SCT Patients (Pts): Early Intervention Improves Outcome - Updated Results of a Treatment IND Expanded Access Protocol." November 18, 2011.
Source
wos-lite
Published In
Blood
Volume
118
Issue
21
Publish Date
2011
Start Page
226
End Page
227

Umbilical cord blood: a guide for primary care physicians.

Umbilical cord blood stem cell transplants are used to treat a variety of oncologic, genetic, hematologic, and immunodeficiency disorders. Physicians have an important role in educating, counseling, and offering umbilical cord blood donation and storage options to patients. Parents may donate their infant's cord blood to a public bank, pay to store it in a private bank, or have it discarded. The federal government and many state governments have passed laws and issued regulations regarding umbilical cord blood, and some states require physicians to discuss cord blood options with pregnant women. Five prominent medical organizations have published recommendations about cord blood donation and storage. Current guidelines recommend donation of umbilical cord blood to public banks when possible, or storage through the Related Donor Cord Blood Program when a sibling has a disease that may require a stem cell transplant. Experts do not currently recommend private banking for unidentified possible future use. Step-by-step guidance and electronic resources are available to physicians whose patients are considering saving or donating their infant's umbilical cord blood.

Authors
Martin, PL; Kurtzberg, J; Hesse, B
MLA Citation
Martin, PL, Kurtzberg, J, and Hesse, B. "Umbilical cord blood: a guide for primary care physicians." Am Fam Physician 84.6 (September 15, 2011): 661-666. (Review)
PMID
21916391
Source
pubmed
Published In
American family physician
Volume
84
Issue
6
Publish Date
2011
Start Page
661
End Page
666

Allogeneic transplantation for patients with high-risk or refractory neuroblastoma.

Authors
Driscoll, TA; Martin, PL; Moffet, J; Daniel, M; Page, K; Parikh, S; Prasad, V; Szabolcs, P; Kurtzberg, J
MLA Citation
Driscoll, TA, Martin, PL, Moffet, J, Daniel, M, Page, K, Parikh, S, Prasad, V, Szabolcs, P, and Kurtzberg, J. "Allogeneic transplantation for patients with high-risk or refractory neuroblastoma." JOURNAL OF CLINICAL ONCOLOGY 29.15 (May 20, 2011).
Source
wos-lite
Published In
Journal of Clinical Oncology
Volume
29
Issue
15
Publish Date
2011

Allogeneic transplantation for patients with high-risk or refractory neuroblastoma.

9552 Background: While the survival of high-risk neuroblastoma patients has improved with myeloablative chemotherapy (MAC) and 13-cis retinoic acid, 60% of children fail this approach, thus other strategies are needed. As a potential platform for future immunologic-based therapies, we report a retrospective, single-center experience using allogeneic hematopoietic stem cell transplantation (allo-HSCT) following MAC for high risk or recurrent neuroblastoma (NB).Between June 1994 and November 2007, 17 pediatric patients with high risk or recurrent NB underwent allo-HSCT: allograft sources were related bone marrow (MRD) in 10 patients (pts) and unrelated umbilical cord blood (UCB) in 7 pts. At initial diagnosis the median age was 3.5 years, 16 pts had stage 4 and 1 patient had stage 3 disease. N-myc was amplified in 4 of the 12 pts with available N-myc status. Prior to allo-HSCT, 4 pts were in CR and 7 pts had failed autologous HSCT. Median time from NB diagnosis to allo-HSCT was 14.9 months. Regarding MAC, 7 pts received total body irradiation (TBI)-based regimens: (5 TBI/melphalan (Mel) and 2 TBI/thiotepa), 6 pts received cyclophosphamide (Cy)-based regimens (5 Cy/Mel and 1 Cy-thiotepa), 1 received carboplatin /etoposide/melphalan (CEM), and 3 pts received therapeutic I-131MIBG based regimens (MIBG /CEM in 1, MIBG /campath /Mel in 1, and MIBG/TBI/Mel in 1).The median day to neutrophil engraftment (ANC > 500) was 10.5 days for MRD BMT and 22 days for UCBT. The median day to platelet engraftment (plt >20k) for MRD BMT was 29.5 days and for UCBT was 62 days. Acute GVHD (grades I-IV) was seen in 6 patients: 3 grade-I skin, 1 grade-II skin, and 2 grade-III involving both skin and gut. Eleven pts (65%) died at a median of 377 days post-allo-HSCT: 2 infections (PCP pneumonia, Pseudomonal sepsis), 2 MSOF, 1 leukemic relapse (child with recurrent NB and secondary AML) and 6 NB progression. Six patients (5 MRD, 1 UCB) are long-term survivors with overall survival of 35% at 10 years.Allo-HSCT provided durable remission in a very high risk group of NB patients. Therefore a strategy that incorporates early allografting with targeted immunologic therapy should be investigated for refractory or ultra-high risk NB patients.

Authors
Driscoll, TA; Martin, PL; Moffet, J; Daniel, M; Page, K; Parikh, S; Prasad, V; Szabolcs, P; Kurtzberg, J
MLA Citation
Driscoll, TA, Martin, PL, Moffet, J, Daniel, M, Page, K, Parikh, S, Prasad, V, Szabolcs, P, and Kurtzberg, J. "Allogeneic transplantation for patients with high-risk or refractory neuroblastoma." Journal of clinical oncology : official journal of the American Society of Clinical Oncology 29.15_suppl (May 2011): 9552-.
PMID
28019759
Source
epmc
Published In
Journal of Clinical Oncology
Volume
29
Issue
15_suppl
Publish Date
2011
Start Page
9552

Caregivers Helping in Relieving Pain – C.H.I.R.P. Project

Authors
Edmisten, JH; Prewitt, J; Martin, PL; Little, K
MLA Citation
Edmisten, JH, Prewitt, J, Martin, PL, and Little, K. "Caregivers Helping in Relieving Pain – C.H.I.R.P. Project." February 2011.
Source
crossref
Published In
Biology of Blood and Marrow Transplantation
Volume
17
Issue
2
Publish Date
2011
Start Page
S361
End Page
S361
DOI
10.1016/j.bbmt.2010.12.621

DIFFERENTIAL IMPACT OF HLA-A, -B, AND-DRB1 MISMATCHING IN RELATION TO OTHER VARIABLES ON THE OUTCOMES OF MYELOABLATIVE UNRELATED SINGLE DONOR UMBILICAL CORD BLOOD TRANSPLANTATION FROM 4/6 MATCHED UNITS IN CHILDREN AND YOUNG ADULTS

Authors
Prasad, VK; Mendizabal, A; Tewari, P; Page, K; Parikh, SH; Szabolcs, P; Driscoll, TA; Martin, PL; Kurtzberg, J
MLA Citation
Prasad, VK, Mendizabal, A, Tewari, P, Page, K, Parikh, SH, Szabolcs, P, Driscoll, TA, Martin, PL, and Kurtzberg, J. "DIFFERENTIAL IMPACT OF HLA-A, -B, AND-DRB1 MISMATCHING IN RELATION TO OTHER VARIABLES ON THE OUTCOMES OF MYELOABLATIVE UNRELATED SINGLE DONOR UMBILICAL CORD BLOOD TRANSPLANTATION FROM 4/6 MATCHED UNITS IN CHILDREN AND YOUNG ADULTS." February 2011.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
17
Issue
2
Publish Date
2011
Start Page
S185
End Page
S186
DOI
10.1016/j.bbmt.2010.12.100

A PROSPECTIVE STUDY OF REDUCED INTENSITY CONDITIONING (RIC) IN CHILDREN UNDERGOING UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) FOR NON-MALIGNANT DISEASES: PRELIMINARY RESULTS DEMONSTRATE A HIGH RATE OF ENGRAFTMENT AND LOW INCIDENCE OF GVHD

Authors
Parikh, SH; Martin, PL; Driscoll, TA; Baker, J; Piersol, K; Moffet, J; Stokhuyzen, A; Cash, J; Kurtzberg, J; Szabolcs, P
MLA Citation
Parikh, SH, Martin, PL, Driscoll, TA, Baker, J, Piersol, K, Moffet, J, Stokhuyzen, A, Cash, J, Kurtzberg, J, and Szabolcs, P. "A PROSPECTIVE STUDY OF REDUCED INTENSITY CONDITIONING (RIC) IN CHILDREN UNDERGOING UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) FOR NON-MALIGNANT DISEASES: PRELIMINARY RESULTS DEMONSTRATE A HIGH RATE OF ENGRAFTMENT AND LOW INCIDENCE OF GVHD." February 2011.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
17
Issue
2
Publish Date
2011
Start Page
S265
End Page
S266
DOI
10.1016/j.bbmt.2010.12.338

DURABLE ENGRAFTMENT AND CORRECTION OF GENETIC DEFECT IN CHILDREN WITH CONGENITAL AMEGAKARYOCYTIC THROMBOCYTOPENIA FOLLOWING MYELOABLATIVE UMBILICAL CORD BLOOD TRANSPLANTATION

Authors
Mahadeo, KM; Parikh, SH; Driscoll, TA; Page, K; Szabolcs, P; Martin, PL; Kurtzberg, J; Prasad, VK
MLA Citation
Mahadeo, KM, Parikh, SH, Driscoll, TA, Page, K, Szabolcs, P, Martin, PL, Kurtzberg, J, and Prasad, VK. "DURABLE ENGRAFTMENT AND CORRECTION OF GENETIC DEFECT IN CHILDREN WITH CONGENITAL AMEGAKARYOCYTIC THROMBOCYTOPENIA FOLLOWING MYELOABLATIVE UMBILICAL CORD BLOOD TRANSPLANTATION." February 2011.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
17
Issue
2
Publish Date
2011
Start Page
S256
End Page
S256
DOI
10.1016/j.bbmt.2010.12.312

MEETING THE CHALLENGE: DEVELOPING A MODEL TO CARE FOR AN ADOLESCENT PATIENT WITH LEUKEMIA AND SEVERE AUTISM

Authors
Bain, ME; Martin, PL; Kojis, S; Trout, SB; Kurtzberg, J
MLA Citation
Bain, ME, Martin, PL, Kojis, S, Trout, SB, and Kurtzberg, J. "MEETING THE CHALLENGE: DEVELOPING A MODEL TO CARE FOR AN ADOLESCENT PATIENT WITH LEUKEMIA AND SEVERE AUTISM." February 2011.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
17
Issue
2
Publish Date
2011
Start Page
S283
End Page
S283
DOI
10.1016/j.bbmt.2010.12.391

Ancestry and pharmacogenomics of relapse in acute lymphoblastic leukemia

Although five-year survival rates for childhood acute lymphoblastic leukemia (ALL) are now over 80% in most industrialized countries1, not all children have benefited equally from this progress2. Ethnic differences in survival after childhood ALL have been reported in many clinical studies3-11, with poorer survival observed among African Americans or those with Hispanic ethnicity when compared with European Americans or Asians3-5. The causes of ethnic differences remain uncertain, although both genetic and non-genetic factors are likely important 4,12. Interrogating genome-wide germline SNP genotypes in an unselected large cohort of children with ALL, we observed that the component of genomic variation that co-segregated with Native American ancestry was associated with risk of relapse (P = 0.0029) even after adjusting for known prognostic factors (P = 0.017). Ancestry-related differences in relapse risk were abrogated by the addition of a single extra phase of chemotherapy, indicating that modifications to therapy can mitigate the ancestry-related risk of relapse. © 2011 Nature America, Inc. All rights reserved.

Authors
Yang, JJ; Cheng, C; Devidas, M; Cao, X; Fan, Y; Campana, D; Yang, W; Neale, G; Cox, NJ; Scheet, P; Borowitz, MJ; Winick, NJ; Martin, PL; Willman, CL; Bowman, WP; Camitta, BM; Carroll, A; Reaman, GH; Carroll, WL; Loh, M; Hunger, SP; Pui, C-H; Evans, WE; Relling, MV
MLA Citation
Yang, JJ, Cheng, C, Devidas, M, Cao, X, Fan, Y, Campana, D, Yang, W, Neale, G, Cox, NJ, Scheet, P, Borowitz, MJ, Winick, NJ, Martin, PL, Willman, CL, Bowman, WP, Camitta, BM, Carroll, A, Reaman, GH, Carroll, WL, Loh, M, Hunger, SP, Pui, C-H, Evans, WE, and Relling, MV. "Ancestry and pharmacogenomics of relapse in acute lymphoblastic leukemia." Nature Genetics 43.3 (2011): 237-241.
PMID
21297632
Source
scival
Published In
Nature Genetics
Volume
43
Issue
3
Publish Date
2011
Start Page
237
End Page
241
DOI
10.1038/ng.763

Defibrotide (DF) In the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF): Results of a Treatment IND Expanded Access Protocol

Authors
Richardson, P; Smith, A; Arai, S; Grupp, SA; Kernan, NA; Adams, R; Martin, PL; Abhyankar, S; III, GA; Grimley, M; Lehmann, LE; Haut, P; Termuhlen, A; Antin, JH; Spitzer, T; Avigan, D; Massaro, J; Hannah, AL; Iacobelli, M; Soiffer, RJ; Grp, DS
MLA Citation
Richardson, P, Smith, A, Arai, S, Grupp, SA, Kernan, NA, Adams, R, Martin, PL, Abhyankar, S, III, GA, Grimley, M, Lehmann, LE, Haut, P, Termuhlen, A, Antin, JH, Spitzer, T, Avigan, D, Massaro, J, Hannah, AL, Iacobelli, M, Soiffer, RJ, and Grp, DS. "Defibrotide (DF) In the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF): Results of a Treatment IND Expanded Access Protocol." November 19, 2010.
Source
wos-lite
Published In
Blood
Volume
116
Issue
21
Publish Date
2010
Start Page
399
End Page
400

Meta-Analysis of Defibrotide (DF) In the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) with Comparison to a Historical Control (HC)

Authors
Richardson, PG; Steinbach, G; Kernan, N; Guinan, EC; Chen, AR; Martin, PL; Krishnan, A; Arai, S; Brochstein, JA; Grupp, SA; Mineishi, S; Termuhlen, A; Tomblyn, M; Antin, JH; Revta, C; Hume, R; Massaro, J; Hannah, AL; Iacobelli, M; Soiffer, RJ; Grp, DS
MLA Citation
Richardson, PG, Steinbach, G, Kernan, N, Guinan, EC, Chen, AR, Martin, PL, Krishnan, A, Arai, S, Brochstein, JA, Grupp, SA, Mineishi, S, Termuhlen, A, Tomblyn, M, Antin, JH, Revta, C, Hume, R, Massaro, J, Hannah, AL, Iacobelli, M, Soiffer, RJ, and Grp, DS. "Meta-Analysis of Defibrotide (DF) In the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) with Comparison to a Historical Control (HC)." November 19, 2010.
Source
wos-lite
Published In
Blood
Volume
116
Issue
21
Publish Date
2010
Start Page
1430
End Page
1431

Reduced Intensity Conditioning In Children Undergoing Unrelated Umbilical Cord Blood Transplantation for Non-Malignant Diseases: Preliminary Results Demonstrate a High Rate of Engraftment and Low Incidence of Gvhd.

Authors
Parikh, SH; Martin, PL; Driscoll, TA; Baker, J; Piersol, K; Moffet, J; Stokhuyzen, A; Kurtzberg, J; Szabolcs, P
MLA Citation
Parikh, SH, Martin, PL, Driscoll, TA, Baker, J, Piersol, K, Moffet, J, Stokhuyzen, A, Kurtzberg, J, and Szabolcs, P. "Reduced Intensity Conditioning In Children Undergoing Unrelated Umbilical Cord Blood Transplantation for Non-Malignant Diseases: Preliminary Results Demonstrate a High Rate of Engraftment and Low Incidence of Gvhd." November 19, 2010.
Source
wos-lite
Published In
Blood
Volume
116
Issue
21
Publish Date
2010
Start Page
1453
End Page
1454

ARID5B Genetic Polymorphisms Contribute to Racial Disparities In Childhood Acute Lymphoblastic Leukemia: A Children's Oncology Group Study

Authors
Yang, J; Xu, H; Cheng, C; Devidas, M; Pei, D; Fan, Y; Yang, W; Neale, GA; Winick, NJ; Martin, PL; Willman, CL; Camitta, BM; Reaman, GH; Carroll, WL; Loh, M; Hunger, SP; Evans, WE; Relling, MV
MLA Citation
Yang, J, Xu, H, Cheng, C, Devidas, M, Pei, D, Fan, Y, Yang, W, Neale, GA, Winick, NJ, Martin, PL, Willman, CL, Camitta, BM, Reaman, GH, Carroll, WL, Loh, M, Hunger, SP, Evans, WE, and Relling, MV. "ARID5B Genetic Polymorphisms Contribute to Racial Disparities In Childhood Acute Lymphoblastic Leukemia: A Children's Oncology Group Study." November 19, 2010.
Source
wos-lite
Published In
Blood
Volume
116
Issue
21
Publish Date
2010
Start Page
9
End Page
10

Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.

Therapeutic options for severe hepatic veno-occlusive disease (VOD) are limited and outcomes are dismal, but early phase I/II studies have suggested promising activity and acceptable toxicity using the novel polydisperse oligonucleotide defibrotide. This randomized phase II dose-finding trial determined the efficacy of defibrotide in patients with severe VOD following hematopoietic stem cell transplantation (HSCT) and identified an appropriate dose for future trials. Adult and pediatric patients received either lower-dose (arm A: 25 mg/kg/day; n = 75) or higher-dose (arm B: 40 mg/kg/day; n = 74) i.v. defibrotide administered in divided doses every 6 hours for > or =14 days or until complete response, VOD progression, or any unacceptable toxicity occurred. Overall complete response and day +100 post-HSCT survival rates were 46% and 42%, respectively, with no significant difference between treatment arms. The incidence of treatment-related adverse events was low (8% overall; 7% in arm A, 10% in arm B); there was no significant difference in the overall rate of adverse events between treatment arms. Early stabilization or decreased bilirubin was associated with better response and day +100 survival, and decreased plasminogen activator inhibitor type 1 (PAI-1) during treatment was associated with better outcome; changes were similar in both treatment arms. Defibrotide 25 or 40 mg/kg/day also appears effective in treating severe VOD following HSCT. In the absence of any differences in activity, toxicity or changes in PAI-1 level, defibrotide 25 mg/kg/day was selected for ongoing phase III trials in VOD.

Authors
Richardson, PG; Soiffer, RJ; Antin, JH; Uno, H; Jin, Z; Kurtzberg, J; Martin, PL; Steinbach, G; Murray, KF; Vogelsang, GB; Chen, AR; Krishnan, A; Kernan, NA; Avigan, DE; Spitzer, TR; Shulman, HM; Di Salvo, DN; Revta, C; Warren, D; Momtaz, P; Bradwin, G; Wei, LJ; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, PG, Soiffer, RJ, Antin, JH, Uno, H, Jin, Z, Kurtzberg, J, Martin, PL, Steinbach, G, Murray, KF, Vogelsang, GB, Chen, AR, Krishnan, A, Kernan, NA, Avigan, DE, Spitzer, TR, Shulman, HM, Di Salvo, DN, Revta, C, Warren, D, Momtaz, P, Bradwin, G, Wei, LJ, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial." Biol Blood Marrow Transplant 16.7 (July 2010): 1005-1017.
PMID
20167278
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
16
Issue
7
Publish Date
2010
Start Page
1005
End Page
1017
DOI
10.1016/j.bbmt.2010.02.009

Down syndrome childhood acute lymphoblastic leukemia has a unique spectrum of sentinel cytogenetic lesions that influences treatment outcome: A report from the Children's Oncology Group

Children with Down syndrome (DS) have an increased risk of acute lymphoblastic leukemia (ALL) and an inferior outcome. We reviewed data from 2811 children with ALL enrolled in Children's Oncology Group P9900, which included prospective testing for the major cytogenetic lesions in childhood ALL: ETV6-RUNX1, TCF3-PBX1, BCR-ABL1, and MLL translocations and trisomies of chromosomes 4 and 10. Eighty (3%) B-precursor ALL patients had DS. Age, sex, white blood cell count, and risk group were similar between DS-ALL and non-DS-ALL but significantly more patients with DS-ALL were white (91.2% vs 76.4%, P = .001). Children with DS-ALL had lower rates of the favorable cytogenetic lesions ETV6-RUNX1 (2.5% vs 24%, P < .001) and trisomies 4 and 10 (7.7% vs 24%, P < .001). Five-year event-free (EFS) and overall survival (OS) were inferior in children with DS-ALL: 69.9% ± 8.6% versus 78.1% ± 1.2% (P = .078), and 85.8% ± 6.5% versus 90.0% ± 0.9% (P = .033). However, when children with MLL translocations, BCR-ABL1, ETV6-RUNX1, and trisomies 4 and 10 were excluded, the EFS and OS were similar for children with and without DS (EFS 68.0 % ± 9.3% vs 70.5% ± 1.9%, P = .817; and OS 86.7% ± 6.7% vs 85.4% ± 1.5%; P = .852), both overall and adjusted for race. DS-ALL displays a unique spectrum of biologic subtypes with different frequencies of sentinel cytogenetic lesions having a large influence on outcome. © 2010 by The American Society of Hematology.

Authors
Maloney, KW; Carroll, WL; Carroll, AJ; Devidas, M; Borowitz, MJ; Martin, PL; Pullen, J; Whitlock, JA; Willman, CL; Winick, NJ; Camitta, BM; Hunger, SP
MLA Citation
Maloney, KW, Carroll, WL, Carroll, AJ, Devidas, M, Borowitz, MJ, Martin, PL, Pullen, J, Whitlock, JA, Willman, CL, Winick, NJ, Camitta, BM, and Hunger, SP. "Down syndrome childhood acute lymphoblastic leukemia has a unique spectrum of sentinel cytogenetic lesions that influences treatment outcome: A report from the Children's Oncology Group." Blood 116.7 (2010): 1045-1050.
PMID
20442364
Source
scival
Published In
Blood
Volume
116
Issue
7
Publish Date
2010
Start Page
1045
End Page
1050
DOI
10.1182/blood-2009-07-235291

Hepatic Veno-Occlusive Disease following Stem Cell Transplantation: Incidence, Clinical Course, and Outcome

The occurrence of hepatic veno-occlusive disease (VOD) has been reported in up to 60% of patients following stem cell transplantation (SCT), with incidence varying widely between studies depending on the type of transplant, conditioning regimen, and criteria used to make the diagnosis. Severe VOD is characterized by high mortality and progression to multiorgan failure (MOF); however, there is no consensus on how to evaluate severity. This review and analysis of published reports attempts to clarify these issues by calculating the overall mean incidence of VOD and mortality from severe VOD, examining the effect of changes in SCT practice on the incidence of VOD over time, and discussing the methods used to evaluate severity. Across 135 studies performed between 1979 and October 2007, the overall mean incidence of VOD was 13.7% (95% confidence interval [CI] = 13.3%-14.1%). The mean incidence of VOD was significantly lower between 1979-1994 than between 1994-2007 (11.5% [95% CI, 10.9%-12.1%] vs 14.6% [95% CI, 14.0%-15.2%]; P < .05). The mortality rate from severe VOD was 84.3% (95% CI, 79.6%-88.9%); most of these patients had MOF, which also was the most frequent cause of death. Thus, VOD is less common than early reports suggested, but the current incidence appears to be relatively stable despite recent advances in SCT, including the advent of reduced-intensity conditioning. The evolution of MOF in the setting of VOD after SCT can be considered a reliable indication of severity and a predictor of poor outcome. © 2010 American Society for Blood and Marrow Transplantation.

Authors
Coppell, JA; Richardson, PG; Soiffer, R; Martin, PL; Kernan, NA; Chen, A; Guinan, E; Vogelsang, G; Krishnan, A; Giralt, S; Revta, C; Carreau, NA; Iacobelli, M; Carreras, E; Ruutu, T; Barbui, T; Antin, JH; Niederwieser, D
MLA Citation
Coppell, JA, Richardson, PG, Soiffer, R, Martin, PL, Kernan, NA, Chen, A, Guinan, E, Vogelsang, G, Krishnan, A, Giralt, S, Revta, C, Carreau, NA, Iacobelli, M, Carreras, E, Ruutu, T, Barbui, T, Antin, JH, and Niederwieser, D. "Hepatic Veno-Occlusive Disease following Stem Cell Transplantation: Incidence, Clinical Course, and Outcome." Biology of Blood and Marrow Transplantation 16.2 (2010): 157-168.
PMID
19766729
Source
scival
Published In
Biology of Blood and Marrow Transplantation
Volume
16
Issue
2
Publish Date
2010
Start Page
157
End Page
168
DOI
10.1016/j.bbmt.2009.08.024

Defibrotide (DF) in the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) Following Stem Cell Transplantation (SCT): Results of a Phase 3 Study Utilizing a Historical Control

Authors
Richardson, P; Tomblyn, M; Kernan, N; Brochstein, JA; Mineishi, S; Termuhlen, A; Arai, S; Grupp, SA; Guinan, E; Martin, PL; Corbacioglu, S; Holler, E; D'Agostino, R; Massaro, J; Hannah, AL; Iacobelli, M; Soiffer, RJ
MLA Citation
Richardson, P, Tomblyn, M, Kernan, N, Brochstein, JA, Mineishi, S, Termuhlen, A, Arai, S, Grupp, SA, Guinan, E, Martin, PL, Corbacioglu, S, Holler, E, D'Agostino, R, Massaro, J, Hannah, AL, Iacobelli, M, and Soiffer, RJ. "Defibrotide (DF) in the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) Following Stem Cell Transplantation (SCT): Results of a Phase 3 Study Utilizing a Historical Control." November 20, 2009.
Source
wos-lite
Published In
Blood
Volume
114
Issue
22
Publish Date
2009
Start Page
272
End Page
273

Transplantation Following Myeloablative Cytoreduction Results in Excellent Survival, Long-Term Engraftment, and Donor Chimerism in Children with Chronic Granulomatous Disease Across Donor and Graft Sources

Authors
Tewari, P; Wood, S; Martin, PL; Parikh, SH; Szabolcs, P; Page, KM; Driscoll, T; Kurtzberg, J; Prasad, VK
MLA Citation
Tewari, P, Wood, S, Martin, PL, Parikh, SH, Szabolcs, P, Page, KM, Driscoll, T, Kurtzberg, J, and Prasad, VK. "Transplantation Following Myeloablative Cytoreduction Results in Excellent Survival, Long-Term Engraftment, and Donor Chimerism in Children with Chronic Granulomatous Disease Across Donor and Graft Sources." November 20, 2009.
Source
wos-lite
Published In
Blood
Volume
114
Issue
22
Publish Date
2009
Start Page
1319
End Page
1319

Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience.

Myelodysplastic syndromes (MDS) respond poorly to chemotherapy. Between 1995 and 2006, 23 pediatric patients with MDS were transplanted with unrelated donor umbilical cord blood (UUCB) at our center. The median age was 11.1 years (range: 1.1-19.7), median weight was 38.6 kg (range: 9.6-62.6), 61% of patients were male, and median time from diagnosis to transplant was 6.6 months (range: 2.0-61.4). Patients were followed for a median of 5.3 years (range: 1.6-12.4 years) posttransplant. MDS stage was refractory anemia (RA) in 12, refractory anemia with excess blasts (RAEB) in 8, and refractory anemia with excess blasts in transformation (RAEB-T) in 3 patients; 18 (78%) patients had primary MDS. Monosomy 7 was present in 17(74%) patients. Patients with acute myelogenous leukemia (AML) were excluded. Preparative regimen was total body irradiation (TBI) based in 18 (78%) patients. Graft-versus-host-disease (GVHD) prophylaxis was cyclosporine (CsA)/steroids (19 patients) or CsA/mycophenolate mofetil (MMF; 4 patients). Grafts were HLA matched at Class I (A and B) at low resolution and Class II (DRB1) at the allelic level, resulting in 16 (70%) 4/6 and 7 (30%) 5/6 matched transplants. The grafts contained a median of 4.0 x 10(7) (range: 1.7-12.6) total nucleated cells (TNC)/kg precryopreservation; 3.6 x 10(7) (range: 1.0-12.0) TNC/kg and 1.7 x 10(5) (range: 0.2-28.5) CD34+ cells/kg were infused. Cumulative incidence of neutrophil engraftment (absolute neutrophil count [ANC] >500/microL) at day 42 and day 100 was 73.9% (95% confidence interval [CI] 55.1%-92.7%) and 91.3% (95% CI 71.3%-100.0%) respectively, and that of platelet engraftment (50 K) at 180 days was 69.6% (95% CI 49.8%-89.4%). Three patients had graft failure whereas 3 patients (13%) engrafted slowly (after day 42). Three patients developed acute GVHD (aGVHD) grades II-IV with a cumulative incidence at 100 days of 13% (95% CI 0.0%-27.1.0%). Four patients relapsed with a cumulative incidence of relapse at 3 years of 13.0% (95% CI 0.0%-27.1%). Cumulative incidence of nonrelapse mortality (NRM) at 1 year was 27% (95% CI 8.0%-46.0%). Ten patients died: 3 graft failure, 4 relapse, 2 infections (1 adenovirus, 1 toxoplasmosis), and 1 Epstein-Barr virus (EBV) lymphoproliferative disorder. Probabilities of event-free survival (EFS) at 1 and 3 years were 69.6% (95% CI 46.6%-84.2%) and 60.9% (95% CI 38.3%-77.4%), respectively. Factors associated with better EFS were age < or = 11 years (P = .05) and weight < or = 38 kg (P = .03). These results, especially in younger patients with monosomy 7 positive MDS, are equivalent to published matched allogeneic bone marrow data. UUCB should be actively considered for pediatric MDS patients lacking matched related or unrelated adult donors.

Authors
Parikh, SH; Mendizabal, A; Martin, PL; Prasad, VK; Szabolcs, P; Driscoll, TA; Kurtzberg, J
MLA Citation
Parikh, SH, Mendizabal, A, Martin, PL, Prasad, VK, Szabolcs, P, Driscoll, TA, and Kurtzberg, J. "Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience." Biol Blood Marrow Transplant 15.8 (August 2009): 948-955.
PMID
19589484
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
8
Publish Date
2009
Start Page
948
End Page
955
DOI
10.1016/j.bbmt.2009.04.010

PERICARDIAL EFFUSION FOLLOWING UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION: ANALYSIS IN A COHORT OF 423 PEDIATRIC PATIENTS TRANSPLANTED AT A SINGLE CENTER

Authors
Hwang, EI; Camitta, MGW; Vinesett, R; Mendizabal, A; Wood, S; Semmel, D; Page, K; Driscoll, TA; Parikh, SH; Szabolcs, P; Kurtzberg, J; Prasad, VK
MLA Citation
Hwang, EI, Camitta, MGW, Vinesett, R, Mendizabal, A, Wood, S, Semmel, D, Page, K, Driscoll, TA, Parikh, SH, Szabolcs, P, Kurtzberg, J, and Prasad, VK. "PERICARDIAL EFFUSION FOLLOWING UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION: ANALYSIS IN A COHORT OF 423 PEDIATRIC PATIENTS TRANSPLANTED AT A SINGLE CENTER." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 15.2 (February 2009): 73-74.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
2
Publish Date
2009
Start Page
73
End Page
74

UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION FROM A SINGLE 4/6 MATCHED UNIT IS AN EFFECTIVE THERAPY FOR CHILDREN WITH MALIGNANT AND NON-MALIGNANT DIAGNOSES: GOOD SURVIVAL, LOW GRAFT FAILURE AND GRAFT-VS-HOST DISEASE IN A SINGLE CENTER ANALYSIS OF 314 PATIENTS

Authors
Prasad, VK; Mendizabal, A; Gill, P; Parikh, SH; Szabolcs, P; Driscoll, TA; Page, K; Wood, S; Semmel, D; Martin, PL; Carter, S; Kurtzberg, J
MLA Citation
Prasad, VK, Mendizabal, A, Gill, P, Parikh, SH, Szabolcs, P, Driscoll, TA, Page, K, Wood, S, Semmel, D, Martin, PL, Carter, S, and Kurtzberg, J. "UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION FROM A SINGLE 4/6 MATCHED UNIT IS AN EFFECTIVE THERAPY FOR CHILDREN WITH MALIGNANT AND NON-MALIGNANT DIAGNOSES: GOOD SURVIVAL, LOW GRAFT FAILURE AND GRAFT-VS-HOST DISEASE IN A SINGLE CENTER ANALYSIS OF 314 PATIENTS." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 15.2 (February 2009): 27-27.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
2
Publish Date
2009
Start Page
27
End Page
27

OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN PEDIATRIC PATIENTS WITH PRIMARY AND SECONDARY MYELODYSPLASTIC SYNDROMES

Authors
Parikh, SH; Mendizabal, A; Betz-Stablein, B; Martin, PL; Szabolcs, P; Prasad, VK; Driscoll, TA; Kurtzberg, J
MLA Citation
Parikh, SH, Mendizabal, A, Betz-Stablein, B, Martin, PL, Szabolcs, P, Prasad, VK, Driscoll, TA, and Kurtzberg, J. "OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN PEDIATRIC PATIENTS WITH PRIMARY AND SECONDARY MYELODYSPLASTIC SYNDROMES." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 15.2 (February 2009): 72-72.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
2
Publish Date
2009
Start Page
72
End Page
72

EASING THE TBI EXPERIENCE IN THE PRE-SCHOOL PATIENT

Authors
Cash, JV; Sweezy, CM; Browne-Topete, A; Martin, PL; Georgas, DL; Larrier, NA
MLA Citation
Cash, JV, Sweezy, CM, Browne-Topete, A, Martin, PL, Georgas, DL, and Larrier, NA. "EASING THE TBI EXPERIENCE IN THE PRE-SCHOOL PATIENT." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 15.2 (February 2009): 32-33.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
2
Publish Date
2009
Start Page
32
End Page
33

UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IS AN EFFECTIVE THERAPY FOR WISKOTT-ALDRICH SYNDROME

Authors
Sun, JM; Driscoll, T; Prasad, K; Parikh, S; Szabolcs, P; Kurtzberg, Y; Martin, PL
MLA Citation
Sun, JM, Driscoll, T, Prasad, K, Parikh, S, Szabolcs, P, Kurtzberg, Y, and Martin, PL. "UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IS AN EFFECTIVE THERAPY FOR WISKOTT-ALDRICH SYNDROME." BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION 15.2 (February 2009): 76-76.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
2
Publish Date
2009
Start Page
76
End Page
76

Single Donor 4/6 Matched Banked Unrelated Cord Blood Is An Effective Graft Source for Children Undergoing Myeloablative Transplantation for Malignant and Nonmalignant Disorders: Single Center Analysis of 318 Patients

Authors
Prasad, VK; Gill, P; Vinesett, R; Parikh, SH; Szabolcs, P; Driscoll, TA; Page, KM; Wood, S; Semmel, D; Martin, PL; Kurtzberg, J
MLA Citation
Prasad, VK, Gill, P, Vinesett, R, Parikh, SH, Szabolcs, P, Driscoll, TA, Page, KM, Wood, S, Semmel, D, Martin, PL, and Kurtzberg, J. "Single Donor 4/6 Matched Banked Unrelated Cord Blood Is An Effective Graft Source for Children Undergoing Myeloablative Transplantation for Malignant and Nonmalignant Disorders: Single Center Analysis of 318 Patients." November 16, 2008.
Source
wos-lite
Published In
Blood
Volume
112
Issue
11
Publish Date
2008
Start Page
690
End Page
690

Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.

Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftment, overall survival (OS), and graft-versus-host disease (GVHD). Patients received myeloablative chemotherapy (busulfan, cyclophosphamide, ATG) and cyclosporine-based GVHD prophylaxis. Infused cell doses were high (7.57 x 10(7)/kg) because of the patients' young age (median, 1.5 years) and small size (median, 12 kg). Median follow-up was 4.2 years (range, 1-11 years). The cumulative incidences of neutrophil and platelet engraftment were 87.1% (95% confidence interval [CI], 81.8%-92.4%) and 71.0% (95% CI, 63.7%-78.3%). A total of 97% achieved high (> 90%) donor chimerism. Serum enzyme normalized in 97% of patients with diseases for which testings exist. Grade III/IV acute GVHD occurred in 10.3% (95% CI, 5.4%-15.2%) of patients. Extensive chronic GVHD occurred in 10.8% (95% CI, 5.7%-15.9%) of patients by 1 year. OS at 1 and 5 years was 71.8% (95% CI, 64.7%-78.9%) and 58.2% (95% CI, 49.7%-66.6%) in all patients and 84.5% (95% CI, 77.0%-92.0%) and 75.7% (95% CI, 66.1%-85.3%) in patients with high (80-100) performance score. In multivariate analysis, favorable factors for OS were high pretransplantation performance status, matched donor/recipient ethnicity, and higher infused colony forming units.

Authors
Prasad, VK; Mendizabal, A; Parikh, SH; Szabolcs, P; Driscoll, TA; Page, K; Lakshminarayanan, S; Allison, J; Wood, S; Semmel, D; Escolar, ML; Martin, PL; Carter, S; Kurtzberg, J
MLA Citation
Prasad, VK, Mendizabal, A, Parikh, SH, Szabolcs, P, Driscoll, TA, Page, K, Lakshminarayanan, S, Allison, J, Wood, S, Semmel, D, Escolar, ML, Martin, PL, Carter, S, and Kurtzberg, J. "Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes." Blood 112.7 (October 1, 2008): 2979-2989.
PMID
18587012
Source
pubmed
Published In
Blood
Volume
112
Issue
7
Publish Date
2008
Start Page
2979
End Page
2989
DOI
10.1182/blood-2008-03-140830

Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation.

Autoimmune cytopenias are a recognized complication of hematopoietic stem cell transplant (HSCT), and are considered to be a feature of chronic graft-versus-host disease (cGVHD). We report on a cohort of very young infants (< or =3 months of age) receiving HSCT from unrelated donor umbilical cord blood for genetic disorders who developed posttransplant autoimmune cytopenias at an increased rate compared to older aged controls. These infants received a conditioning regimen consisting of busulfan, cyclophosphamide, and antithymocyte globulin (ATG). All infants received HLA mismatched unrelated umbilical cord blood as graft source. GVHD prophylaxis was either cyclosporine + methylprednisolone (n = 16) or cyclosporine + mycophenolate mofetil (n = 3). Engraftment, acute GVHD (aGVHD) and cGVHD, survival, treatment-related mortality (TRM), and deaths were evaluated. Ten patients developed cGVHD manifesting as autoimmune cytopenias at a median 247 days posttransplant with a cumulative incidence of 44% (95% confidence interval [CI] 21%-68%) and 56% (95% CI 32%-80%) at 1 and 2 years, respectively. In 6 of 10 patients developing autoimmune cytopenias, cGVHD presented as autoimmune cytopenia de novo. The cytopenias observed included anemia (n = 4), thrombocytopenia (n = 1), anemia with thrombocytopenia (n = 3), and pancytopenia (n = 2). No graft factors were identified as being significant to development of cGVHD. All patients responded to treatment with methylprednisolone, azithioprine +/- rituximab. One patient required splenectomy. We hypothesize that posttransplant immunosuppression interferes with normal immune ontogeny creating immune dysregulation and graft directed cell destruction. Alternative strategies to prevent GVHD should be considered for this unique patient population.

Authors
Page, KM; Mendizabal, AM; Prasad, VK; Martin, PL; Parikh, S; Wood, S; Sempowski, GD; Szabolcs, P; Kurtzberg, J
MLA Citation
Page, KM, Mendizabal, AM, Prasad, VK, Martin, PL, Parikh, S, Wood, S, Sempowski, GD, Szabolcs, P, and Kurtzberg, J. "Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation." Biol Blood Marrow Transplant 14.10 (October 2008): 1108-1117.
PMID
18804040
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
14
Issue
10
Publish Date
2008
Start Page
1108
End Page
1117
DOI
10.1016/j.bbmt.2008.07.006

Clinical significance of minimal residual disease in childhood acute lymphoblastic leukemia and its relationship to other prognostic factors: a Children's Oncology Group study.

Minimal residual disease (MRD) is an important predictor of relapse in acute lymphoblastic leukemia (ALL), but its relationship to other prognostic variables has not been fully assessed. The Children's Oncology Group studied the prognostic impact of MRD measured by flow cytometry in the peripheral blood at day 8, and in end-induction (day 29) and end-consolidation marrows in 2143 children with precursor B-cell ALL (B-ALL). The presence of MRD in day-8 blood and day-29 marrow MRD was associated with shorter event-free survival (EFS) in all risk groups; even patients with 0.01% to 0.1% day-29 MRD had poor outcome compared with patients negative for MRD patients (59% +/- 5% vs 88% +/- 1% 5-year EFS). Presence of good prognostic markers TEL-AML1 or trisomies of chromosomes 4 and 10 still provided additional prognostic information, but not in National Cancer Institute high-risk (NCI HR) patients who were MRD(+). The few patients with detectable MRD at end of consolidation fared especially poorly, with only a 43% plus or minus 7% 5-year EFS. Day-29 marrow MRD was the most important prognostic variable in multi-variate analysis. The 12% of patients with all favorable risk factors, including NCI risk group, genetics, and absence of days 8 and 29 MRD, had a 97% plus or minus 1% 5-year EFS with nonintensive therapy. These studies are registered at www.clinicaltrials.gov as NCT00005585, NCT00005596, and NCT00005603.

Authors
Borowitz, MJ; Devidas, M; Hunger, SP; Bowman, WP; Carroll, AJ; Carroll, WL; Linda, S; Martin, PL; Pullen, DJ; Viswanatha, D; Willman, CL; Winick, N; Camitta, BM; Children's Oncology Group,
MLA Citation
Borowitz, MJ, Devidas, M, Hunger, SP, Bowman, WP, Carroll, AJ, Carroll, WL, Linda, S, Martin, PL, Pullen, DJ, Viswanatha, D, Willman, CL, Winick, N, Camitta, BM, and Children's Oncology Group, . "Clinical significance of minimal residual disease in childhood acute lymphoblastic leukemia and its relationship to other prognostic factors: a Children's Oncology Group study." Blood 111.12 (June 15, 2008): 5477-5485.
PMID
18388178
Source
pubmed
Published In
Blood
Volume
111
Issue
12
Publish Date
2008
Start Page
5477
End Page
5485
DOI
10.1182/blood-2008-01-132837

Comparison of the biology of Down syndrome (DS) acute lymphoblastic leukemia (ALL) and non-DS ALL: Children's Oncology Group (COG) study P9900

Authors
Maloney, K; Carroll, WL; Carroll, A; Devidas, M; Hunger, SP; Martin, PL; Willman, CL; Winick, N; Whitlock, J; Camitta, BM
MLA Citation
Maloney, K, Carroll, WL, Carroll, A, Devidas, M, Hunger, SP, Martin, PL, Willman, CL, Winick, N, Whitlock, J, and Camitta, BM. "Comparison of the biology of Down syndrome (DS) acute lymphoblastic leukemia (ALL) and non-DS ALL: Children's Oncology Group (COG) study P9900." JOURNAL OF CLINICAL ONCOLOGY 26.15 (May 20, 2008).
Source
wos-lite
Published In
Journal of Clinical Oncology
Volume
26
Issue
15
Publish Date
2008

Comparison of the biology of Down syndrome (DS) acute lymphoblastic leukemia (ALL) and non-DS ALL: Children's Oncology Group (COG) study P9900.

10003 Background: DS ALL is a heterogeneous disorder with inferior survival compared to non-DS. Controversy exists regarding the relative frequencies of sentinel cytogenetic lesions in children with DS and ALL. METHODS: COG P9900 was a laboratory classification study for ALL therapeutic trials from 12/99 to 2/05. Flow cytometry for DNA index; molecular testing for TEL-AML1, E2A-PBX1, BCR-ABL and FISH testing for trisomies 4 and 10 and for MLL rearrangements were done centrally for risk group stratification in a prospective manner to assign continuing treatment. RESULTS: Eighty of 2811 (3%) consecutively enrolled eligible B-precursor ALL patients (pts) had DS. Age > or < than 10 years of age, gender, presenting white blood cell count (WBC) and NCI risk group were similar between ALL patients with/without DS. However, the genetic lesions present in DS-ALL patients and the event-free survival (EFS) and overall survival (OS) differed from those of non-DS patients. No BCR/ABL or MLL translocations were found in the DS pts. TEL-AML1 fusion was present in 2.5% (2/80) of DS-ALL pts vs. 24% (651/2710) (p<0.0001) of non-DS-ALL pts. Trisomies of chromosomes 4 and 10 were present in 7.7% (6/78) of DS-ALL pts vs. 24% (643/2689) (p=0.0004) of non-DS-ALL pts. Five-year EFS and OS were inferior in pts with DS-ALL; 70% vs. 76% (p=0.078), and 86% vs. 90% (p=0.0333). However, when pts with MLL, BCR/ABL, TEL-AML 1 and trisomies 4 and 10 were excluded, the EFS and OS for both groups were equivalent with EFS for DS 68% vs. 70% for non-DS (p=0.8174), and OS for DS 87% vs. 85% for non-DS (p=0.8519). CONCLUSIONS: The inferior outcome of patients with ALL and DS is related to a much lower incidence of favorable genetic features (TEL- AML 1 and trisomies of chromosomes 4 and 10). Further investigation into the distinct pathogenesis of DS ALL is warranted to improve treatment response. No significant financial relationships to disclose.

Authors
Maloney, K; Carroll, WL; Carroll, A; Devidas, M; Hunger, SP; Martin, PL; Willman, CL; Winick, N; Whitlock, J; Camitta, BM
MLA Citation
Maloney, K, Carroll, WL, Carroll, A, Devidas, M, Hunger, SP, Martin, PL, Willman, CL, Winick, N, Whitlock, J, and Camitta, BM. "Comparison of the biology of Down syndrome (DS) acute lymphoblastic leukemia (ALL) and non-DS ALL: Children's Oncology Group (COG) study P9900." J Clin Oncol 26.15_suppl (May 20, 2008): 10003-.
PMID
27951259
Source
pubmed
Published In
Journal of Clinical Oncology
Volume
26
Issue
15_suppl
Publish Date
2008
Start Page
10003

Pharmacogenetics of minimal residual disease response in children with B-precursor acute lymphoblastic leukemia: a report from the Children's Oncology Group.

Minimal residual disease (MRD) as a marker of antileukemic drug efficacy is being used to assess risk status and, in some cases, to adjust the intensity of therapy. Within known prognostic categories, the determinants of MRD are not known. We measured MRD by flow cytometry at day 8 (in blood) and at day 28 (in bone marrow) of induction therapy in more than 1000 children enrolled in Pediatric Oncology Group therapy protocols 9904, 9905, and 9906. We classified patients as "best risk" if they had cleared MRD by day 8 of therapy and as "worst risk" if they had MRD remaining in bone marrow at day 28, and tested whether MRD was related to polymorphisms in 16 loci in genes hypothesized to influence response to therapy in acute lymphoblastic leukemia (ALL). After adjusting for known prognostic features such as presence of the TEL-AML1 rearrangement, National Cancer Institute (NCI) risk status, ploidy, and race, the G allele of a common polymorphism in chemokine receptor 5 (CCR5) was associated with more favorable MRD status than the A allele (P = .009, logistic regression), when comparing "best" and "worst" risk groups. These data are consistent with growing evidence that both acquired and host genetics influence response to cancer therapy.

Authors
Davies, SM; Borowitz, MJ; Rosner, GL; Ritz, K; Devidas, M; Winick, N; Martin, PL; Bowman, P; Elliott, J; Willman, C; Das, S; Cook, EH; Relling, MV
MLA Citation
Davies, SM, Borowitz, MJ, Rosner, GL, Ritz, K, Devidas, M, Winick, N, Martin, PL, Bowman, P, Elliott, J, Willman, C, Das, S, Cook, EH, and Relling, MV. "Pharmacogenetics of minimal residual disease response in children with B-precursor acute lymphoblastic leukemia: a report from the Children's Oncology Group." Blood 111.6 (March 15, 2008): 2984-2990.
PMID
18182569
Source
pubmed
Published In
Blood
Volume
111
Issue
6
Publish Date
2008
Start Page
2984
End Page
2990
DOI
10.1182/blood-2007-09-114082

Incidence of hepatic veno-occlusive disease following stem cell transplantation: systematic review of literature from 1979-2007

Authors
Coppell, JA; Richardson, PG; Martin, PL; Iacobelli, M; Carreras, E; Ruutu, T; Barbui, T; Soiffer, R; Niederwieser, D
MLA Citation
Coppell, JA, Richardson, PG, Martin, PL, Iacobelli, M, Carreras, E, Ruutu, T, Barbui, T, Soiffer, R, and Niederwieser, D. "Incidence of hepatic veno-occlusive disease following stem cell transplantation: systematic review of literature from 1979-2007." March 2008.
Source
wos-lite
Published In
Bone Marrow Transplantation
Volume
41
Publish Date
2008
Start Page
S91
End Page
S91

Acute myelogenous leukemia associated with Ollier disease.

Ollier disease is a rare disorder characterized by the presence of multiple enchondromas and a propensity to develop malignancies. We report the case of a 7-year-old Caucasian male with Ollier disease who developed acute myelogenous leukemia (AML). This report describes a patient with Ollier disease and AML and may offer a clue into the genetic pathogenesis of these disorders.

Authors
White, MS; Martin, PL; McLean, TW
MLA Citation
White, MS, Martin, PL, and McLean, TW. "Acute myelogenous leukemia associated with Ollier disease." Pediatr Blood Cancer 50.3 (March 2008): 645-646.
PMID
16991136
Source
pubmed
Published In
Pediatric Blood & Cancer
Volume
50
Issue
3
Publish Date
2008
Start Page
645
End Page
646
DOI
10.1002/pbc.21050

THE USE, SAFETY, AND EFFICACY OF DACLIZUMAB FOR STEROID REFRACTORY GRAFT-VERSUS-HOST-DISEASE (GVHD) AFTER UNRELATED CORD BLOOD TRANSPLANTATION (UCBT)

Authors
Mohamed, A; Niedzwiecki, D; Baker, JH; Vinesett, R; Martin, PL; Driscoll, TA; Prasad, VK; Parikh, S; Kurtzberg, J; Szabolcs, P
MLA Citation
Mohamed, A, Niedzwiecki, D, Baker, JH, Vinesett, R, Martin, PL, Driscoll, TA, Prasad, VK, Parikh, S, Kurtzberg, J, and Szabolcs, P. "THE USE, SAFETY, AND EFFICACY OF DACLIZUMAB FOR STEROID REFRACTORY GRAFT-VERSUS-HOST-DISEASE (GVHD) AFTER UNRELATED CORD BLOOD TRANSPLANTATION (UCBT)." February 2008.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
14
Issue
2
Publish Date
2008
Start Page
77
End Page
77
DOI
10.1016/j.bbmt.2007.12.217

OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN PEDIATRIC PATIENTS WITH MYELODYSPLASTIC SYNDROME

Authors
Parikh, SH; Mendizabal, A; Martin, PL; Szabolcs, P; Prasad, VK; Driscoll, TA; Kurtzberg, J
MLA Citation
Parikh, SH, Mendizabal, A, Martin, PL, Szabolcs, P, Prasad, VK, Driscoll, TA, and Kurtzberg, J. "OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN PEDIATRIC PATIENTS WITH MYELODYSPLASTIC SYNDROME." February 2008.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
14
Issue
2
Publish Date
2008
Start Page
83
End Page
84
DOI
10.1016/j.bbmt.2007.12.233

OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN 159 YOUNG PATIENTS WITH PEROXISOMAL AND LYSOSOMAL STORAGE DISORDERS AT A SINGLE CENTER

Authors
Prasad, VK; Mendizabal, A; Parikh, SH; Szabolcs, P; Driscoll, T; Page, K; Lakshbinarayanan, S; Allison, J; Wood, S; Semmel, D; Escolar, ML; Martin, PL; Carter, S; Kurtzberg, J
MLA Citation
Prasad, VK, Mendizabal, A, Parikh, SH, Szabolcs, P, Driscoll, T, Page, K, Lakshbinarayanan, S, Allison, J, Wood, S, Semmel, D, Escolar, ML, Martin, PL, Carter, S, and Kurtzberg, J. "OUTCOMES OF UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION IN 159 YOUNG PATIENTS WITH PEROXISOMAL AND LYSOSOMAL STORAGE DISORDERS AT A SINGLE CENTER." February 2008.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
14
Issue
2
Publish Date
2008
Start Page
26
End Page
26
DOI
10.1016/j.bbmt.2007.12.071

UNRELATED DONOR UMBILICAL CORD BLOOD TRANSPLANT FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)

Authors
Kelly, SS; Shahlaee, A; Szabolcs, P; Driscoll, T; Eslin, D; Kurtzberg, J; Martin, PL
MLA Citation
Kelly, SS, Shahlaee, A, Szabolcs, P, Driscoll, T, Eslin, D, Kurtzberg, J, and Martin, PL. "UNRELATED DONOR UMBILICAL CORD BLOOD TRANSPLANT FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)." February 2008.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
14
Issue
2
Publish Date
2008
Start Page
75
End Page
75

Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation.

Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for chronic granulomatous disease (CGD), but many patients lack a suitably matched related donor. We report successful outcomes after mismatched, unrelated-donor umbilical cord blood transplantation (uUCBT) in two boys with X-linked CGD. Both patients experienced autologous recovery after first transplants, required second transplants to achieve durable donor engraftment, and are alive 27 and 15 months post-transplant. Both had invasive fungal disease and received granulocyte transfusions. In conclusion, uUCBT is effective in children with CGD, but immunosuppression in the conditioning regimen may need to be increased to decrease the risk of graft rejection.

Authors
Parikh, SH; Szabolcs, P; Prasad, VK; Lakshminarayanan, S; Martin, PL; Driscoll, TA; Kurtzberg, J
MLA Citation
Parikh, SH, Szabolcs, P, Prasad, VK, Lakshminarayanan, S, Martin, PL, Driscoll, TA, and Kurtzberg, J. "Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation." Pediatr Blood Cancer 49.7 (December 2007): 982-984.
PMID
17941061
Source
pubmed
Published In
Pediatric Blood & Cancer
Volume
49
Issue
7
Publish Date
2007
Start Page
982
End Page
984
DOI
10.1002/pbc.21365

Analysis of the cellular components of the graft and clinical characteristics of 159 children with lysosomal and peroxisomal disorders (LSD) undergoing unrelated umbilical cord blood transplantation at a single center

Authors
Prasad, VK; Medizabal, A; Parikh, SH; Szaboles, P; Driscoll, TA; Page, K; Lakshminarayan, S; Allison, J; Wood, S; Semmell, D; Escolar, ML; Martin, PL; Carter, S; Kurtzberg, J
MLA Citation
Prasad, VK, Medizabal, A, Parikh, SH, Szaboles, P, Driscoll, TA, Page, K, Lakshminarayan, S, Allison, J, Wood, S, Semmell, D, Escolar, ML, Martin, PL, Carter, S, and Kurtzberg, J. "Analysis of the cellular components of the graft and clinical characteristics of 159 children with lysosomal and peroxisomal disorders (LSD) undergoing unrelated umbilical cord blood transplantation at a single center." November 16, 2007.
Source
wos-lite
Published In
Blood
Volume
110
Issue
11
Publish Date
2007
Start Page
106A
End Page
106A

Antimetabolite therapy for lesser-risk B-lineage acute lymphoblastic leukemia of childhood: a report from Children's Oncology Group Study P9201.

Pediatric Oncology Group (POG) protocol 9201 enrolled children with lesser-risk B-lineage acute lymphoblastic leukemia (ALL) defined by age (1-9), white blood cell count (WBC) less than 50 x 10(9)/L (50,000/microL), DNA findings of trisomies 4 and 10 (or DNA index > 1.16), and lack of overt central nervous system (CNS) leukemia. After vincristine, prednisone, and asparaginase induction, 650 of 653 eligible patients attained remission (3 induction deaths) and received 6 courses of intravenous methotrexate (1 g/m(2)) with daily mercaptopurine. Weekly intramuscular methotrexate was added during maintenance; pulses of vincristine and prednisone were administered with periodic intrathecal chemotherapy. Treatment duration was 2.5 years. No alkylators, epipodophylotoxins, anthracyclines, or radiation were given. The 6-year event-free survival (EFS) was 86.6% with overall survival (OS) of 97.2%. Patients with less than 5% marrow blasts on induction day 15 had superior EFS. A difference not reaching conventional statistical significance (P = .068) was noted for superior outcomes in patients with trisomies of chromosomes 4 and 10 versus those lacking double trisomies. Sex, ethnicity, CNS status, and WBC were not predictive. This indicates the great majority of children with lesser-risk B-lineage ALL are curable without agents with substantial late effects.

Authors
Chauvenet, AR; Martin, PL; Devidas, M; Linda, SB; Bell, BA; Kurtzberg, J; Pullen, J; Pettenati, MJ; Carroll, AJ; Shuster, JJ; Camitta, B
MLA Citation
Chauvenet, AR, Martin, PL, Devidas, M, Linda, SB, Bell, BA, Kurtzberg, J, Pullen, J, Pettenati, MJ, Carroll, AJ, Shuster, JJ, and Camitta, B. "Antimetabolite therapy for lesser-risk B-lineage acute lymphoblastic leukemia of childhood: a report from Children's Oncology Group Study P9201." Blood 110.4 (August 15, 2007): 1105-1111.
PMID
17442849
Source
pubmed
Published In
Blood
Volume
110
Issue
4
Publish Date
2007
Start Page
1105
End Page
1111
DOI
10.1182/blood-2006-12-061689

Prospective Aspergillus galactomannan antigen testing in pediatric hematopoietic stem cell transplant recipients.

BACKGROUND: The galactomannan (GM) assay is an approved noninvasive test for detection of invasive aspergillosis (IA) that has been validated in adult patients with hematologic malignancies who are undergoing bone marrow transplantation. There have been few studies with this assay in pediatric patients, but early reports suggest that there may be differences in the performance such that false-positive GM tests in pediatric patients are more common than in adult patients. METHODS: We performed a prospective study in pediatric hematopoietic stem cell transplant recipients with twice-weekly sampling for GM detection during the highest risk periods of neutropenia and graft-versus-host disease. We analyzed 826 serum samples from 64 patients, including 15 serum samples from one patient diagnosed with probable IA according to defined criteria. RESULTS: Twenty of 811 samples tested positive on repeat testing (specificity, 97.5%; 95% CI: 96.2-98.4%) including samples from 8 of 63 patients without clinical evidence of IA according to study criteria (specificity, 87.3%; 95% CI: 76.9-93.4%). Eleven patients received piperacillin/tazobactam therapy, and 4 of the 11 patients had a positive assay result coinciding with the dates of piperacillin/tazobactam administration. When samples from these patients were excluded, specificity increased to 98.4% (95% CI: 97.2-99.1%) by sample and to 91.5% (95% CI: 81.6-96.3%) by patient. CONCLUSIONS: The GM assay holds promise for early, noninvasive diagnosis of IA in high-risk children and false-positive results were not common or unexplainable. This study supports further validation of this assay in a large-scale, pediatric-dedicated format.

Authors
Steinbach, WJ; Addison, RM; McLaughlin, L; Gerrald, Q; Martin, PL; Driscoll, T; Bentsen, C; Perfect, JR; Alexander, BD
MLA Citation
Steinbach, WJ, Addison, RM, McLaughlin, L, Gerrald, Q, Martin, PL, Driscoll, T, Bentsen, C, Perfect, JR, and Alexander, BD. "Prospective Aspergillus galactomannan antigen testing in pediatric hematopoietic stem cell transplant recipients." Pediatr Infect Dis J 26.7 (July 2007): 558-564.
PMID
17596794
Source
pubmed
Published In
Pediatric Infectious Disease Journal
Volume
26
Issue
7
Publish Date
2007
Start Page
558
End Page
564
DOI
10.1097/INF.0b013e3180616cbb

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marrow transplantation has been used to halt progression of the disease. However, many patients lack suitable HLA- matched related donors and must rely on unmatched donors for a source of stem cells. The purpose of this study was to evaluate outcomes of unrelated donor umbilical cord blood transplantation after chemotherapy-based myeloablative conditioning and retrospectively determine if baseline studies correlate and help predict outcome. Between November 22, 1996, and November 3, 2005, 12 boys with X-linked ALD who lacked HL- matched related donors were referred to Duke University Medical Center for transplantation. These children were conditioned with myeloablative therapy including busulfan, cyclophosphamide, and antithymocyte globulin before receiving umbilical cord-blood transplants from unrelated donors. Baseline studies of neurophysiologic, neuroimaging, and neurodevelopmental status were performed and patients were subsequently evaluated for survival, engraftment, graft-versus-host disease, and neurodevelopmental outcomes. A substudy evaluated whether baseline neuroimaging and neurophysiologic studies correlated with cognitive and motor function and if these studies were predictive of posttransplantation outcomes. The umbilical cord blood grafts had normal levels of very long chain fatty acids. They delivered a median of 6.98 x 10(7) nucleated cells per kilogram of recipient body weight and were discordant for up to 4 of 6 HLA markers. Neutrophil engraftment occurred at a median of 22.9 days after transplantation. Three patients had grade II-IV acute graft-versus-host disease; 2 had extensive chronic graft-versus-host disease. Cumulative incidence of overall survival of the group at 6 months is 66.7% (95% confidence interval 39.9-93.3%). Median follow-up was 3.3 years (range 12 days to 6.3 years). As previously reported with bone marrow transplantation, symptomatic patients faired poorly with lower survival and rapid deterioration of neurologic function. This study included 3 patients transplanted at a very young age (2.6-3.5 years) before the onset of clinical symptoms who continue to develop at a normal rate for 3-5 years posttransplant. Although baseline Loes scores correlated with cognitive and motor outcome, neurophysiologic studies failed to show statistically significant differences. Transplantation of boys with X-linked ALD using partial HLA-matched umbilical cord blood yields similar results to those previously reported after bone marrow transplantation. Superior outcomes were seen in neurologically asymptomatic boys less than 3.5 years of age at the time of transplantation. Baseline Loes scores were a strong predictor of cognitive and motor outcome.

Authors
Beam, D; Poe, MD; Provenzale, JM; Szabolcs, P; Martin, PL; Prasad, V; Parikh, S; Driscoll, T; Mukundan, S; Kurtzberg, J; Escolar, ML
MLA Citation
Beam, D, Poe, MD, Provenzale, JM, Szabolcs, P, Martin, PL, Prasad, V, Parikh, S, Driscoll, T, Mukundan, S, Kurtzberg, J, and Escolar, ML. "Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy." Biol Blood Marrow Transplant 13.6 (June 2007): 665-674.
PMID
17531776
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
13
Issue
6
Publish Date
2007
Start Page
665
End Page
674
DOI
10.1016/j.bbmt.2007.01.082

Outcomes of unrelated umbilical cord blood transplantation in pediatric patients with myelodysplastic syndrome

Authors
Parikh, SH; Mendizabal, A; Martin, PL; Szaboles, P; Prasad, VK; Driscoll, TA; Kurtzberg, J
MLA Citation
Parikh, SH, Mendizabal, A, Martin, PL, Szaboles, P, Prasad, VK, Driscoll, TA, and Kurtzberg, J. "Outcomes of unrelated umbilical cord blood transplantation in pediatric patients with myelodysplastic syndrome." February 2007.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
13
Issue
2
Publish Date
2007
Start Page
64
End Page
65

Outcomes of matched related donor bone marrow transplantation in pediatric patients with myelodysplastic syndrome

Authors
Parikh, SH; Martin, PL; Prasad, VK; Szaboles, P; Driscoll, TA; Kurtzbeig, J
MLA Citation
Parikh, SH, Martin, PL, Prasad, VK, Szaboles, P, Driscoll, TA, and Kurtzbeig, J. "Outcomes of matched related donor bone marrow transplantation in pediatric patients with myelodysplastic syndrome." February 2007.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
13
Issue
2
Publish Date
2007
Start Page
64
End Page
64

Outcomes of pediatric patients transplanted a second time after experiencing graft failure with unrelated donor umbilical cord blood transplantation

Authors
Baker, JH; Martin, PL; Driscoll, T; Szabolcs, P; Allison, J; Gurganus, K; Ciocci, G; Parikh, S; Prasad, V; Kurtzberg, J
MLA Citation
Baker, JH, Martin, PL, Driscoll, T, Szabolcs, P, Allison, J, Gurganus, K, Ciocci, G, Parikh, S, Prasad, V, and Kurtzberg, J. "Outcomes of pediatric patients transplanted a second time after experiencing graft failure with unrelated donor umbilical cord blood transplantation." February 2007.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
13
Issue
2
Publish Date
2007
Start Page
137
End Page
137
DOI
10.1016/j.bbmt.2007.01.007

Prognostic significance of minimal residual disease (MRD) in childhood B-precursor ALL and its relation to other risk factors. A Children's Oncology Group (COG) study.

Authors
Borowitz, MJ; Devidas, M; Bowman, WP; Carroll, WL; Chen, I-M; Harvey, R; Hunger, S; Martin, PL; Pullen, J; Viswanatha, D; Willman, CL; Winick, N; Camitta, B
MLA Citation
Borowitz, MJ, Devidas, M, Bowman, WP, Carroll, WL, Chen, I-M, Harvey, R, Hunger, S, Martin, PL, Pullen, J, Viswanatha, D, Willman, CL, Winick, N, and Camitta, B. "Prognostic significance of minimal residual disease (MRD) in childhood B-precursor ALL and its relation to other risk factors. A Children's Oncology Group (COG) study." November 16, 2006.
Source
wos-lite
Published In
Blood
Volume
108
Issue
11
Publish Date
2006
Start Page
69A
End Page
69A

Acute graft versus host disease (GVHD) is increased in patients receiving cyclosporine/celicept as frontline prophylaxis against GVKD in pediatric patients undergoing allogeneic stem cell transplantation.

Authors
Lakshminarayanan, S; Szabolcs, P; Prasad, V; Parikh, S; Wood, S; Ciocci, G; Stafford, L; Boyd, L; Driscoll, T; Martin, PL; Kurtzberg, J
MLA Citation
Lakshminarayanan, S, Szabolcs, P, Prasad, V, Parikh, S, Wood, S, Ciocci, G, Stafford, L, Boyd, L, Driscoll, T, Martin, PL, and Kurtzberg, J. "Acute graft versus host disease (GVHD) is increased in patients receiving cyclosporine/celicept as frontline prophylaxis against GVKD in pediatric patients undergoing allogeneic stem cell transplantation." November 16, 2006.
Source
wos-lite
Published In
Blood
Volume
108
Issue
11
Publish Date
2006
Start Page
820A
End Page
820A

Melphalan containing cytoreduction results in a good overall survival (OS) in pediatric patients with relapse or refractory AML undergoing unrelated umbilical cord blood transplantation (UCBT).

Authors
Prasad, VK; Wu, J; Martin, PL; Driscoll, TA; Parikh, SH; Semmel, D; Moffet, J; Stafford, L; Shonkwiler, A; Carter, S; Szabolcs, P; Kurtzberg, J
MLA Citation
Prasad, VK, Wu, J, Martin, PL, Driscoll, TA, Parikh, SH, Semmel, D, Moffet, J, Stafford, L, Shonkwiler, A, Carter, S, Szabolcs, P, and Kurtzberg, J. "Melphalan containing cytoreduction results in a good overall survival (OS) in pediatric patients with relapse or refractory AML undergoing unrelated umbilical cord blood transplantation (UCBT)." November 16, 2006.
Source
wos-lite
Published In
Blood
Volume
108
Issue
11
Publish Date
2006
Start Page
899A
End Page
899A

Defibrotide (DF) for the treatment of severe veno-occlusive disease (sVOD) and multi-organ failure (MOF) post SCT: Final results of a multi-center, randomized, dose-finding trial.

Authors
Richardson, P; Soiffer, RJ; Antin, JH; Jin, Z; Kurtzberg, J; Martin, PL; Steinbach, G; Murray, KF; Vogelsang, GB; Chen, A; Krishnan, A; Kernan, NA; Avigan, D; Spitzer, TR; Warren, D; Revta, C; Wei, LJ; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, P, Soiffer, RJ, Antin, JH, Jin, Z, Kurtzberg, J, Martin, PL, Steinbach, G, Murray, KF, Vogelsang, GB, Chen, A, Krishnan, A, Kernan, NA, Avigan, D, Spitzer, TR, Warren, D, Revta, C, Wei, LJ, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide (DF) for the treatment of severe veno-occlusive disease (sVOD) and multi-organ failure (MOF) post SCT: Final results of a multi-center, randomized, dose-finding trial." November 16, 2006.
Source
wos-lite
Published In
Blood
Volume
108
Issue
11
Publish Date
2006
Start Page
17A
End Page
18A

The addition of fludarabine to a conventional Busulfan/Melphalan/Anti-Thymocyte globulin (Flu/Bu/Mel/ATG) preparative regimen increased engraftment without additional toxicity.

Authors
Lakshminarayanan, S; Mendizabal, A; Prasad, V; Parikh, S; Szabolcs, P; Driscoll, T; Schweitzer, M; Kassmann, B; Paden, M; Robinette, D; Martin, PL; Kurtzberg, J
MLA Citation
Lakshminarayanan, S, Mendizabal, A, Prasad, V, Parikh, S, Szabolcs, P, Driscoll, T, Schweitzer, M, Kassmann, B, Paden, M, Robinette, D, Martin, PL, and Kurtzberg, J. "The addition of fludarabine to a conventional Busulfan/Melphalan/Anti-Thymocyte globulin (Flu/Bu/Mel/ATG) preparative regimen increased engraftment without additional toxicity." BLOOD 108.11 (November 16, 2006): 833A-834A.
Source
wos-lite
Published In
Blood
Volume
108
Issue
11
Publish Date
2006
Start Page
833A
End Page
834A

To stop or not to stop: how much support should be provided to mechanically ventilated pediatric bone marrow and stem cell transplant patients?

Every publication to date reporting the outcome of intensive care support for pediatric SCT patients must be viewed with caution because all are single-institution, retrospective reports. Nevertheless, some of the conclusions made by these investigators appear to be clinically relevant. First, an SCT patient who requires intensive care support does not automatically have a dismal chance of survival. Survival rates in recent reports range from 15% to 36%, which is reasonable when the overall post-transplant survival rate for non-ICU patients may be only 50%. Second, adverse risk factors differ from center to center, likely due to the wide variation in patient populations, donor source, and transplant preparation regimens. Third, MSOF is a consistent adverse risk factor for survival. An additional conclusion that can be drawn from the data presented in this article is that patients who do not show significant, objective improvement by the second week of PICU care are unlikely to survive. The limitation or withdrawal of life-sustaining medical support should be recommended to the patient, the patient's family, and the patient's doctors. Although there are no predictive models that are 100% reliable for these clinical situations, in the author's experience, most families and physicians view critical care support beyond 2 weeks, in the absence of clinical improvement, as futile care. It is clear that better data are needed in the form of prospective, multi-institutional studies that include the therapeutic efficacy of interventions such as high-frequency oscillatory ventilation, continuous venovenous hemodialysis, early use of noninvasive ventilation (ie, noninvasive positive pressure ventilation), the use of biologic agents to decrease inflammation, the impact of new antifungal medications, and lung-protective ventilation with permissive hypercapnia. Of these potential therapies, the author is aware of only one multi-institutional study involving continuous venovenous hemodialysis at this time.

Authors
Martin, PL
MLA Citation
Martin, PL. "To stop or not to stop: how much support should be provided to mechanically ventilated pediatric bone marrow and stem cell transplant patients?." Respir Care Clin N Am 12.3 (September 2006): 403-419. (Review)
PMID
16952801
Source
pubmed
Published In
Respiratory care clinics of North America
Volume
12
Issue
3
Publish Date
2006
Start Page
403
End Page
419
DOI
10.1016/j.rcc.2006.06.002

Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.

The Cord Blood Transplantation Study (COBLT), sponsored by the National Heart, Lung, and Blood Institute, is a phase II multicenter study designed to evaluate the use of cord blood in allogeneic transplantation. In this report, we evaluated the outcomes of cord blood transplantation in 69 patients with lysosomal and peroxisomal storage diseases. Patients with mucopolysaccharidoses I to III, mucolipidoses (ML) II (n = 36), adrenoleukodystrophy (n = 8), metachromatic leukodystrophy (n = 6), Krabbe disease (n = 16), and Tay-Sachs disease (n = 3) were enrolled between August 1999 and June 2004. All patients received the same preparative regimen, graft-versus-host disease (GVHD) prophylaxis, and supportive care. End points included survival, engraftment, GVHD, and toxicity. Sixty-nine patients (64% men; 81% white) with a median age of 1.8 years underwent transplantation with a median cell dose of 8.7 x 10(7)/kg. One-year survival was 72% (95% confidence interval, 61%-83%). The cumulative incidence of neutrophil engraftment by day 42 was 78% (95% confidence interval, 67%-87%) at a median of 25 days. Grade II to IV acute GVHD occurred in 36% of patients. Cord blood donors are readily available for rapid transplantation. Cord blood transplantation should be considered as frontline therapy for young patients with lysosomal and peroxisomal storage diseases.

Authors
Martin, PL; Carter, SL; Kernan, NA; Sahdev, I; Wall, D; Pietryga, D; Wagner, JE; Kurtzberg, J
MLA Citation
Martin, PL, Carter, SL, Kernan, NA, Sahdev, I, Wall, D, Pietryga, D, Wagner, JE, and Kurtzberg, J. "Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases." Biol Blood Marrow Transplant 12.2 (February 2006): 184-194.
PMID
16443516
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
184
End Page
194
DOI
10.1016/j.bbmt.2005.09.016

Busulfan/melphalan is an effective and well tolerated conditioning regimen for pediatric AML and MDS patients receiving a matched sibling bmt

Authors
Martin, PL; Driscoll, TA; Szabolcs, P; Parikh, SH; Prasad, VK; Kurtzberg, J
MLA Citation
Martin, PL, Driscoll, TA, Szabolcs, P, Parikh, SH, Prasad, VK, and Kurtzberg, J. "Busulfan/melphalan is an effective and well tolerated conditioning regimen for pediatric AML and MDS patients receiving a matched sibling bmt." February 2006.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
127
End Page
127
DOI
10.1016/j.bbmt.2005.11.389

Single center experience of unrelated umbilical cord blood transplantation (UCBDT) for acute myeloid leukemia (AML) in 97 pediatric patients: Impact of disease status and cytoreductive regimen

Authors
Prasad, VK; Wu, J; Parikh, SH; Driscoll, TA; Szabolcs, P; Martin, PL; Carter, S; Kurtzberg, J
MLA Citation
Prasad, VK, Wu, J, Parikh, SH, Driscoll, TA, Szabolcs, P, Martin, PL, Carter, S, and Kurtzberg, J. "Single center experience of unrelated umbilical cord blood transplantation (UCBDT) for acute myeloid leukemia (AML) in 97 pediatric patients: Impact of disease status and cytoreductive regimen." February 2006.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
124
End Page
124
DOI
10.1016/j.bbmt.2005.11.381

Patient controlled analgesia by proxy

Authors
Guess, C; Frey, MA; Martin, PL; Kurtzberg, J
MLA Citation
Guess, C, Frey, MA, Martin, PL, and Kurtzberg, J. "Patient controlled analgesia by proxy." February 2006.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
159
End Page
160
DOI
10.1016/j.bbmt.2005.11.495

Cellcept/cyclosporine as prophylaxis against graft- versus-host disease in pediatric patients undergoing allogeneic stem cell transplantation

Authors
Lakshminarayanan, S; Martin, PL; Szabolcs, P; Driscoll, T; Parikh, S; Prasad, V; Kurtzberg, J
MLA Citation
Lakshminarayanan, S, Martin, PL, Szabolcs, P, Driscoll, T, Parikh, S, Prasad, V, and Kurtzberg, J. "Cellcept/cyclosporine as prophylaxis against graft- versus-host disease in pediatric patients undergoing allogeneic stem cell transplantation." February 2006.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
67
End Page
68
DOI
10.1016/j.bbmt.2005.11.212

Vend occulsive disease: A potentially life threatening complication of hematopoietic stem cell transplantation

Authors
Summers, E; Edmisten, J; Martin, PL; Frey, M; Kurtzberg, J
MLA Citation
Summers, E, Edmisten, J, Martin, PL, Frey, M, and Kurtzberg, J. "Vend occulsive disease: A potentially life threatening complication of hematopoietic stem cell transplantation." February 2006.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
12
Issue
2
Publish Date
2006
Start Page
161
End Page
161
DOI
10.1016/j.bbmt.2005.11.500

Defibrotide (DF) for the treatment of Veno-Occlusive Disease (VOD) and multi-system organ failure (MOF) post SCT: Analysis of response and survival according to degree and type of MOV.

Authors
Richardson, PG; Soiffer, RJ; Antin, JH; Jin, Z; Kurtzberg, J; Martin, PL; Hockenbery, D; Murray, KF; Vogelsang, GB; Chen, A; Krishnan, A; Kernan, NA; Avigan, D; Spitzer, TR; Warren, D; Momtaz, P; Revta, C; Wei, LJ; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, PG, Soiffer, RJ, Antin, JH, Jin, Z, Kurtzberg, J, Martin, PL, Hockenbery, D, Murray, KF, Vogelsang, GB, Chen, A, Krishnan, A, Kernan, NA, Avigan, D, Spitzer, TR, Warren, D, Momtaz, P, Revta, C, Wei, LJ, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide (DF) for the treatment of Veno-Occlusive Disease (VOD) and multi-system organ failure (MOF) post SCT: Analysis of response and survival according to degree and type of MOV." November 16, 2005.
Source
wos-lite
Published In
Blood
Volume
106
Issue
11
Publish Date
2005
Start Page
120A
End Page
120A

Comparison of diagnostic and relapse flow cytometry phenotypes in childhood acute lymphoblastic leukemia: implications for residual disease detection: a report from the children's oncology group.

BACKGROUND: Flow cytometric analysis of minimal residual disease (MRD) depends on detecting phenotypically abnormal populations. However, little is known about how phenotypic shifts between diagnosis and relapse affect MRD detection in childhood acute lymphoid leukemia (ALL). METHODS: We compared diagnostic and relapse bone marrow specimens in 42 children with precursor B-ALL studied with the two-tube panel CD19-APC/CD45-PerCP/CD10-PE/CD20-FITC and CD19-APC/CD45-PerCP/CD9-PE/CD34-FITC. RESULTS: At least 29 cases had phenotypic shifts of intensity or coefficient of variation of distribution of one or more markers. Shifts were complex and could not be explained by change in maturation stage. In the majority of cases MRD populations more closely resembled the diagnostic than the relapse specimen. In 6 of 7 MRD negative cases we did not identify an abnormal population that resembled diagnosis or relapse. In the remaining case, in which CD34 and CD10 were lost between diagnosis and relapse, it is possible that we could have missed an MRD population resembling relapse. CONCLUSIONS: Phenotypic shifts are common, but do not affect MRD recognition. At most 1 of 42 cases might have harbored an abnormal population undetected because of shift. However, MRD analysis with rigid gating (looking strictly for abnormal phenotypes at diagnosis) might have missed many positive cases, 8 of 22 (36%) in this series.

Authors
Borowitz, MJ; Pullen, DJ; Winick, N; Martin, PL; Bowman, WP; Camitta, B
MLA Citation
Borowitz, MJ, Pullen, DJ, Winick, N, Martin, PL, Bowman, WP, and Camitta, B. "Comparison of diagnostic and relapse flow cytometry phenotypes in childhood acute lymphoblastic leukemia: implications for residual disease detection: a report from the children's oncology group." Cytometry B Clin Cytom 68.1 (November 2005): 18-24.
PMID
16184615
Source
pubmed
Published In
Cytometry
Volume
68
Issue
1
Publish Date
2005
Start Page
18
End Page
24
DOI
10.1002/cyto.b.20071

Management of cytomegalovirus in the pediatric bone marrow/stem cell transplantation population

Authors
Stanton, TJ; Martin, PL; Talbert, G
MLA Citation
Stanton, TJ, Martin, PL, and Talbert, G. "Management of cytomegalovirus in the pediatric bone marrow/stem cell transplantation population." February 2005.
Source
crossref
Published In
Biology of Blood and Marrow Transplantation
Volume
11
Issue
2
Publish Date
2005
Start Page
104
End Page
104
DOI
10.1016/j.bbmt.2004.12.314

Treatment of pediatric patients with Sanfilippo syndrome (MPS IIIA and IIIB) with unrelated umbilical cord blood transplantation

Authors
Kurtzberg, J; Szabolcs, P; Wood, S; Ciocci, G; Driscoll, T; Prasad, V; Parikh, S; Martin, PL; Allison, J; Escolar, ML
MLA Citation
Kurtzberg, J, Szabolcs, P, Wood, S, Ciocci, G, Driscoll, T, Prasad, V, Parikh, S, Martin, PL, Allison, J, and Escolar, ML. "Treatment of pediatric patients with Sanfilippo syndrome (MPS IIIA and IIIB) with unrelated umbilical cord blood transplantation." February 2005.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
11
Issue
2
Publish Date
2005
Start Page
83
End Page
84
DOI
10.1016/j.bbmt.2004.12.246

Infection in the pediatric patient after bone marrow or stem cell transplantation: Nursing considerations

Authors
Kokoszka, AK; Frey, MA; Talbert, G; Martin, PL
MLA Citation
Kokoszka, AK, Frey, MA, Talbert, G, and Martin, PL. "Infection in the pediatric patient after bone marrow or stem cell transplantation: Nursing considerations." February 2005.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
11
Issue
2
Publish Date
2005
Start Page
100
End Page
100
DOI
10.1016/j.bbmt.2004.12.297

Outcomes of unrelated umbilical cord blood transplantation in pediatric patients with myelodysplastic syndrome

Authors
Parikh, SH; Martin, PL; Szabolcs, P; Ann, S; Prasad, V; Driscoll, T; Kurtzberg, J
MLA Citation
Parikh, SH, Martin, PL, Szabolcs, P, Ann, S, Prasad, V, Driscoll, T, and Kurtzberg, J. "Outcomes of unrelated umbilical cord blood transplantation in pediatric patients with myelodysplastic syndrome." February 2005.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
11
Issue
2
Publish Date
2005
Start Page
80
End Page
80
DOI
10.1016/j.bbmt.2004.12.236

Pharmacogenetics of minimal residual disease response in children with acute lymphoblastic leukemia (ALL).

Authors
Davies, SM; Borowitz, M; Devidas, M; Winick, N; Martin, PL; Bowman, P; Elliott, J; Linda, S; Cook, EH; Relling, MV
MLA Citation
Davies, SM, Borowitz, M, Devidas, M, Winick, N, Martin, PL, Bowman, P, Elliott, J, Linda, S, Cook, EH, and Relling, MV. "Pharmacogenetics of minimal residual disease response in children with acute lymphoblastic leukemia (ALL)." November 16, 2004.
Source
wos-lite
Published In
Blood
Volume
104
Issue
11
Publish Date
2004
Start Page
132A
End Page
132A

Comparison of diagnostic and relapse flow cytometry phenotypes in childhood ALL: Implications for residual disease (MRD) detection

Authors
Borowitz, MJ; Pullen, DJ; Winick, N; Martin, PL; Bowman, WP; Camitta, B
MLA Citation
Borowitz, MJ, Pullen, DJ, Winick, N, Martin, PL, Bowman, WP, and Camitta, B. "Comparison of diagnostic and relapse flow cytometry phenotypes in childhood ALL: Implications for residual disease (MRD) detection." November 16, 2004.
Source
wos-lite
Published In
Blood
Volume
104
Issue
11
Publish Date
2004
Start Page
310A
End Page
310A

Defibrotide (DF) for the treatment of severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post SCT: Final results of a phase II, multicenter, randomized study and preliminary analyses of surrogate markers and ultrasound findings

Authors
Richardson, PG; Soiffer, RJ; Antin, JH; Voss, SD; Jin, Z; Kurtzberg, J; Martin, PL; Hockenbery, D; Murray, KF; Vogelsang, GB; Chen, A; Krishnan, A; Kernan, NA; Avigan, D; Spitzer, TR; Iannone, R; Giralt, S; Warren, D; Momtaz, P; Bradwin, G; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, PG, Soiffer, RJ, Antin, JH, Voss, SD, Jin, Z, Kurtzberg, J, Martin, PL, Hockenbery, D, Murray, KF, Vogelsang, GB, Chen, A, Krishnan, A, Kernan, NA, Avigan, D, Spitzer, TR, Iannone, R, Giralt, S, Warren, D, Momtaz, P, Bradwin, G, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide (DF) for the treatment of severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post SCT: Final results of a phase II, multicenter, randomized study and preliminary analyses of surrogate markers and ultrasound findings." November 16, 2004.
Source
wos-lite
Published In
Blood
Volume
104
Issue
11
Publish Date
2004
Start Page
103A
End Page
104A

The incidence of testicular recurrence in boys with acute leukemia treated with total body and testicular irradiation and stem cell transplantation.

BACKGROUND: The incidence of testicular recurrence of childhood acute leukemia after total body irradiation (TBI) in conjunction with stem cell transplantation (SCT) has been reported to be as high as 24%. The authors studied the incidence of testicular failure in a large series of male patients who underwent SCT using either TBI and a testicular irradiation boost or chemotherapy alone. METHODS: One hundred thirty-one boys with either acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL) were treated with SCT with either TBI with testicular boost (n = 94 patients), TBI without testicular boost (n = 1 patient), or chemotherapy alone (n = 36 patients) between 1991 and 1999. RESULTS: The median follow-up was 26.5 months (range, 0.6-99.5 months) from the date of bone marrow infusion. Two patients in the study had a primary testicular failure after TBI with testicular boost followed by an umbilical cord blood transplantation. The first patient had ALL, did not engraft, and was rescued with autologous cells. He developed disease in the testicle 15 months afterward and subsequently died. The second patient had Philadelphia chromosome-positive ALL and developed a testicular recurrence 26 months after SCT. He was treated with orchiectomy, further testicular irradiation, and chemotherapy and remained in complete remission > 3 year after his failure. The incidence of testicular failure in boys who received TBI and testicular irradiation who survived > or = 1 year was 4.2%. There were no primary testicular failures reported in boys who received chemotherapy alone. CONCLUSIONS: Boys with AML or ALL had a low incidence of primary testicular failure when they were treated with TBI plus a testicular boost or with chemotherapy alone.

Authors
Quaranta, BP; Halperin, EC; Kurtzberg, J; Clough, R; Martin, PL
MLA Citation
Quaranta, BP, Halperin, EC, Kurtzberg, J, Clough, R, and Martin, PL. "The incidence of testicular recurrence in boys with acute leukemia treated with total body and testicular irradiation and stem cell transplantation." Cancer 101.4 (August 15, 2004): 845-850.
PMID
15305418
Source
pubmed
Published In
Cancer
Volume
101
Issue
4
Publish Date
2004
Start Page
845
End Page
850
DOI
10.1002/cncr.20413

Unrelated umbilical cord blood transplantation for an infant with beta-thalassemia major.

BACKGROUND: beta-thalassemia major, one of the most prevalent hemoglobinopathies throughout the world, can be cured by allogeneic stem cell transplantation therapy. Many patients, however, lack a suitably matched related donor. Unrelated umbilical cord blood can be used as an alternative stem cell source for some of these patients. This report describes the successful transplantation of a 2-month-old infant with beta-thalassemia major using partially HLA-matched unrelated umbilical cord blood. METHODS: After cytoreduction with busulfan, cyclophosphamide, and antithymocyte globulin (ATG), the patient underwent transplantation at the age of 2 months with a 4/6 HLA matching umbilical cord blood unit from an unrelated donor. RESULTS: The patient engrafted promptly with 100% donor chimerism. His only major complication was an autoimmune hemolytic anemia that resolved 2 years after transplantation. He is currently surviving, event-free, 5 years after transplantation with normal growth and cognitive development and full donor chimerism without evidence of beta-thalassemia. CONCLUSIONS: Umbilical cord blood transplantation from related and unrelated donors should be considered for patients with beta-thalassemia major who lack traditional bone marrow donors. As most newborns undergo screening for hemoglobinopathies, those with disease could be transplanted early in life before experiencing the morbidity and mortality caused by transfusion therapy, alloimmunization, and iron overload, increasing the likelihood of successful transplantation therapy.

Authors
Hall, JG; Martin, PL; Wood, S; Kurtzberg, J
MLA Citation
Hall, JG, Martin, PL, Wood, S, and Kurtzberg, J. "Unrelated umbilical cord blood transplantation for an infant with beta-thalassemia major." J Pediatr Hematol Oncol 26.6 (June 2004): 382-385.
PMID
15167353
Source
pubmed
Published In
Journal of Pediatric Hematology/Oncology
Volume
26
Issue
6
Publish Date
2004
Start Page
382
End Page
385

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.

BACKGROUND: Hurler's syndrome (the most severe form of mucopolysaccharidosis type I) causes progressive deterioration of the central nervous system and death in childhood. Allogeneic bone marrow transplantation before the age of two years halts disease progression and prolongs life, but many children lack a bone marrow donor. We investigated the feasibility of using cord-blood transplants from unrelated donors and a myeloablative preparative regimen that did not involve total-body irradiation in young children with Hurler's syndrome. METHODS: Between December 1995 and October 2002, 20 consecutive children with Hurler's syndrome received busulfan, cyclophosphamide, and antithymocyte globulin before receiving cord-blood transplants from unrelated donors. The children were subsequently evaluated for engraftment, adverse effects, and effects on disease symptoms. RESULTS: Cord-blood donors had normal alpha-L-iduronidase activity (mean number of cells, 10.53x10(7) per kilogram of body weight) and were discordant for up to three of six HLA markers. Neutrophil engraftment occurred a median of 24 days after transplantation. Five patients had grade II or grade III acute graft-versus-host disease; none had extensive chronic graft-versus-host disease. Seventeen of the 20 children were alive a median of 905 days after transplantation, with complete donor chimerism and normal peripheral-blood alpha-L-iduronidase activity (event-free survival rate, 85 percent). Transplantation improved neurocognitive performance and decreased somatic features of Hurler's syndrome. CONCLUSIONS: Cord blood from unrelated donors appears to be an excellent source of stem cells for transplantation in patients with Hurler's syndrome. Sustained engraftment can be achieved without total-body irradiation. Cord-blood transplantation favorably altered the natural history of Hurler's syndrome and thus may be important to consider in young children with this form of the disease.

Authors
Staba, SL; Escolar, ML; Poe, M; Kim, Y; Martin, PL; Szabolcs, P; Allison-Thacker, J; Wood, S; Wenger, DA; Rubinstein, P; Hopwood, JJ; Krivit, W; Kurtzberg, J
MLA Citation
Staba, SL, Escolar, ML, Poe, M, Kim, Y, Martin, PL, Szabolcs, P, Allison-Thacker, J, Wood, S, Wenger, DA, Rubinstein, P, Hopwood, JJ, Krivit, W, and Kurtzberg, J. "Cord-blood transplants from unrelated donors in patients with Hurler's syndrome." N Engl J Med 350.19 (May 6, 2004): 1960-1969.
PMID
15128896
Source
pubmed
Published In
The New England journal of medicine
Volume
350
Issue
19
Publish Date
2004
Start Page
1960
End Page
1969
DOI
10.1056/NEJMoa032613

Unrelated umbilical cord blood transplantation for familial erythrophagocytic lymphohistiocytosis

Authors
Archambault, BL; Driscoll, TA; Stafford, LA; Szabolcs, P; Kurtzberg, J; Martin, PL
MLA Citation
Archambault, BL, Driscoll, TA, Stafford, LA, Szabolcs, P, Kurtzberg, J, and Martin, PL. "Unrelated umbilical cord blood transplantation for familial erythrophagocytic lymphohistiocytosis." February 2004.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
10
Issue
2
Publish Date
2004
Start Page
79
End Page
80
DOI
10.1016/j.bbmt.2003.12.132

Second hematopoietic stem cell transplant using unrelated donor umbilical cord blood cures 50% of pediatric patients with leukemia relapsing after prior allogeneic transplant from a matched related sibling

Authors
Staba, SL; Szabolcs, P; Driscoll, T; Martin, PL; Kurtzberg, J
MLA Citation
Staba, SL, Szabolcs, P, Driscoll, T, Martin, PL, and Kurtzberg, J. "Second hematopoietic stem cell transplant using unrelated donor umbilical cord blood cures 50% of pediatric patients with leukemia relapsing after prior allogeneic transplant from a matched related sibling." February 2004.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
10
Issue
2
Publish Date
2004
Start Page
38
End Page
38
DOI
10.1016/j.bbmt.2003.12.179

Transplantation of boys with X-linked adrenoleukodystrophy with unrelated-donor, partially HLA-mismatched banked umbilical cord blood

Authors
Allison, J; Martin, PL; Szabolcs, P; Driscoll, T; Parikh, S; Wood, S; Kurtzberg, J
MLA Citation
Allison, J, Martin, PL, Szabolcs, P, Driscoll, T, Parikh, S, Wood, S, and Kurtzberg, J. "Transplantation of boys with X-linked adrenoleukodystrophy with unrelated-donor, partially HLA-mismatched banked umbilical cord blood." February 2004.
Source
wos-lite
Published In
Biology of Blood and Marrow Transplantation
Volume
10
Issue
2
Publish Date
2004
Start Page
74
End Page
74
DOI
10.1016/j.bbmt.2003.12.100

Fatal Scopulariopsis brevicaulis infection in a paediatric stem-cell transplant patient treated with voriconazole and caspofungin and a review of Scopulariopsis infections in immunocompromised patients.

Authors
Steinbach, WJ; Schell, WA; Miller, JL; Perfect, JR; Martin, PL
MLA Citation
Steinbach, WJ, Schell, WA, Miller, JL, Perfect, JR, and Martin, PL. "Fatal Scopulariopsis brevicaulis infection in a paediatric stem-cell transplant patient treated with voriconazole and caspofungin and a review of Scopulariopsis infections in immunocompromised patients." J Infect 48.1 (January 2004): 112-116. (Review)
PMID
14667801
Source
pubmed
Published In
Journal of Infection
Volume
48
Issue
1
Publish Date
2004
Start Page
112
End Page
116

Unrelated donor umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

Authors
Kurtzberg, J; Szabolcs, P; Martin, PL; Driscoll, T; Kelly, T; Ciocci, G; Escolar, ML
MLA Citation
Kurtzberg, J, Szabolcs, P, Martin, PL, Driscoll, T, Kelly, T, Ciocci, G, and Escolar, ML. "Unrelated donor umbilical cord blood transplantation for X-linked adrenoleukodystrophy." November 16, 2003.
Source
wos-lite
Published In
Blood
Volume
102
Issue
11
Publish Date
2003
Start Page
479B
End Page
479B

Defibrotide (DF) is effective in the treatment of severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT): Results of a phase II, multicenter, randomized study.

Authors
Richardson, P; Soiffer, RJ; Antin, JH; Jin, Z; Vredenburgh, JJ; Kurtzberg, J; Martin, PL; Hockenbery, D; Steinbach, G; Murray, KF; Vogelsang, GB; Chen, A; Krishnan, A; Prasad, VK; Warren, DL; Momtaz, P; Batchelder, A; Wei, LJ; Rifai, N; Bradwin, G; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, P, Soiffer, RJ, Antin, JH, Jin, Z, Vredenburgh, JJ, Kurtzberg, J, Martin, PL, Hockenbery, D, Steinbach, G, Murray, KF, Vogelsang, GB, Chen, A, Krishnan, A, Prasad, VK, Warren, DL, Momtaz, P, Batchelder, A, Wei, LJ, Rifai, N, Bradwin, G, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide (DF) is effective in the treatment of severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT): Results of a phase II, multicenter, randomized study." November 16, 2003.
Source
wos-lite
Published In
Blood
Volume
102
Issue
11
Publish Date
2003
Start Page
193A
End Page
193A

Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.

Allogeneic stem cell transplantation with umbilical cord blood (UCB) cells is limited by the cell dose a single unit provides recipients. Ex vivo expansion is one strategy to increase the number of cells available for transplantation. Aastrom Biosciences developed an automated continuous perfusion culture device for expansion of hematopoietic stem cells (HSCs). Cells are expanded in media supplemented with fetal bovine serum, horse serum, PIXY321, flt-3 ligand, and erythropoietin. We performed a phase 1 trial augmenting conventional UCB transplants with ex vivo-expanded cells. The 28 patients were enrolled on the trial between October 8, 1997 and September 30, 1998. UCB cells were expanded in the device, then administered as a boost to the conventional graft on posttransplantation day 12. While expansion of total cells and colony-forming units (CFUs) occurred in all cases, the magnitude of expansion varied considerably. The median fold increase was 2.4 (range, 1.0-8.5) in nucleated cells, 82 (range, 4.6-266.4) in CFU granulocyte-macrophages, and 0.5 (range, 0.09-2.45) in CD34+ lineage negative (lin-) cells. CD3+ cells did not expand under these conditions. Clinical-scale ex vivo expansion of UCB is feasible, and the administration of ex vivo-expanded cells is well tolerated. Augmentation of UCB transplants with ex vivo-expanded cells did not alter the time to myeloid, erythroid, or platelet engraftment in 21 evaluable patients. Recipients of ex vivo-expanded cells continue to have durable engraftment with a median follow-up of 47 months (range, 41-51 months). A randomized phase 2 study will determine whether augmenting UCB transplants with ex vivo-expanded UCB cells is beneficial.

Authors
Jaroscak, J; Goltry, K; Smith, A; Waters-Pick, B; Martin, PL; Driscoll, TA; Howrey, R; Chao, N; Douville, J; Burhop, S; Fu, P; Kurtzberg, J
MLA Citation
Jaroscak, J, Goltry, K, Smith, A, Waters-Pick, B, Martin, PL, Driscoll, TA, Howrey, R, Chao, N, Douville, J, Burhop, S, Fu, P, and Kurtzberg, J. "Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System." Blood 101.12 (June 15, 2003): 5061-5067.
PMID
12595310
Source
pubmed
Published In
Blood
Volume
101
Issue
12
Publish Date
2003
Start Page
5061
End Page
5067
DOI
10.1182/blood-2001-12-0290

High-dose chemotherapy with autologous stem-cell rescue in children and adults with newly diagnosed pineoblastomas.

PURPOSE: We evaluated the usefulness of a treatment regimen that included high-dose chemotherapy (HDC) with autologous stem-cell rescue (ASCR) in patients with newly diagnosed pineoblastoma (PBL). PATIENTS AND METHODS: Twelve patients with PBL were initially treated with surgery and induction chemotherapy. All but two patients underwent radiotherapy. Subsequently, all patients received HDC using cyclophosphamide (CTX) + melphalan (MEL) or busulfan (Bu) + MEL regimens and ASCR. RESULTS: A total of six children and six adults with median ages of 4.2 (range, 0.3 to 19.8 years) and 23 years (range, 23 to 43.7 years), respectively, were treated according to this strategy. Four patients had metastatic disease confined to the neuraxis. Five of 12 patients (42%) had a complete tumor resection at diagnosis. Ten patients received radiotherapy at median doses of 36.0 and 59.4 Gy to the neuraxis and pineal region, respectively. Eleven patients received HDC with CTX + MEL, and one patient received BU + MEL followed by ASCR. Nine patients are alive with no evidence of disease recurrence at a median of 62 months from diagnosis (range, 28 to 125 months), including three patients with metastatic disease and two infants who did not receive any radiotherapy. Three patients have died of progressive disease at 19, 32, and 37 months from diagnosis, respectively. The actuarial 4-year progression-free and overall survivals are 69% (95% confidence interval [CI], 39% to 99%) and 71% (95% CI, 43% to 99%), respectively. CONCLUSION: The use of HDC in addition to radiotherapy seems to be an effective treatment for patients with newly diagnosed pineoblastoma.

Authors
Gururangan, S; McLaughlin, C; Quinn, J; Rich, J; Reardon, D; Halperin, EC; Herndon, J; Fuchs, H; George, T; Provenzale, J; Watral, M; McLendon, RE; Friedman, A; Friedman, HS; Kurtzberg, J; Vredenbergh, J; Martin, PL
MLA Citation
Gururangan, S, McLaughlin, C, Quinn, J, Rich, J, Reardon, D, Halperin, EC, Herndon, J, Fuchs, H, George, T, Provenzale, J, Watral, M, McLendon, RE, Friedman, A, Friedman, HS, Kurtzberg, J, Vredenbergh, J, and Martin, PL. "High-dose chemotherapy with autologous stem-cell rescue in children and adults with newly diagnosed pineoblastomas." J Clin Oncol 21.11 (June 1, 2003): 2187-2191.
PMID
12775745
Source
pubmed
Published In
Journal of Clinical Oncology
Volume
21
Issue
11
Publish Date
2003
Start Page
2187
End Page
2191
DOI
10.1200/JCO.2003.10.096

Marrow cell transplantation for infantile hypophosphatasia.

An 8-month-old girl who seemed certain to die from the infantile form of hypophosphatasia, an inborn error of metabolism characterized by deficient activity of the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP), underwent the first trial of bone marrow cell transplantation for this heritable type of rickets. After cytoreduction, she was given T-cell-depleted, haplo-identical marrow from her healthy sister. Chimerism in peripheral blood and bone marrow became 100% donor. Three months later, she was clinically improved, with considerable healing of rickets and generalized skeletal remineralization. However, 6 months post-transplantation, worsening skeletal disease recurred, with partial return of host hematopoiesis. At the age of 21 months, without additional chemotherapy or immunosuppressive treatment, she received a boost of donor marrow cells expanded ex vivo to enrich for stromal cells. Significant, prolonged clinical and radiographic improvement followed soon after. Nevertheless, biochemical features of hypophosphatasia have remained unchanged to date. Skeletal biopsy specimens were not performed. Now, at 6 years of age, she is intelligent and ambulatory but remains small. Among several hypotheses for our patient's survival and progress, the most plausible seems to be the transient and long-term engraftment of sufficient numbers of donor marrow mesenchymal cells, forming functional osteoblasts and perhaps chondrocytes, to ameliorate her skeletal disease.

Authors
Whyte, MP; Kurtzberg, J; McAlister, WH; Mumm, S; Podgornik, MN; Coburn, SP; Ryan, LM; Miller, CR; Gottesman, GS; Smith, AK; Douville, J; Waters-Pick, B; Armstrong, RD; Martin, PL
MLA Citation
Whyte, MP, Kurtzberg, J, McAlister, WH, Mumm, S, Podgornik, MN, Coburn, SP, Ryan, LM, Miller, CR, Gottesman, GS, Smith, AK, Douville, J, Waters-Pick, B, Armstrong, RD, and Martin, PL. "Marrow cell transplantation for infantile hypophosphatasia." J Bone Miner Res 18.4 (April 2003): 624-636.
PMID
12674323
Source
pubmed
Published In
Journal of Bone and Mineral Research
Volume
18
Issue
4
Publish Date
2003
Start Page
624
End Page
636
DOI
10.1359/jbmr.2003.18.4.624

Mechanically ventilated pediatric stem cell transplant recipients: effect of cord blood transplant and organ dysfunction on outcome.

OBJECTIVES: To compare survival of pediatric umbilical cord blood and bone marrow transplant recipients requiring admission to a pediatric intensive care unit for mechanical ventilation and to determine the effect of organ dysfunction on outcome. DESIGN: Retrospective chart review. SETTING: Tertiary care referral center for pediatric stem cell transplants. PATIENTS: All children 0-18 yrs old admitted to the pediatric intensive care unit for mechanical ventilation after receiving a stem cell transplant. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: Data were collected from medical records of 86 patients who received a stem cell transplant and were subsequently admitted to the pediatric intensive care unit for mechanical ventilation. Demographic data were collected at the time of intubation, and physiologic data were collected at 6 hrs and 96 hrs after intubation. The pediatric intensive care unit, hospital, and 2-yr survival rates for umbilical cord blood transplant recipients were 37%, 25%, and 19%, respectively. The survival rates for bone marrow transplant recipients were 47%, 32%, and 21% for the same time periods. Umbilical cord blood and bone marrow transplant recipients with hepatic dysfunction had a significantly worse outcome, as did patients admitted for respiratory failure or sepsis. CONCLUSIONS: Pediatric recipients of an umbilical cord blood transplant who subsequently required mechanical ventilation had lower pediatric intensive care unit and hospital survival rates compared with patients receiving bone marrow transplantation. Survival at 2 yrs for umbilical cord blood transplant and bone marrow transplant patients was similar. Predictors of outcome for all stem cell transplant recipients requiring mechanical ventilation included pediatric intensive care unit diagnosis requiring intubation and hepatic function. Predictors of outcome can be identified shortly after intubation in pediatric stem cell transplant recipients and may aid in therapeutic decision making and family counseling.

Authors
Hagen, SA; Craig, DM; Martin, PL; Plumer, DD; Gentile, MA; Schulman, SR; Cheifetz, IM
MLA Citation
Hagen, SA, Craig, DM, Martin, PL, Plumer, DD, Gentile, MA, Schulman, SR, and Cheifetz, IM. "Mechanically ventilated pediatric stem cell transplant recipients: effect of cord blood transplant and organ dysfunction on outcome." Pediatr Crit Care Med 4.2 (April 2003): 206-213.
PMID
12749654
Source
pubmed
Published In
Pediatric Critical Care Medicine
Volume
4
Issue
2
Publish Date
2003
Start Page
206
End Page
213
DOI
10.1097/01.PCC.0000043293.83440.79

Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome.

Veno-occlusive disease (VOD) is the most common regimen-related toxicity accompanying stem cell transplantation (SCT). Severe VOD complicated by multisystem organ failure (MOF) remains almost uniformly fatal. Preliminary experience with defibrotide (DF), a single-stranded polydeoxyribonucleotide with fibrinolytic, antithrombotic, and anti-ischemic properties, in the treatment for severe VOD has suggested safety and activity. Eighty-eight patients who developed severe VOD after SCT were treated with DF under a defined treatment plan. At diagnosis, median bilirubin was 76.95 microM (4.5 mg/dL), median weight gain was 7%, ascites was present in 84%, and abnormal hepatic portal venous flow was present in 35%. At DF initiation, median bilirubin had increased to 215.46 microM (12.6 mg/dL), and MOF was present in 97%. DF was administered intravenously in doses ranging from 5 to 60 mg/kg per day for a median of 15 days. No severe hemorrhage or other serious toxicity related to DF was reported. Complete resolution of VOD was seen in 36%, with 35% survival at day +100. Predictors of survival included younger age, autologous SCT, and abnormal portal flow, whereas busulfan-based conditioning and encephalopathy predicted worse outcome. Decreases in mean creatinine and plasminogen activator inhibitor 1(PAI-1) levels during DF therapy predicted better survival. The complete response rate, survival to day +100, and absence of significant DF-associated toxicity in this largest patient cohort reported to date confirm the results of earlier studies. Certain features associated with successful outcome may correlate with DF-related treatment effects, and prospective evaluation of DF therapy for severe VOD should allow better definition of predictors of response or failure.

Authors
Richardson, PG; Murakami, C; Jin, Z; Warren, D; Momtaz, P; Hoppensteadt, D; Elias, AD; Antin, JH; Soiffer, R; Spitzer, T; Avigan, D; Bearman, SI; Martin, PL; Kurtzberg, J; Vredenburgh, J; Chen, AR; Arai, S; Vogelsang, G; McDonald, GB; Guinan, EC
MLA Citation
Richardson, PG, Murakami, C, Jin, Z, Warren, D, Momtaz, P, Hoppensteadt, D, Elias, AD, Antin, JH, Soiffer, R, Spitzer, T, Avigan, D, Bearman, SI, Martin, PL, Kurtzberg, J, Vredenburgh, J, Chen, AR, Arai, S, Vogelsang, G, McDonald, GB, and Guinan, EC. "Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome." Blood 100.13 (December 15, 2002): 4337-4343.
PMID
12393437
Source
pubmed
Published In
Blood
Volume
100
Issue
13
Publish Date
2002
Start Page
4337
End Page
4343
DOI
10.1182/blood-2002-04-1216

Correction of congenital immunodeficiency disease with umbilical cord blood transplantation.

Authors
Kurtzberg, J; Martin, PL; Driscoll, T; Mustafa, M; Wood, S; Kelly, T; Szabolcs, P
MLA Citation
Kurtzberg, J, Martin, PL, Driscoll, T, Mustafa, M, Wood, S, Kelly, T, and Szabolcs, P. "Correction of congenital immunodeficiency disease with umbilical cord blood transplantation." November 16, 2002.
Source
wos-lite
Published In
Blood
Volume
100
Issue
11
Publish Date
2002
Start Page
858A
End Page
858A

Defibrotide (DF) appears effective and safe in a phase II, randomized study of patients (pts) with severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT).

Authors
Richardson, PG; Soiffer, R; Antin, JH; Vredenburgh, J; Kurtzberg, J; Martin, PL; Hockenbery, D; Steinbach, G; Murray, K; Vogelsang, G; Chen, A; Warren, DL; Momtaz, P; Wei, LJ; Jin, Z; Rifai, N; Bradwin, G; Iacobelli, M; McDonald, GB; Guinan, EC
MLA Citation
Richardson, PG, Soiffer, R, Antin, JH, Vredenburgh, J, Kurtzberg, J, Martin, PL, Hockenbery, D, Steinbach, G, Murray, K, Vogelsang, G, Chen, A, Warren, DL, Momtaz, P, Wei, LJ, Jin, Z, Rifai, N, Bradwin, G, Iacobelli, M, McDonald, GB, and Guinan, EC. "Defibrotide (DF) appears effective and safe in a phase II, randomized study of patients (pts) with severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT)." November 16, 2002.
Source
wos-lite
Published In
Blood
Volume
100
Issue
11
Publish Date
2002
Start Page
112A
End Page
112A

Unrelated umbilical cord blood transplant for Wiskott-Aldrich syndrome.

Authors
Archambault, BL; Driscoll, T; Szabolcs, P; Kurtzberg, J; Martin, PL
MLA Citation
Archambault, BL, Driscoll, T, Szabolcs, P, Kurtzberg, J, and Martin, PL. "Unrelated umbilical cord blood transplant for Wiskott-Aldrich syndrome." November 16, 2002.
Source
wos-lite
Published In
Blood
Volume
100
Issue
11
Publish Date
2002
Start Page
855A
End Page
855A

Infections diagnosed in the first year after pediatric stem cell transplantation.

BACKGROUND: Cumulative incidence of infections in the first year posttransplantation in adult patients has been well-described. Such description is less than complete for pediatric stem cell transplantation (SCT) patients. Further among those patients who have been infected, analysis of risk factors for infection has not been well-described for a large cohort of pediatric SCT patients. METHODS: We conducted a retrospective cohort study of infections in the first year after SCT at Duke University Medical Center. We recorded all infections in the first year after transplantation. We determined incidences for 6 categories of infection: gram-negative rods; gram-positive cocci; yeast species; Aspergillus sp.; adenovirus; and cytomegalovirus. We determined incidences based on type of transplant and days post transplantation. We also completed bivariable and multivariable analysis of risk factors [neutropenia, graft vs. host disease (GVHD) and GVHD treatment] for infection type among those children who were infected. RESULTS: We evaluated 510 transplants in 485 children. There were 584 infections in the first year after transplantation. During the first 30 days posttransplantation, type of transplantation did not predict incidence of infection or type of infection. After 30 days children who received unrelated cord blood transplant and matched unrelated donor transplant were at much higher risk of infection than were patients who received autologous, matched sibling or haploidentical transplant (P < 0.001). Patients who received unrelated cord blood or matched unrelated donor transplantation were at higher risk of aspergillosis (P = 0.002), candidiasis (P = 0.005) and adenovirus (P < 0.0001) but not cytomegalovirus (P = 0.18). In analysis of risk factors among those infected, patients with aspergillosis were more likely to have severe GVHD: multivariable 1 year risk ratio, 7.5; 95% confidence interval, 3.0, 18.4. Neutropenia was more strongly associated with gram-negative rod infection than any other type of infection. CONCLUSIONS: The incidence of infection immediately after transplantation did not differ significantly by type of transplant in this pediatric population. Type of transplant predicted increased incidence of infection 30 days posttransplantation and increased incidence of infection with several organisms traditionally associated with a high mortality rate in the transplant population.

Authors
Benjamin, DK; Miller, WC; Bayliff, S; Martel, L; Alexander, KA; Martin, PL
MLA Citation
Benjamin, DK, Miller, WC, Bayliff, S, Martel, L, Alexander, KA, and Martin, PL. "Infections diagnosed in the first year after pediatric stem cell transplantation." Pediatr Infect Dis J 21.3 (March 2002): 227-234.
PMID
12005087
Source
pubmed
Published In
Pediatric Infectious Disease Journal
Volume
21
Issue
3
Publish Date
2002
Start Page
227
End Page
234

Survival of pediatric bone marrow transplant recipients requiring mechanical ventilation and the influence of unrelated umbilical cord blood transplant on outcome

Authors
Hagen, SA; Plumer, D; Gentile, MA; Craig, DM; Meliones, JN; Martin, PL; Cheifetz, IM
MLA Citation
Hagen, SA, Plumer, D, Gentile, MA, Craig, DM, Meliones, JN, Martin, PL, and Cheifetz, IM. "Survival of pediatric bone marrow transplant recipients requiring mechanical ventilation and the influence of unrelated umbilical cord blood transplant on outcome." CRITICAL CARE MEDICINE 29.12 (December 2001): A131-A131.
Source
wos-lite
Published In
Critical Care Medicine
Volume
29
Issue
12
Publish Date
2001
Start Page
A131
End Page
A131

Correction of Hurler Syndrome with unrelated umbilical cord blood transplantation.

Authors
Staba, SL; Martin, PL; Ciocci, GH; Allison-Thacker, J; Kurtzberg, J
MLA Citation
Staba, SL, Martin, PL, Ciocci, GH, Allison-Thacker, J, and Kurtzberg, J. "Correction of Hurler Syndrome with unrelated umbilical cord blood transplantation." BLOOD 98.11 (November 16, 2001): 667A-667A.
Source
wos-lite
Published In
Blood
Volume
98
Issue
11
Publish Date
2001
Start Page
667A
End Page
667A

Multi-institutional phase II, randomized dose finding study of defibrotide (DF) in patients (pts) with severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT): Promising response rate without significant toxicity in a high risk population.

Authors
Richardson, PG; Warren, DL; Momtaz, P; Soiffer, R; Antin, JH; Spitzer, T; Avigan, D; Wei, LJ; Jin, Z; Rifai, N; Bradwin, G; Vredenburgh, J; Kurtzberg, J; Martin, PL; Vogelsang, G; Arai, S; Chen, A; McDonald, GB; Murray, K; Iacobelli, M; Guinan, EC
MLA Citation
Richardson, PG, Warren, DL, Momtaz, P, Soiffer, R, Antin, JH, Spitzer, T, Avigan, D, Wei, LJ, Jin, Z, Rifai, N, Bradwin, G, Vredenburgh, J, Kurtzberg, J, Martin, PL, Vogelsang, G, Arai, S, Chen, A, McDonald, GB, Murray, K, Iacobelli, M, and Guinan, EC. "Multi-institutional phase II, randomized dose finding study of defibrotide (DF) in patients (pts) with severe veno-occlusive disease (VOD) and multi-system organ failure (MOF) post stem cell transplantation (SCT): Promising response rate without significant toxicity in a high risk population." BLOOD 98.11 (November 16, 2001): 853A-853A.
Source
wos-lite
Published In
Blood
Volume
98
Issue
11
Publish Date
2001
Start Page
853A
End Page
853A

Human herpesvirus 6 limbic encephalitis after stem cell transplantation.

Central nervous system complications are common in stem cell transplant recipients, but selective involvement of the medial temporal area is unusual. The 5 patients reported here presented after stem cell transplantation with increased hippocampal T2 signal on magnetic resonance imaging and increased hippocampal glucose uptake on [F-18]fluorodeoxyglucose-positron emission tomography (FDG-PET) associated with short-term memory loss, insomnia, and temporal lobe electrographic seizure activity. The initial scalp electroencephalograms (EEGs) failed to detect seizure activity in these patients, although the memory dysfunction along with the magnetic resonance imaging and FDG-PET findings suggested subcortical seizure activity. However, extended EEG monitoring revealed repetitive temporal lobe electrographic seizure activity. Follow-up MRIs in 2 patients and postmortem findings on 1 patient suggested that hippocampal sclerosis had developed following the clinical syndrome. Cerebrospinal fluid studies revealed the presence of human herpesvirus 6, variant B, DNA in all of 3 patients who had lumbar punctures. Immunohistochemical staining for the P41 and P101 human herpesvirus 6 protein antigens showed numerous immunoreactive astrocytes and neurons in the hippocampus of 1 of the patients who died from other causes. Because of its subtle clinical presentation, this syndrome may be underrecognized, but can be diagnosed with appropriate magnetic resonance imaging techniques, EEG monitoring, and cerebrospinal fluid viral studies.

Authors
Wainwright, MS; Martin, PL; Morse, RP; Lacaze, M; Provenzale, JM; Coleman, RE; Morgan, MA; Hulette, C; Kurtzberg, J; Bushnell, C; Epstein, L; Lewis, DV
MLA Citation
Wainwright, MS, Martin, PL, Morse, RP, Lacaze, M, Provenzale, JM, Coleman, RE, Morgan, MA, Hulette, C, Kurtzberg, J, Bushnell, C, Epstein, L, and Lewis, DV. "Human herpesvirus 6 limbic encephalitis after stem cell transplantation." Ann Neurol 50.5 (November 2001): 612-619.
PMID
11706967
Source
pubmed
Published In
Annals of Neurology
Volume
50
Issue
5
Publish Date
2001
Start Page
612
End Page
619

Management of cholelithiasis in pediatric patients who undergo bone marrow transplantation.

PURPOSE: The aim of this study was to determine the incidence, risk factors, and proper management for asymptomatic cholelithiasis in children undergoing bone marrow transplantation (BMT). METHODS: The authors reviewed retrospectively the records of 575 children who underwent bone marrow transplantation at a University bone marrow transplantation unit (BMT) unit between February 1991 and October 1999. Of these patients, 235 underwent abdominal ultrasonography for evaluation of jaundice, sepsis, abdominal pain, or metastasis. To identify risk factors for cholelithiasis, the authors stratified the patients based on their disease and treatment regimen. Finally, the authors analyzed the natural history and management of BMT children with cholelithiasis. RESULTS: The authors identified 20 cases of cholelithiasis (8.5%) in the 235 BMT patients who underwent ultrasonography. Children who underwent BMT to treat bone marrow failure showed a significantly increased risk of cholelithiasis compared with children treated for malignancy (27% v 7.4%; P<.01). Most children (85%) with gallstones did not require surgical intervention. Specifically, 9 (45%) died from their primary disease, 5 (25%) showed sonographic resolution of their gallstones, and 3 (15%) underwent follow-up nonoperatively with persistent cholelithiasis. Three of the 20 patients with gallstones (15%) had signs of acute cholecystitis and underwent surgery. There were no surgical complications or deaths in the operative group. CONCLUSIONS: Cholelithiasis occurs at a high incidence in pediatric bone marrow transplant patients. Children undergoing BMT for bone marrow failure are at higher risk of having gallstones than those being treated for malignancy. Finally, these data support a strategy of nonoperative management for asymptomatic cholelithiasis in this highly selected group of patients.

Authors
Safford, SD; Safford, KM; Martin, P; Rice, H; Kurtzberg, J; Skinner, MA
MLA Citation
Safford, SD, Safford, KM, Martin, P, Rice, H, Kurtzberg, J, and Skinner, MA. "Management of cholelithiasis in pediatric patients who undergo bone marrow transplantation." J Pediatr Surg 36.1 (January 2001): 86-90.
PMID
11150443
Source
pubmed
Published In
Journal of Pediatric Surgery
Volume
36
Issue
1
Publish Date
2001
Start Page
86
End Page
90
DOI
10.1053/jpsu.2001.20016

Low incidence of Epstein-Barr virus-associated posttransplantation lymphoproliferative disorders in 272 unrelated-donor umbilical cord blood transplant recipients.

Umbilical cord blood (UCB) is being increasingly used for transplantation, but the ability of neonatal T cells to regulate Epstein-Barr virus (EBV)-associated lymphoproliferation is unknown. Because UCB transplantation (UCBT) is associated with a relatively low infused dose of donor T cells, frequent donor-recipient HLA disparity, and use of antithymocyte globulin during conditioning, we hypothesized that the risk of EBV-associated posttransplantation lymphoproliferative disorders (EVB-PTLD) after UCBT may be increased. To investigate the incidence of EBV-PTLD after UCBT, we analyzed 272 unrelated-donor UCBTs performed from August 1993 to December 1999 at Duke University Medical Center and the University of Minnesota. Five cases of EBV-PTLD were identified, with a cumulative incidence of 2% (95% confidence interval, 0.3%-3.7%) at 2 years. EBV-PTLD affected UCB recipients aged 1 to 49 years (median, 8 years), with 4 patients undergoing transplantation for leukemia and 1 for immunodeficiency. Patients received UCB grafts that were HLA matched (n = 1) or mismatched at 1 (n = 1) or 2 (n = 3) HLA loci. Diagnoses occurred at 4 to 14 months (median, 6 months) after UCBT, with 4 of 5 patients having preceding grade II to IV acute graft-versus-host disease and 1 being diagnosed at autopsy. Treatment of 4 patients consisted of withdrawal of immunosuppressive treatment and administration of rituximab, with 2 of 4 patients responding. Thus, the incidence of EBV-PTLD after unrelated-donor UCBT appears similar to that observed after transplantation using unrelated bone marrow (BM) and compares favorably with unrelated-donor T-cell-depleted BM transplantation. Because adoptive immunotherapy with donor lymphocytes is not an available option for recipients of unrelated-donor UCBT, new therapeutic strategies are needed, and rituximab appears promising.

Authors
Barker, JN; Martin, PL; Coad, JE; DeFor, T; Trigg, ME; Kurtzberg, J; Weisdorf, DJ; Wagner, J
MLA Citation
Barker, JN, Martin, PL, Coad, JE, DeFor, T, Trigg, ME, Kurtzberg, J, Weisdorf, DJ, and Wagner, J. "Low incidence of Epstein-Barr virus-associated posttransplantation lymphoproliferative disorders in 272 unrelated-donor umbilical cord blood transplant recipients." Biol Blood Marrow Transplant 7.7 (2001): 395-399.
PMID
11529490
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
7
Issue
7
Publish Date
2001
Start Page
395
End Page
399

Graft-versus-leukemia-induced complete remission following unrelated umbilical cord blood transplantation for acute leukemia.

A 15-year-old female received an unrelated three of six HLA antigen matched umbilical cord blood (UCB) transplant for refractory, relapsed T-cell ALL. Conditioning consisted of TBI, melphalan, and anti-thymocyte globulin (ATG), with cyclosporin A (CsA) and solumedrol for GVHD prophylaxis. She engrafted and a day 34 bone marrow aspirate showed 100% donor cells and no evidence of leukemia. The post-transplant course was complicated by mild grade I acute GVHD involving skin, and limited chronic GVHD of the gut which resolved with the addition of 1 mg/kg/day of steroids to her CsA prophylaxis. One hundred and ninety days after transplantation the patient developed pancytopenia and was subsequently found to have a leukemic relapse. Immunosuppression was discontinued and she was started on G-CSF and erythropoietin. Moderate skin and gut GVHD developed which was treated with both topical and low-dose oral steroids. Over the next few weeks she became transfusion independent and a follow-up bone marrow aspirate showed complete remission. She continued in complete remission for 4 months, at which time localized leukemic relapse was found in a soft tissue breast mass in spite of continued bone marrow remission. While the patient ultimately died of progressive disease, this case demonstrates that mismatched UCB in conjunction with G-CSF is capable of generating a GVL effect that can induce a complete remission.

Authors
Howrey, RP; Martin, PL; Driscoll, T; Szabolcs, P; Kelly, T; Shpall, EJ; Bearman, SI; Slat-Vasquez, V; Rubinstein, P; Stevens, CE; Kurtzberg, J
MLA Citation
Howrey, RP, Martin, PL, Driscoll, T, Szabolcs, P, Kelly, T, Shpall, EJ, Bearman, SI, Slat-Vasquez, V, Rubinstein, P, Stevens, CE, and Kurtzberg, J. "Graft-versus-leukemia-induced complete remission following unrelated umbilical cord blood transplantation for acute leukemia." Bone Marrow Transplant 26.11 (December 2000): 1251-1254.
PMID
11149743
Source
pubmed
Published In
Bone Marrow Transplantation
Volume
26
Issue
11
Publish Date
2000
Start Page
1251
End Page
1254
DOI
10.1038/sj.bmt.1702697

High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis (FEL).

Authors
Kurtzberg, J; Szabolcs, P; Howrey, RP; Driscoll, TA; Martin, PL
MLA Citation
Kurtzberg, J, Szabolcs, P, Howrey, RP, Driscoll, TA, and Martin, PL. "High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis (FEL)." BLOOD 96.11 (November 16, 2000): 362B-362B.
Source
wos-lite
Published In
Blood
Volume
96
Issue
11
Publish Date
2000
Start Page
362B
End Page
362B

Unrelated umbilical cord blood transplant for osteopetrosis.

Authors
Martin, PL; Lacaze, M; Driscoll, TA; Szaboles, P; Kurtzberg, J; Wiley, J; Rubinstein, P; Howrey, RP
MLA Citation
Martin, PL, Lacaze, M, Driscoll, TA, Szaboles, P, Kurtzberg, J, Wiley, J, Rubinstein, P, and Howrey, RP. "Unrelated umbilical cord blood transplant for osteopetrosis." BLOOD 96.11 (November 16, 2000): 207A-207A.
Source
wos-lite
Published In
Blood
Volume
96
Issue
11
Publish Date
2000
Start Page
207A
End Page
207A

Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank.

Authors
Kurtzberg, J; Martin, PL; Szabolcs, PM; Howrey, RP; Driscoll, TA; Stevens, CE; Rubinstein, P
MLA Citation
Kurtzberg, J, Martin, PL, Szabolcs, PM, Howrey, RP, Driscoll, TA, Stevens, CE, and Rubinstein, P. "Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank." BLOOD 96.11 (November 16, 2000): 206A-206A.
Source
wos-lite
Published In
Blood
Volume
96
Issue
11
Publish Date
2000
Start Page
206A
End Page
206A

Low incidence of Epstein-Barr virus-associated post-transplant lymphoproliferative disorders (EBV-PTLD) in 263 unrelated donor umbilical cord blood transplant recipients.

Authors
Barker, JN; Martin, PL; Defor, T; Weisdorf, DJ; Kurtzberg, J; Wagner, JE
MLA Citation
Barker, JN, Martin, PL, Defor, T, Weisdorf, DJ, Kurtzberg, J, and Wagner, JE. "Low incidence of Epstein-Barr virus-associated post-transplant lymphoproliferative disorders (EBV-PTLD) in 263 unrelated donor umbilical cord blood transplant recipients." BLOOD 96.11 (November 16, 2000): 206A-206A.
Source
wos-lite
Published In
Blood
Volume
96
Issue
11
Publish Date
2000
Start Page
206A
End Page
206A

When you hear hoof beats...do not forget the zebras.

Authors
Klock, B; Pham, T; Smith, LS; Beris, S; Lobo, FM; Martin, PL; Rappeport, J; Fuleihan, RL
MLA Citation
Klock, B, Pham, T, Smith, LS, Beris, S, Lobo, FM, Martin, PL, Rappeport, J, and Fuleihan, RL. "When you hear hoof beats..do not forget the zebras." Curr Opin Pediatr 12.2 (April 2000): 172-177.
PMID
10763769
Source
pubmed
Published In
Current Opinion in Pediatrics
Volume
12
Issue
2
Publish Date
2000
Start Page
172
End Page
177

Unrelated placental blood in marrow transplantation.

Authors
Kurtzberg, J; Martin, P; Chao, N; Stevens, C; Rubinstein, P
MLA Citation
Kurtzberg, J, Martin, P, Chao, N, Stevens, C, and Rubinstein, P. "Unrelated placental blood in marrow transplantation." Stem Cells 18.2 (2000): 153-154.
PMID
10742391
Source
pubmed
Published In
Stem Cells
Volume
18
Issue
2
Publish Date
2000
Start Page
153
End Page
154
DOI
10.1634/stemcells.18-2-153

High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis fed

Familial erythrophagocytic lymphohistiocytosis (PEL) is an inherited disease that is uniformly fatal without myeloablation and transplantation therapy. Frequently babies and young children with this condition need to proceed to transplant rapidly before disease progression. Banked unrelated umbilical cord blood (uUCB)is a readily available source of allogeneic unrelated hematopoietic stem cells which can be used for patients who lack a matched related donor. Over the past 2 years, we have transplanted 4 babies with PEL with uUCB after cytoreduction with busulfan (40mg/mg/m2/dose PO q6 hours x 16 doses, days -9 to -6), cyclophosphamide (50mg/kg/dose IV daily, days -5 to -2) and ATG (30mg/kg/ dose, daily days -3 to -1). Prior to transplant, each baby had received at least 1 month of therapy with prednisone, VP-16 and intrathecal methotrexate. All had CNS involvement at diagnosis, but negative CSF cytopathology at the start of the preparative therapy for transplant. Prophylaxis for GvHD was provided with conventional dose cyclosporine and intermediate dose solumedrol. Supportive care was provided with G-CSF, prophylactic antiviral and antifungal therapy. TPN, RBC and platelet transfusions, and nursing under HEPA filtration. The median age and weights of the patients were 0.5 years (range 0.3-0.8) and 8.2kg (range 4.7-11.6), respectively. 75% were female and 50% were CMV negative. Two patients received a 4/6 and 2 received a 5/6 antigen matching graft. The median nucleated cell dose administered was 137 million cells/kg (range 70-215) and the median CD34 dose was 381,000 cells/kg (range 250-644K). All patients engrafted achieving an ANC of 500/mm3 at 12,12, 13, and 18 days, respectively. They achieved platelet and RBC transfusion independence in a median of 48 and 44 days repectively and achieved an untransfused platelet count of 50K/ mm3 in 31,49,69,and 75 days, respectively. 3/4 patients developed grade 1 GvHD involving skin only while the fourth had no GvHD. The average hospital stay was 45 days. All patients engrafted with 100% donor cells and are survi ving event-free and free of signs or symptoms of PEL from 75-660 days (median 451 days). These preliminary data suggest that unrelated UCB should be strongly considered as an allogeneic donor source for rapid transplantation of patients with PEL. Furthermore, it is not necessary to use total body irradiation in the preparative regimen to cure these babies of this disease.

Authors
Kurtzberg, J; Szabolcs, P; Howrey, RP; Driscoll, TA; Martin, PL
MLA Citation
Kurtzberg, J, Szabolcs, P, Howrey, RP, Driscoll, TA, and Martin, PL. "High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis fed." Blood 96.11 PART II (2000): 362b-.
Source
scival
Published In
Blood
Volume
96
Issue
11 PART II
Publish Date
2000
Start Page
362b

Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank

Over the past decade, umbilical cord blood (UCB) has been shown to provide adequate stem cells for bone marrow reconstitution after myeloablative therapy. Reports to date include 2 from registries and a few small pilot studies, but there have been no reports of a large series of patients transplanted at a single institution. We now report the outcomes of 158 consecutively transplanted pédiatrie patients (<18 years of age) with hématologie malignancies, bone marrow failure or immunodeficiency syndromes, or inborn errors of metabolism treated at a single institution and transplanted with UCB units from a single bank over the past 7 years. Factors associated with a favorable outcome are identified. Ninety-eight patients were transplanted for a high risk or recurrent malignancy (48 ALL, 29 ANLL, 6 MDS, 2 JCML, 6 CML, 5 Neuroblastoma, 2 NHL) and 60 for non-malignant conditions (13 for BM failure, 18 for immunodeficiency, 29 for inborn errors). The median age was 5.13 yrs (range 0.08-17.9 yrs), median weight 16.7 kg (range 3.9-93.3), 54% were CMV positive, 63% male, 26 % were of minority ethnic background, and 84% had high risk characteristics. Four pts received 6/6, 45 pts 4/6 and 15 pts 3/6 matched grafts. The median cell dose was 55 million cells/kg and median CD34 dose 489,000 cells/kg. The median days to an ANC of 500/mm3, platelet count of 50K/mm3 and RBC transfusion independence were 22, 74,and 48, respectively. The probabilities of reaching an ANC of 500 by days 42 and 60 were 89% and 95% while the probability of reaching an untransfused platelet count of 50K/mm3 by day 180 was 88%. The incidence of acute GvHD, 35% developed moderate (17%) to severe (18%) disease. The probability of chronic GvHD was 14%, 85%limited. Relapses occurred in 16% of patients with malignancies within a median of 5 months (range 1-22). The probability of overall EFS at 3 years is 45%. In univariate analysis.the factors associated with a favorable outcome were cell dose and age, in multivariate analysis, cell dose/kg was the only statistically significant variable predictive of a favorable outcome. We conclude that banked, unrelated UCB (even if HLA mismatched at 1-2 antigens) should be strongly considered as the unrelated donor source for pédiatrie patients lacking a matched related stem cell donor.

Authors
Kurtzberg, J; Martin, PL; Szabolcs, PM
MLA Citation
Kurtzberg, J, Martin, PL, and Szabolcs, PM. "Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank." Blood 96.11 PART I (2000): 205a-.
Source
scival
Published In
Blood
Volume
96
Issue
11 PART I
Publish Date
2000
Start Page
205a

Unrelated umbilical cord blood transplant for osteopetrosis

Severe osteopetrosis is a uniformly fatal disease that affects infants and young children. Stem cell transplantation can be curative since the patient's defective osteoclasts are replaced by functional donor osteoclasts. Due to encroachment of cranial nerves leading to blindness and deafness, rapid intervention is necessary to decrease morbidity. We have investigated use of unrelated umbilical cord blood (UUCB) as the source of stem cells for 7 patients with osteopetrosis who lacked matched family donors. Sufficiently matched UUCB units were identified for all 7 osteopetrosis patients referred to our center. Four patients received UCB units with 2 antigen mismatches, while 2 patients received a single antigen mismatched unit. The median age at the time of transplant was 7-months (range 4-27) and the median mononuclear cell dose was 10xlOe7 cells/kg (range 2.3-21.1xlOe7). All patients were prepared with Busulfan, Cyclophosphamide and ATG. Steroids and Cyclosporine were used for GvHD prophylaxis. All évaluable cases engrafted with a median time to ANC 500 of 22 days (range 12-68). Five patients had grade I-I1 acute GvHD, while 2 had limited chronic GvHD which responded to therapy. Five of seven patients are currently alive, with a median follow-up of 23 months (range 1-49). Our results show that use of UUCB for severe osteopetrosis is an effective treatment for patients who lack matched family donors. The advantages of UUCB are low risk of chronic GvHD and the rapidity of identifying a match. For osteopetrosis patients, it is critical to begin transplantation as soon as possible after diagnosis to decrease the risk of cranial nerve damage. Age MNC Match ANC Pit. RBC aGvHD cGvHD Survival (Mo) (10e7/kg) 500 Indep. Indep. 7 2.3 5/6 12 23 30 D limited 23 mo 13 5.9 4/6 20 67 56 None None 49 mo 27 12.3 4/6 21 150 151 0 None 23 mo 5 5.6 5/6 23 47 39 None limited 12 mo 8 18.3 4/6 40 ME ME u NE died d 92 6 10.0 6/6 68 NE NE IINE died dl 10 4 21.1 4/6 Too early TE TE TE TE 1 mo.

Authors
Martin, PL; Lacaze, M
MLA Citation
Martin, PL, and Lacaze, M. "Unrelated umbilical cord blood transplant for osteopetrosis." Blood 96.11 PART I (2000): 207a-.
Source
scival
Published In
Blood
Volume
96
Issue
11 PART I
Publish Date
2000
Start Page
207a

Augmentation of umbilical cord blood (UCB) transplantation with ex vivo expaned cells, a phase I trial using the replicell system.

Authors
Kurtzberg, J; Jaroscak, J; Martin, PL; Driscoll, T; Waters-Pick, B; Douville, J; Howrey, R; Goltry, KL; Smith, AK
MLA Citation
Kurtzberg, J, Jaroscak, J, Martin, PL, Driscoll, T, Waters-Pick, B, Douville, J, Howrey, R, Goltry, KL, and Smith, AK. "Augmentation of umbilical cord blood (UCB) transplantation with ex vivo expaned cells, a phase I trial using the replicell system." BLOOD 94.10 (November 15, 1999): 571A-571A.
Source
wos-lite
Published In
Blood
Volume
94
Issue
10
Publish Date
1999
Start Page
571A
End Page
571A

Transplantation of a patient with congenital neutropenia and invasive fungal disease with unrelated umbilical cord blood using paternal G-CSF mobilized, irradiated granulocyte support.

Authors
Kurtzberg, J; Szabolcs, P; Driscoll, T; Howrey, R; Waters-Pick, B; Martin, PL
MLA Citation
Kurtzberg, J, Szabolcs, P, Driscoll, T, Howrey, R, Waters-Pick, B, and Martin, PL. "Transplantation of a patient with congenital neutropenia and invasive fungal disease with unrelated umbilical cord blood using paternal G-CSF mobilized, irradiated granulocyte support." BLOOD 94.10 (November 15, 1999): 368B-369B.
Source
wos-lite
Published In
Blood
Volume
94
Issue
10
Publish Date
1999
Start Page
368B
End Page
369B

Unrelated cord blood transplantation for correction of genetic diseases.

Authors
Howrey, RP; Martin, PL; Ciocci, G; Driscoll, TA; Frey, M; Buckley, RH; Hickling, WH; Wiley, J; Provenzale, J; Morse, R; Rubinstein, P; Krivit, W; Kurtzberg, J
MLA Citation
Howrey, RP, Martin, PL, Ciocci, G, Driscoll, TA, Frey, M, Buckley, RH, Hickling, WH, Wiley, J, Provenzale, J, Morse, R, Rubinstein, P, Krivit, W, and Kurtzberg, J. "Unrelated cord blood transplantation for correction of genetic diseases." BLOOD 92.10 (November 15, 1998): 291A-291A.
Source
wos-lite
Published In
Blood
Volume
92
Issue
10
Publish Date
1998
Start Page
291A
End Page
291A

Transient engraftment of maternal T-cells in recipients of unrelated cord blood transplants.

Authors
Kurtzberg, J; Carrier, C; Martin, PL; Stevens, CE; Howrey, RP; Driscoll, TA; Reismoen, N; Rubinstein, P
MLA Citation
Kurtzberg, J, Carrier, C, Martin, PL, Stevens, CE, Howrey, RP, Driscoll, TA, Reismoen, N, and Rubinstein, P. "Transient engraftment of maternal T-cells in recipients of unrelated cord blood transplants." BLOOD 92.10 (November 15, 1998): 652A-653A.
Source
wos-lite
Published In
Blood
Volume
92
Issue
10
Publish Date
1998
Start Page
652A
End Page
653A

Busulfan and melphalan as preparative therapy for BMT in childhood leukemias.

Authors
Graham, ML; Katsanis, E; Shapiro, TW; Hutter, JJ; Martin, PL; Kurtzberg, J
MLA Citation
Graham, ML, Katsanis, E, Shapiro, TW, Hutter, JJ, Martin, PL, and Kurtzberg, J. "Busulfan and melphalan as preparative therapy for BMT in childhood leukemias." BLOOD 92.10 (November 15, 1998): 128A-128A.
Source
wos-lite
Published In
Blood
Volume
92
Issue
10
Publish Date
1998
Start Page
128A
End Page
128A

Transplantation of related, haplo-identical umbilical cord blood in pediatric patients with genetic hematological diseases.

Authors
Martin, PL; Howrey, R; Driscoll, T; Frothingham, B; Owen, W; Kelly, P; Lee, Y; Hamerschlak, N; Kurtzberg, J
MLA Citation
Martin, PL, Howrey, R, Driscoll, T, Frothingham, B, Owen, W, Kelly, P, Lee, Y, Hamerschlak, N, and Kurtzberg, J. "Transplantation of related, haplo-identical umbilical cord blood in pediatric patients with genetic hematological diseases." BLOOD 92.10 (November 15, 1998): 358B-358B.
Source
wos-lite
Published In
Blood
Volume
92
Issue
10
Publish Date
1998
Start Page
358B
End Page
358B

A phase I trial of augmentation of unrelated umbilical cord blood transplantation with ex-vivo expanded cells.

Authors
Jaroscak, J; Martin, PL; Waters-Pick, B; Armstrong, RD; Driscoll, T; Howrey, RP; Castellino, S; Douville, J; Burhop, S; Goltry, K; Rubinstein, P; Smith, A; Kurtzberg, J
MLA Citation
Jaroscak, J, Martin, PL, Waters-Pick, B, Armstrong, RD, Driscoll, T, Howrey, RP, Castellino, S, Douville, J, Burhop, S, Goltry, K, Rubinstein, P, Smith, A, and Kurtzberg, J. "A phase I trial of augmentation of unrelated umbilical cord blood transplantation with ex-vivo expanded cells." BLOOD 92.10 (November 15, 1998): 646A-646A.
Source
wos-lite
Published In
Blood
Volume
92
Issue
10
Publish Date
1998
Start Page
646A
End Page
646A

Toxicity, pharmacology and feasibility of administration of PEG-L-asparaginase as consolidation therapy in patients undergoing bone marrow transplantation for acute lymphoblastic leukemia.

We attempted to administer PEG-L-asparaginase (PEG-L-A) following hematologic recovery to 38 patients undergoing autologous or allogeneic marrow transplantation for acute lymphoblastic leukemia (ALL). Twenty-four patients (12 of 22 receiving allogeneic and 12 of 16 receiving autologous transplants) received between one and 12 doses of PEG-L-A, including nine who completed the planned 12 doses of therapy. The toxicities encountered were similar to those observed in non-transplanted patients undergoing therapy with PEG-L-A and included allergic reactions, pancreatitis, weight loss, hypoalbuminemia, and low levels of anti-thrombin III. Of the 24 who received the drug, eight remain in remission. Of 12 patients in second remission at the time of transplantation who received PEG-L-A, five of seven who received allogeneic and two of five who received autologous transplants remain in remission, 16+ to 46+ months from transplant. While PEG-L-A could be administered to most of the patients undergoing marrow transplantation for ALL, most patients either relapsed while receiving the drug or developed toxicities which resulted in abbreviated courses. At this time, we cannot recommend PEG-L-A as single agent, post-BMT chemotherapy.

Authors
Graham, ML; Asselin, BL; Herndon, JE; Casey, JR; Chaffee, S; Ciocci, GH; Daeschner, CW; Davis, AR; Gold, S; Halperin, EC; Laughlin, MJ; Martin, PL; Olson, JF; Kurtzberg, J
MLA Citation
Graham, ML, Asselin, BL, Herndon, JE, Casey, JR, Chaffee, S, Ciocci, GH, Daeschner, CW, Davis, AR, Gold, S, Halperin, EC, Laughlin, MJ, Martin, PL, Olson, JF, and Kurtzberg, J. "Toxicity, pharmacology and feasibility of administration of PEG-L-asparaginase as consolidation therapy in patients undergoing bone marrow transplantation for acute lymphoblastic leukemia." Bone Marrow Transplant 21.9 (May 1998): 879-885.
PMID
9613779
Source
pubmed
Published In
Bone Marrow Transplantation
Volume
21
Issue
9
Publish Date
1998
Start Page
879
End Page
885
DOI
10.1038/sj.bmt.1701223

Hematologic engraftment and reconstitution of immune function post unrelated placental cord blood transplant in an adult with acute lymphocytic leukemia.

Authors
Laughlin, MJ; Rizzieri, DA; Smith, CA; Moore, JO; Lilly, S; McGaughey, D; Martin, P; Carrier, C; Stevens, CE; Rubinstein, P; Buckley, R; Kurtzberg, J
MLA Citation
Laughlin, MJ, Rizzieri, DA, Smith, CA, Moore, JO, Lilly, S, McGaughey, D, Martin, P, Carrier, C, Stevens, CE, Rubinstein, P, Buckley, R, and Kurtzberg, J. "Hematologic engraftment and reconstitution of immune function post unrelated placental cord blood transplant in an adult with acute lymphocytic leukemia." Leuk Res 22.3 (March 1998): 215-219.
PMID
9619913
Source
pubmed
Published In
Leukemia Research
Volume
22
Issue
3
Publish Date
1998
Start Page
215
End Page
219

Marked radiographic improvement in infantile hypophosphatasia with T-depleted bone marrow transplantation from a related haploidentical donor.

Authors
Martin, PL; Thompson, J; Gottesman, G; Reid, J; Whyte, MP; Miller, C; Kurtzberg, J
MLA Citation
Martin, PL, Thompson, J, Gottesman, G, Reid, J, Whyte, MP, Miller, C, and Kurtzberg, J. "Marked radiographic improvement in infantile hypophosphatasia with T-depleted bone marrow transplantation from a related haploidentical donor." BLOOD 90.10 (November 15, 1997): 4537-4537.
Source
wos-lite
Published In
Blood
Volume
90
Issue
10
Publish Date
1997
Start Page
4537
End Page
4537

Correction of beta thalassemia major with umbilical cord blood transplantation from a related haploidentical donor.

Authors
Martin, PL; Jaroscak, J; Hamerschlak, N; Kurtzberg, J
MLA Citation
Martin, PL, Jaroscak, J, Hamerschlak, N, and Kurtzberg, J. "Correction of beta thalassemia major with umbilical cord blood transplantation from a related haploidentical donor." BLOOD 90.10 (November 15, 1997): 4536-4536.
Source
wos-lite
Published In
Blood
Volume
90
Issue
10
Publish Date
1997
Start Page
4536
End Page
4536

Comparison of fluorescence in situ hybridization, cytogenetic analysis, and DNA index analysis to detect chromosomes 4 and 10 aneuploidy in pediatric acute lymphoblastic leukemia: a Pediatric Oncology Group study.

PURPOSE: Chromosome abnormalities are an important prognostic factor in childhood acute lymphoblastic leukemia (ALL). Recently, a subset of patients with hyperdiploid ALL and trisomy of chromosomes 4 and 10 has been reported to have a very favorable event-free survival. Rapid and accurate detection of these patients will allow them to be treated with highly effective and relatively nontoxic antimetabolite therapy. Because of inherent problems associated with conventional cancer cytogenetics, we examined the efficacy of fluorescence in situ hybridization (FISH) to identify this ALL subgroup. PATIENTS AND METHODS: Fifty uncultured bone marrow specimens from children with newly diagnosed ALL were examined for chromosomes 4 and 10 aneuploidy with FISH. These results were compared with routine cytogenetics and DNA Index (DI). RESULTS: Interphase FISH cytogenetics identified the abnormal cell line(s) in all cases in which cytogenetics showed aneuploidy of chromosomes 4 and 10. In cases in which cytogenetics was not informative, FISH identified the presence of an aneuploid chromosome 4 and/or 10 cell line in concordance with the DI. CONCLUSIONS: FISH interphase cytogenetics can accurately detect chromosome 4 and 10 aneuploidy in leukemic cells. It is a rapid and clinically applicable technique that can reliably identify childhood ALL cases who have trisomy of chromosomes 4 and 10 and who have very favorable event-free survival.

Authors
Martin, PL; Look, AT; Schnell, S; Harris, MB; Pullen, J; Shuster, JJ; Carroll, AJ; Pettenati, MJ; Rao, PN
MLA Citation
Martin, PL, Look, AT, Schnell, S, Harris, MB, Pullen, J, Shuster, JJ, Carroll, AJ, Pettenati, MJ, and Rao, PN. "Comparison of fluorescence in situ hybridization, cytogenetic analysis, and DNA index analysis to detect chromosomes 4 and 10 aneuploidy in pediatric acute lymphoblastic leukemia: a Pediatric Oncology Group study." J Pediatr Hematol Oncol 18.2 (May 1996): 113-121.
PMID
8846121
Source
pubmed
Published In
Journal of Pediatric Hematology/Oncology
Volume
18
Issue
2
Publish Date
1996
Start Page
113
End Page
121

Relationship of DNA ploidy to histology and prognosis in rhabdomyosarcoma. Comparison of flow cytometry and image analysis.

BACKGROUND: Although DNA ploidy correlates with prognosis in certain childhood cancers, e.g., neuroblastoma, its significance in rhabdomyosarcoma (RMS) is unclear and controversial. METHODS: Ploidy by flow cytometry (FCM) and image analysis (IA) in 26 of 27 children with RMS (17 embryonal, 3 mixed embryonal/alveolar, 5 alveolar, 1 anaplastic, 1 ectomesenchymoma) and 4 adults with pleomorphic RMS were evaluated. Statistical comparisons were analyzed between DNA content and gender, age, localization, Intergroup Rhabdomyosarcoma Study (IRS) group, and histopathologic subtype. Survival analyses were performed by the Kaplan-Meier test using the approximate chi-square statistic for the log rank test. RESULTS: The concordance rate between FCM and IA was 26 of 30 (87%); FCM was not performed in one tumor. Image analysis was more sensitive than FCM in detecting aneuploidy. Furthermore, DNA content was associated significantly with histologic subtype (P = 0.031); embryonal histology commonly was hyperdiploid (mean, 1.44; median, 1.27), whereas alveolar histology usually was near-tetraploid (mean, 1.83; median, 1.95). All four adult patients with pleomorphic RMS were aneuploid, with one showing multiple DNA peaks. No correlation between DNA content and survival was observed in the children with RMS. However, IRS group (P = 0.011) and patient age (P = 0.036) were independent prognostic indicators significantly related to survival. All adult patients died of their disease. CONCLUSIONS: Although ploidy correlates with histologic subtype, DNA content is not significantly predictive of prognosis in patients with RMS. Age at diagnosis and IRS group are independent predictors of clinical outcome in children with RMS.

Authors
Kilpatrick, SE; Teot, LA; Geisinger, KR; Martin, PL; Shumate, DK; Zbieranski, N; Russell, GB; Fletcher, CD
MLA Citation
Kilpatrick, SE, Teot, LA, Geisinger, KR, Martin, PL, Shumate, DK, Zbieranski, N, Russell, GB, and Fletcher, CD. "Relationship of DNA ploidy to histology and prognosis in rhabdomyosarcoma. Comparison of flow cytometry and image analysis." Cancer 74.12 (December 15, 1994): 3227-3233.
PMID
7526971
Source
pubmed
Published In
Cancer
Volume
74
Issue
12
Publish Date
1994
Start Page
3227
End Page
3233

Bone marrow transplantation in the perinatal period.

Authors
Martin, PL; Rappeport, JM
MLA Citation
Martin, PL, and Rappeport, JM. "Bone marrow transplantation in the perinatal period." Semin Perinatol 14.5 (October 1990): 416-422.
PMID
2287956
Source
pubmed
Published In
Seminars in Perinatology
Volume
14
Issue
5
Publish Date
1990
Start Page
416
End Page
422

Formation of a rate-limiting intermediate in 5S RNA gene transcription.

We have performed in vitro kinetic analyses of transcription from the 5S gene promoter in order to resolve the rate-limiting events which lead to accurate transcription of the 5S RNA gene. We demonstrate that a rate-limiting intermediate can be formed during an extended incubation prior to initiation of transcription. Formation of such a complex is temperature-dependent, requires magnesium and ATP, consists of stoichiometric amounts of the known class III transcription factors and RNA polymerase III on 5S DNA, and eliminates the normal lag in attainment of a steady-state rate of transcription. This complex is therefore different from the "stable complex" minimally required for template commitment. Further analyses demonstrate that TFIIIB, like TFIIIA and TFIIIC, can be stably sequestered on the 5S gene and also allow us to formulate the following order of factor interactions on the 5S gene: TFIIIA, TFIIIC, TFIIIB, RNA polymerase III.

Authors
Bieker, JJ; Martin, PL; Roeder, RG
MLA Citation
Bieker, JJ, Martin, PL, and Roeder, RG. "Formation of a rate-limiting intermediate in 5S RNA gene transcription." Cell 40.1 (January 1985): 119-127.
PMID
3967290
Source
pubmed
Published In
Cell
Volume
40
Issue
1
Publish Date
1985
Start Page
119
End Page
127

Transcription of class III genes: formation of preinitiation complexes.

Class III genes require multiple cellular factors for transcription by RNA polymerase III; these genes form stable transcription complexes, which in the case of Xenopus 5S genes are correlated with differential expression in vivo. The minimal number and identity of the factors required to form both stable and metastable complexes on three class III genes (encoding, respectively, 5S RNA, transfer RNA, and adenovirus VA RNA species) were determined. Stable complex formation requires one common factor, whose recognition site was analyzed, and either no additional factors (the VA gene), a second common factor (the transfer RNA gene), or a third gene-specific factor (the 5S gene). The mechanism of stable complex formation and its relevance to transcriptional regulation were examined in light of the various factors and the promoter sequences recognized by these factors.

Authors
Lassar, AB; Martin, PL; Roeder, RG
MLA Citation
Lassar, AB, Martin, PL, and Roeder, RG. "Transcription of class III genes: formation of preinitiation complexes." Science 222.4625 (November 18, 1983): 740-748. (Review)
PMID
6356356
Source
pubmed
Published In
Science
Volume
222
Issue
4625
Publish Date
1983
Start Page
740
End Page
748

Eukaryotic gene transcription with purified components.

Authors
Dignam, JD; Martin, PL; Shastry, BS; Roeder, RG
MLA Citation
Dignam, JD, Martin, PL, Shastry, BS, and Roeder, RG. "Eukaryotic gene transcription with purified components." Methods Enzymol 101 (1983): 582-598.
PMID
6888276
Source
pubmed
Published In
Methods in Enzymology
Volume
101
Publish Date
1983
Start Page
582
End Page
598
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