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Parikh, Suhag H.

Overview:

Stem cell transplantation for a variety of disorders - ranging from malignant diseases such as leukemia, lymphoma and myelodysplastic syndrome to nonmalignant diseases such as sickle cell disease, thalassemias, aplastic anemia, histiocytosis and leukodystrophies. My clinical research interest is stem cell transplantation for children with primary immune deficiency disorders and hemoglobinopathies such as sickle cell anemia,thalassemia and other non-malignant disorders. In addition,I am interested in developing startegies, such as reduced intensity conditioning, to make transplant safer.

Positions:

Associate Professor of Pediatrics

Pediatrics, Blood and Marrow Transplantation
School of Medicine

Member of the Duke Cancer Institute

Duke Cancer Institute
School of Medicine

Education:

M.B.B.S. 1988

M.B.B.S. — Nagpur University (India)

Grants:

Prospective Study of SCID Infants who receive Hematopoietic Cell Therapy

Administered By
Pediatrics, Allergy and Immunology
AwardedBy
University of California - San Francisco
Role
Assistant Research Professor
Start Date
September 01, 2014
End Date
August 31, 2019

Prospective Study of SCID Infants who receive Hematopoietic Cell Therapy

Administered By
Pediatrics, Allergy and Immunology
AwardedBy
University of California - San Francisco
Role
Co Investigator
Start Date
September 12, 2009
End Date
August 31, 2014

Publications:

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

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

Congenital hemophagocytic lymphohistiocytosis presenting as thrombocytopenia in a newborn.

Hemophagocytic lymphohistiocytosis (HLH) is a disease caused by dysregulation and hyperactivation of the immune system, and can be familial or acquired. HLH presenting in infancy can be rapidly fatal if not promptly recognized and treated. Congenital HLH can be caused by various genetic mutations or part of immunodeficiency syndromes. We present an infant with Griscelli syndrome and familial HLH with atypical genetic mutations, presenting as thrombocytopenia on the first day of life, cured with chemotherapy and unrelated cord blood transplant.

Authors
Hinson, A; Owen, W; Prose, N; Parikh, S; Thornburg, C
MLA Citation
Hinson, A, Owen, W, Prose, N, Parikh, S, and Thornburg, C. "Congenital hemophagocytic lymphohistiocytosis presenting as thrombocytopenia in a newborn." Journal of pediatric hematology/oncology 37.4 (May 2015): 300-303.
PMID
25121636
Source
epmc
Published In
Journal of Pediatric Hematology/Oncology
Volume
37
Issue
4
Publish Date
2015
Start Page
300
End Page
303
DOI
10.1097/mph.0000000000000234

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

Pre-engraftment syndrome after myeloablative dual umbilical cord blood transplantation: risk factors and response to treatment.

High fevers and/or rashes prior to neutrophil engraftment are frequently observed after umbilical cord blood (UCB) transplantation, and the condition is referred to as pre-engraftment syndrome (PES). Few studies have evaluated the risk factors for and treatment response to PES. Therefore, we retrospectively characterized PES in 57 consecutive engrafted patients (≥ 12 years old) who received myeloablative dual UCB transplantation. All patients received TBI (≥ 13.2 Gy)-based myeloablative conditioning. Tacrolimus (n=35) or CYA (n=22) combined with mycophenolate mofetil was used as GVHD prophylaxis. PES was defined as the presence of non-infectious fever (≥ 38.5 °C) and/or rash prior to or on the day of neutrophil engraftment. The incidence (95% confidence interval) of PES was 77% (66-88%). The incidence of PES was significantly higher in patients who received CYA as a GVHD prophylaxis than those who received tacrolimus (P<0.001), and this association was confirmed in the multivariate analysis. The occurrence of PES did not impact OS or tumor relapse, although it may have increased non-relapse mortality (P=0.071). The incidence of acute GHVD or treatment-related mortality was not influenced by the choice to use corticosteroids to treat PES. This study suggests that use of CYA for GVHD prophylaxis increases the risk of PES following dual UCB transplantation.

Authors
Kanda, J; Kaynar, L; Kanda, Y; Prasad, VK; Parikh, SH; Lan, L; Shen, T; Rizzieri, DA; Long, GD; Sullivan, KM; Gasparetto, C; Chute, JP; Morris, A; Winkel, S; McPherson, J; Kurtzberg, J; Chao, NJ; Horwitz, ME
MLA Citation
Kanda, J, Kaynar, L, Kanda, Y, Prasad, VK, Parikh, SH, Lan, L, Shen, T, Rizzieri, DA, Long, GD, Sullivan, KM, Gasparetto, C, Chute, JP, Morris, A, Winkel, S, McPherson, J, Kurtzberg, J, Chao, NJ, and Horwitz, ME. "Pre-engraftment syndrome after myeloablative dual umbilical cord blood transplantation: risk factors and response to treatment." Bone Marrow Transplant 48.7 (July 2013): 926-931.
PMID
23334274
Source
pubmed
Published In
Bone Marrow Transplantation
Volume
48
Issue
7
Publish Date
2013
Start Page
926
End Page
931
DOI
10.1038/bmt.2012.279

Diagnosis of 22q11.2 deletion syndrome and artemis deficiency in two children with T-B-NK+ immunodeficiency.

Two infants are described who presented with 22q11.2 deletion and a T(-)B(-)NK(+) immune phenotype. For both infants, the initial diagnosis was athymia secondary to complete DiGeorge anomaly. The first infant underwent thymus transplantation but 6 months after transplantation had circulating thymus donor T cells; the patient did not develop recipient naïve T cells. Genetic analyses revealed that both patients had Artemis deficiency, a rare form of severe combined immunodeficiency (SCID). Both infants have subsequently undergone bone marrow transplantation. These cases illustrate the importance and paradox of differentiating SCID from complete DiGeorge anomaly because hematopoietic stem cell transplantation (HSCT) is the preferred treatment for SCID but is ineffective for complete DiGeorge anomaly. However, if the thymus is completely absent, donor stem cells from a HSCT would not be able to be educated.

Authors
Heimall, J; Keller, M; Saltzman, R; Bunin, N; McDonald-McGinn, D; Zakai, E; de Villartay, J-P; Moshous, D; Ariue, B; McCarthy, EA; Devlin, BH; Parikh, S; Buckley, RH; Markert, ML
MLA Citation
Heimall, J, Keller, M, Saltzman, R, Bunin, N, McDonald-McGinn, D, Zakai, E, de Villartay, J-P, Moshous, D, Ariue, B, McCarthy, EA, Devlin, BH, Parikh, S, Buckley, RH, and Markert, ML. "Diagnosis of 22q11.2 deletion syndrome and artemis deficiency in two children with T-B-NK+ immunodeficiency." J Clin Immunol 32.5 (October 2012): 1141-1144.
PMID
22864628
Source
pubmed
Published In
Journal of Clinical Immunology
Volume
32
Issue
5
Publish Date
2012
Start Page
1141
End Page
1144
DOI
10.1007/s10875-012-9741-9

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

Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).

The Sickle Cell Unrelated Donor Transplant Trial (SCURT trial) of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) is a phase II study of the toxicity and efficacy of unrelated donor hematopoietic cell transplantation in children with severe sickle cell disease (SCD) using a reduced-intensity conditioning regimen. Here we report the results for the cord blood cohort of this trial. Eight children with severe SCD underwent unrelated donor cord blood transplantation (CBT) following alemtuzumab, fludarabine, and melphalan. Cyclosporine or tacrolimus and mycophenolate mofetil were administered for graft-versus-host disease (GVHD) prophylaxis. Donor/recipient HLA match status was 6 of 6 (n = 1) or 5 of 6 (n = 7), based on low/intermediate-resolution molecular typing at HLA -A, -B, and high-resolution typing at -DRB1. Median recipient age was 13.7 years (range: 7.4-16.2 years), and median weight was 35.0 kg (range: 25.2-90.2 kg). The median pre-cryopreservation total nucleated cell dose was 6.4 × 10(7) /kg (range: 3.1-7.6), and the median postthaw infused CD34 cell dose was 1.5 × 10(5) /kg (range: 0.2-2.3). All patients achieved neutrophil recovery (absolute neutrophil count >500/mm(3)) by day 33 (median: 22 days). Three patients who engrafted had 100% donor cells by day 100, which was sustained, and 5 patients had autologous hematopoietic recovery. Six of 8 patients had a platelet recovery to >50,000/mm(3) by day 100. Two patients developed grade II acute GVHD. Of these, 1 developed extensive chronic GVHD and died of respiratory failure 14 months posttransplantation. With a median follow-up of 1.8 years (range: 1-2.6), 7 patients are alive with a 1-year survival of 100%, and 3 of 8 are alive without graft failure or disease recurrence. Based upon the high incidence of graft rejection after unrelated donor CBT, enrollment onto the cord blood arm of the SCURT trial was suspended. However, because this reduced-intensity regimen has demonstrated a favorable safety profile, this trial remains open to enrollment for unrelated marrow donor transplants. Novel approaches aimed at improving engraftment will be needed before unrelated CBT can be widely adopted for transplanting patients with severe SCD.

Authors
Kamani, NR; Walters, MC; Carter, S; Aquino, V; Brochstein, JA; Chaudhury, S; Eapen, M; Freed, BM; Grimley, M; Levine, JE; Logan, B; Moore, T; Panepinto, J; Parikh, S; Pulsipher, MA; Sande, J; Schultz, KR; Spellman, S; Shenoy, S
MLA Citation
Kamani, NR, Walters, MC, Carter, S, Aquino, V, Brochstein, JA, Chaudhury, S, Eapen, M, Freed, BM, Grimley, M, Levine, JE, Logan, B, Moore, T, Panepinto, J, Parikh, S, Pulsipher, MA, Sande, J, Schultz, KR, Spellman, S, and Shenoy, S. "Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN)." Biol Blood Marrow Transplant 18.8 (August 2012): 1265-1272.
PMID
22343376
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
18
Issue
8
Publish Date
2012
Start Page
1265
End Page
1272
DOI
10.1016/j.bbmt.2012.01.019

Reduced-intensity conditioning (RIC) in children with nonmalignant disorders (NMD) undergoing unrelated donor umbilical cord blood transplantation (UCBT).

Authors
Parikh, S; Szabolcs, P
MLA Citation
Parikh, S, and Szabolcs, P. "Reduced-intensity conditioning (RIC) in children with nonmalignant disorders (NMD) undergoing unrelated donor umbilical cord blood transplantation (UCBT)." Biol Blood Marrow Transplant 18.1 Suppl (January 2012): S53-S55. (Review)
PMID
22226113
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
18
Issue
1 Suppl
Publish Date
2012
Start Page
S53
End Page
S55
DOI
10.1016/j.bbmt.2011.10.013

Stem cell source and outcome after hematopoietic stem cell transplantation (HSCT) in children and adolescents with acute leukemia.

Allogeneic hematopoietic stem cell transplantation from siblings, unrelated donors or HLA mismatched family members has become an important procedure to offer a chance of cure to children and adolescents with acute leukemia at high risk of relapse and those with certain genetic diseases. Bone marrow (BM) was the only stem cell source for many years. During the past 15 years, peripheral blood stem cells from granulocyte colony-stimulating factor (G-CSF) mobilized healthy donors, or umbilical cord blood from related or unrelated donors, have become available. Each stem cell source has different risks/benefits for patients and donors, the choice depending not only on availability, but also on HLA compatibility and urgency of the HSCT. This review will analyze the advantages and limitations of each of these options, and the main criteria which can be applied when choosing the appropriate stem cell source for pediatric transplant recipients with acute leukemia.

Authors
Peters, C; Cornish, JM; Parikh, SH; Kurtzberg, J
MLA Citation
Peters, C, Cornish, JM, Parikh, SH, and Kurtzberg, J. "Stem cell source and outcome after hematopoietic stem cell transplantation (HSCT) in children and adolescents with acute leukemia." Pediatr Clin North Am 57.1 (February 2010): 27-46. (Review)
PMID
20307710
Source
pubmed
Published In
Pediatric Clinics of North America
Volume
57
Issue
1
Publish Date
2010
Start Page
27
End Page
46
DOI
10.1016/j.pcl.2010.01.004

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

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome.

Allogeneic stem cell transplantation (SCT) is considered effective in preventing disease progression in patients with Hurler syndrome (HS). Unrelated umbilical cord blood (UCB) grafts are suggested as an alternative to bone marrow (BM) or peripheral blood stem cells (PBSC). We studied 93 HS patients receiving an UCB graft to analyze risk factors for outcomes. The median time from diagnosis to transplant was 4.6 months, median follow-up was 29 months, and median number of nucleated CB cells infused was 7.6 x 10(7)/kg. Most of the patients received 1 or 2 HLA disparate grafts, and the most frequently used conditioning regimen was cyclophosphamide + busulfan (Bu/Cy). All patients received anti-T cell antibody. At post transplant day +60, the cumulative incidence of neutrophil engraftment was 85%. A younger age at transplant and a higher CD34(+) dose at infusion were favorably associated with engraftment. With the exception of 2 patients, all engrafted patients achieved full and sustained donor chimerism. The 3-year event-free survival (EFS) and 3-year overall survival (OS) rates were 70% and 77%, respectively. In a multivariate analyses, use of Bu/Cy and a shorter interval from diagnosis to transplant were predictors for improved EFS rate (82% for patients transplanted within 4.6 months after diagnosis compared to 57% for the rest). Improved outcomes from early transplantation and immediate availability of CB unit lead us to conclude that CB transplantation is a beneficial option, which should be considered expediently for children with HS.

Authors
Boelens, JJ; Rocha, V; Aldenhoven, M; Wynn, R; O'Meara, A; Michel, G; Ionescu, I; Parikh, S; Prasad, VK; Szabolcs, P; Escolar, M; Gluckman, E; Cavazzana-Calvo, M; Kurtzberg, J; EUROCORD, Inborn error Working Party of EBMT and Duke University,
MLA Citation
Boelens, JJ, Rocha, V, Aldenhoven, M, Wynn, R, O'Meara, A, Michel, G, Ionescu, I, Parikh, S, Prasad, VK, Szabolcs, P, Escolar, M, Gluckman, E, Cavazzana-Calvo, M, Kurtzberg, J, EUROCORD, and Inborn error Working Party of EBMT and Duke University, . "Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome." Biol Blood Marrow Transplant 15.5 (May 2009): 618-625.
PMID
19361754
Source
pubmed
Published In
Biology of Blood and Marrow Transplantation
Volume
15
Issue
5
Publish Date
2009
Start Page
618
End Page
625
DOI
10.1016/j.bbmt.2009.01.020

Umbilical Cord Blood: A Reliable Source of Stem and Progenitor Cells for Human Transplantation

Authors
Parikh, SH; Kurtzberg, J
MLA Citation
Parikh, SH, and Kurtzberg, J. "Umbilical Cord Blood: A Reliable Source of Stem and Progenitor Cells for Human Transplantation." Rossi's Principles of Transfusion Medicine: Fourth Edition. March 5, 2009. 559-565.
Source
scopus
Publish Date
2009
Start Page
559
End Page
565
DOI
10.1002/9781444303513.ch36

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

Dasatinib crosses the blood-brain barrier and is an efficient therapy for central nervous system Philadelphia chromosome-positive leukemia.

Although imatinib, a BCR-ABL tyrosine kinase inhibitor, is used to treat acute Philadelphia chromosome-positive (Ph(+)) leukemia, it does not prevent central nervous system (CNS) relapses resulting from poor drug penetration through the blood-brain barrier. Imatinib and dasa-tinib (a dual-specific SRC/BCR-ABL kinase inhibitor) were compared in a preclinical mouse model of intracranial Ph(+) leukemia. Clinical dasatinib treatment in patients with CNS Ph(+) leukemia was assessed. In preclinical studies, dasatinib increased survival, whereas imatinib failed to inhibit intracranial tumor growth. Stabilization and regression of CNS disease were achieved with continued dasa-tinib administration. The drug also demonstrated substantial activity in 11 adult and pediatric patients with CNS Ph(+) leukemia. Eleven evaluable patients had clinically significant, long-lasting responses, which were complete in 7 patients. In 3 additional patients, isolated CNS relapse occurred during dasatinib therapy; and in 2 of them, it was caused by expansion of a BCR-ABL-mutated dasatinib-resistant clone, implying selection pressure exerted by the compound in the CNS. Dasatinib has promising therapeutic potential in managing intracranial leukemic disease and substantial clinical activity in patients who experience CNS relapse while on imatinib therapy. This study is registered at ClinicalTrials.gov as CA180006 (#NCT00108719) and CA180015 (#NCT00110097).

Authors
Porkka, K; Koskenvesa, P; Lundán, T; Rimpiläinen, J; Mustjoki, S; Smykla, R; Wild, R; Luo, R; Arnan, M; Brethon, B; Eccersley, L; Hjorth-Hansen, H; Höglund, M; Klamova, H; Knutsen, H; Parikh, S; Raffoux, E; Gruber, F; Brito-Babapulle, F; Dombret, H; Duarte, RF; Elonen, E; Paquette, R; Zwaan, CM; Lee, FYF
MLA Citation
Porkka, K, Koskenvesa, P, Lundán, T, Rimpiläinen, J, Mustjoki, S, Smykla, R, Wild, R, Luo, R, Arnan, M, Brethon, B, Eccersley, L, Hjorth-Hansen, H, Höglund, M, Klamova, H, Knutsen, H, Parikh, S, Raffoux, E, Gruber, F, Brito-Babapulle, F, Dombret, H, Duarte, RF, Elonen, E, Paquette, R, Zwaan, CM, and Lee, FYF. "Dasatinib crosses the blood-brain barrier and is an efficient therapy for central nervous system Philadelphia chromosome-positive leukemia." Blood 112.4 (August 15, 2008): 1005-1012.
PMID
18477770
Source
pubmed
Published In
Blood
Volume
112
Issue
4
Publish Date
2008
Start Page
1005
End Page
1012
DOI
10.1182/blood-2008-02-140665

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

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

Phenylbutyrate and phenylacetate induce differentiation and inhibit proliferation of human medulloblastoma cells.

PURPOSE: Phenylbutyrate (PB) and phenylacetate (PA) have antiproliferative and differentiation-inducing effects in malignant tumors, and had been evaluated in Phase I/II clinical trials. This study was undertaken to evaluate their antitumor activities in medulloblastomas. EXPERIMENTAL DESIGN: The biological effects of PB and PA, ranging from 0.1 mM to 3 mM, on two medulloblastoma cell lines (DAOY and D283-MED) were examined using various long-term in vitro and in vivo assays for morphology, proliferation, differentiation, anchorage-independent growth, apoptosis, and tumorigenicity. RESULTS: PB and PA can both induce morphological changes and suppress proliferation in a time- and dose-dependent manner. These effects were more pronounced with PB and became irreversible in D283-MED cells after continuous exposure to 3 mM PB for 28 days. Both PB and PA were able to increase expression of glial marker glial fibriliary acidic protein and neuronal marker synaptophysin in two cell lines. For anchorage-independent growth, PB showed a more significant suppression than PA in D283-MED cells. PB caused more pronounced cell cycle arrest and remarkably reduced tumorigenicity in D283-MED cells than in DAOY cells. Apoptosis was readily induced in D283-MED cells with either low dose of PB or short-term treatment. In contrast, much higher concentrations of PB or longer treatment were required to achieve similar effect with DAOY cells. PB induced increased histones H3 acetylation in both cell lines, but histone H4 acetylation was only observed in D283-MED cells. CONCLUSIONS: PB, through induction of hyperacetylation of histone H3 and H4, is a much more potent antitumor agent than PA. 283-MED cells are more responsive to PB than DAOY cells, which may be dependent on their original state of differentiation as well as the changes of histone H4 acetylation status.

Authors
Li, X-N; Parikh, S; Shu, Q; Jung, H-L; Chow, C-W; Perlaky, L; Leung, H-CE; Su, J; Blaney, S; Lau, CC
MLA Citation
Li, X-N, Parikh, S, Shu, Q, Jung, H-L, Chow, C-W, Perlaky, L, Leung, H-CE, Su, J, Blaney, S, and Lau, CC. "Phenylbutyrate and phenylacetate induce differentiation and inhibit proliferation of human medulloblastoma cells." Clin Cancer Res 10.3 (February 1, 2004): 1150-1159.
PMID
14871995
Source
pubmed
Published In
Clinical cancer research : an official journal of the American Association for Cancer Research
Volume
10
Issue
3
Publish Date
2004
Start Page
1150
End Page
1159

Clear cell sarcoma of the kidney: an unusual presentation and review of the literature.

PURPOSE: To describe a child with clear cell sarcoma of the kidney (CCSK) with an unusual presentation, including a primary tumor of the left kidney with metastases to the right kidney and soft tissues of the lower extremities, and to review the literature. PATIENT AND METHODS: An 8-month-old infant presented with hypertension, an abdominal mass, and soft tissue masses in the left thigh and right foot. Imaging studies revealed a large left-sided renal tumor, left paravertebral soft tissue masses, and left thigh mass. At laparotomy, a lesion was noted in the lower pole of the contralateral kidney. CCSK with metastases to the contralateral kidney and to the soft tissues of left thigh, right foot, and left paravertebral region was diagnosed on histopathologic examination. RESULTS: Multimodal oncologic treatment included surgery, chemotherapy, and radiotherapy. Three months after completion of therapy, a soft tissue lesion in the left arm and, later, soft tissue lesions involving multiple parts of the body developed. The patient died 18 months after diagnosis without clinical or radiographic evidence of bone involvement. CONCLUSIONS: In a review of the literature, CCSK is most commonly associated with bone and lung metastases. Soft tissue involvement is uncommon. Metastasis to the contralateral kidney at initial diagnosis has not previously been reported. This case represents an unusual metastatic pattern of CCSK.

Authors
Parikh, SH; Chintagumpala, M; Hicks, MJ; Trautwein, LM; Blaney, S; Minifee, P; Woo, SY
MLA Citation
Parikh, SH, Chintagumpala, M, Hicks, MJ, Trautwein, LM, Blaney, S, Minifee, P, and Woo, SY. "Clear cell sarcoma of the kidney: an unusual presentation and review of the literature." J Pediatr Hematol Oncol 20.2 (March 1998): 165-168. (Review)
PMID
9544171
Source
pubmed
Published In
Journal of Pediatric Hematology/Oncology
Volume
20
Issue
2
Publish Date
1998
Start Page
165
End Page
168

Polymorphonuclear leukocyte functions in children with cyanotic and acyanotic congenital heart disease.

Eighteen cyanotic congenital heart disease (CCHD) and 17 acyanotic congenital heart disease (ACHD) patients in the age range of 2 months to 10 years along with their age and nutrition matched controls were studied for bactericidal, chemotactic and phagocytic functions. Bactericidal and phagocytic functions were significantly depressed in CCHD (p < 0.001) as well as ACHD group (p < 0.001) compared with controls. Chemotactic function was not significantly affected in either. Arterial oxygen content (as a measure of hypoxia) was calculated for each patient and correlated with each immune parameter by univariate linear regression analysis. In CCHD patients linear correlation of borderline significance (p = 0.07) was found between arterial oxygen content and bactericidal activity, but no correlation could be established with phagocytic and chemotactic functions. No correlation was obtained between hematocrit and any of the immune parameters. In ACHD patients no correlations were obtained between the immune parameters and arterial oxygen content or hematocrit. Iron deficiency anemia, known to affect bactericidal function, did not seem to affect the immune parameters in CCHD and ACHD groups. Altered oxygen content of the blood owing to hypoxia in CCHD patients may be an important etiological factor in the genesis of bacteremia and cerebral abscess. The affection of immune functions in ACHD cannot be adequately explained.

Authors
Parikh, S; Bharucha, B; Kamdar, S; Kshirsagar, N
MLA Citation
Parikh, S, Bharucha, B, Kamdar, S, and Kshirsagar, N. "Polymorphonuclear leukocyte functions in children with cyanotic and acyanotic congenital heart disease." Indian Pediatr 30.7 (July 1993): 883-890.
PMID
8132280
Source
pubmed
Published In
Indian Pediatrics
Volume
30
Issue
7
Publish Date
1993
Start Page
883
End Page
890
Show More

Research Areas:

  • Adolescent
  • Adrenoleukodystrophy
  • Anemia
  • Anemia, Hemolytic, Autoimmune
  • Blood Platelets
  • Bone Marrow Transplantation
  • Cause of Death
  • Cell Division
  • Central Nervous System Neoplasms
  • Child
  • Child, Preschool
  • Cognition
  • Common Variable Immunodeficiency
  • Cord Blood Stem Cell Transplantation
  • DiGeorge Syndrome
  • Disease-Free Survival
  • Drug Evaluation, Preclinical
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease
  • Granulocytes
  • Granulomatous Disease, Chronic
  • HLA Antigens
  • Hematologic Neoplasms
  • Hemoglobinopathies
  • Humans
  • Immunologic Deficiency Syndromes
  • Infant
  • Infant, Newborn
  • Kaplan-Meier Estimate
  • Leukemia
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Lymphoproliferative Disorders
  • Male
  • Metabolic Diseases
  • Metabolism, Inborn Errors
  • Mucopolysaccharidoses
  • Multivariate Analysis
  • Mycophenolic Acid
  • Neoplasm Transplantation
  • Neutrophils
  • Pancytopenia
  • Quality of Life
  • Risk Factors
  • Severe Combined Immunodeficiency
  • Sickle Cell Trait
  • Stem Cell Transplantation
  • Stem Cells
  • Survival Rate
  • Thalassemia
  • Tissue and Organ Harvesting
  • Transplantation Chimera
  • Transplantation, Homologous
  • Treatment Outcome
  • Umbilical Cord
  • United States
  • Unrelated Donors
  • Whole-Body Irradiation