BMT CTN 2402 HOPE Resource
What is the Purpose of this Study?
This study is not testing a new treatment. People will continue to receive their usual medical care, such as a stem cell transplant or gene therapy. If they join the study, they will give blood samples during their regular visits over about five years. They may also choose to give other samples, such as stool, skin, hair, nails, or bone marrow. These samples will help researchers learn more about how the body responds to these treatments over time.
Non-cancer blood disorders such as sickle cell disease, thalassemia, aplastic anemia, and other bone marrow failure diseases.
Who Can Participate in the Study?
People can join this study if they have a blood disease that is not cancer, such as sickle cell disease, thalassemia, or aplastic anemia. They must be planning to receive a stem cell transplant or gene therapy. They need to be able to come to study visits and follow study steps. They must be willing to give blood samples and agree to be followed for up to five years so researchers can learn how treatments work over time.
What is Involved?
This study collects samples from people who have serious blood diseases and are getting treatments like a stem cell transplant or gene therapy. The goal is to learn why these treatments work better for some people and not for others. Researchers also want to understand why some people have problems after treatment. By studying these samples over time, doctors hope to improve care and make treatments safer and more effective for future patients.