Thalassemia Clinical Trial
Official title:
Allogeneic Stem Cell Transplantation Following Nonmyeloablative Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.
Hemoglobinopathies, such as sickle cell disease and thalassemia major, are genetic diseases
associated with significant morbidity and premature death. Allogeneic bone marrow
transplantation (BMT) is the only potential cure for severe hemoglobinopathies. Typical
regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient
hematopoiesis and to prevent graft rejection. The widespread use of this treatment has been
limited by toxicity, risk of end-organ damage, and donor availability. This study will use a
nonmyeloablative regimen of fludarabine and busulfan to attempt to generate consistent
engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy.
G-CSF mobilization of the donor's peripheral blood white blood cells will precede donor
apheresis. A nonmyeloablative conditioning regimen of fludarabine and busulfan will be
administered to patients prior to allogeneic peripheral blood stem cell infusions. FK506 and
prednisone will be administered for graft versus host disease (GVHD) prophylaxis. Patients
will be evaluated for engraftment, donor: host hematopoietic chimerism, toxicity, and
hemoglobinopathy.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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