Sickle Cell Anemia Clinical Trial
Official title:
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
The purpose of this study is to determine and confirm the role of bone marrow transplantation in the treatment of disorders of the red cell and hemoglobin including sickle cell anemia, thalassemia and diamond blackfan anemia.
The trial proposed is a single armed phase II treatment protocol designed to examine the
engraftment,toxicity and graft-versus-host disease following a novel cytoreductive regimen
including cyclophosphamide and Busulfan for the treatment of patients with Severe Sickle
Cell Anemia,Thalassemia, and Diamond Blackfan Anemia using stem cell transplants derived
from HLA-genotypically identical siblings.
Patients will be conditioned for transplantation with cyclophosphamide (50 mg/kg/day x 4
days), and busulfan [(if < 4 years of age 1 mg/kg 4 times per day x 4 days), (if > 4 years
of age 0.8 mg/kg 4 times per day x 4 days)]. Patients will receive Methotrexate &
Cyclosporin-A for prophylaxis against GvHD and GCSF to promote engraftment.
The preferred source of stem cells from related HLA-matched related donors will be
unmodified bone marrow stem cells.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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