Finding studies
Finding studies
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Lead
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
With
OBJECTIVES: * Determine the transplant-related mortality and progression-free survival of patients with severe hemoglobinopathies receiving nonmyeloablative conditioning comprising fludarabine phosphate, cyclophosphamide, and total-body irradiation followed by partially HLA-mismatched bone marrow transplantation from first-degree relatives or HLA-matched donors. * Characterize donor hematopoietic chimerism at 30, 60, and 180 days after transplantation in these patients. * Determine the hematologic and non-hematologic toxicity of this regimen in these patients. OUTLINE: * Preparative regimen: Patients receive fludarabine phosphate IV over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1-2 hours on days -6 and -5. Patients also undergo total-body irradiation on day -1. * Bone marrow transplantation: Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV over 1-2 hours on days 3 and 4. * Graft-versus-host disease prophylaxis: Patients receive sirolimus orally daily on days 5-365 and oral mycophenolate mofetil 3 times a day on days 5-35. After completion of study treatment, patients are followed periodically. PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study.
Age
2–70
Sex
ALL
Healthy volunteers
Accepted
