A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Anemia and Other Hemoglobinopathies
- 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
- 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
- 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. Donors
receive filgrastim (G-CSF) subcutaneously beginning on day -5 to day -1.
- 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 25 patients will be accrued for this study.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Transplant-related mortality at day 100 and 1 year after bone marrow transplantation (BMT)
1 year after bone marrow transplant
Javier Bolanos-Meade, MD
Sidney Kimmel Comprehensive Cancer Center
United States: Institutional Review Board
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Baltimore, Maryland 21231-2410|