HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
- To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell
transplantation after reduced-intensity conditioning regimen comprising busulfan,
fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure
- Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7
and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte
globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30
minutes starting 30 minutes before ATG on days -4 to -1.
- HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive
donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.
- Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4
hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral
medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the
absence of GVHD, cyclosporine is tapered starting between days 30 to 60.
After completion of study treatment, patients are followed periodically for 1 year.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Donor cell engraftment
neutrophil count over 500/ul
10-35 days after transplantation
Kyoo H. Lee, MD
Asan Medical Center
Korea: Food and Drug Administration