Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders
Prior to transplantation, subjects will receive the drugs busulfan (orally or through the
catheter), as well as fludarabine and anti-thymocyte globulin (ATG) via the catheter.
Busulfan, fludarabine and ATG will be given with Total Lymphoid Irradiation (TLI) to help
the new donor bone marrow take and grow after transplantation.
Those patients receiving donor marrow will have the T cells (a type of white blood cell in
the donor marrow) removed to lower the risk that the new marrow will react to their body, a
condition called Graft-Versus-Host-Disease (GVHD). After bone marrow transplantation,
subjects will receive drugs to help prevent GVHD, including cyclosporin and mycophenolate
Blood samples are taken at day 28, day 60, day 100, 1 year and as required by medical status
yearly for five years after transplant to evaluate how well the new marrow is growing. A
bone marrow biopsy is required at day 21, at day 100 and 1 year.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Patients Alive (Survival) at 2 Years
Calculated from day 1 of transplant to last contact.
Paul Orchard, MD
University of Minnesota Medical Center
United States: Institutional Review Board
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