Diamond-Blackfan Anemia Clinical Trial
Official title:
Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment 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
mofetil (MMF).
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.
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