Hematopoietic Stem Cell Transplant for Fanconi Anemia
Status:
Completed
Trial end date:
2016-08-30
Target enrollment:
Participant gender:
Summary
The trial proposed is a single arm phase II treatment protocol designed to examine
engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival
following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine
and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune
system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia
(SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking
HLA-genotypically identical donors using stem cell transplants derived from (1)
HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.