Hematopoietic Stem Cell Transplant for Fanconi Anemia

A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

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.

We are currently recruiting patients.

Inclusion Criteria:

• Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia
• Karnofsky or Lansy performance scale > or = to 70%.
• Must have adequate cardiac, hepatic, renal and pulmonary function.
• Must have 7/8 or 8/8 available unrelated donor.

Exclusion Criteria:

• Pregnant or breastfeeding.
• Active CNS leukemic involvement
• Active uncontrolled viral, bacterial or fungal infection
• Positive for HIV.