Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

Inclusion Criteria

Diagnosis:

• Research participants must be have a diagnosis of Fanconi anemia (confirmed by mitomycin or diepoxybutane [DEB] chromosomal breakage testing).

Hematologic Diagnosis and Status:

• Research participants must have one of the following hematologic diagnoses:
• Severe Aplastic Anemia (SAA)/Severe Isolated Single lineage Cytopenia
• Myelodysplastic Syndrome(MDS)
• Acute leukemia.

HLA-compatible Unrelated volunteer donors:

• Research participants who do not have a related HLA-matched donor but have an unrelated donor who is either matched at all A, B and DRB1 loci or who is mismatched at 1/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.

HLA-mismatched Related donors:

• Research participants who do not have a related or unrelated HLA-compatible donor must have a healthy family member who is at least HLA-haplotype identical to the recipient. First degree related donors must have a normal DEB test.
• The donor must be healthy and willing and able (1) to receive a 5 day course of G-CSF and undergo 2 daily leukaphereses, or (2) to undergo general anesthesia and bone marrow donation. In order to undergo a Tcell depletion, a donor should be able to have a volume of 15 ml/Kg of the research participant's body weight harvested safely.

HLA-compatible Cord Blood Units:

• Research participants who do not have a related HLA-matched donor but have an unrelated placental cord blood unit which matched at all A, B and DRB1 loci or who is mismatched at 1/6 or 2/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
• Research participants may be of either gender or any ethnic background.
• Research participants must have a Karnofsky adult, or Lansky pediatric performance scale status > 70%.
• At the time of referral for transplantation, research participants must be in good clinical condition without co-existing medical problems that would significantly increase the risk of the transplant procedure. Research participants must be free of infections at the time of transplant. Research participants must have a life expectancy that is greater than 8 weeks.
• Research participants must have adequate physical function measured by cardiac, Hepatic, Renal, Pulmonary.
• Research participants must be available for follow-up evaluations at 30, 60, 180 days post BMT and yearly for 5 years.

Exclusion Criteria

• Active CNS leukemic involvement
• Female research participants who are pregnant or breast-feeding
• Active viral, bacterial or fungal infection
• Research participant seropositive for HIV-I/II; HTLV -I/II