Stem Cell Transplantation for Fanconi Anemia

All subjects will be given the same treatment regimen of total body irradiation (TBI),
Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate
donor stem cell transplant. Since this treatment regimen has been given before, without
thymic shielding, we will compare the outcomes of these patients with the historical data
from subjects who did not receive thymic shielding.

Inclusion Criteria:

- Patients must be less than (<) 18 years of age with a diagnosis of Fanconi anemia.

- Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal
to (≤)1 antigen mismatched related (non-HLA-matched sibling) or <1 antigen mismatched
unrelated UCB donor. Patients and donors will be typed for HLA-A and B using
serological or molecular techniques and for DRB1 using high resolution molecular
typing.

- Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without
excess blasts, or high risk genotype as defined below.

- Aplastic anemia is defined as having at least one of the following when not
receiving growth factors or transfusions

- Platelet count <20 x 10^9/L

- ANC <5 x 10^8/L

- Hgb <8 g/dL

- Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal
anomalies

- High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2
mutations)

- Adequate major organ function including

- Cardiac: ejection fraction greater than (>)45%

- Hepatic: bilirubin, AST/ALT, ALP <2 x normal

- Karnofsky performance status >70% or Lansky performance status >50%

- Women of child-bearing age must be using adequate birth control and have a negative
pregnancy test

Exclusion Criteria:

- Available HLA-genotypically identical related donor

- History of gram negative sepsis or systemic fungal infection (proven or suspected
based on radiographic studies)

- Refractory anemia with excess blasts, or leukemia

- Active central nervous system (CNS) leukemia at time of hematopoietic cell transplant
(HCT)

- History of squamous cell carcinoma of the head/neck/cervix within 2 years of HCT

- Pregnant or lactating female

- Prior radiation therapy preventing use of total body irradiation (TBI) 450 centigray
(cGy)