Combination Chemotherapy and Stem Cell Transplantation in Treating Patients with Fanconi Anemia

Inclusion Criteria

• Diagnosis of FA
• Patients must have an HLA-A, B, DRB1 identical sibling donor; related donors will be evaluated under the University of Minnesota donor protocol MT2012-14C; patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing; if a matched sibling marrow donor is not available, umbilical cord blood (UCB) may be used as a cell source; if unlicensed cord blood unit is used, patients will co-enroll on MT2011-13R: Infusion of Cell Populations from Unlicensed Umbilical Cord Blood Units
• Patient with FA must have moderately severe aplastic anemia (AA), early myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal abnormalities
• Platelet count < 20 x 10^9/L
• Absolute neutrophil count (ANC) < 5 x 10^8/L
• Hemoglobin (Hgb) < 8 g/dL
• Early myelodysplastic syndrome with multilineage dysplasia with < 5% blasts, with or without chromosomal anomalies
• Ejection fraction >= 45%
• No clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
• Karnofsky performance status >= 70% or Lansky >= 50% (for patients < 16 years)
• Women of child bearing age must be using adequate birth control and have a negative pregnancy test
• Written consent

Exclusion Criteria

• Active bacterial infection within one week of HCT
• Active fungal infection at time of HCT
• Late MDS with greater than 5% blasts in bone marrow
• Acute myeloid leukemia (AML) or history of AML
• Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2 years of HCT
• Pregnant or lactating female