Last updated on January 2019

Cytoxan Fludara and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Fanconi's Anemia
  • Age: - 59 Years
  • Gender: Male or Female

Inclusion Criteria:

  • Patients must be <60 years of age with a diagnosis of Fanconi Anemia (FA).
  • Patients must have an HLA-A, B, DRB1 identical sibling 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 moderately severe aplastic anemia (AA), early myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal abnormalities.
  • In patients <18 years of age, moderately severe aplastic anemia is defined as having at least one of the following:
  • platelet count <40 x 10^9/L
  • absolute neutrophil count (ANC) <10 x 10^8/L
  • Hgb <9 g/dL
  • In patients 18-60 years of age, moderately severe aplastic anemia is defined as having at least one of the following:
  • platelet count <20 x 10^9/L
  • absolute neutrophil count ANC <5 x 10^8/L
  • Hgb <8 g/dL
  • Early myelodysplastic syndrome, with multilineage dysplasia with < 5% blasts, with or without chromosomal anomalies.
  • Adequate major organ function including:
  • Cardiac: ejection fraction >45%
  • Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
  • Karnofsky performance status >70% or Lansky >50%
  • Women of child bearing age must be using adequate birth control and have a negative pregnancy test.

Exclusion Criteria:

  • Active bacterial infection within one week of hematopoietic cell transplant (HCT)
  • Active fungal infection at time of HCT.
  • Late MDS with greater than 5% blasts in bone marrow.
  • Acute myelogenous 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.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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