Last updated on February 2019

Hematopoietic Stem Cell Transplantation in High Risk Patients With 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: - 44 Years
  • Gender: Male or Female

Inclusion Criteria:

  • Patients must be <45 years of age with a diagnosis of Fanconi anemia with:
  • Biallelic BRCA2 mutations, or
  • Aplastic anemia, or advanced myelodysplastic syndrome (MDS) (MDS with 5% blasts), or acute leukemia who are ineligible for total body irradiation. Aplastic anemia is defined as having at least one of the following (with or without cytogenetic abnormalities): platelet count <20 * 10^9, - absolute neutrophil count (ANC) <5 * 10^8/L, - Hgb <8 g/dL /
  • Patients must have an HLA-A, B, DRB1 identical or 1 antigen mismatched related or unrelated BM donor or have an HLA-A, B, DRB1 identical, 1 antigen or 2 antigen mismatched related or unrelated umbilical cord blood (UCB) donor. Patients and donors will be typed for HLA-A and B using serological level typing and for DRB1 using high resolution molecular typing.
  • Adequate major organ function including:
  • Cardiac: ejection fraction >45%
  • Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites, no cirrhosis)
  • Karnofsky performance status >70% or Lansky >50%
  • Women of child bearing potential must be using adequate birth control and have a negative pregnancy test.

Exclusion Criteria:

  • Active CNS leukemia at time of HSCT.
  • Active uncontrolled infection within one week of hematopoietic stem cell transplant (HSCT).
  • Pregnant or lactating female.

Donor Inclusion Criteria:

  • Donor must be in good health based on review of systems and results of physical examination.
  • Donor must have a normal hemoglobin, white count, platelet count and partial thromboplastin time (PTT), and a negative diepoxybutane (DEB) test.
  • HIV-NAT negative, HTLV-1, HTLV-2 negative, Hepatitis B and C negative.
  • Female donors of childbearing potential must have a negative pregnancy test.
  • Unrelated donors must agree to peripheral blood stem cell (PBSC) donation

Donor Exclusion Criteria:

  • Donor is a lactating female.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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