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E. Gluckman, Bone marrow transplantation in fanconi's anemia, Stem Cells, Volume 11, Issue S2, 1993, Pages 180–183, https://doi.org/10.1002/stem.5530110829
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Abstract
Fanconi's anemia (FA), a disease characterized by malformations and progressive pancytopenia, can be successfully cured by allogeneic bone marrow transplantation. Due to the sensitivity of FA cells to alkylating agents, a modified conditioning regimen including low-dose cyclophosphamide (20 mg/kg) and 5 Gy thoracoabdominal irradiation has been used. We report here our experience with bone marrow transplantation in a series of 49 patients. In HLA-identical sibling transplants, the long-term survival was 75%. Results with matched unrelated transplants are limited by the small number of patients.