Bone marrow transplantation in fanconi's anemia

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.