Open AccessBone marrow transplantation in fanconi's anemia
Author(s) -
Gluckman E.
Publication year - 1993
Publication title -
stem cells
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.159
H-Index - 229
eISSN - 1549-4918
pISSN - 1066-5099
DOI - 10.1002/stem.5530110829
Subject(s) - pancytopenia , fanconi anemia , biology , cyclophosphamide , transplantation , savior sibling , bone marrow failure , bone marrow , aplastic anemia , anemia , conditioning regimen , bone marrow transplantation , total body irradiation , immunology , gastroenterology , medicine , haematopoiesis , stem cell , chemotherapy , hematopoietic stem cell transplantation , dna repair , genetics , gene
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.