Bone marrow transplantation in Fanconi's anemia.
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.
Gluckman
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Unité de Greffe de Moelle, Hôpital Saint Louis, Paris, France.
D000206:Actuarial Analysis; D000293:Adolescent; D000328:Adult; D016913:Blood Component Transfusion; D016465:Bone Marrow Purging; D016026:Bone Marrow Transplantation / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide; D005199:Fanconi Anemia / Q000401:mortality / Q000601:surgery*; D005312:Fetal Blood / Q000166:cytology; D006084:Graft Rejection; D006086:Graft vs Host Disease / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015182:Lymphatic Irradiation; D015996:Survival Rate; D016896:Treatment Outcome
D003520:Cyclophosphamide