title: Bone marrow transplantation in patients with Fanconi anemia: experience with cyclophosphamide and total body irradiation conditioning regimen.
abstract: Eleven patients with Fanconi anemia (FA) underwent bone marrow transplantation (BMT) between March 1985 and May 1990 in a single institution. Ten patients received bone marrow from healthy full human leukocyte antigen (HLA) matched siblings and one patient from her father (one antigen mismatch). Ten patients were conditioned with cyclophosphamide (Cy) at a dose of 5 mg/kg per day for 4 days followed by total body irradiation (TBI) for a total of 600 cGy over 3 days. Six of the 11 patients are alive and have normal reconstitution of their bone marrow. Median follow-up was 72 months (range 42-84). Three of the 10 patients who received Cy and TBI (two HLA compatible, one antigen mismatch) had graft failure. Five patients developed at least grade III acute graft-versus-host disease (GVHD). The rates of graft failure and GVHD are, however, still significantly high. Modification of the conditioning regimen and GVHD prophylaxis is needed to improve the outcome.
authors.affiliation: Department of Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
authors.forename: H
authors.initials: H
authors.lastname: Solh
authors.forename: K
authors.initials: K
authors.lastname: Rao
authors.forename: A M
authors.initials: AM
authors.lastname: Martins da Cunha
authors.forename: A
authors.initials: A
authors.lastname: Padmos
authors.forename: K
authors.initials: K
authors.lastname: Sackey
authors.forename: P
authors.initials: P
authors.lastname: Ernst
authors.forename: D
authors.initials: D
authors.lastname: Spence
authors.forename: H
authors.initials: H
authors.lastname: Clink
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014916:Whole-Body Irradiation
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide