title: [Bone marrow transplantation in Fanconi's anemia. Results in 5 patients].
abstract: Fanconi's anaemia (FA) is a form of congenital bone marrow failure characterised by progressive pancytopenia and course usually fatal. Bone marrow transplantation (BMT) offers a chance to restore haemopoiesis, as in bone marrow aplasia of any other aetiology. Five children (4M, 1F) with FA were subjected to BMT. Their ages ranged between 4 and 13 years. The time elapsed between diagnosis and BMT ranged between 6 months and 10 years. Three patients had been previously treated with prednisolone and oxymetholone. The donor age ranged from 20 months to 25 years. The correlation donor sex/recipient sex was female/male in 4 cases (siblings, 3; mother, 1) and male/female in the remainder (one brother). All the siblings involved were HLA-identical, with a negative mixed lymphocyte culture; the mother shared 5 antigens with a different DR. The conditioning regimen was that proposed by Gluckman. BMT was performed by infusion of 2.2 to 5.3 x 10(8) bone marrow cells per kg. Cyclosporin A was used in the prophylaxis of graft versus cells host disease (GVHD), associated to methotrexate in one case and to prednisone in another. One patient showed no signs of GVHD; the onset of GVHD took place between days +8 and +18, and was grade II-III in the other four. Three patients had limited chronic GVHD. Four patients are alive and in good clinical conditions at 11 to 63 months post-BMT. The remainder, who had stage III GVHD, died on day +45. The favourable results of BMT in FA have been confirmed, so this procedure should be considered as the best choice in the treatment of FA.
authors.affiliation: Unidad de Hematología, Hospital Matermo-Infantil, C.S. Vall d'Hebron, Barcelona.
authors.forename: J J
authors.initials: JJ
authors.lastname: Ortega
authors.forename: T
authors.initials: T
authors.lastname: Olivé
authors.forename: C
authors.initials: C
authors.lastname: Sánchez
authors.forename: J
authors.initials: J
authors.lastname: Giralt
authors.forename: E
authors.initials: E
authors.lastname: Serret
mesh_terms: D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003524:Cyclosporins / Q000627:therapeutic use; D005069:Evaluation Studies as Topic; D005199:Fanconi Anemia / Q000150:complications / Q000601:surgery*; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D008113:Liver Neoplasms / Q000150:complications; D008297:Male
chemical_list: D003524:Cyclosporins
vernacular_title: Trasplante de medula ósea en anemia de Fanconi. Resultados en cinco pacientes.