title: Marrow transplantation for Fanconi anaemia: conditioning with reduced doses of cyclophosphamide without radiation.
abstract: Nine patients with Fanconi anaemia (FA) were conditioned for HLA-identical sibling bone marrow transplant (BMT) with reduced dose of cyclophosphamide (Cy) without radiation or antithymocyte globulin (ATG). The total dose of Cy was 140 mg/kg (n = 2) or 120 mg/kg (n = 7). The median patient age was 8 years (range 4-19). Graft-versus-host disease (GVHD) prophylaxis was with methotrexate and cyclosporine (n = 8) or cyclosporine alone (n = 1). All patients had sustained engraftment and two developed grade>/= II acute GVHD. Cy toxicity included grade >/= 2 mucositis seen in all evaluable patients and haemorrhagic cystitis in two patients. The Kaplan-Meler survival estimate is 89% with a median follow-up of 285 d (range 56-528). For the purpose of comparison, this report also reviews and updates long-term follow-up data on 32 previously reported FA patients conditioned with 140-200 mg Cy/kg without radiation. The lowest dose of Cy (without radiation or ATG) after which HLA-identical sibling marrow transplant can be successfully performed in FA patients has yet to be determined, but it appears that uniform and sustained engraftment can be achieved with a Cy dose of as low as 120 mg/kg.
authors.affiliation: Fred Hutchinson Cancer Research Center, Seattle, Washington 98104, USA.
authors.forename: M E
authors.initials: ME
authors.lastname: Flowers
authors.forename: J
authors.initials: J
authors.lastname: Zanis
authors.forename: R
authors.initials: R
authors.lastname: Pasquini
authors.forename: H J
authors.initials: HJ
authors.lastname: Deeg
authors.forename: R
authors.initials: R
authors.lastname: Ribeiro
authors.forename: G
authors.initials: G
authors.lastname: Longton
authors.forename: C R
authors.initials: CR
authors.lastname: Medeiros
authors.forename: K
authors.initials: K
authors.lastname: Doney
authors.forename: J
authors.initials: J
authors.lastname: Sanders
authors.forename: J
authors.initials: J
authors.lastname: Bryant
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male
chemical_list: D003520:Cyclophosphamide