title: Bone marrow transplantation for thalassaemia: experience of two British centres.
abstract: Bone marrow transplantation (BMT) was carried out on 38 patients with thalassaemia major over a period of 9 years; 30 were Asian. In all cases, the donor was an HLA-identical relative. The mean age at transplant was 6.4 years (range 0.5-20 years). Conditioning was busulphan and cyclophosphamide (CY). Cyclosporin (CsA) (n = 30), CsA + methotrexate (n = 6) or CsA + T cell depletion (n = 2) were used for prophylaxis against graft-versus-host disease (GVHD). Thirty-four patients successfully engrafted. Two patients required a second transplant and two achieved mixed chimerism, eventually rejecting their grafts. Nine patients (23.6) developed acute GVHD grade III-IV. Eleven patients (28.9) developed chronic GVHD. There were 11 deaths, 7 within the first 100 days post-BMT. Twenty-seven patients are alive from 156 to 3213 days post-BMT. The actuarial survival at 9 years post-BMT was 70%. The mortality is higher than in previously reported series; possible reasons for this are discussed.
authors.affiliation: Westminster Children's Hospital, London, UK.
authors.forename: A
authors.initials: A
authors.lastname: Vellodi
authors.forename: S
authors.initials: S
authors.lastname: Picton
authors.forename: C J
authors.initials: CJ
authors.lastname: Downie
authors.forename: M
authors.initials: M
authors.lastname: Eltumi
authors.forename: R
authors.initials: R
authors.lastname: Stevens
authors.forename: D I
authors.initials: DI
authors.lastname: Evans
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D013789:Thalassemia / Q000401:mortality / Q000628:therapy*