BONE-MARROW TRANSPLANTATION FOR THALASSEMIA - EXPERIENCE OF 2 BRITISHCENTERS

Bone marrow transplantation (BMT) was carried out on 38 patients with thalassaemia major over a period of 9 years; 30 were Asian. In all cas es, the donor was an HLA-identical relative. The mean age at transplan t was 6.4 years (range 0.5-20 years). Conditioning was busulphan and c yclophosphamide (CY). Cyclosporin (CsA) (n = 30), CsA + methotrexate ( n = 6) or CsA + T cell depletion (n = 2) were used for prophylaxis aga inst graft-versus-host disease (GVHD). Thirty-four patients successful ly engrafted. Two patients required a second transplant and two achiev ed mixed chimerism, eventually rejecting their grafts. Nine patients ( 23.6) developed acute GVHD grade III-IV. Eleven patients (28.9) develo ped chronic GVHD. There were 11 deaths, 7 within the first 100 days po st-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.