Bone marrow transplantation for β-thalassaemia major: the UK experience in two paediatric centres

Summary. Stem cell transplantation (SCT) remains the only cure for thalassaemia major. Recent advances in medical treatment make it even more important that accurate information is available regarding outcome of SCT in relevant patient populations in order to guide informed decisions regarding the most appropriate treatment for individual thalassaemia patients. We report the results of 55 consecutive first related allogeneic bone marrow transplants (BMT) for children with β-thalassaemia major performed in two UK paediatric centres over 10 years. Between February 1991 and February 2001, 55 children underwent 57 allogeneic BMT. The median age at BMT was 6·4 years and the majority of patients (73%) originated from the Indian subcontinent. Using the Pesaro risk classification, 17 patients were class 1, 27 were class 2 and 11 were class 3. Actuarial overall survival and thalassaemia-free survival at 8 years were 94·5% (95% CI 85·1–98·1) and 81·8% (95% CI 69·7–89·8) respectively. Despite the majority of patients being in class 2 or 3, transplant-related mortality was low (5·4%). The principal complication was graft rejection accompanied by autologous reconstitution that occurred in 13·2% of transplants. Following modification of the conditioning regimen in 1993, the rejection rate fell to 4·6% and remained low. Acute graft-versus-host disease (GVHD) of grade II–IV occurred in 31% and chronic GVHD in 14·5%. These data compare favourably with survival with medical treatment for thalassaemia major and suggest that allogeneic BMT remains an important treatment option for children with β-thalassaemia major, particularly when compliance with iron chelation is poor.