Bone-marrow transplantation for thalassaemia.

An 18-month-old boy with beta-thalassaemia major underwent bone-marrow transplantation with marrow from his 30-month-old brother. The brother was HLA-identical, mixed-lymphocyte culture non-reactive and had thalassaemia minor. The patient was "conditioned" with busulphan and cyclophosphamide before transplantation and received methotrexate to prevent graft-versus-host disease. Immediately after the transplant, complications arose, which included mild graft-versus-host disease, gastrointestinal bleeding and fever. The boy is alive 18 months after transplantation, is leading a normal life, is receiving no therapy and has a normally functioning donor marrow with thalassaemia minor. Bone-marrow transplantation may be considered as alternative therapy in patients with beta-thalassaemia who are young, and who have no organ dysfunction or iron overload. Chronic transfusion and chelation therapy and its problems must be weighed against the 13% risk of mortality and the 73% chance of a normal life that are associated with transplantation. Older patients, who have received multiple blood transfusions, have iron overload or have organ dysfunction, have a low survival rate after transplantation and this therapy is inappropriate for such patients.