Bone marrow transplantation for beta-thalassemia: The University of California San Francisco experience

We report the results of allogeneic HLA-matched family donor stem cell tran splantation in 17 North American children with beta -thalassemia major or h emoglobin E/beta -thalassemia who received transplants at the University of California San Francisco. Pretransplantation conditioning was with busulfa n, cyclophosphamide, and antithymocyte globulin and graft-versus-host proph ylaxis used cyclosporine, usually with added methotrexate. Twelve children are alive, well, and free of the clinical manifestations of thalassemia. Fo ur experienced graft rejection and autologous recovery, and one died from c omplications shortly after transplantation. Overall survival was 94% and ev ent-free survival 71%, results similar to those obtained by other transplan tation centers. Improved conditioning regimes and enlargement of the donor pool will be needed to improve the outcome of transplantation and to make t his treatment available to more children with thalassemia.