title: Bone marrow transplantation for beta-thalassemia: the University of California San Francisco experience.
abstract: We report the results of allogeneic HLA-matched family donor stem cell transplantation in 17 North American children with beta-thalassemia major or hemoglobin E/beta-thalassemia who received transplants at the University of California San Francisco. Pretransplantation conditioning was with busulfan, cyclophosphamide, and antithymocyte globulin and graft-versus-host prophylaxis used cyclosporine, usually with added methotrexate. Twelve children are alive, well, and free of the clinical manifestations of thalassemia. Four experienced graft rejection and autologous recovery, and one died from complications shortly after transplantation. Overall survival was 94% and event-free survival 71%, results similar to those obtained by other transplantation centers. Improved conditioning regimes and enlargement of the donor pool will be needed to improve the outcome of transplantation and to make this treatment available to more children with thalassemia.
authors.affiliation: Department of Pediatrics, University of California San Francisco, USA. wmentzer@sfghpeds.ucsf.edu
authors.forename: W C
authors.initials: WC
authors.lastname: Mentzer
authors.forename: M J
authors.initials: MJ
authors.lastname: Cowan
mesh_terms: D000293:Adolescent; D001208:Asia / Q000208:ethnology; D016026:Bone Marrow Transplantation / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D006084:Graft Rejection / Q000453:epidemiology; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006785:Hospitals, University; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008877:Middle East / Q000208:ethnology; D012189:Retrospective Studies; D012495:San Francisco; D015996:Survival Rate; D014184:Transplantation, Homologous; D017086:beta-Thalassemia / Q000401:mortality / Q000628:therapy*
chemical_list: D007166:Immunosuppressive Agents