title: Bone marrow transplantation for severe aplastic anemia: influence of conditioning and graft-versus-host disease prophylaxis regimens on outcome.
abstract: Data for 595 patients with severe aplastic anemia receiving HLA-identical sibling bone marrow transplants were analyzed to determine the effect of pretransplant conditioning and graft-versus-host disease (GVHD) prophylaxis on outcome. Transplants were performed between 1980 and 1987 and reported to the International Bone Marrow Transplant Registry. Three conditioning regimens (cyclophosphamide alone, cyclophosphamide plus limited field radiation, and cyclophosphamide plus total body radiation) were studied; none was associated with superior long-term survival. Three GVHD prophylaxis regimens (methotrexate, cyclosporine, and methotrexate plus cyclosporine) were studied. Recipients of cyclosporine with or without methotrexate had a significantly higher probability of 5-year survival (69%, 95% confidence interval 63% to 74%) than patients receiving methotrexate only (56%, 49% to 62%, P less than .003). Higher survival with cyclosporine resulted from decreased risks of interstitial pneumonia (P less than .0002) and chronic GVHD (P less than .005). Additional risk factors adversely associated with survival included infection pretransplant (P less than .004), use of parous or transfused female donors (P less than .005), older patient age (P less than .005), and 20 or more pretransplant transfusions (P less than .006). These data may prove useful in planning randomized clinical trials and in identifying patients at high-risk of treatment failure.
authors.affiliation: International Bone Marrow Transplant Registry, Medical College of Wisconsin 53226.
authors.forename: E
authors.initials: E
authors.lastname: Gluckman
authors.forename: M M
authors.initials: MM
authors.lastname: Horowitz
authors.forename: R E
authors.initials: RE
authors.lastname: Champlin
authors.forename: J M
authors.initials: JM
authors.lastname: Hows
authors.forename: A
authors.initials: A
authors.lastname: Bacigalupo
authors.forename: J C
authors.initials: JC
authors.lastname: Biggs
authors.forename: B M
authors.initials: BM
authors.lastname: Camitta
authors.forename: R P
authors.initials: RP
authors.lastname: Gale
authors.forename: E C
authors.initials: EC
authors.lastname: Gordon-Smith
authors.forename: A M
authors.initials: AM
authors.lastname: Marmont
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000601:surgery*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006801:Humans; D007165:Immunosuppression Therapy; D007223:Infant; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D014916:Whole-Body Irradiation
chemical_list: D016572:Cyclosporine; D003520:Cyclophosphamide; D008727:Methotrexate