title: Excellent long-term survival after allogeneic marrow transplantation in patients with severe aplastic anemia.
abstract: Between 1982 and 1996, 20 patients (10 male, 10 female) with severe aplastic anemia (SAA) with a median age of 25 years (17-37 years), received grafts from an HLA-identical sibling (n = 17), HLA-identical unrelated donor (n = 2) or identical twin (n = 1). The median time from diagnosis to marrow transplantation (BMT) was 15 months (range 1-96 months). More than half of the patients had received more than 10 units of red blood cells or platelet transfusions prior to BMT. Pretransplant immunosuppression consisted of cyclophosphamide (CY) alone (n = 10), CY in combination with total body irradiation (n = 8), and CY and antithymocyte globulin (n = 2). For graft-versus-host disease (GVHD) prophylaxis methotrexate (MTX) alone (n = 9) or MTX with cyclosporin A (n = 10) were given. One patient died on day 18 after marrow grafting due to infection; all other patients had complete and sustained engraftment (95%). Eight patients developed acute GVHD (42%), nine patients chronic GVHD (53%) including four with extensive disease manifestation. One patient experienced a secondary malignancy 11 years after BMT. Eighteen patients followed for a median of 9.45 years (0.42-14.7 years) have sustained hematological reconstitution and are alive and well with a Karnofsky performance score of at least 90%. Thus, excellent long-term survival and low morbidity make allogeneic or syngeneic BMT the treatment of choice for younger patients with severe aplastic anemia.
authors.affiliation: University of Vienna, Department of Medicine I, Austria.
authors.forename: E
authors.initials: E
authors.lastname: Reiter
authors.forename: F
authors.initials: F
authors.lastname: Keil
authors.forename: S
authors.initials: S
authors.lastname: Brugger
authors.forename: P
authors.initials: P
authors.lastname: Kalhs
authors.forename: W
authors.initials: W
authors.lastname: Rabitsch
authors.forename: W
authors.initials: W
authors.lastname: Hinterberger
authors.forename: G
authors.initials: G
authors.lastname: Fischer
authors.forename: A
authors.initials: A
authors.lastname: Rosenmayr
authors.forename: O
authors.initials: O
authors.lastname: Haas
authors.forename: B
authors.initials: B
authors.lastname: Volc
authors.forename: K
authors.initials: K
authors.lastname: Lechner
authors.forename: H T
authors.initials: HT
authors.lastname: Greinix
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000276:immunology; D004200:Diseases in Twins; D005190:Family; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006680:HLA Antigens; D006801:Humans; D019520:Living Donors; D008297:Male; D015996:Survival Rate; D014184:Transplantation, Homologous; D014185:Transplantation, Isogeneic; D014430:Twins, Monozygotic
chemical_list: D006680:HLA Antigens