EXCELLENT LONG-TERM SURVIVAL AFTER ALLOGENEIC MARROW TRANSPLANTATION IN PATIENTS WITH SEVERE APLASTIC-ANEMIA

Between 1982 and 1996, 20 patients (10 male, 10 female) with severe ap lastic anemia (SAA) with a median age of 25 years (17-37 years), recei ved grafts from an HLA-identical sibling (n = 17), HLA-identical unrel ated donor (n = 2) or identical twin (n = 1), The median time from dia gnosis to marrow transplantation (BMT) was 15 months (range 1-96 month s), More than half of the patients had received more than 10 units of red blood cells or platelet transfusions pries to BMT, Pretransplant i mmunosuppression consisted of cyclophosphamide (CY) alone (n = 10), CY in combination with total body irradiation (n = 8), and CY and antith ymocyte globulin (n = 2), For graft-versus-host disease (GVHD) prophyl axis methotrexate (MTX) alone (n = 9) or MTX with cyclosporin A (n = 1 0) 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 chroni c GVHD (53%) including four with extensive disease manifestation, One patient experienced a secondary malignancy 11 years after BMT. Eightee n patients followed for a median of 9.45 years (0.42-14.7 Sears) have sustained hematological reconstitution and are alive and well with a K arnofsky performance score of at least 90%, Thus, excellent long-term survival and low morbidity make allogeneic or syngeneic BMT the treatm ent of choice for younger patients with severe aplastic anemia.