BMT FOR SEVERE APLASTIC-ANEMIA USING CYCLOSPORINE

Between 1984 and 1991 24 patients with severe aplastic anemia (SAA) we re transplanted with HLA identical sibling donor BM. The overall long- term survival was 79 +/- 8%. The average age was 21 years (range 4-53 years) and the median pre-transplant disease duration was 35 days (ran ge 12-2998 days). Over one-half (15 of 24) of the patients had receive d > 10 units of blood product transfusions prior to BMT. The pre-trans plant conditioning regimen consisted of 200 mg/kg cyclophosphamide (CY ). Cyclosporine (CYA) was administered from 2 days prior to BMT and co ntinued for 6-12 months. Two of the 24 patients failed to achieve prim ary engraftment (FTE). One of these patients had autologous recovery o f BM function and is alive and well. Five of the 22 patients who engra fted failed to sustain engraftment (FTSE). Of these, three are alive a nd well following a second BMT or marrow boost. Only 1 of the 22 patie nts who engrafted had clinically significant (i.e. Stage 11-IV) acute GVHD. No patient developed chronic GVHD. Our results indicate that BMT following a regimen consisting of CY with the continuous use of CYA i n the post-transplant period is well tolerated and associated with exc ellent long-term survival. The high incidence of secondary graft insta bility (i.e. FTSE), however, suggests that future studies should focus on post-transplanation immunomodulation.