ABSTRACT
BACKGROUND: We performed this study to compare response rate and survival according to treatment modalities, such as allogeneic bone marrow transplantation (BMT), immunosuppressive therapy (IS) and androgen therapy in patients with aplastic anemia (AA). METHODS: Medical records of one hundred and thirty-seven patients who diagnosed with aplastic anemia at the Asan Medical Center from September 1989 to December 2000 were retrospectively analyzed. RESULTS: Forty-one patients received supportive care only. Ninety-five out of 96 treated patients were evaluable for response. In severe AA group (N=79), the response rate according to treatment modalities was 25.0% in the androgen group (N=4), 30.3% in the ALG or ATG group (N=33), 25.0% in the cyclosporine (CSA) group (N=8), 44.4% in the ATG plus CSA group (N=9), and 73.8% in the BMT group (N=23) (P<0.001). Five- and 10-year survival of overall patients was 67.5% and 50.9%, respectively. Ten-year survival according to disease severity was 42.3% in severe AA group and 75.8% in non-severe AA group (P=0.0043). Five- year survival according to treatment modalities in patients with severe AA was 75.0% in the androgen group, 49.0% in the ALG or ATG group, 75.0% in the CSA group, and 88.9% in the ATG plus CSA group. Five-year survival was 82.6% in the BMT group and 57.7% in the IS group (P=0.0259). CONCLUSION: This study showes that BMT resulted in significantly better response rate and overall survival compared to IS in patients with severe AA.