Outcome of Severe Aplastic Anemia Treated with Immunosuppressive Therapy Compared with Bone Marrow Transplantation

Abstract

Background: The response rates and survival following allogeneic bone marrow transplantation (BMT) or immunosuppressive treatment were compared in severe aplastic anemia (SAA) and the prognostic factors related with survival identified. Methods: Medical data of SAA patients, treated with BMT or immunosuppressive therapy (IST) at the Ajou University Hospital, between January 1995 and December 2005, were retrospectively analyzed. Results: A total of 43 patients were evaluable; 18 (41.9%) were treated with IST (antithymocyte globulin plus cyclosporine A plus steroid) and 25 (58.1%) with allogeneic BMT. In the IST group, the response rate was 77.8% (2 complete and 12 partial remissions), with two treatment failures. As later complications, acute myeloid leukemia developed in 1 patient and myelodysplastic syndrome developed in 2. In the BMT group, the response rate was 92.0% (18 complete and 5 partial remissions) (P<0.001). Six patients developed grade II to III acute graft-versus-host-disease (GVHD) and 3 developed chronic GVHD. The median survival time in all patients was 60.27 months, and the 5-year survival rates were 61.0 and 81.9% in the IST and BMT groups, respectively (P=0.144). The factors influencing the overall survival were an age under 40-years and a positive treatment response. Conclusion: This study shows that allogeneic BMT, compared to IST, resulted in good response andoverall survival rates in patients with SAA. However, the overall survival rate between the two groups was statistically insignificant. Our study suggests that younger age SAA patients, with HLA-matched BMT donors, may benefit more from allogeneic BMT. (Korean J Hematol 2006;41:259-265.)