Successful treatment of severe aplastic anemia in children using standardized immunosuppressive therapy with antithymocyte globulin and cyclosporine A

Background Given the heterogeneity of published data in US children, we sought to evaluate outcomes of a standardized immunosuppressive therapy (IST) regimen for severe aplastic anemia (SAA) at The Children's Hospital (Denver, CO). Methods. We retrospectively analyzed the records of 16 children diagnosed from 1990 to 2003 and treated by IST, among whom 14 received the standardized regimen of antithymocyte globulin (ATG) and cyclosporine A (CsA). Serial hematologic parameters, complications, transfusion requirements, and time to response were assessed. Results. One child who died from a pre-existing Aspergillus infection prior to expected IST response was excluded from the analysis. Overall (transfusion-independent) response to IST was 100% (13/13), without any relapses or clinically evident leukemic/ myelodysplastic transformations after a median follow-up time of 4.4 years (range: 10 months-13.3 years). Conclusions. This report documents excellent outcome using combination ATG and CsA IST for pediatric SAA. (C) 2004 Wiley-Liss, Inc.