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. 2004 Wiley-Liss, Inc.