Objective: This study was designed to evaluate the efficacy of immunosuppressive therapy (IST) regimens as treatment of children with acquired severe aplastic anemia (SAA).
Methods: Data of consecutive 112 children with SAA who had no HLA-matched sibling seen from January 2000 to June 2006 were retrospectively analyzed. The patients were randomized to receive one of the following IST regimens: cyclosporine A (CSA) alone (IST regimen I); CSA and intravenous immunoglobulin (IVIG) [400 mg/(kg x d) x 5 d] (IST regimen II); rabbit anti-T-lymphocyte globulin (R-ATG) [3-5 mg/(kg x d) x 5 d] and CSA (IST regimen III). No repeated courses of R-ATG were given for nonresponders. All the patients also received stanozolol or testosterone propionate. The dose of CSA was adjusted to maintain trough drug levels above 100 microg/L and peak drug levels above 300 microg/L.
Results: The overall rate of response to IST regimen I was 26.92% and to IST regimen II was 33.33%. The response to IST regimen III (62.5%) was significantly higher (P = 0.001). The response to IST regimen I and IST regimen II had no significant difference. The 5-year overall survival for IST regimens I, II, and III was 20.50% +/- 15.41%, 39.77% +/- 9.77%, and 66.27% +/- 6.84%, respectively.
Conclusion: If patients had no HLA-matched sibling, the combination of R-ATG and CSA remains the best combination for the treatment of children with SAA, providing a survival advantage at 5 years.