OBJECTIVEThis study was designed to evaluate the efficacy of immunosuppressive therapy (IST) regimens as treatment of children with acquired severe aplastic anemia (SAA).

METHODSData 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.

RESULTSThe 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.

CONCLUSIONIf 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.