Wang SC, Zou Y, Chen XJ, Yang WY, Liu TF, Zhang L, Chen YM, Guo Y, Zhu XF
[Efficacy of immunosuppresive therapy in children with acquired aplastic anemia.] [English Abstract, Journal Article]
Zhonghua Er Ke Za Zhi 2009 Jan; 47(1):53-6.
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.d) x 5 d] (IST regimen II); rabbit anti-T-lymphocyte globulin (R-ATG) [3 - 5 mg/(kg.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 significient 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.
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