Objective: To investigate prognostic factors of immunosuppressive therapy (IST) in children acquired severe aplastic anemia(SAA).
Methods: Data of 56 consecutive children cases with SAA who had received rabbit anti-thymocyte globulin (R-ATG) [3-5 mg/( kg x d) x 5 d] and cyclosporine A (CSA) from January 2000 to June 2006 were retrospectively analyzed. 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 response rate to the immunosuppressive therapy (IST) was 62.5% and the complete remission rate was 37.5%. The 5-year overall survival for IST regimens was 66.27% +/- 6.84%, patients who had infections when using ATG had significantly lower response and higher mortality. Patients whose nucleated erythrocyte population in bone marrow was > or =10% had good prognosis. Patients whose granulocytes population in bone marrow was > or =10% had lower mortality.
Conclusion: Patients who had infections when using ATG had significantly lower response and higher mortality. Patients whose nucleated erythrocyte population in bone marrow was > or =10% had good prognosis.