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  • Title: Immunosuppressive therapy for acquired severe aplastic anemia (SAA): a prospective comparison of four different regimens.
    Author: Zheng Y, Liu Y, Chu Y.
    Journal: Exp Hematol; 2006 Jul; 34(7):826-31. PubMed ID: 16797409.
    Abstract:
    OBJECTIVE: This study was designed to investigate four different immunosuppressive therapy (IST) regimens as treatment of acquired severe aplastic anemia (SAA). PATIENTS AND METHODS: 142 consecutive SAA patients were randomized to receive one of the following IST regimens: equine anti-human thymocyte immunoglobulin (E-ATG) alone (IST regimen I); E-ATG and cyclosporine A (CSA) (IST regimen II); E-ATG, CSA plus recombinant human granulocyte-macrophage colony-stimulating factor (rhuGM-CSF) and rhu erythropoietin (rhuEPO) (IST regimen III); or rabbit ATG (ATG-F), CSA, rhuGM-CSF, and rhuEPO (IST regimen IV). No repeated courses of E-ATG or ATG-F were given for nonresponders. All patients also received stanozolol or testosteron propionate. RESULTS: The overall response rate to IST regimen I was 58%. The response to IST regimen II (79%) was significantly higher (p = 0.04), more rapid and complete than after IST regimen I. The response rate to IST regimen IV (53%) was significantly lower than that of IST regimen III (73%, p = 0.039). The additional use of growth factors did not reduce early deaths and did not accelerate hematopoietic recovery after IST. Of the 142 patients enrolled in this trial, 92 (65%) are alive at a median follow-up time of 102 months (range, 54-166 months). The 5-year actuarial survival for IST regimens I, II, III, and IV was 58%, 81%, 80%, and 66%, respectively. CONCLUSION: The combination of E-ATG and CSA remains the best combination for the treatment of SAA patients, producing a survival advantage at 5 years. The addition of growth factors did not improve these results. Rabbit ATG-F appeared less effective than E-ATG.
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