A modified regimen of high-dose cyclophosphamide (CTX) plus cyclosporine (CsA) was adopted for patients with severe or very severe aplastic anemia, and the effectiveness was compared with a regimen of antithymocyte globulin (ATG) plus CsA. A total of 121 patients enrolled in this study received either CTX plus CsA (CTX group, 48 cases) or ATG plus CsA (ATG group, 73 cases). The early death rate was 4.2% in the CTX group and 8.2% in the ATG group, showing no significant difference (p = 0.312). The total response rate in the CTX and ATG groups was 54.2% and 57.5% at 3 months, 64.6% and 72.6% at 6 months, and 72.9% and 78.1% at 12 months, respectively (p > 0.05). The overall 5-year survival rate was 81.2% and 80.7%, and the event-free survival rate was 68.2% and 67.3% in the CTX and ATG groups, respectively (p > 0.05). The total medical cost of the CTX group was 54.8% less than that of ATG regimen (p = 0.000). In summary, treatment of severe or very severe aplastic anemia with CTX plus CsA has effectiveness that is comparable to a conventional regimen and less costly.
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