Cyclosporine monotherapy for severe aplastic anemia: a developing country experience

BACKGROUND: Immunosuppression is the most effective treatment for aplastic anemia after hematopoietic stem cell transplantation. Although the combination of cyclosporine and antithymocyte globulin (ATG) is superior to either agent alone, cyclosporine monotherapy is an easily available, safe and cheap immunosuppressive therapy (IST) option. These advantages are particularly valuable in developing countries where ATG is frequently not available. PATIENTS AND METHODS: In the referral hematology center in Yemen, 20 patients (16 males and 4 females) with severe aplastic anemia (SAA) were prospectively identified and managed with cyclosporine monotherapy during the period between April 2001 and November 2004. RESULTS: Data from 14 patients who received cyclosporine for at least 3 months were analyzed. At 6 months, 2 (14.3%) patients achieved complete remission (CR) and 5 (35.7%) patients achieved partial remission (PR) and at 1 year, 4 (28.6%) patients achieved CR and 3 (21.4%) patients remained in PR. The overall response rate was 50% and the cumulative survival rate at I year was 78.6%. The median time to remission was 120 days (range, 46 to 131 days). Side effects were modest and easily monitored. CONCLUSION: Our results support findings that cyclosporine monotherapy is an effective and safe immunosuppressive therapy for SAA, and that it could be a reasonable IST option for patients in developing countries.