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 su-perior to either agent alone, cyclosporine monotherapy is an easily avail-able, 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 mono-therapy 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 com-plete 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 1 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 mono-therapy is an effective and safe immunosuppressive therapy for SAA, and that it could be a reasonable IST option for patients in developing countries.