Background: Human leukocyte antigen-matched bone marrow transplant in the treatment of aplastic anemia is generally not feasible in developing countries due to lack of resources and expertise and immunosuppressive therapy (IST) has been used as an alternative. This study aims to report the long-term outcome of children with aplastic anemia treated with IST [antithymocyte globulin (ATG) and cyclosporine] in our hospital.
Procedure: Case files of children with aplastic anemia who received IST from January 2001 to November 2009 were reviewed.
Results: Thirty-five patients with aplastic anemia (14 very severe aplastic anemia; 21 severe aplastic anemia) were given IST. Seven patients expired within 3 months of therapy and were excluded. The analysis was done in 28 patients (24 male and 4 female; 12 very severe aplastic anemia and 16 severe aplastic anemia). The median age was 10 years (range, 5 to 12 y). Ten patients achieved partial response and 4 patients complete response at 1 year with overall response rate of 50%. Three nonresponders received a second course of ATG after 12 months out of which 2 responded. Hence, overall response including second course was 16 (57%). Three patients relapsed after a median interval of 23 months. The median duration of follow-up of 16 responders was 40 months (range, 15 to 119 mo). In the patients with long-term follow-up for >4 years (n=7), all were surviving and independent of transfusions.
Conclusions: In a developing country setting, IST with ATG and cyclosporine seems to be a good alternative treatment for aplastic anemia in children.