Aplastic anemia in children: How good is immunosuppressive therapy?

Aplastic anemia (AA) is an uncommon disorder in children, with hematopoietic stem cell transplant being the 1st line therapy; immunosuppressive therapy (IST) is the alternative therapy and is the most commonly used modality of treatment. There is paucity of data from the developing countries regarding treatment outcome with IST. We aimed to assess the outcome of IST in children with AA. Data for 43 children treated with IST from January 2012 to January 2017 (5 years) were retrieved from clinic records. IST included equine antithymocyte globulin (ATG) along with cyclosporine A. Complete response, partial response and nonresponse was seen in 9 (21%), 14 (32.5%) and 20(46.5%) patients, respectively. The median time to best response in the whole cohort was 19.1 months. However, complete response occurred nearly 2-year post-IST. There was no difference in outcome related to severity of AA, the presence of PNH clone, higher ALC or different available brands of ATG. There was a significantly better rate of response (