Outcomes after Immunosuppressive Therapy for Aplastic Anemia: A Single Centre Experience from Northern India

Aplastic anemia (AA) in children is a life-threatening disorder with very few therapeutic options. This study was carried out to assess the response of Immunosuppressive therapy (IST) in children with non-inherited AA.

It was a prospective observational study carried out among 35 children aged less than 18 years, recently diagnosed with AA by bone marrow biopsy. Those suffering from inherited bone marrow failure syndromes and secondary AA were excluded. IST included equine anti-thymocyte globulin (eATG), steroids, and cyclosporine. Response to immunosuppressive therapy was assessed after four months.

The mean age was 10.77 ± 3.6 years. Non-severe AA was diagnosed in 65.71% of children with severe and very severe AA in 17.14%. The overall response rate was found to be 65.7%, with 54.28% of children achieving a partial response and 11.42% of children achieving a complete response. Whereas 17.14% did not respond to treatment, and another 17.14% of children expired before the time point of response assessment. Hemoglobin levels, total leucocyte count, platelet count, and reticulocyte count showed statistically significant elevation following IST.

IST can be safely and effectively used as the first-line treatment option in children with non-inherited aplastic anemia, especially when stem cell transplant is not a feasible option.