Outcome of immunosuppressive therapy in pediatric patients with acquired aplastic anemia

A BSTRACT Aim: To assess the response to immunosuppressive therapy (IST) in children with acquired aplastic anemia (AAA) and factors affecting the outcome. Methods: Ambispective observational study was conducted from Aug 2022 to May 2024 prospectively. Records from June 2012 to July 2022 were analyzed for retrospective cases. Children 2–18 years meeting Camitta’s criteria were included. Hematopoietic stem cell transplant (HSCT) was advised if feasible and affordable, rest were offered IST. They received IST with horse anti-thymocyte globulin (ATG) and cyclosporine. Thrombopoietin (TPO) agonists were used in few patients only. Response was assessed at 3 months and 6 months after IST. Results: Among 52 prospective cases 43 (83%) were AAA and 9 (17%) were Fanconi anemia (FA). Among 48 retrospective cases, 42 (87%) were AAA and 6 (12%) were FA. FA patients were excluded from analysis. Majority of patients were severe aplastic anemia (SAA) (37%) followed by very severe (VSAA) (32%) and non-severe aplastic anemia (NSAA) (30%). Overall response rate (ORR) was 58.8% with complete response (CR) of 35.3% and partial response (PR) of 23.0% in prospective group and CR of 55.2% and PR of 3.8% in retrospective group. SAA and NSAA patients responded 2.5 times better than VSAA patients to IST (RR = 2.514, 95% CI = 1.145–7.076). Other variables age, gender, duration of illness, duration from diagnosis to IST and type of IST had no correlation with outcome ( P > 0.05). Conclusion: The ORR was found to be 58.8%, which is similar to published reports.