Clinical outcomes of pediatric patients with severe acquired aplastic anemia treated at Siriraj Hospital

Background: Severe acquired aplastic anemia (SAA) is a life-threatening condition. Thai treatment guidelines recommend matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and immunosuppressive therapy (IST) combined with eltrombopag (EPAG). Objective: This study aimed to investigate the clinical outcomes of pediatric patients with SAA treated at Siriraj Hospital. Method: This retrospective study included pediatric patients with SAA treated at Siriraj Hospital from 1 January 2013 to 31 December 2023. Result: Twenty-seven patients were enrolled. Treatment modalities included IST in 13 patients (48.2%), IST+EPAG in 7 patients (25.9%) and MSD-HSCT in 7 patients (25.9%). At 3 and 6 months, overall response (OR) rates were 28.6% and 42.9% in the IST+EPAG group and 0% and 7.7% in the IST-only group, respectively. However, these differences were not statistically significant (p = 0.111 and 0.197, respectively). Overall survival (OS) was similar between the IST+EPAG (62.5%, 95%CI: 0.14-0.89) and the IST group (63.3%, 95%CI: 0.29–0.84). The mean time to response and mean time to transfusion independence were shorter in the IST+EPAG group compared with the IST group, but the differences were not statically significance (p = 0.085 and 0.162, respectively). The OS for the MSD-HSCT group was 42.9%. Conclusion: The combination of EPAG with IST was associated with a trend toward higher OR rates at 3 and 6 months. The IST+EPAG group also demonstrated shorter mean times to hematologic response and transfusion independence. OS was comparable between groups.