A Multicenter, Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Front-Line Treatment for Pediatric Severe Aplastic Anemia.

Eltrombopag (EPAG) added to standard immunosuppressive therapy (IST) was associated with higher overall (OR) and complete response (CR) rates in patients with treatment-naïve severe aplastic anemia (SAA) in adults, but the clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial. We compared the efficacy and safety of EPAG combined with IST (n=38) versus IST alone (n=57) as a front-line treatment for pediatric patients with SAA. The EPAG+IST group had higher CR and OR rates at 3 and 6 months, although 1-year OR, CR, and partial response (PR) rates showed no significant difference between two groups. Older age at diagnosis (>8.95 years) was associated in the EPAG+IST group with higher OR rates at 6 months and 1-year (p=0.007, p=0.005, respectively). The addition of EPAG to IST did not demonstrate superiority over IST alone in terms of overall survival (OS) and event-free survival (EFS) in this study; with 1-year EFS of 81.1% for EPAG+IST and 71.3% for IST, and with 1-year OS of 89.2% vs. 80.4%. Eltrombopag induced a faster response compared to those receiving IST alone without increasing toxic effects, but EPAG does not confer additional benefits regarding OS or relapse rates in children. Notably, older age at diagnosis in the EPAG+IST group was significantly associated with improved response rates.