Abstract
BACKGROUND: To compare the efficacy of eltrombopag (EPAG) combined with standard immunosuppressive therapy (IST), EPAG with cyclosporine or cyclosporine alone in children with severe aplastic anemia (SAA). METHODS: This is a retrospective study. The patients were categorized as three groups: Group A (EPAG + rabbit antithymocyte globulin + cyclosporine, n = 12), Group B (EPAG + cyclosporine, n = 13), and Group C (cyclosporine alone, n = 16). The overall remission rate (ORR) of each group at 1, 3, 6, and 12 months of treatment was evaluated. RESULTS: There was no significant difference in the ORR among Groups A, B, and C at 1, 3, 6 and 12 months (P > 0.05). The incidence rates of adverse reactions in each group at 6 months were 36.4% (4/11), 58.3% (7/12), and 69.2% (9/13), respectively (P = 0.264). Patients with a duration from diagnosis to receiving EPAG treatment of ≤ 60 days, a diagnosis of SAA, a platelet (PLT) count ≥ 15 × 10(9)/L, a white blood cell (WBC) count of ≥ 2.0 × 10(9)/L, N% of ≥ 40%, a lymphocyte count of ≥ 1.0 × 10(9)/L, and a CD4(+)/CD8(+) ratio of ≥ 1.5 were more likely to achieve a hematopoietic response. No clonal evolution was observed in any of the patients. CONCLUSION: EPAG combined with IST shows comparable efficacy to cyclosporine alone in children with SAA, along with a favorable safety profile. Patients with earlier initiation of EPAG and preserved residual hematopoietic function are more likely to achieve remission, without serious adverse reactions or significant clonal evolution.