Table 1_Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia.docx

BackgroundTo 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).
MethodsThis 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.
ResultsThere 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 × 109/L, a white blood cell (WBC) count of ≥ 2.0 × 109/L, N% of ≥ 40%, a lymphocyte count of ≥ 1.0 × 109/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.
ConclusionEPAG 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.