Efficacy and safety of romiplostim with horse anti-thymocyte globulin and cyclosporine in acquired aplastic anemia

Abstract

This study evaluated the efficacy and safety of high-dose romiplostim (ROMI) combined with horse anti-thymocyte globulin (hATG) and cyclosporine A (CyA) as first-line immunosuppressive therapy for aplastic anemia (AA). We retrospectively analyzed eight patients who received hATG + CyA + ROMI at a single institution between October 2023 and January 2025. ROMI was initiated at 10 μg/kg/week and escalated to 20 μg/kg. Hematologic responses were evaluated at weeks 14 and 27. At week 27, the overall response rate was 100% and the complete response rate was 85.7%. Only one episode of grade ≥ 3 anaphylaxis attributed to hATG was observed. High-dose ROMI combined with hATG and CyA demonstrated favorable efficacy and tolerability as a first-line treatment for transfusion-dependent AA. However, larger prospective studies are required to confirm these findings.

Keywords: Aplastic anemia; Horse ATG; Romiplostim.