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<p>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&#xa0;μg/kg/week and escalated to 20&#xa0;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.</p>

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

  • 期刊论文

  • 发表日期：

    2026年3月11日

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  • Shuku Sato,
  • Emi Sawazaki,
  • Shun Tsunoda,
  • Wataru Kamata,
  • Tomiteru Togano,
  • Yotaro Tamai,
  • Ryuji Tanosaki

摘要

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.