Nishimori Hisakazu, Nakazawa Hideyuki, Tamura Shinobu, Uchida Toshiki, Usuki Kensuke, Szamosi Johan, de Latour Régis Peffault, Röth Alexander, Panse Jens
Department of Hematology and Oncology, Okayama University Hospital, Okayama, Japan.
Department of Hematology and Medical Oncology, Shinshu University School of Medicine, Matsumoto, Japan.
Acta Haematol. 2025;148(1):22-35. doi: 10.1159/000537696. Epub 2024 Apr 17.
Pegcetacoplan, the first approved proximal complement C3 inhibitor, showed superiority to eculizumab in improving hemoglobin levels and clinical outcomes in the phase 3 PEGASUS study in patients with paroxysmal nocturnal hemoglobinuria (PNH) and inadequate response to eculizumab.
This analysis evaluates the efficacy and safety of pegcetacoplan for Japanese patients in PEGASUS, as they are known for different clinicopathologic features compared to non-Asian patients. Ten Japanese patients were enrolled to receive pegcetacoplan (n = 5) or eculizumab (n = 5) during the 16-week randomized controlled period. All patients received pegcetacoplan monotherapy during the open-label period until week 48.
Treatment with pegcetacoplan improved hemoglobin with a mean change from baseline of 2.4 g/dL at week 16, which was sustained through 48 weeks. Pegcetacoplan-treated Japanese patients experienced sustained improvements in key secondary efficacy endpoints, including freedom from transfusion, lactate dehydrogenase level, reticulocyte count, and FACIT-Fatigue score. The safety profile was consistent with previously reported data from pegcetacoplan studies. No events of hemolysis, meningococcal infection, or thrombosis were reported in the Japanese population and all Japanese patients remained on treatment throughout the study.
These data suggest that Japanese patients with PNH can be effectively and safely managed with pegcetacoplan.
Pegcetacoplan, the first approved proximal complement C3 inhibitor, showed superiority to eculizumab in improving hemoglobin levels and clinical outcomes in the phase 3 PEGASUS study in patients with paroxysmal nocturnal hemoglobinuria (PNH) and inadequate response to eculizumab.
This analysis evaluates the efficacy and safety of pegcetacoplan for Japanese patients in PEGASUS, as they are known for different clinicopathologic features compared to non-Asian patients. Ten Japanese patients were enrolled to receive pegcetacoplan (n = 5) or eculizumab (n = 5) during the 16-week randomized controlled period. All patients received pegcetacoplan monotherapy during the open-label period until week 48.
Treatment with pegcetacoplan improved hemoglobin with a mean change from baseline of 2.4 g/dL at week 16, which was sustained through 48 weeks. Pegcetacoplan-treated Japanese patients experienced sustained improvements in key secondary efficacy endpoints, including freedom from transfusion, lactate dehydrogenase level, reticulocyte count, and FACIT-Fatigue score. The safety profile was consistent with previously reported data from pegcetacoplan studies. No events of hemolysis, meningococcal infection, or thrombosis were reported in the Japanese population and all Japanese patients remained on treatment throughout the study.
These data suggest that Japanese patients with PNH can be effectively and safely managed with pegcetacoplan.
培塞妥单抗是首个获批的近端补体C3抑制剂，在阵发性夜间血红蛋白尿（PNH）且对依库珠单抗反应不足的患者进行的3期PEGASUS研究中，在改善血红蛋白水平和临床结局方面显示出优于依库珠单抗的效果。
本分析评估了培塞妥单抗对PEGASUS研究中日本患者的疗效和安全性，因为已知日本患者与非亚洲患者相比具有不同的临床病理特征。在为期16周的随机对照期内，招募了10名日本患者接受培塞妥单抗（n = 5）或依库珠单抗（n = 5）治疗。在开放标签期直至第48周，所有患者均接受培塞妥单抗单药治疗。
培塞妥单抗治疗可改善血红蛋白水平，在第16周时与基线相比平均变化为2.4 g/dL，且这种改善持续至48周。接受培塞妥单抗治疗的日本患者在关键次要疗效终点方面持续改善，包括无需输血、乳酸脱氢酶水平、网织红细胞计数和功能性评估癌症治疗-疲劳量表（FACIT-Fatigue）评分。安全性概况与培塞妥单抗研究先前报告的数据一致。在日本人群中未报告溶血、脑膜炎球菌感染或血栓形成事件，并且所有日本患者在整个研究过程中均持续接受治疗。
这些数据表明，培塞妥单抗可有效且安全地治疗日本PNH患者。
培塞妥单抗是首个获批的近端补体C3抑制剂，在阵发性夜间血红蛋白尿（PNH）且对依库珠单抗反应不足的患者进行的3期PEGASUS研究中，在改善血红蛋白水平和临床结局方面显示出优于依库珠单抗的效果。
本分析评估了培塞妥单抗对PEGASUS研究中日本患者的疗效和安全性，因为已知日本患者与非亚洲患者相比具有不同的临床病理特征。在为期16周的随机对照期内，招募了10名日本患者接受培塞妥单抗（n = 5）或依库珠单抗（n = 5）治疗。在开放标签期直至第48周，所有患者均接受培塞妥单抗单药治疗。
培塞妥单抗治疗可改善血红蛋白水平，在第16周时与基线相比平均变化为2.4 g/dL，且这种改善持续至48周。接受培塞妥单抗治疗的日本患者在关键次要疗效终点方面持续改善，包括无需输血、乳酸脱氢酶水平、网织红细胞计数和功能性评估癌症治疗-疲劳量表（FACIT-Fatigue）评分。安全性概况与培塞妥单抗研究先前报告的数据一致。在日本人群中未报告溶血、脑膜炎球菌感染或血栓形成事件，并且所有日本患者在整个研究过程中均持续接受治疗。
这些数据表明，培塞妥单抗可有效且安全地治疗日本PNH患者。
