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The long-acting C5 inhibitor, Ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment.
Kidney International ( IF 14.8 ) Pub Date : 2020-03-06 , DOI: 10.1016/j.kint.2020.01.035 Eric Rondeau 1 , Marie Scully 2 , Gema Ariceta 3 , Tom Barbour 4 , Spero Cataland 5 , Nils Heyne 6 , Yoshitaka Miyakawa 7 , Stephan Ortiz 8 , Eugene Swenson 9 , Marc Vallee 10 , Sung-Soo Yoon 11 , David Kavanagh 12 , Hermann Haller 13 ,
Kidney International ( IF 14.8 ) Pub Date : 2020-03-06 , DOI: 10.1016/j.kint.2020.01.035 Eric Rondeau 1 , Marie Scully 2 , Gema Ariceta 3 , Tom Barbour 4 , Spero Cataland 5 , Nils Heyne 6 , Yoshitaka Miyakawa 7 , Stephan Ortiz 8 , Eugene Swenson 9 , Marc Vallee 10 , Sung-Soo Yoon 11 , David Kavanagh 12 , Hermann Haller 13 ,
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Ravulizumab is a long-acting C5 inhibitor engineered from eculizumab with increased elimination half-life, allowing an extended dosing interval from two to eight weeks. Here we evaluate the efficacy and safety of ravulizumab in adults with atypical hemolytic uremic syndrome presenting with thrombotic microangiopathy. In this global, phase 3, single arm study in complement inhibitor-naïve adults (18 years and older) who fulfilled diagnostic criteria for atypical hemolytic uremic syndrome, enrolled patients received ravulizumab through a 26-week initial evaluation period. The primary endpoint was complete thrombotic microangiopathy response defined as normalization of platelet count and lactate dehydrogenase and 25% or more improvement in serum creatinine. Secondary endpoints included changes in hematologic variables and renal function. Safety was also evaluated. Ravulizumab treatment resulted in an immediate, complete, and sustained C5 inhibition in all patients. Complete thrombotic microangiopathy response was achieved in 53.6% of patients. Normalization of platelet count, lactate dehydrogenase and 25% or more improvement in serum creatinine was achieved in 83.9%, 76.8% and 58.9% of patients, respectively. Improvement in estimated glomerular filtration rate by one or more stage was achieved in 68.1% of patients by day 183. No unexpected adverse events were reported across a safety analysis set of 58 patients. Four deaths occurred (three within one month of study initiation, including one in a patient excluded based on eligibility criteria after the first dose) with none considered treatment-related by the study investigator. Thus, treatment with ravulizumab once every eight weeks resulted in rapidly improved hematologic and renal endpoints with no unexpected adverse events in adults with atypical hemolytic uremic syndrome.
中文翻译：
长效 C5 抑制剂 Ravulizumab 对未接受过抑制剂治疗的非典型溶血性尿毒症综合征成年患者有效且安全。
Ravulizumab 是一种由依库珠单抗设计的长效 C5 抑制剂，具有更长的消除半衰期，可将给药间隔从 2 周延长至 8 周。在这里，我们评估了 ravulizumab 对伴有血栓性微血管病的非典型溶血性尿毒症综合征成人的疗效和安全性。在这项针对满足非典型溶血性尿毒症综合征诊断标准的未使用补体抑制剂的成人（18 岁及以上）进行的全球 3 期单臂研究中，入组患者在 26 周的初始评估期内接受了 ravulizumab。主要终点是完全血栓性微血管病反应，定义为血小板计数和乳酸脱氢酶正常化以及血清肌酐改善 25% 或更多。次要终点包括血液学变量和肾功能的变化。还评估了安全性。Ravulizumab 治疗导致所有患者立即、完全和持续地抑制 C5。53.6% 的患者实现了完全的血栓性微血管病反应。分别有 83.9%、76.8% 和 58.9% 的患者实现了血小板计数、乳酸脱氢酶和血清肌酐 25% 或更多改善的正常化。到第 183 天，68.1% 的患者的估计肾小球滤过率提高了一个或多个阶段。在 58 名患者的安全性分析中没有报告意外的不良事件。发生了 4 例死亡（研究开始后一个月内 3 例，包括在第一次给药后根据合格标准排除的患者中的 1 例），研究人员认为无一例与治疗相关。因此，
更新日期：2020-03-06
中文翻译：
长效 C5 抑制剂 Ravulizumab 对未接受过抑制剂治疗的非典型溶血性尿毒症综合征成年患者有效且安全。
Ravulizumab 是一种由依库珠单抗设计的长效 C5 抑制剂，具有更长的消除半衰期，可将给药间隔从 2 周延长至 8 周。在这里，我们评估了 ravulizumab 对伴有血栓性微血管病的非典型溶血性尿毒症综合征成人的疗效和安全性。在这项针对满足非典型溶血性尿毒症综合征诊断标准的未使用补体抑制剂的成人（18 岁及以上）进行的全球 3 期单臂研究中，入组患者在 26 周的初始评估期内接受了 ravulizumab。主要终点是完全血栓性微血管病反应，定义为血小板计数和乳酸脱氢酶正常化以及血清肌酐改善 25% 或更多。次要终点包括血液学变量和肾功能的变化。还评估了安全性。Ravulizumab 治疗导致所有患者立即、完全和持续地抑制 C5。53.6% 的患者实现了完全的血栓性微血管病反应。分别有 83.9%、76.8% 和 58.9% 的患者实现了血小板计数、乳酸脱氢酶和血清肌酐 25% 或更多改善的正常化。到第 183 天，68.1% 的患者的估计肾小球滤过率提高了一个或多个阶段。在 58 名患者的安全性分析中没有报告意外的不良事件。发生了 4 例死亡（研究开始后一个月内 3 例，包括在第一次给药后根据合格标准排除的患者中的 1 例），研究人员认为无一例与治疗相关。因此，
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