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在遗传性血管方面将长期预防转换为Donidalorsen:一年的OASISplus结果

Marc A Riedl¹, Jonathan A Bernstein², Joshua S Jacobs³

¹Division of Allergy and Immunology, University of California at San Diego, La Jolla, California, USA.

|March 14, 2026

概括

多尼达尔森有效地减少了遗传性血管 (HAE) 发作,并改善了从其他长期预防治疗转换的患者的生活质量. 这种治疗在一年内表现出良好的耐受性.

科学领域:

药理学和治疗学药理学和治疗学
临床医学临床医学
遗传学和基因组学遗传学和基因组学

背景情况:

多尼达尔森是一种针对血前性蛋白的反感性寡核酸,用于遗传性血管 (HAE).
转换队列的OASISplus研究 (NCT05392114) 评估了从其他预防疗法过渡的HAE患者的长期使用.

研究的目的:

评估多尼达尔森在HAE患者的1年安全性和有效性,这些患者从长期预防性治疗 (LTP) 转换.
评估治疗出现的不良事件 (TEAE),HAE发作率,生活质量 (AE-QoL) 和疾病控制 (AECT).

主要方法:

注册的HAE患者 (≥12年) 在稳定的LTP (lanadelumab,berotralstat,C1抑制剂) 上.
患者转换为每4周服用多尼达洛森80毫克皮下注射.
在基线和第52周评估了TEAE,每月HAE攻击率,AE-QoL和AECT.

主要成果:

与基线LTP相比,Donidalorsen在52周内将HAE攻击率降低了67.6%,比起基线LTP.
87.5%的患者经历了TEAE,大多为轻度至中度;注射部位反应是最常见的.
在52周观察到AE-QoL (12.2分) 和疾病控制 (90.4%与AECT≥10) 的显著改善.

结论:

在HAE患者从其他LTPs切换时,Donidalorsen被很好地容忍.
治疗显著改善了长期HAE攻击率,生活质量和疾病控制.
多尼达洛森为治疗遗传性血管提供了一种可行的长期治疗选择.

关键词:

血管发炎血管发炎临床免疫学临床免疫学这是一种炎症炎症炎症炎症.生活的质量生活的质量.

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