在以前接受长期预防的患者中治疗遗传性血管

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Marc A Riedl¹, Jonathan A Bernstein², Joshua S Jacobs³

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

The journal of allergy and clinical immunology. In practice

|July 17, 2025

概括

多尼达尔森显著减少了遗传性血管 (HAE) 发作,并改善了从其他治疗方法转换的患者的生活质量. 大多数参与者更喜欢donidalorsen,报告了更好的疾病控制和满意度.

科学领域:

免疫学免疫学免疫学
遗传学遗传学是一个
药理学药理学是指药理学的学科.

背景情况:

遗传性血管 (HAE) 是一种罕见的,危及生命的遗传性疾病,导致组织胀.
唐尼多尔森是一种正在研究中的反感性寡核酸,准了血前基蛋白的产生.

研究的目的:

评估多尼达洛森的安全性,疗效和患者报告的结果,用于从现有疗法转换HAE患者.
与之前的HAE治疗相比,评估治疗偏好和对donidalorsen的满意度.

主要方法:

一项开放式的第3期研究 (OASISplus Switch队列) 招募了65名HAE患者.
患者从拉纳德鲁马布,C1抑制剂或贝罗特拉斯塔特转换为每4周服用80毫克的多尼达洛森.
主要终点:安全性;次要终点:HAE发作率,QoL,疾病控制和16周治疗偏好.

主要成果:

多尼达洛森的安全性表现良好,大多数不良事件与该药物无关.
到第16周,观察到HAE攻击率总体减少了62%.
报告说,血管-QoL得分和疾病控制有显著改善,93%的人实现了良好的疾病控制.

结论:

多尼达洛森耐受性很好,有效降低HAE发作率并改善生活质量.
大多数患者更喜欢donidalorsen而不是以前的治疗方法,这表明了高度满意度.
在第52周进行进一步分析将提供长期数据.

关键词:

疾病控制疾病控制没有道达尔或森.遗传性血管腺遗传性血管腺遗传性血管腺这是一个开放的标签.第三阶段第三阶段治疗偏好治疗偏好

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