患者的口服伊普塔科潘治疗:一个随机,双盲,并行组,多中心,安慰剂控制的第三期研究
David Kavanagh1, Andrew S Bomback2, Marina Vivarelli3
1Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK; National Renal Complement Therapeutics Centre, Royal Victoria Infirmary, Newcastle upon Tyne, UK.
Lancet (London, England)
|September 28, 2025
概括
通过抑制替代补充路径,伊普塔科潘显著降低了C3型血球病患者的蛋白尿症. 这种口服治疗在3期临床试验中显示出良好的安全性.
科学领域:
- 肝脏病学
- 免疫学
- 药理学
背景情况:
- 这是一种罕见的,严重的病,由替代补体通路过度激活引起.
- 针对替代补充途径为C3G提供了潜在的治疗策略.
- 伊普塔科潘是一种口服的近位补体抑制剂,旨在选择性地向B因子.
研究的目的:
- 通过活检证实C3球体病变的成年患者的伊普塔科班的疗效和安全性.
- 在接受iptacopan与标准治疗同时治疗的患者中,评估蛋白尿的减少作为主要终点.
主要方法:
- 一项多中心,随机,双盲,安慰剂控制的第三期研究 (APPEAR- C3G) 招募了74名成年C3G患者.
- 参与者接受了iptacopan或安慰剂,以及6个月的支持性治疗 (RAAS抑制剂) 和免疫抑制.
- 主要终点是24小时尿蛋白- 肌素比率 (UPCR) 在6个月内相对降低.
主要成果:
- 与安慰剂相比,伊普塔科潘在6个月后显示UPCR的相对显著降低为35. 1% (p=0. 0014).
- 在Iptacopan组,UPCR的几何平均值从3. 33g/ g降至2. 17g/ g,而在安慰剂组则从2. 58g/ g增加至2. 80g/ g.
- 伊普塔科潘的耐受性很好,不良反应大多为轻度/ 中度;由于不良反应而导致的死亡或停止治疗并未发生.
结论:
- 在C3G患者中,Iptacopan可提供统计学显著和临床意义上的蛋白尿减少.
- 在这种患者群体中,口服补充抑制剂iptacopan具有可接受的安全性.
- 伊普塔科潘是一种有前途的治疗方案.
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