由于正常的C1INH与F12/PLG突变而导致的遗传性血管
Danny M Cohn1, Avner Reshef2, Petra Staubach3
1The Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam.
在使用正常C1抑制剂 (HAE- nC1INH),特别是HAE- FXII时,加拉达西马布有效降低了遗传性血管的发作. 在HAE- nC1INH患者中,加拉达西马布的长期预防显示出良好的安全性.
科学领域:
- 免疫学
- 遗传学
- 药理学
背景情况:
- 具有正常C1抑制剂 (HAE- nC1INH) 的遗传性血管具有显著的未满足的治疗需求.
- HAE-nC1INH包括由于XII因子 (HAE-FXII) 或PLG基因突变引起的HAE等疾病.
研究的目的:
- 在HAE-FXII或HAE-PLG患者中使用每月皮下加拉迪马布进行长期预防.
- 加拉达西马布是一种向激活XII因子的单克隆抗体.
主要方法:
- 一个开放的第二阶段研究,使用了600毫克的加拉达西马布治疗期 (TP1) 和随后的延长期 (TP2).
- 一个正在进行的第三阶段的开放扩展 (OLE),使用200毫克的加拉达西马布.
- 在HAE-FXII和HAE-PLG患者中评估了疗效和安全性.
主要成果:
- 在第二阶段,两名HAE- FXII患者的发作率 (AR) 显著降低,在OLE中保持无发作状态.
- 一名HAE-PLG患者的AR降低,而两名患者的AR增加.
- 与治疗相关的不良事件通常是轻微的,报告的一次严重的无关事件.
结论:
- 在所有评估的HAE- nC1INH患者中,加拉达西马布表现出良好的安全性.
- 在HAE- FXII患者中显示出有效性,在1名HAE- PLG患者中显著减少了AR.
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