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儿科患者的Evolocumab治疗同卵性家族高胆固醇血症:三项开放标签研究的数据汇总

Frederick J Raal¹, Robert A Hegele², Andrea Ruzza³

¹Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa (F.J.R.).

Arteriosclerosis, thrombosis, and vascular biology

|March 28, 2024

概括

埃沃洛库马布在患有同卵性家族高胆固醇血症 (HoFH) 的儿科患者中耐受性良好,没有显示新的安全问题. 然而,LDL-C降低在患者之间有显著的差异,支持PCSK9抑制剂试验,无论残留的LDLR功能.

科学领域:

心血管医学心血管医学
遗传学是一个遗传学.
药理学药理学是指药理学的学科.

背景情况:

患有同卵性家族高胆固醇血症 (HoFH) 的儿科患者面临高风险的动脉样硬化心血管疾病.
达到目标低密度脂蛋白胆固醇 (LDL-C) 水平对于这些年轻患者来说是一项挑战.
作为PCSK9抑制剂的Evolocumab是一种潜在的治疗选择,用于控制HoFH.

研究的目的:

评估evolocumab在患有HoFH的儿科患者的综合安全性和疗效.
评估治疗出现的不良事件和evolocumab的降脂效应.
为了确定剩余的LDL受体 (LDLR) 活性是否影响治疗反应.

主要方法:

从3项临床研究 (TAUSSIG,RAMAN,HAUSER-OLE) 的儿童HoFH患者的安全性和疗效数据进行了汇总.
10至17岁的患者接受了埃沃洛库马布 (420毫克每月或每两周一次) 和背景他类药物+/-埃泽提米布.
主要终点:治疗出现的不良事件;次要终点:脂质和PCSK9水平的变化.

主要成果:

埃沃洛库马布在39名小儿HoFH患者中耐受良好,中位暴露时间为18.2个月.
常见的不良事件包括上呼吸道感染,流感和.
观察到LDL-C降低的显著变化;42.9%的LDL-C降低达到≥15%,但残留LDLR活性与反应无关.

结论:

埃沃洛库马布在儿科HoFH患者中表现出良好的安全性,与之前的研究一致.
没有发现新的安全信号,加强了它的耐受性.
可变的LDL-C反应支持启动PCSK9抑制剂治疗,无论估计的剩余LDLR功能如何.

关键词:

动脉样硬化动脉样硬化心血管疾病心血管疾病过高脂血症是什么意思脂蛋白,LDL以及其他类型的蛋白质.

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