埃维纳库马布在同卵性家族高胆固醇血症:长期的安全性和有效性
Daniel Gaudet1, Susanne Greber-Platzer2, Laurens F Reeskamp3
1Clinical Lipidology and Rare Lipid Disorders Unit, Community Gene Medicine Center, Department of Medicine, Université de Montréal and ECOGENE-21, 930 Jacques-Cartier, Suite 210-B, Chicoutimi, Québec G7H 7K9, Canada.
European heart journal
|June 10, 2024
概括
埃维纳库马布在患有同卵性家族高胆固醇血症 (HoFH) 的患者中显著降低了LDL胆固醇. 长期治疗在成人和青少年中显示了持续的疗效和一般耐受良好的安全性.
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:
- 同胞性家族性高胆固醇血症 (HoFH) 是一种罕见的遗传疾病,导致极高的LDL胆固醇 (LDL-C) 和早期心血管疾病.
- 埃维纳库马布在对HoFH患者的关键第3期试验中显示出显著的LDL-C降低.
研究的目的:
- 评估埃维纳库马布在被诊断患有HoFH的成年和青少年患者的长期安全性和疗效.
- 评估埃维纳库马布在长期治疗期间对LDL-C水平和患者整体福祉的持续影响.
主要方法:
- 一个开放的,单臂的,第三期试验 (NCT03409744) 涉及12岁及以上的HoFH患者.
- 患者每4周内服用15mg/kg的静脉注射埃维纳库马布,并与稳定的降脂疗法相结合.
- 包括从未服用过埃维纳库马布的患者和以前在其他试验中接受过该药物的患者.
主要成果:
- 116名患者 (102名成年人,14名青少年) 接受治疗,平均治疗时间为104.3周.
- 治疗引起的不良事件发生在80.2%的患者中;严重的TEAE发生在23.3%的患者中.
- 在总人口中,埃维纳库马布从基线到第24周的平均LDL-C降低了43.6%,并观察到持续的降低.
结论:
- 埃维纳库马布在大量的HoFH患者中,无论年龄或性别,一般都能耐受.
- 该药物显著降低了LDL-C水平,证明了长期持续的疗效.
- 埃维纳库马布的安全性和疗效概况支持其用于管理HoFH.
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