萨西兰是一种针对APOC3的RNA干扰剂,用于混合超脂血症
Christie M Ballantyne1, Szilard Vasas1, Masoud Azizad1
1From the Baylor College of Medicine and the Texas Heart Institute, Houston (C.M.B.); Borbánya Praxis, Nyíregyháza, Hungary (S.V.); Valley Clinical Trials, Northridge (M.A.), Arrowhead Pharmaceuticals, Pasadena (T.C., S.M., R.Z., M.M., J.H.), and the Stanford School of Medicine, Stanford (N.J.L.) - all in California; the Royal Adelaide Hospital, Adelaide, SA, Australia (P.C.); the Icahn School of Medicine at Mount Sinai, New York (R.S.R.); and the Department of Medicine, Université de Montréal and Ecogene-21, Quebec, QC, Canada (D.G.).
在患有混合高脂血症的患者中,Plozasiran显著降低了甘油三水平. 这种向肝细胞的疗法在控制动脉样硬化心血管疾病风险方面表现有前途.
科学领域:
- 心血管医学 心血管医学
- 药理学 药理学是指药理学的学科.
- 遗传学 遗传学 是一个
背景情况:
- 混合型高脂血症增加了动脉样硬化心血管疾病的风险,原因是高非高密度脂蛋白 (HDL) 胆固醇.
- 富含三糖的脂蛋白代谢是由阿波利波蛋白C3 (APOC3) 调节的,它抑制了脂蛋白脂酶.
研究的目的:
- 评估Plozasiran的安全性和有效性,一个针对APOC3的小干扰RNA,在患有混合超脂血症的患者中.
- 评估Plozasiran对48周的禁食甘油三水平的影响.
主要方法:
- 进行了一项为期48周的2b期,双盲,随机,安慰剂对照试验.
- 参与者每季度或半年接受皮肤下布洛萨西兰 (10-50毫克) 或安慰剂.
- 主要终点是24周的禁食甘油三水平的百分比变化.
主要成果:
- 与安慰剂相比,Plozasiran在24周内在所有测试剂量和治疗方案中显著降低了禁食甘油三水平.
- 减少范围从-44.2至-62.4个百分点 (P<0.001).
- 普洛萨西兰接受者的7-21%观察到血糖控制恶化,而安慰剂接受者的10%观察到.
结论:
- 在患有混合高脂血症的患者中,Plozasiran显著降低了甘油三水平.
- 需要进一步的临床结局试验来证实plozasiran的长期疗效和安全性.
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