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JACC:阿利昔单抗对纯合子家族性高胆固醇血症患者的疗效研究

2020-07-09 MedSci原创 MedSci原创

纯合子家族性高胆固醇血症(HoFH)的特点是低密度脂蛋白胆固醇(LDL-C)水平异常升高和早期动脉粥样硬化性心血管疾病。本研究旨在评估前蛋白转化酶枯草杆菌素9抑制剂阿利昔单抗对成人HoFH患者低密度脂

纯合子家族性高胆固醇血症(HoFH)的特点是低密度脂蛋白胆固醇(LDL-C)水平异常升高和早期动脉粥样硬化性血管疾病。本研究旨在评估前蛋白转化酶枯草杆菌素9抑制剂阿利昔单抗对成人HoFH患者低密度脂蛋白胆固醇(LDL-C)的降低作用。

本研究将69名纯合子家族性高胆固醇血症患者随机2:1分至阿利昔单抗治疗组和安慰剂对照组。基线水平时,67名患者接受了他汀类药物治疗,安慰剂组的平均基线LDL-C为259.6毫克/分升,阿利昔单抗组为295.0毫克/分升。在第12周,与基线检查相比,LDL-C百分比变化的最小平方平均差为-35.6%(阿利罗库单抗[-26.9%]与安慰剂[8.6%];p<0.0001)。其他致动脉粥样硬化脂质的降低分别为:载脂蛋白B-29.8%;非高密度脂蛋白胆固醇-32.9%;总胆固醇-26.5%;脂蛋白(a)-28.4%(p<0.0001)。另外,在双盲治疗期间,未报告严重不良事件、永久性停药或因治疗引起的不良事件而死亡。

研究结果显示,对于纯合子家族性高胆固醇血症患者,阿利昔单抗能明显降低患者12周时的低密度脂蛋白胆固醇,安全性较好。

原始出处:

Dirk J. B et al.Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia.JACC.2020 July.

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    2020-11-05 hbwxf
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    2020-07-11 wetgdt
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    2020-07-09 内科新手

    谢谢梅斯提供这么好的信息,学到很多

    0

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