Long-Term Evolocumab in Patients With Familial Hypercholesterolemia
Long-Term Evolocumab in Patients With Familial Hypercholesterolemia
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DOI：
10.1016/j.jacc.2019.12.020
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发表时间：
2020-02-18
影响因子：
24
通讯作者：
Raal, Frederick J.
中科院分区：
文献类型：
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作者：
Santos, Raul D.;Stein, Evan A.;Raal, Frederick J.
背景蛋白原转化酶枯草杆菌蛋白酶/kexin 9型抑制剂疗法是不能达到低密度脂蛋白胆固醇（LDL-C）目标的家族性高胆固醇血症（FH）患者的治疗选择。HoFH或重度HeFH ≥ 12岁且接受稳定降脂治疗的患者开始皮下注射依洛尤单抗420 mg每月一次或420 mg每2周一次（如果接受脂蛋白单采术）。12周后，未进行单采的患者可以每2周将剂量上调至420 mg。主要终点是治疗后出现的不良事件的发生率;次要终点是LDL-C和其他血脂的变化。
BACKGROUND Proprotein convertase subtilisin/kexin type 9 inhibitor therapy is a treatment option for patients with familial hypercholesterolemia (FH) who are unable to reach low-density lipoprotein cholesterol (LDL-C) goals.OBJECTIVES The aim of this study was to provide long-term safety and efficacy data for evolocumab in patients with homozygous FH (HoFH) and severe heterozygous FH (HeFH).METHODS In this open-label, single-arm study, patients with HoFH or severe HeFH >= 12 years of age and on stable lipid-lowering therapy began subcutaneous evolocumab 420 mg monthly or 420 mg every 2 weeks if on lipoprotein apheresis. After 12 weeks, those not on apheresis could be up-titrated to 420 mg every 2 weeks. The primary endpoint was the incidence of treatment-emergent adverse events; secondary endpoints were changes in LDL-C and other lipids.RESULTS In total, 300 patients (106 with HoFH, including 14