Butt Waleed Z, Yee Jennifer K
Division of Endocrinology, Department of Internal Medicine, Harbor-UCLA Medical Center, 1000 W. Carson Street, Harbor Box 446, Torrance, CA, 90509, USA.
The Lundquist Institute of Biomedical Innovation at Harbor, UCLA Medical Center, 1124 W. Carson Street, Martin Building, Torrance, CA, 90502, USA.
Curr Atheroscler Rep. 2022 May;24(5):379-389. doi: 10.1007/s11883-022-01013-x. Epub 2022 Mar 28.
Lifestyle modification is additive to lipid-lowering medications in the treatment of heterozygous familial hypercholesterolemia (HeFH), which does not respond sufficiently to statin therapy. While both are also important in homozygous familial hypercholesterolemia (HoFH), additional measures such as apheresis may be needed. The purpose of this review is to identify non-statin medications to lower cholesterol that are available for children and adolescents as adjunctive therapy.
Ezetimibe is commonly used as second-line pharmacotherapy for treatment of HeFH and HoFH. Colesevelam, a bile acid sequestrant, may be considered for adjunct therapy. Since 2015, the PCSK9 inhibitor evolocumab has been available for adolescents, and its FDA approval has now expanded to age 10 years. The ANGPTL3 inhibitor evinacumab has been approved for children age 12 years and older. A clinical trial for lomitapide is in progress. Approvals for PCSK9 and ANGPTL3 inhibitors have expanded opportunities for children and adolescents with HeFH and HoFH to achieve lower LDL-C levels.
生活方式的改变对于杂合子家族性高胆固醇血症（HeFH）的治疗是降低血脂药物的补充，HeFH对他汀类药物治疗反应不足。虽然生活方式改变和药物治疗对纯合子家族性高胆固醇血症（HoFH）也很重要，但可能还需要诸如血液分离术等其他措施。本综述的目的是确定可用于儿童和青少年作为辅助治疗的非他汀类降胆固醇药物。
依折麦布通常用作治疗HeFH和HoFH的二线药物治疗。考来维仑，一种胆汁酸螯合剂，可考虑用于辅助治疗。自2015年以来，前蛋白转化酶枯草溶菌素9（PCSK9）抑制剂依洛尤单抗已可用于青少年，其美国食品药品监督管理局（FDA）的批准范围现已扩大到10岁。血管生成素样蛋白3（ANGPTL3）抑制剂evinacumab已被批准用于12岁及以上的儿童。洛米他派的一项临床试验正在进行中。PCSK9和ANGPTL3抑制剂的批准为患有HeFH和HoFH的儿童和青少年提供了降低低密度脂蛋白胆固醇（LDL-C）水平的更多机会。
