Won Jane I, Zhang Jun, Tecson Kristen M, McCullough Peter A
Baylor University Medical Center, Baylor Jack and Jane Hamilton Heart and Vascular Hospital, Baylor Heart and Vascular Institute, Dallas, TX; The Heart Hospital, Plano, TX.
Rev Cardiovasc Med. 2017;18(1):21-28. doi: 10.3909/ricm0834.
Homozygous familial hypercholesterolemia (HoFH) is an autosomal codominant disorder manifested by high concentrations of total cholesterol and low-density lipoprotein (LDL) cholesterol, and premature cardiovascular disease. Despite conventional lipid-lowering therapy, LDL cholesterol levels remain elevated in patients with HoFH; these patients are considered to be at high risk for cardiovascular events. In 2012-2013, two drugs with novel mechanisms of action were approved by the US Food and Drug Administration for the treatment of HoFH: lomitapide mesylate and mipomersen. Both of these treatments reduce total cholesterol, LDL cholesterol, non-high-density lipoprotein cholesterol, apolipoprotein B, lipoprotein a, and triglyceride levels. This review describes the clinical tradeoffs in efficacy and hepatotoxicity of these drugs in two cases of HoFH.
纯合子家族性高胆固醇血症（HoFH）是一种常染色体共显性疾病，表现为总胆固醇和低密度脂蛋白（LDL）胆固醇浓度升高，以及心血管疾病早发。尽管采用了传统的降脂治疗，但HoFH患者的LDL胆固醇水平仍居高不下；这些患者被认为发生心血管事件的风险很高。2012年至2013年，美国食品药品监督管理局批准了两种作用机制新颖的药物用于治疗HoFH：甲磺酸洛美他派和米泊美生。这两种治疗方法均可降低总胆固醇、LDL胆固醇、非高密度脂蛋白胆固醇、载脂蛋白B、脂蛋白a和甘油三酯水平。本综述描述了这两种药物在两例HoFH患者中的疗效和肝毒性方面的临床权衡。
