Universidad Científica del Sur, Lima, Peru.
Department of Cardiology Research, Torres de Salud National Research Center, Lima, Peru.
Vasc Health Risk Manag. 2022 Feb 21;18:73-80. doi: 10.2147/VHRM.S191965. eCollection 2022.
Familial hypercholesterolemia (FH) is an autosomal dominant condition that leads to significantly elevated low-density lipoprotein cholesterol (LDL-C) levels and an elevated risk for cardiovascular disease. Mipomersen is an antisense oligonucleotide inhibitor targeted to apolipoprotein B-100 (apoB-100) mRNA that is administered via subcutaneous injection. Once administered, mipomersen causes selective degradation of the apoB-100 mRNA and inhibition of protein translation. This ultimately results in substantial reductions in LDL-C and other lipoprotein levels. Mipomersen is approved for the treatment of homozygous FH. In this review, we discuss its mechanism, current evidence, limitations of use including adverse events, and impact on health-related quality of life.
家族性高胆固醇血症（FH）是一种常染色体显性遗传疾病，可导致低密度脂蛋白胆固醇（LDL-C）水平显著升高，并使心血管疾病风险升高。米泊美生是一种针对载脂蛋白 B-100（apoB-100）mRNA 的反义寡核苷酸抑制剂，通过皮下注射给药。给药后，米泊美生可导致 apoB-100 mRNA 的选择性降解和蛋白质翻译抑制。这最终导致 LDL-C 和其他脂蛋白水平的大幅降低。米泊美生已被批准用于治疗纯合子家族性高胆固醇血症。在这篇综述中，我们讨论了它的作用机制、现有证据、包括不良反应在内的使用限制，以及对健康相关生活质量的影响。
