Cuevas Ada, Farías María Magdalena, Alonso Rodrigo
Rev Med Chil. 2014 Jul;142(7):880-8. doi: 10.4067/S0034-98872014000700008.
Statins are the preferred treatment for hypercholesterolemia and several studies have demonstrated their long-term safety and efficacy in reducing cardiovascular morbidity and mortality. However, in some cases of severe hypercholesterolemia such as homozygous and heterozygous familial hypercholesterolemia or statin intolerant patients, statins can be less efficient. In recent years, new lipid-lowering agents with novel mechanisms of action have been developed to reduce LDL-cholesterol in patients with severe hypercholesterolemia, associated or not to conventional lipid-lowering therapy. These therapies include microsomal transfer protein inhibitor (Lomitapide), antisense oligonucleotide to Apo B100 (Mipomersen) and monoclonal antibodies against Proprotein convertase subtilisin/kexin type 9 (PCSK9). Different studies have shown the great effectiveness of these new therapies. Short-term studies confirmed their adequate security profile, especially in patients with homozygous familiar hypercholesterolemia or severe hypercholesterolemia. Some of these agents have been also tested in statin-intolerant patients. However, long-term studies are needed to evaluate their safety, effectiveness and impact on cardiovascular risk reduction.
他汀类药物是高胆固醇血症的首选治疗药物,多项研究已证明其在降低心血管发病率和死亡率方面的长期安全性和有效性。然而,在某些严重高胆固醇血症病例中,如纯合子和杂合子家族性高胆固醇血症或他汀类药物不耐受患者,他汀类药物的效果可能较差。近年来,已开发出具有新型作用机制的新型降脂药物,用于降低严重高胆固醇血症患者的低密度脂蛋白胆固醇,无论是否与传统降脂治疗联合使用。这些疗法包括微粒体转移蛋白抑制剂(洛美他派)、载脂蛋白B100反义寡核苷酸(米泊美生)和抗前蛋白转化酶枯草溶菌素/kexin 9型(PCSK9)单克隆抗体。不同的研究已表明这些新疗法具有显著疗效。短期研究证实了它们具有足够的安全性,尤其是在纯合子家族性高胆固醇血症或严重高胆固醇血症患者中。其中一些药物也已在他汀类药物不耐受患者中进行了测试。然而,需要进行长期研究以评估它们的安全性、有效性以及对降低心血管风险的影响。