抗前蛋白转化酶枯草溶菌素9单克隆抗体临床研究进展

Clinical Research Progress of Anti-Proprotein Convertase Subtilisin/Kexin Type 9( PCSK9 ) Monoclonal Antibodies

系统查阅近年采抗前蛋白转化酶枯草溶菌素9（proprotein convertase subtilisin/kexin type 9, PCSK9）单克隆抗体（单抗）治疗高胆固醇血症的临床研究，分析和归纳其结果发现，在Ⅰ，Ⅱ，Ⅲ期临床试验中，抗前蛋白转化酶枯草溶茵素9单抗与安慰剂或依折麦布相比，可降低高胆固醇血症患者低密度脂蛋白胆固醇（low-density lipoprotein-cholesterol, LDL-C）50%-60%，总体不良反应较少。抗前蛋白转化酶枯草溶茵素9单抗治疗高胆固醇血症的有效性和安全性已得到短期（最长52周）临床研究证实。目前正在进行扩大的Ⅲ期临床实验以评估长期用药的安全性和有效性，以及是否具有心血管疾病的保护作用。

To summarize the data from clinical trials of anti-proprotein convertase subtilisin/kexin type 9 （PCSK9） monoclonal antibodies （mAbs） in patients with hypercholesterolaemia. Based on systematic literature review, theresults of clinical trials of different anti-PCSK9 mAbs were presented and evaluated. In phase Ⅰ,Ⅱ,Ⅲ trials, anti-PCSK9 mAbs resulted in 50% -60% reduction in low-density lipoprotein-cbolesterol （LDL-C） level and bad a lower incidence of adverse effects, compared with placebo or ezetimibein patients with hypercholesterolaemia. Anti-PCSK9 mAbs have demonstrated a significant effect of reducing LDL-C and were well tolerated in short-term （up to 52 weeks） trials. Large phase Ⅲ trials are ongoing to evaluate the long-term safety and efficacy as well as the impact of these promising new agents on cardiovascular outcomes.