Efficacy and safety of proprotein convertase subtilisin/kexin type 9 monoclonal antibody in adults with familial hypercholesterolemia

// Bin Li 1 , Pan-Pan Hao 2 , Yong Zhang 3 , Rui-Hong Yin 4 , Qing-Zan Kong 1 , Xiao-Jun Cai 1 , Zhuo Zhao 1 , Jian-Ni Qi 5 , Ying Li 6 , Jie Xiao 2 , Fu Wang 2 , Wei Yi 7 , Xiao-Ping Ji 2 and Guo-Hai Su 1 1 Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China 2 Key Laboratory of Cardiovascular Remodeling and Function Research, Chinese Ministry of Education and Chinese Ministry of Health, Department of Cardiology, Shandong University Qilu Hospital, Jinan, Shandong, China 3 Department of Neurology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China 4 Department of Gastroenterology, First People’s Hospital of Jinan, Jinan, Shandong, China 5 Central Laboratory, Shandong Provincial Hospital affiliated to Shandong University, Jinan, Shandong, China 6 Central Laboratory, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China 7 Engineering Training Center, Shandong University, Jinan, Shandong, China Correspondence to: Guo-Hai Su, email: // Keywords : efficacy, safety, proprotein convertase subtilisin/kexin type 9 monoclonal antibody, familial hypercholesterolemia Received : April 27, 2016 Accepted : July 10, 2016 Published : July 21, 2016 Abstract Proprotein convertase-subtilisin/kexin type 9 (PCSK9) monoclonal antibody is a new therapy to reduce low-density lipoprotein cholesterol (LDL-C) level in patients with familial hypercholesterolemia (FH). This pooled analysis aimed to estimate the efficacy and safety of PCSK9 antibody therapy in FH. Reports of randomized controlled trials (RCTs) comparing PCSK9 antibody to placebo were retrieved by a search of MEDLINE via PubMed, EMBASE, the Cochrane Library databases, ClinicalTrials.gov and Clinical Trial Results (up to November 30, 2015) with no language restriction. Data were abstracted by a standardized protocol. We found eight RCTs (1,879 patients with FH) for the pooled analysis. As compared with placebo, PCSK9 antibody therapy remarkably reduced LDL-C level (mean reduction: −48.54 %, 95 % CI: −53.19 to −43.88), total cholesterol (mean reduction: -31.08%, 95 % CI: -35.20 to -26.95), lipoprotein (a) (mean reduction: -20.44%, 95 % CI: -25.21 to -15.66), and apolipoprotein B (mean reduction: -36.32%, 95 % CI: -40.75 to -31.90) and elevated the level of high-density lipoprotein cholesterol (mean change: 6.29 %, 95 % CI: 5.12 to 7.46) and apolipoprotein A1(mean change: 4.86%, 95 % CI: 3.77 to 5.95). Therapy with and without PCSK9 antibodies did not differ in rate of adverse events (pooled rate: 50.86 % vs. 48.63%; RR: 1.03; 95 % CI: 0.92 to 1.15; P = 0.64; heterogeneity P = 0.13; I 2 = 40%) or serious adverse events (pooled rate: 7.14% vs. 6.74%; RR: 1.05; 95 % CI: 0.70 to 1.58; P = 0.80; heterogeneity P = 0.69; I 2 = 0%). PCSK9 antibody may be an effective and safe treatment for FH.