title: Efficacy and safety of proprotein convertase subtilisin/kexin type 9 monoclonal antibody in adults with familial hypercholesterolemia.
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; I2= 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; I2= 0%). PCSK9 antibody may be an effective and safe treatment for FH.
authors.affiliation: Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Bin
authors.initials: B
authors.lastname: Li
authors.affiliation: 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.
authors.forename: Pan-Pan
authors.initials: PP
authors.lastname: Hao
authors.affiliation: Department of Neurology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Yong
authors.initials: Y
authors.lastname: Zhang
authors.affiliation: Department of Gastroenterology, First People's Hospital of Jinan, Jinan, Shandong, China.
authors.forename: Rui-Hong
authors.initials: RH
authors.lastname: Yin
authors.affiliation: Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Qing-Zan
authors.initials: QZ
authors.lastname: Kong
authors.affiliation: Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Xiao-Jun
authors.initials: XJ
authors.lastname: Cai
authors.affiliation: Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Zhuo
authors.initials: Z
authors.lastname: Zhao
authors.affiliation: Central Laboratory, Shandong Provincial Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Jian-Ni
authors.initials: JN
authors.lastname: Qi
authors.affiliation: Central Laboratory, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Ying
authors.initials: Y
authors.lastname: Li
authors.affiliation: 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.
authors.forename: Jie
authors.initials: J
authors.lastname: Xiao
authors.affiliation: 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.
authors.forename: Fu
authors.initials: F
authors.lastname: Wang
authors.affiliation: Engineering Training Center, Shandong University, Jinan, Shandong, China.
authors.forename: Wei
authors.initials: W
authors.lastname: Yi
authors.affiliation: 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.
authors.forename: Xiao-Ping
authors.initials: XP
authors.lastname: Ji
authors.affiliation: Department of Cardiology, Jinan Central Hospital affiliated to Shandong University, Jinan, Shandong, China.
authors.forename: Guo-Hai
authors.initials: GH
authors.lastname: Su
mesh_terms: D000818:Animals; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D015415:Biomarkers; D002986:Clinical Trials as Topic; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy* / Q000378:metabolism; D008055:Lipids / Q000097:blood; D000091362:PCSK9 Inhibitors; D017594:Publication Bias; D016896:Treatment Outcome; D000071449:Proprotein Convertase 9
chemical_list: D000911:Antibodies, Monoclonal; D015415:Biomarkers; D008055:Lipids; D000091362:PCSK9 Inhibitors; C472125:PCSK9 protein, human; D000071449:Proprotein Convertase 9
keywords: efficacy; familial hypercholesterolemia; proprotein convertase subtilisin/kexin type 9 monoclonal antibody; safety