ObjectivesTo systematically review the efficacy and safety of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor on hypercholesteremia.MethodsDatabases including The Cochrane Library, PubMed, EMbase, CNKI, WanFang Data and CBM were searched electronically for randomized controlled trials (RCTs) of PCSK9 inhibitor on the treatment of hypercholesteremia from inception to June 2019. Two reviewers independently screened literatures, extracted data, and assessed risk of bias of included studies. Then, meta-analysis was performed using RevMan 5.3 software.ResultsA total of 42 RCTs involving 90 058 patients were included. Alirocumab, evolocumab, bococizumab and LY3015014 were retrieved. Placebo, statin or ezetimibe were used to treat the patient in the control group. The efficacy results showed that there were significant differences between alirocumab combined statin and placebo combined statin (MD=−49.89, 95%CI −56.34 to −43.44, P<0.00001) or evolocumab combined statin and placebo combined statin (MD=−56.54, 95%CI −61.09 to −52.00, P<0.00001) in reducing LDL-C levels. There was significant difference between alirocumab combined statin and ezetimibe combined statin (MD=−29.41, 95%CI −36.08 to −22.75, P<0.00001) in reducing LDL-C levels. The results of Safety showed that there were no significant differences between alirocumab and placebo (RR=0.99, 95%CI 0.97 to 1.01, P=0.37) or evolocumab and placebo (RR=1.05, 95%CI 1.00 to 1.10, P=0.07) in the incidence of adverse events (AEs). There was significant difference between bococizumab and placebo (RR=1.09, 95%CI 1.01 to 1.18, P=0.03) in the incidence of AEs. There was significant difference between evolocumab and ezetimibe (RR=0.89, 95%CI 0.80 to 0.99, P=0.04) in the incidence of AEs. There was no significant difference between alirocumab and statin (RR=0.93, 95%CI 0.84 to 1.03, P=0.90) in the incidence of AEs.ConclusionsPCSK9 inhibitor can reduce LDL-C levels in patients with hypercholesterolemia and has good safety. Due to the limitation of the quantity and quality of the included studies, the above conclusions are needed to be verified by more high quality studies.