{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T04:55:22Z","timestamp":1787028922535,"version":"build-2736575974"},"reference-count":49,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2024,10,8]],"date-time":"2024-10-08T00:00:00Z","timestamp":1728345600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Natural Science Foundation General Project of the Sichuan Provincial Science and Technology Department","award":["2024NSFSC0633"],"award-info":[{"award-number":["2024NSFSC0633"]}]},{"name":"Natural Science Foundation General Project of the Sichuan Provincial Science and Technology Department","award":["SCU2022F4080"],"award-info":[{"award-number":["SCU2022F4080"]}]},{"name":"Natural Science Foundation General Project of the Sichuan Provincial Science and Technology Department","award":["Q23055"],"award-info":[{"award-number":["Q23055"]}]},{"name":"The Fundamental Research Funds for the Central Universities","award":["2024NSFSC0633"],"award-info":[{"award-number":["2024NSFSC0633"]}]},{"name":"The Fundamental Research Funds for the Central Universities","award":["SCU2022F4080"],"award-info":[{"award-number":["SCU2022F4080"]}]},{"name":"The Fundamental Research Funds for the Central Universities","award":["Q23055"],"award-info":[{"award-number":["Q23055"]}]},{"name":"Sichuan Medical Youth Innovation Project","award":["2024NSFSC0633"],"award-info":[{"award-number":["2024NSFSC0633"]}]},{"name":"Sichuan Medical Youth Innovation Project","award":["SCU2022F4080"],"award-info":[{"award-number":["SCU2022F4080"]}]},{"name":"Sichuan Medical Youth Innovation Project","award":["Q23055"],"award-info":[{"award-number":["Q23055"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Medicina"],"abstract":"<jats:p>Background and Objectives: The proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibitors evolocumab and alirocumab are recently developed promising drugs used for treatment of familial hypercholesterolemia (FH). This systematic review and meta-analysis aimed to thoroughly evaluate the efficacy and safety of evolocumab and alirocumab among pediatric patients with FH. Materials and Methods: A comprehensive search was conducted in PubMed, Embase, CENTRAL (Cochrane Central Register of Controlled Trials), and ClinicalTrials.gov from inception through July 2024 to identify primary interventional studies among pediatric patients with FH. Meta-analyses were performed if appropriate. Statistics were analyzed using Review Manager version 5.4 and Stata version 16.0. Results: Fourteen articles reporting nine unique studies were included. There were three randomized controlled trials (RCTs) assessing evolocumab or alirocumab involving a total of 320 pediatric patients, one cross-over trial and five single-arm or observational studies. Pooled results showed significant efficacy of evolocumab\/alirocumab in reducing low-density lipoprotein cholesterol (LDL-C) (weighted mean difference [WMD]: \u221237.92%, 95% confidence interval [CI]: \u221243.06% to \u221232.78%; I2 = 0.0%, p = 0.60), apolipoprotein B (WMD: \u221233.67%, 95% CI: \u221238.12% to \u221229.22%; I2 = 0.0%, p = 0.71), and also lipoprotein(a) (WMD: \u221216.94%, 95% CI: \u221226.20% to \u22127.69%; I2 = 0.0%, p = 0.71) among pediatric patients with FH. The efficacies of evolocumab\/alirocumab on LDL-C reduction within pediatric patients with heterozygous FH (HeFH) were consistent between studies, whereas in patients with homozygous FH (HoFH), it varied dramatically. Pediatric patients with the null\/null variant may respond to the treatment. PCSK9 inhibitors were generally well tolerated within most pediatric patients, in line with previous studies among adult populations. Conclusions: The PCSK9 inhibitors evolocumab\/alirocumab significantly reduced LDL-C and some other lipid parameters, such as apolipoprotein B, in pediatric patients with HeFH. These drugs may be appropriate as a potential therapy for pediatric patients with HoFH who cannot achieve LDL-C targets with other treatments. Evolocumab\/alirocumab was generally well tolerated in the pediatric population.<\/jats:p>","DOI":"10.3390\/medicina60101646","type":"journal-article","created":{"date-parts":[[2024,10,8]],"date-time":"2024-10-08T12:01:54Z","timestamp":1728388914000},"page":"1646","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Efficacy and Safety of Evolocumab and Alirocumab as PCSK9 Inhibitors in Pediatric Patients with Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis"],"prefix":"10.3390","volume":"60","author":[{"given":"Guoguang","family":"Xiao","sequence":"first","affiliation":[{"name":"Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China"},{"name":"Department of Pediatrics, West China Xiamen Hospital of Sichuan University, Xiamen 361022, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shan","family":"Gao","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yongmei","family":"Xie","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhiling","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China"},{"name":"Key Laboratory of Birth Defects and Related Diseases of Women and Children (Sichuan University), Ministry of Education, Chengdu 610041, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Min","family":"Shu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China"},{"name":"Department of Pediatrics, West China Xiamen Hospital of Sichuan University, Xiamen 361022, China"},{"name":"Key Laboratory of Birth Defects and Related Diseases of Women and Children (Sichuan University), Ministry of Education, Chengdu 610041, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,10,8]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"17093","DOI":"10.1038\/nrdp.2017.93","article-title":"Familial hypercholesterolaemia","volume":"3","author":"Defesche","year":"2017","journal-title":"Nat. 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