{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,7]],"date-time":"2026-06-07T00:10:52Z","timestamp":1780791052795,"version":"3.54.1"},"reference-count":22,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2024,9,6]],"date-time":"2024-09-06T00:00:00Z","timestamp":1725580800000},"content-version":"vor","delay-in-days":5,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["accpjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacotherapy"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Heterozygous familial hypercholesterolemia (HeFH) is a genetic condition that is associated with a high risk of atherosclerotic cardiovascular disease (ASCVD) due to elevated lipid levels. Proprotein convertase subtilisin\/kexin type 9 (PCSK9) monoclonal antibody inhibitors have been shown to reduce low\u2010density lipoprotein cholesterol (LDL\u2010C) substantially. This study aimed to assess the real\u2010world effectiveness of PCSK9 inhibitor therapy among patients with HeFH.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Retrospective cohort study of patients with probable or definite HeFH on a PCSK9 inhibitor at a specialized lipid clinic between 2015 and 2022. The primary objective was the proportion of patients who attained a \u226550% reduction in LDL\u2010C after 12\u2009months of treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In total, 141 patients were screened and 95 were included. Mean age was 63\u2009years, 51% were female, and mean baseline LDL\u2010C level was 4.0\u2009mmol\/L (155\u2009mg\/dL). A majority of patients (60%) had statin intolerance, and 73% were on ezetimibe. The most common PCSK9 inhibitor was evolocumab (94%). Overall, 74% of patients achieved a \u226550% reduction in LDL\u2010C after 12\u2009months of therapy. Mean LDL\u2010C concentration decreased to 1.7\u2009mmol\/L (66\u2009mg\/dL) (approximately 59% reduction from baseline) after 12\u2009months of follow\u2010up but increased to 1.9\u2009mmol\/L (73\u2009mg\/dL) after \u226524\u2009months of follow\u2010up. Similar trends were observed in non\u2010high\u2010density lipoprotein cholesterol and apolipoprotein B. Lipoprotein(a) was significantly reduced by 45% over 12\u2009months. Twelve percent of patients permanently discontinued therapy. Barriers to PCSK9i use were mostly related to cost.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In a real\u2010world cohort of HeFH patients, most of which were intolerant to statins, a high majority were able to achieve a \u226550% reduction in LDL\u2010C after 12\u2009months of PCSK9 inhibitor therapy (mean reduction of approximately 59%), which is similar to clinical trial data of patients with ASCVD. A significant reduction in non\u2010high\u2010density lipoprotein cholesterol, apolipoprotein B, and lipoprotein(a) were also observed.<\/jats:p><\/jats:sec>","DOI":"10.1002\/phar.4609","type":"journal-article","created":{"date-parts":[[2024,9,6]],"date-time":"2024-09-06T05:30:44Z","timestamp":1725600644000},"page":"730-737","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Real\u2010world effectiveness of monoclonal antibody inhibitors of <scp>PCSK<\/scp>9 in patients with heterozygous familial hypercholesterolemia: A retrospective cohort study"],"prefix":"10.1002","volume":"44","author":[{"given":"Rebecca","family":"Siemens","sequence":"first","affiliation":[{"name":"Surrey Memorial Hospital, Fraser Health (Lower Mainland Pharmacy Services)  Surrey British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Pryjma","sequence":"additional","affiliation":[{"name":"Jim Pattison Outpatient Care and Surgery Centre, Fraser Health (Lower Mainland Pharmacy Services)  Surrey British\u00a0Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Buchkowsky","sequence":"additional","affiliation":[{"name":"Richmond Hospital, Vancouver Coastal Health (Lower Mainland Pharmacy Services)  Richmond British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0287-898X","authenticated-orcid":false,"given":"Arden R.","family":"Barry","sequence":"additional","affiliation":[{"name":"Jim Pattison Outpatient Care and Surgery Centre, Fraser Health (Lower Mainland Pharmacy Services)  Surrey British\u00a0Columbia Canada"},{"name":"Faculty of Pharmaceutical Sciences The University of British Columbia  Vancouver British Columbia 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FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia","volume":"36","author":"Kastelein JJ","year":"2015","journal-title":"Eur Heart J"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cjca.2018.04.014"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cjco.2022.03.003"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/clc.23600"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1161\/JAHA.119.014129"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2019.10.057"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.5603\/EP.a2023.0036"}],"container-title":["Pharmacotherapy: The Journal of Human Pharmacology and Drug 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