{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,3]],"date-time":"2026-09-03T17:32:19Z","timestamp":1788456739630,"version":"build-2803163510"},"reference-count":39,"publisher":"Oxford University Press (OUP)","issue":"17","license":[{"start":{"date-parts":[[2023,6,14]],"date-time":"2023-06-14T00:00:00Z","timestamp":1686700800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009857","name":"Regeneron Pharmaceuticals, Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009857","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,11,30]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>Despite intensive lipid-lowering therapies (LLTs), most patients with homozygous familial hypercholesterolaemia (HoFH) do not achieve guideline recommended low-density lipoprotein cholesterol (LDL-C) targets and are at increased risk of premature cardiovascular death. This analysis aimed to predict the impact of evinacumab and standard-of-care LLTs on life expectancy in an HoFH population using mathematical modelling.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods and results<\/jats:title>\n                    <jats:p>Mathematical models were developed using efficacy data for evinacumab from the phase 3 ELIPSE HoFH trial plus efficacy data for standard-of-care LLTs from peer-reviewed publications. Treatment strategies evaluated included (i) untreated, (ii) high-intensity statin (HIS) only, (iii) HIS plus ezetimibe, (iv) HIS plus ezetimibe plus proprotein convertase subtilisin\/kexin type 9 inhibitor (PCSK9i), and (v) HIS plus ezetimibe plus PCSK9i plus evinacumab. Markov analyses were used to assess differences in survival probability for different LLT strategies. The median survival for untreated HoFH patients was only 33\u201343 years, depending on different assumptions on baseline untreated LDL-C levels. In the most robust model, we estimated that HIS increased median survival by 9 years and ezetimibe further increased median survival by an additional 9 years. When PCSK9i was added on top of HIS plus ezetimibe, median survival was further improved by 14 years. Finally, the addition of evinacumab to standard-of-care LLTs was estimated to increase median survival by \u223c12 years.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>In this mathematical modelling analysis, evinacumab treatment could potentially increase long-term survival vs. standard-of-care LLTs for patients with HoFH.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/eurjpc\/zwad203","type":"journal-article","created":{"date-parts":[[2023,6,13]],"date-time":"2023-06-13T19:31:57Z","timestamp":1686684717000},"page":"1874-1880","source":"Crossref","is-referenced-by-count":10,"title":["Modelling the potential long-term survival benefit of evinacumab treatment vs. standard of care in patients with homozygous familial hypercholesterolaemia"],"prefix":"10.1093","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5735-8482","authenticated-orcid":false,"given":"Jing","family":"Gu","sequence":"first","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc. , 777 Old Saw Mill, River Road, Tarrytown, NY 10591 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreas","family":"Kuznik","sequence":"additional","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc. , 777 Old Saw Mill, River Road, Tarrytown, NY 10591 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Quon","sequence":"additional","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc. , 777 Old Saw Mill, River Road, Tarrytown, NY 10591 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankita","family":"Chauhan","sequence":"additional","affiliation":[{"name":"Axtria , Berkeley Heights, NJ , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tupili S","family":"Sravya","sequence":"additional","affiliation":[{"name":"Axtria , Berkeley Heights, NJ , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frederick J","family":"Raal","sequence":"additional","affiliation":[{"name":"Carbohydrate and Lipid Metabolism Research Unit, Division of Endocrinology and Metabolism, Department of Medicine, Faculty of Health Sciences, University of the Witwatersrand , Johannesburg , South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2023,6,14]]},"reference":[{"key":"2023113004072259200_zwad203-B1","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1093\/eurheartj\/ehz455","article-title":"2019 ESC\/EAS guidelines for the management of dyslipidaemias: lipid modification to reduce cardiovascular risk","volume":"41","author":"Mach","year":"2020","journal-title":"Eur Heart J"},{"key":"2023113004072259200_zwad203-B2","doi-asserted-by":"crossref","first-page":"1067","DOI":"10.1161\/CIRCULATIONAHA.115.018791","article-title":"Prevalence of familial hypercholesterolemia in the 1999 to 2012 United States National Health and Nutrition Examination Surveys (NHANES)","volume":"133","author":"de Ferranti","year":"2016","journal-title":"Circulation"},{"key":"2023113004072259200_zwad203-B3","doi-asserted-by":"crossref","first-page":"2146","DOI":"10.1093\/eurheartj\/ehu274","article-title":"Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. 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