{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T01:43:13Z","timestamp":1792633393646,"version":"4.5.0"},"reference-count":33,"publisher":"Public Library of Science (PLoS)","issue":"3","license":[{"start":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T00:00:00Z","timestamp":1647993600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:sec id=\"sec001\">\n<jats:title>Background and aims<\/jats:title>\n<jats:p>Familial dysbetalipoproteinemia (FDBL) is a rare inborn lipid disorder characterized by the formation of abnormal triglyceride- and cholesterol-rich lipoproteins (remnant particles). Patients with FDBL have a high risk for atherosclerotic disease. The effect of PCSK9 inhibition on lipoproteins and its subfractions has not been evaluated in FDBL.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec002\">\n<jats:title>Methods<\/jats:title>\n<jats:p>Three patients (65\u00b17 years, 23\u00b13 kg\/m<jats:sup>2<\/jats:sup>, 2 females) with FDBL (diagnosed by isoelectrofocusing) and atherosclerosis (coronary and\/or cerebro-vascular and\/or peripheral arterial disease) resistant or intolerant to statin and fibrate therapy received evolocumab (140mg every 14 days). In addition to a fasting lipid profile (preparative ultracentrifugation), apoB and cholesterol concentrations were determined in 15 lipoprotein-subfractions (density gradient ultracentrifugation; d 1.006\u20131.21g\/ml) before and after 12 weeks of evolocumab treatment. Patients with LDL-hypercholesterolemia (n = 8, 56\u00b18 years, 31\u00b17 kg\/m<jats:sup>2<\/jats:sup>) and mixed hyperlipidemia (n = 5, 68\u00b112 years, 30\u00b11 kg\/m<jats:sup>2<\/jats:sup>) also receiving evolocumab for 12 weeks were used for comparison.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec003\">\n<jats:title>Results<\/jats:title>\n<jats:p>All patients tolerated PCSK9 inhibition well. PCSK9 inhibitors reduced cholesterol (29\u201337%), non-HDL-cholesterol (36\u201350%) and apoB (40\u201352%) in all patient groups including FDBL. In FDBL, PCSK9 inhibition reduced VLDL-cholesterol and the concentration of apoB containing lipoproteins throughout the whole density spectrum (VLDL, IDL, remnants, LDL). Lipoprotein(a) was decreased in all patient groups to a similar extent.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec004\">\n<jats:title>Conclusions<\/jats:title>\n<jats:p>This indicates that the dominant fraction of apoB-containing lipoproteins is reduced with PCSK9 inhibition, <jats:italic>i<\/jats:italic>.<jats:italic>e<\/jats:italic>. LDL in hypercholesterolemia and mixed hyperlipidemia, and cholesterol-rich VLDL, remnants and LDL in FDBL. PCSK9 inhibition may be a treatment option in patients with FDBL resistant or intolerant to statin and\/or fibrate therapy.<\/jats:p>\n<\/jats:sec>","DOI":"10.1371\/journal.pone.0265838","type":"journal-article","created":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T17:37:57Z","timestamp":1648057077000},"page":"e0265838","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":13,"title":["Effect of PCSK9 inhibition with evolocumab on lipoprotein subfractions in familial dysbetalipoproteinemia (type III hyperlipidemia)"],"prefix":"10.1371","volume":"17","author":[{"given":"Elisa","family":"Waldmann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liya","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristina","family":"Busygina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julia","family":"Altenhofer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kerstin","family":"Henze","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"Folwaczny","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9873-0412","authenticated-orcid":true,"given":"Klaus G.","family":"Parhofer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2022,3,23]]},"reference":[{"key":"pone.0265838.ref001","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1097\/MOL.0000000000000192","article-title":"Dysbetalipoproteinemia: an extreme disorder of remnant metabolism","volume":"26","author":"D. 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