{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T20:50:25Z","timestamp":1789505425815,"version":"build-2803163510"},"reference-count":17,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"7","content-domain":{"domain":["www.ahajournals.org"],"crossmark-restriction":true},"short-container-title":["ATVB"],"published-print":{"date-parts":[[2025,7]]},"abstract":"<jats:sec>\n                    <jats:title>BACKGROUND:<\/jats:title>\n                    <jats:p>Homozygous familial hypercholesterolemia (HoFH) is an autosomal semidominant disorder characterized by extreme elevations in LDL-C (low-density lipoprotein cholesterol) and early-onset atherosclerotic cardiovascular disease. Evinacumab is a monoclonal antibody administered by monthly intravenous infusion that binds ANGPTL3 (angiopoietin-like 3) and when added to standard lipid-lowering therapies lowers LDL-C by \u224850% in HoFH clinical trials. Studies examining the real-world effectiveness and safety of evinacumab are limited.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>METHODS:<\/jats:title>\n                    <jats:p>We performed a retrospective study to assess the effectiveness and safety of evinacumab in patients with HoFH in clinical practice at 6 US academic medical centers. The primary end point was the percent change in LDL-C from baseline to first follow-up and to the most recent follow-up after evinacumab initiation. Secondary end points were percent change in non\u2013high-density lipoprotein cholesterol (non\u2013HDL-C), triglycerides, total cholesterol, HDL-C, and achievement of an LDL-C &lt;70 mg\/dL. Adverse events were recorded.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>RESULTS:<\/jats:title>\n                    <jats:p>\n                      Twenty-four patients (mean age, 40 [range, 5\u201384] years) with HoFH were followed for a median of 48 weeks. Fifty percent were female, 66.7% had atherosclerotic cardiovascular disease, 87.5% were on a statin, 83.3% were on ezetimibe, 66.7% were on PCSK9i (proprotein convertase subtilisin\/kexin type 9 inhibitors), 24% were on lomitapide, and 33.3% were undergoing lipoprotein apheresis. Significant reductions in LDL-C, non\u2013HDL-C, total cholesterol, triglycerides, and HDL-C were observed both at the first follow-up (4 weeks) and the most recent follow-up (48 weeks); mean\u00b1SEM percent change from baseline to the most recent follow-up was as follows: LDL-C, \u221253.2% (\u00b14.1); non\u2013HDL-C, \u221252.7% (\u00b13.9); triglycerides, \u221247.4% (\u00b15.1); total cholesterol, \u221248.9% (\u00b14.0); and HDL-C, \u221230.2% (\u00b14.1);\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      &lt;0.001 for all. Significantly more patients achieved LDL-C &lt;70 mg\/dL after evinacumab was added. Nine (37.5%) patients reported adverse events during or following evinacumab infusions. Treatment was discontinued by 1 patient because of back pain.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>CONCLUSIONS:<\/jats:title>\n                    <jats:p>Across 6 US academic medical centers, evinacumab was generally well tolerated by patients with HoFH and lowered LDL-C by \u224850%, consistent with results from clinical trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1161\/atvbaha.124.322364","type":"journal-article","created":{"date-parts":[[2025,5,29]],"date-time":"2025-05-29T05:00:18Z","timestamp":1748494818000},"page":"1310-1315","update-policy":"https:\/\/doi.org\/10.1161\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":14,"title":["Real-World Effectiveness and Safety of Evinacumab in Children and Adults With Homozygous Familial Hypercholesterolemia: A Multisite US Perspective\u2014Brief Report"],"prefix":"10.1161","volume":"45","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3800-7369","authenticated-orcid":true,"given":"Michael J.","family":"Wilkinson","sequence":"first","affiliation":[{"name":"University of California San Diego, La Jolla (M.J.W., P.B., R.S.N.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9996-6686","authenticated-orcid":true,"given":"Priyesha","family":"Bijlani","sequence":"additional","affiliation":[{"name":"University of California San Diego, La Jolla (M.J.W., P.B., R.S.N.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael H.","family":"Davidson","sequence":"additional","affiliation":[{"name":"University of Chicago, IL (M.H.D., M.H., P.P.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0208-5154","authenticated-orcid":true,"given":"P. Barton","family":"Duell","sequence":"additional","affiliation":[{"name":"Oregon Health &amp; Science University, Portland (P.B.D., B.A.W.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Horan","sequence":"additional","affiliation":[{"name":"University of Chicago, IL (M.H.D., M.H., P.P.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6189-5993","authenticated-orcid":true,"given":"Mary J.","family":"Malloy","sequence":"additional","affiliation":[{"name":"University of California San Francisco (M.J.M., E.O.S.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6274-8530","authenticated-orcid":true,"given":"Ron S.","family":"Newfield","sequence":"additional","affiliation":[{"name":"University of California San Diego, La Jolla (M.J.W., P.B., R.S.N.)."},{"name":"Rady Children\u2019s Hospital, San Diego, CA (R.S.N.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pallavi","family":"Pradeep","sequence":"additional","affiliation":[{"name":"University of Chicago, IL (M.H.D., M.H., P.P.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prediman K.","family":"Shah","sequence":"additional","affiliation":[{"name":"Cedars-Sinai Medical Center, Los Angeles, CA (P.K.S.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7085-5790","authenticated-orcid":true,"given":"Eveline O.","family":"Stock","sequence":"additional","affiliation":[{"name":"University of California San Francisco (M.J.M., E.O.S.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3635-7506","authenticated-orcid":true,"given":"Bruce A.","family":"Warden","sequence":"additional","affiliation":[{"name":"Oregon Health &amp; Science University, Portland (P.B.D., B.A.W.)."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew K.","family":"Ito","sequence":"additional","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc, Tarrytown, NY (M.K.I.)."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.123.065529"},{"key":"e_1_3_3_3_2","unstructured":"U.S. Food and Drug Administration. EVKEEZA (evinacumab-dgnb) prescribing Information. 2023. 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