{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T16:57:01Z","timestamp":1787590621078,"version":"build-2736575974"},"reference-count":64,"publisher":"The Endocrine Society","issue":"11","license":[{"start":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T00:00:00Z","timestamp":1741824000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["(UL1TR001105)"],"award-info":[{"award-number":["(UL1TR001105)"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018262","name":"Intercept Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018262","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,10,16]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context<\/jats:title>\n                  <jats:p>Patients with familial partial lipodystrophy (FPLD) have increased risk of hepatic steatosis and its complications, for which there is no approved therapy.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>This work aimed to investigate the efficacy and safety of obeticholic acid (OCA), a farnesoid X receptor agonist, for reducing hepatic steatosis in patients with FPLD.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>A randomized, double-blind, placebo-controlled, crossover trial was conducted at an academic referral center. Ten women (age 19-60 years) with the Dunnigan variety of FPLD (FPLD2), harboring pathogenic heterozygous variants in the lamin A\/C gene and hepatic steatosis (liver fat &amp;gt;5.6% by proton-density fat fraction mapping by magnetic resonance imaging), were included. Intervention included OCA 25\u2005mg daily vs matched placebo for 4 months each with a 4-month washout period in between. The primary end point variable was liver fat. Secondary end point variables were serum triglycerides (TGs) and transaminase levels.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>All patients completed the trial. OCA therapy caused significant (39.6%) reduction in liver fat as compared to placebo (median liver fat [minimum-maximum]; 6.4% [2.4%-18.0%] vs 10.6% [3.4%-29.3%], respectively; P value for treatment \u00d7 month interaction = .03). There were no significant differences in serum TGs or transaminase levels during OCA and placebo therapy. Overall, OCA was well tolerated except for itching in 4 patients compared to 2 on placebo. OCA, as compared to placebo, caused 24% increase in serum low-density lipoprotein cholesterol (mean 129\u2005mg\/dL vs 104\u2005mg\/dL, respectively; P = .0016).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>OCA is safe and effective in lowering hepatic TG levels in patients with FPLD2.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/clinem\/dgaf173","type":"journal-article","created":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T15:08:03Z","timestamp":1741878483000},"page":"e3617-e3625","source":"Crossref","is-referenced-by-count":4,"title":["Efficacy and Safety of Obeticholic Acid for Treating Hepatic Steatosis in Patients With Familial Partial Lipodystrophy"],"prefix":"10.1210","volume":"110","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7209-6986","authenticated-orcid":false,"given":"Abhimanyu","family":"Garg","sequence":"first","affiliation":[{"name":"Section of Nutrition and Metabolic Diseases, Division of Endocrinology, Department of Internal Medicine, Center for Human Nutrition, UT Southwestern Medical Center , Dallas, TX 75390-8537 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4014-2831","authenticated-orcid":false,"given":"Chandna","family":"Vasandani","sequence":"additional","affiliation":[{"name":"Section of Nutrition and Metabolic Diseases, Division of Endocrinology, Department of Internal Medicine, Center for Human Nutrition, UT Southwestern Medical Center , Dallas, TX 75390-8537 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1833-6077","authenticated-orcid":false,"given":"Xilong","family":"Li","sequence":"additional","affiliation":[{"name":"Peter O\u2019Donnell Jr. School of Public Health, UT Southwestern Medical Center , Dallas, TX 75390-9066 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Quittner","sequence":"additional","affiliation":[{"name":"Section of Nutrition and Metabolic Diseases, Division of Endocrinology, Department of Internal Medicine, Center for Human Nutrition, UT Southwestern Medical Center , Dallas, TX 75390-8537 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5092-6931","authenticated-orcid":false,"given":"Takeshi","family":"Yokoo","sequence":"additional","affiliation":[{"name":"Department of Radiology, UT Southwestern Medical Center , Dallas, TX 75390-9085 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2025,3,13]]},"reference":[{"issue":"12","key":"2025101519582928500_dgaf173-B1","doi-asserted-by":"crossref","first-page":"1220","DOI":"10.1056\/NEJMra025261","article-title":"Acquired and inherited 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