{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T07:17:22Z","timestamp":1790752642857,"version":"4.1.0"},"reference-count":22,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T00:00:00Z","timestamp":1696982400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Aliment Pharmacol Ther"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Summary<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Low phospholipid\u2010associated cholelithiasis (LPAC) syndrome is a rare genetic cause of hepatolithiasis. A pathogenic variant of the <jats:italic>ABCB4<\/jats:italic> gene is reported in half of all patients. Ursodeoxycholic acid (UDCA) is the only drug approved. However, in some patients, UDCA fails to prevent recurrence of symptoms and complications. Experimental evidence suggests that agonists of the farnesoid\u2010X receptor (FXR), the main transcription factor regulating <jats:italic>ABCB4<\/jats:italic>, may be beneficial in this context.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To study the efficacy of obeticholic acid (OCA) in patients with LPAC syndrome with an inadequate response or intolerance to UDCA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a retrospective study of patients with LPAC syndrome treated with OCA, a selective FXR agonist.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We reviewed the records of five OCA\u2010treated patients (4 women; median age 29; <jats:italic>ABCB4<\/jats:italic> variant in 4; no hepatic fibrosis). All patients received OCA at an initial dose of 5\u2009mg daily and then 10\u2009mg daily for a median period of 36\u2009months in combination with UDCA (4 patients) or as a monotherapy (one patient). There were no adverse effects reported. Four patients had improvement in their symptoms \u2010 three completely and one partially. One patient had no clinical benefit. Abnormalities of blood liver tests persisted in one patient despite resolution of symptoms. Radiological signs of hepatolithiasis persisted in three of the four patients who responded clinically to OCA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>These preliminary observations suggest that OCA may have the potential to effectively treat LPAC syndrome in patients with inadequate response or intolerance to UDCA. Larger studies are needed to confirm these data.<\/jats:p><\/jats:sec>","DOI":"10.1111\/apt.17761","type":"journal-article","created":{"date-parts":[[2023,10,12]],"date-time":"2023-10-12T05:08:01Z","timestamp":1697087281000},"page":"113-117","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Obeticholic acid as a second\u2010line treatment for low phospholipid\u2010associated cholelithiasis syndrome"],"prefix":"10.1111","volume":"59","author":[{"given":"Pierre\u2010Antoine","family":"Soret","sequence":"first","affiliation":[{"name":"Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare\u2010Liver) Saint\u2010Antoine Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"},{"name":"Saint\u2010Antoine Research Center (CRSA) INSERM, Sorbonne University  Paris France"},{"name":"French National Cohort of Patients with LPAC syndrome (RaDiCo\u2010COLPAC)  Paris France"},{"name":"RaDiCo, Inserm U933, Armand Trousseau Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6947-2709","authenticated-orcid":false,"given":"Sara","family":"Lemoinne","sequence":"additional","affiliation":[{"name":"Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare\u2010Liver) Saint\u2010Antoine Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"},{"name":"Saint\u2010Antoine Research Center (CRSA) INSERM, Sorbonne University  Paris France"},{"name":"French National Cohort of Patients with LPAC syndrome (RaDiCo\u2010COLPAC)  Paris France"},{"name":"RaDiCo, Inserm U933, Armand Trousseau Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maxime","family":"Mallet","sequence":"additional","affiliation":[{"name":"Hepatology and Gastroenterology Department Piti\u00e9\u2010Salp\u00eatri\u00e8re Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karima Ben","family":"Belkacem","sequence":"additional","affiliation":[{"name":"Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare\u2010Liver) Saint\u2010Antoine Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"},{"name":"Saint\u2010Antoine Research Center (CRSA) INSERM, Sorbonne University  Paris France"},{"name":"French National Cohort of Patients with LPAC syndrome (RaDiCo\u2010COLPAC)  Paris France"},{"name":"RaDiCo, Inserm U933, Armand Trousseau Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Chazouill\u00e8res","sequence":"additional","affiliation":[{"name":"Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare\u2010Liver) Saint\u2010Antoine Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"},{"name":"Saint\u2010Antoine Research Center (CRSA) INSERM, Sorbonne University  Paris France"},{"name":"French National Cohort of Patients with LPAC syndrome (RaDiCo\u2010COLPAC)  Paris France"},{"name":"RaDiCo, Inserm U933, Armand Trousseau Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7920-9784","authenticated-orcid":false,"given":"Christophe","family":"Corpechot","sequence":"additional","affiliation":[{"name":"Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare\u2010Liver) Saint\u2010Antoine Hospital, Assistance Publique \u2013 H\u00f4pitaux de Paris  Paris France"},{"name":"Saint\u2010Antoine Research Center (CRSA) INSERM, Sorbonne University  Paris France"},{"name":"French National Cohort of Patients with LPAC syndrome (RaDiCo\u2010COLPAC)  Paris France"},{"name":"RaDiCo, Inserm U933, Armand Trousseau Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,10,11]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1053\/gast.2001.23947"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhepr.2020.100201"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0016-5085(03)00898-9"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-2-29"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gastro.2005.05.009"},{"key":"e_1_2_6_7_1","first-page":"S1113","article-title":"Low phospholipid\u2010associated cholelithiasis syndrome and interventional endoscopy","volume":"76","author":"Salin G","year":"2022","journal-title":"Hepatology"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2022.10.007"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M308321200"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm1138"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1509840"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2007.07.027"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2016.01.016"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)33041-7"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2019.06.011"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.gastro.2008.03.011"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.3390\/genes13061047"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1042\/bj3140781"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.5414\/CPP48022"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00228-003-0704-1"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF01072597"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005643014395"},{"key":"e_1_2_6_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/apt.16336"}],"container-title":["Alimentary Pharmacology &amp; Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/apt.17761","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,12,29]],"date-time":"2023-12-29T05:55:41Z","timestamp":1703829341000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/apt.17761"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,11]]},"references-count":22,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,1]]}},"alternative-id":["10.1111\/apt.17761"],"URL":"https:\/\/doi.org\/10.1111\/apt.17761","archive":["Portico"],"relation":{},"ISSN":["0269-2813","1365-2036"],"issn-type":[{"value":"0269-2813","type":"print"},{"value":"1365-2036","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,10,11]]},"assertion":[{"value":"2023-06-15","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-10-01","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-10-11","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}