{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T22:05:18Z","timestamp":1780351518493,"version":"3.54.1"},"reference-count":16,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"5","license":[{"start":{"date-parts":[[2018,3,23]],"date-time":"2018-03-23T00:00:00Z","timestamp":1521763200000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Hepatology Communications"],"published-print":{"date-parts":[[2018,5]]},"abstract":"<jats:p>Primary biliary cholangitis (PBC) is a rare chronic cholestatic liver disease that may progress to biliary cirrhosis if left untreated. The first\u2010line therapy for PBC is ursodeoxycholic acid (UDCA). Unfortunately, 1 of 3 patients does not respond to UDCA. These patients are at risk for developing clinical events, including cirrhosis, complications of portal hypertension, hepatocellular carcinoma, liver transplant, or death. Recently, the U.S. Food and Drug Administration approved obeticholic acid to be used in certain patients with PBC. Off\u2010label therapies are also used, and several other therapies are currently under evaluation. Real\u2010world effectiveness of newly approved and off\u2010label therapies remains unknown. TARGET\u2010PBC is a 5\u2010year, longitudinal, observational study of patients with PBC that will evaluate the effectiveness of clinical practice interventions and provide practical information unobtainable in registration trials. Enrollment will take place at both academic and community sites. In addition to consenting to medical records review, participants will be asked to provide an annual blood sample and complete patient reported outcome surveys at predetermined intervals. Any available liver biopsies will be digitally preserved. <jats:italic toggle=\"yes\">Conclusion:<\/jats:italic> Key study outcomes will be the evaluation of the safety and effectiveness of PBC interventions and the assessment of disease progression under real\u2010world conditions. (<jats:italic toggle=\"yes\">Hepatology Communications<\/jats:italic> 2018;2:484\u2010491)<\/jats:p>","DOI":"10.1002\/hep4.1173","type":"journal-article","created":{"date-parts":[[2018,3,24]],"date-time":"2018-03-24T03:40:40Z","timestamp":1521862840000},"page":"484-491","source":"Crossref","is-referenced-by-count":16,"title":["A real\u2010world observational cohort of patients with primary biliary cholangitis: TARGET\u2010primary biliary cholangitis study design and rationale"],"prefix":"10.1097","volume":"2","author":[{"given":"Cynthia","family":"Levy","sequence":"first","affiliation":[{"name":"Department of Medicine,University of Miami,Miami,FL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3906-6811","authenticated-orcid":false,"given":"Christopher L.","family":"Bowlus","sequence":"additional","affiliation":[{"name":"University of California Davis,Davis,CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Carey","sequence":"additional","affiliation":[{"name":"Department of Transplantation,Mayo Clinic,Phoenix,AZ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie M.","family":"Crawford","sequence":"additional","affiliation":[{"name":"TARGET PharmaSolutions, Inc.,Chapel Hill,NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen","family":"Deane","sequence":"additional","affiliation":[{"name":"TARGET PharmaSolutions, Inc.,Chapel Hill,NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marlyn J.","family":"Mayo","sequence":"additional","affiliation":[{"name":"University of Texas Southwestern Medical Center,Dallas,TX"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"W. Ray","family":"Kim","sequence":"additional","affiliation":[{"name":"Stanford University,Stanford,CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael W.","family":"Fried","sequence":"additional","affiliation":[{"name":"University of North Carolina,Chapel Hill,NC"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2018,3,23]]},"reference":[{"key":"hep41173-bib-0001-20240220","doi-asserted-by":"crossref","first-page":"1565","DOI":"10.1016\/S0140-6736(15)00154-3","article-title":"Primary biliary cirrhosis","volume":"386","year":"2015","journal-title":"Lancet"},{"key":"hep41173-bib-0002-20240220","doi-asserted-by":"crossref","first-page":"926","DOI":"10.4254\/wjh.v7.i7.926","article-title":"Primary biliary cirrhosis: pathophysiology, clinical presentation and therapy","volume":"7","year":"2015","journal-title":"World J Hepatol"},{"key":"hep41173-bib-0003-20240220","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1016\/j.cld.2012.12.003","article-title":"Primary biliary cirrhosis: therapeutic advances","volume":"17","year":"2013","journal-title":"Clin Liver Dis"},{"key":"hep41173-bib-0004-20240220","doi-asserted-by":"crossref","first-page":"715","DOI":"10.1053\/j.gastro.2005.12.029","article-title":"Excellent long\u2010term survival in patients with primary biliary cirrhosis and biochemical response to ursodeoxycholic acid","volume":"130","year":"2006","journal-title":"Gastroenterology"},{"key":"hep41173-bib-0005-20240220","doi-asserted-by":"crossref","first-page":"1342","DOI":"10.1056\/NEJM199405123301903","article-title":"Ursodiol for the long\u2010term treatment of primary biliary cirrhosis. 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