{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T23:48:43Z","timestamp":1789170523177,"version":"build-2803163510"},"reference-count":36,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"10","license":[{"start":{"date-parts":[[2020,8,1]],"date-time":"2020-08-01T00:00:00Z","timestamp":1596240000000},"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":["Hepatol. Commun."],"published-print":{"date-parts":[[2020,10]]},"abstract":"<jats:p>\n                    Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are associated with decreased health\u2010related quality of life and debilitating symptoms. These experiences can be defined as patient\u2010reported outcome (PRO) concepts and measured using PRO instruments. We identified all PRO concepts and instruments used in the PBC and PSC literature. This systematic review identified PBC and\/or PSC studies from January 1, 1990, to May 6, 2019, that measured at least one PRO concept. Study population, design, PRO concept, PRO instrument, and validation data for PRO instruments were investigated. We provided descriptive statistics of PRO concepts and instruments used, stratified by population type. Use of PRO concepts and instruments were assessed over time. The search yielded 318 articles (69% in PBC, 18% in PSC, 13% in both, and 24% in drug trials). Forty\u2010nine unique PRO concepts were identified. The five most common PRO concepts included pruritus (25%), fatigue (19%), broad health\u2010related quality of life (16%), gastrointestinal adverse events (6%), and physical adverse events (6%). Only 60% of PRO concepts were measured with a PRO instrument, most of which were nonvalidated visual analogue or numeric rating scales. Only three of 83 PRO instruments were developed with feedback from the target populations (one for PBC, one for PSC, and one for both), and only six documented any psychometric testing in the target populations. Use of PRO instruments increased over time from 30% in the 1990s to 67% by 2019.\n                    <jats:italic toggle=\"yes\">Conclusion:<\/jats:italic>\n                    The overwhelming majority of PRO instruments used in PBC\/PSC were nonspecific and lacked patient validation or empirical justification. Significant opportunities exist to use qualitative methods to better understand patient experiences, and translate this knowledge into meaningful, patient\u2010driven study outcomes.\n                  <\/jats:p>","DOI":"10.1002\/hep4.1567","type":"journal-article","created":{"date-parts":[[2020,8,1]],"date-time":"2020-08-01T15:04:15Z","timestamp":1596294255000},"page":"1502-1515","source":"Crossref","is-referenced-by-count":26,"title":["A Systematic Review of Patient\u2010Reported Outcomes in Primary Biliary Cholangitis and Primary Sclerosing Cholangitis"],"prefix":"10.1097","volume":"4","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5518-452X","authenticated-orcid":false,"given":"Hannah P.","family":"Kim","sequence":"first","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Department of Medicine,University of North Carolina,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8304-4479","authenticated-orcid":false,"given":"Sarah R.","family":"Lieber","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Department of Medicine,University of North Carolina,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael E.","family":"Rogers","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology,Department of Pediatrics,University of North Carolina,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7163-2062","authenticated-orcid":false,"given":"Andrew M.","family":"Moon","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Department of Medicine,University of North Carolina,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marci","family":"Loiselle","sequence":"additional","affiliation":[{"name":"Department of Psychiatry and Behavioral Sciences,Division of Behavioral Medicine,Duke University Medical Center,Durham,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Walker","sequence":"additional","affiliation":[{"name":"University of North Carolina,Health Sciences Library,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David N.","family":"Assis","sequence":"additional","affiliation":[{"name":"Digestive Diseases,Department of Medicine,Yale School of Medicine,New Haven,CT,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ricky","family":"Safer","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure,Greenwood Village,CO,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel","family":"Gomel","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure,Greenwood Village,CO,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1414-1846","authenticated-orcid":false,"given":"Donna M.","family":"Evon","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Department of Medicine,University of North Carolina,Chapel Hill,NC,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2020,8,1]]},"reference":[{"key":"hep41567-bib-0001-20240114","doi-asserted-by":"crossref","first-page":"1836","DOI":"10.1002\/hep.21953","article-title":"Quality of life and everyday activities in patients with primary biliary 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