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These trials will require a meaningful, validated and fit for purpose patient\u2010reported outcome measure (<jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>) to quantify the symptomatic improvement of patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To evaluate existing <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s for suitability in a Food and Drug Administration (<jats:styled-content style=\"fixed-case\">FDA<\/jats:styled-content>) approval trial for a coeliac disease therapeutic.<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>We performed a systematic search in five online databases (MedLine, EmBase, Web of Science, <jats:styled-content style=\"fixed-case\">CENTRAL<\/jats:styled-content>,<jats:styled-content style=\"fixed-case\"> CINAHL<\/jats:styled-content>) for studies that enrolled patients with coeliac disease and used <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s. Studies included in this review had to measure some <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content> concept, be patient administered and based upon a previously validated instrument with published measurement properties.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Our literature search identified 2706 unique records of which 199 ultimately qualified for abstraction. The majority of <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s used in studies of coeliac disease was generic and did not measure numerous symptoms or concerns of interest to patients. Four <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s were found to contain appropriate content for use in an <jats:styled-content style=\"fixed-case\">FDA<\/jats:styled-content> trial: the coeliac disease\u2010specific modification of the Gastrointestinal Symptoms Rating Scale (CeD\u2010<jats:styled-content style=\"fixed-case\">GSRS<\/jats:styled-content>), Psychological General Well\u2010Being Index (<jats:styled-content style=\"fixed-case\">PGWB<\/jats:styled-content>), the Celiac Disease Symptom Diary (<jats:styled-content style=\"fixed-case\">CDSD<\/jats:styled-content>) and the Celiac Disease Patient Reported Outcome (CeD\u2010<jats:styled-content style=\"fixed-case\">PRO<\/jats:styled-content>). The <jats:styled-content style=\"fixed-case\">GSRS<\/jats:styled-content> and <jats:styled-content style=\"fixed-case\">PGWB<\/jats:styled-content> are most often used together and are two of the most extensively used measures in coeliac disease. The <jats:styled-content style=\"fixed-case\">CDSD<\/jats:styled-content> and CeD\u2010<jats:styled-content style=\"fixed-case\">PRO<\/jats:styled-content> were developed exclusively for trials in coeliac disease but have much less published information on their measurement properties.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>While we did not find <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s that currently meet the stated expectations of the <jats:styled-content style=\"fixed-case\">FDA<\/jats:styled-content> for regulatory purposes, four <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>s (CeD\u2010<jats:styled-content style=\"fixed-case\">GSRS<\/jats:styled-content>,<jats:styled-content style=\"fixed-case\"> PGWB<\/jats:styled-content>,<jats:styled-content style=\"fixed-case\"> CDSD<\/jats:styled-content> and CeD\u2010<jats:styled-content style=\"fixed-case\">PRO<\/jats:styled-content>) appear to contain appropriate content and with modest additional validation work could meet scientific standards for valid and sensitive measures of disease and treatment outcome. Specifically, what is needed for these instruments is an understanding of how sensitive they are to real changes in\u2010patient condition, how stable they are over a period of time when health status should not have changed (test\u2013retest reliability) as well as how they correlate with other measures of patient functioning such as intestinal biopsy. All of these objectives could feasibly be accomplished over a short cohort study of patients with biopsy\u2010defined coeliac disease undergoing gluten challenge.<\/jats:p><\/jats:sec>","DOI":"10.1111\/apt.13703","type":"journal-article","created":{"date-parts":[[2016,6,29]],"date-time":"2016-06-29T05:38:02Z","timestamp":1467178682000},"page":"313-331","source":"Crossref","is-referenced-by-count":35,"title":["Systematic review: patient\u2010reported outcome measures in coeliac disease for regulatory submissions"],"prefix":"10.1111","volume":"44","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6577-1047","authenticated-orcid":false,"given":"W. J.","family":"Canestaro","sequence":"first","affiliation":[{"name":"Pharmaceutical Outcomes Research and Policy Program University of Washington School of Pharmacy Seattle WA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. 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