Literature DB >> 19706292

Psychometric evaluation of patient-reported outcomes in irritable bowel syndrome randomized controlled trials: a Rome Foundation report.

Brennan Spiegel1, Michael Camilleri, Roger Bolus, Viola Andresen, William D Chey, Sheri Fehnel, Allen Mangel, Nicholas J Talley, William E Whitehead.   

Abstract

BACKGROUND & AIMS: There is debate about how best to measure patient-reported outcomes (PROs) in irritable bowel syndrome (IBS). We pooled data to measure the psychometric properties of IBS end points, including binary responses (eg, "adequate relief") and 50% improvement in symptom severity.
METHODS: We pooled data from 12 IBS drug trials involving 10,066 participants. We tested the properties of binary response and 50% improvement end points, including the impact of baseline severity on performance, and measured construct validity using clinical anchors.
RESULTS: There were 9044 evaluable subjects (age, 44 years; 85% female; 58% IBS constipation-prominent [IBS-C]; 31% IBS diarrhea-prominent [IBS-D]). Using the binary end point, the proportion responding in the mild, moderate, and severe groups was 42%, 40%, and 38%, respectively (P = .0008). There was no effect of baseline severity on binary response (odds ratio [OR], 0.99; 95% confidence interval [CI], 0.99-1.0; P = .07). The proportions reaching 50% improvement in pain were 45%, 41%, and 41%, respectively; there was a small, yet significant, impact of baseline severity (OR, 1.04; 95% CI, 1.03-1.05; P < .0001) that did not meet clinical relevance criteria. Both end points revealed strong construct validity and detected "minimally clinically important differences" in symptoms. Both provided better discriminant spread in IBS-D than IBS-C.
CONCLUSIONS: Both the traditional binary and 50% improvement end points are equivalent in their psychometric properties. Neither is impacted by baseline severity, and both demonstrate excellent construct validity. They are optimized for the IBS-D population but also appear valid in IBS-C.

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Year:  2009        PMID: 19706292      PMCID: PMC2793276          DOI: 10.1053/j.gastro.2009.08.047

Source DB:  PubMed          Journal:  Gastroenterology        ISSN: 0016-5085            Impact factor:   22.682


  16 in total

1.  Adequate relief as an endpoint in clinical trials in irritable bowel syndrome.

Authors:  A W Mangel; B A Hahn; A T Heath; A R Northcutt; S Kong; G E Dukes; D McSorley
Journal:  J Int Med Res       Date:  1998 Mar-Apr       Impact factor: 1.671

2.  Health-related quality of life among persons with irritable bowel syndrome: a systematic review.

Authors:  H B El-Serag; K Olden; D Bjorkman
Journal:  Aliment Pharmacol Ther       Date:  2002-06       Impact factor: 8.171

3.  The central role of gastrointestinal-specific anxiety in irritable bowel syndrome: further validation of the visceral sensitivity index.

Authors:  Jennifer S Labus; Emeran A Mayer; Lin Chang; Roger Bolus; Bruce D Naliboff
Journal:  Psychosom Med       Date:  2007-01       Impact factor: 4.312

4.  Reports of "satisfactory relief" by IBS patients receiving usual medical care are confounded by baseline symptom severity and do not accurately reflect symptom improvement.

Authors:  William E Whitehead; Olafur S Palsson; Rona L Levy; Andrew D Feld; Michael VonKorff; Marsha Turner
Journal:  Am J Gastroenterol       Date:  2006-05       Impact factor: 10.864

Review 5.  Design of treatment trials for functional gastrointestinal disorders.

Authors:  E Jan Irvine; William E Whitehead; William D Chey; Kei Matsueda; Michael Shaw; Nicholas J Talley; Sander J O Veldhuyzen van Zanten
Journal:  Gastroenterology       Date:  2006-04       Impact factor: 22.682

Review 6.  Irritable bowel syndrome: toward an understanding of severity.

Authors:  Anthony Lembo; Vanessa Z Ameen; Douglas A Drossman
Journal:  Clin Gastroenterol Hepatol       Date:  2005-08       Impact factor: 11.382

7.  Clinical trial: dextofisopam in the treatment of patients with diarrhoea-predominant or alternating irritable bowel syndrome.

Authors:  S M Leventer; K Raudibaugh; C L Frissora; N Kassem; J C Keogh; J Phillips; A W Mangel
Journal:  Aliment Pharmacol Ther       Date:  2007-10-31       Impact factor: 8.171

8.  Validation of irritable bowel syndrome Global Improvement Scale: an integrated symptom end point for assessing treatment efficacy.

Authors:  Susan Gordon; Vanessa Ameen; Barbara Bagby; Britt Shahan; Priti Jhingran; Eric Carter
Journal:  Dig Dis Sci       Date:  2003-07       Impact factor: 3.199

Review 9.  Primary endpoints for irritable bowel syndrome trials: a review of performance of endpoints.

Authors:  Michael Camilleri; Allen W Mangel; Sheri E Fehnel; Douglas A Drossman; Emeran A Mayer; Nicholas J Talley
Journal:  Clin Gastroenterol Hepatol       Date:  2007-04-11       Impact factor: 11.382

10.  Characterization of health related quality of life (HRQOL) for patients with functional bowel disorder (FBD) and its response to treatment.

Authors:  Douglas Drossman; Carolyn B Morris; Yuming Hu; Brenda B Toner; Nicholas Diamant; William E Whitehead; Christine B Dalton; Jane Leserman; Donald L Patrick; Shrikant I Bangdiwala
Journal:  Am J Gastroenterol       Date:  2007-05-17       Impact factor: 10.864

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  25 in total

Review 1.  [Visceral pain].

Authors:  S Elsenbruch; W Häuser; W Jänig
Journal:  Schmerz       Date:  2015-10       Impact factor: 1.107

2.  A cognitive-behavioral treatment for irritable bowel syndrome using interoceptive exposure to visceral sensations.

Authors:  Michelle G Craske; Kate B Wolitzky-Taylor; Jennifer Labus; Stephen Wu; Michael Frese; Emeran A Mayer; Bruce D Naliboff
Journal:  Behav Res Ther       Date:  2011-04-19

3.  Factors affecting response to proton pump inhibitor therapy in patients with gastroesophageal reflux disease: a multicenter prospective observational study.

Authors:  Nobuyuki Matsuhashi; Mineo Kudo; Norimasa Yoshida; Kazunari Murakami; Mototsugu Kato; Tsuyoshi Sanuki; Atsushi Oshio; Takashi Joh; Kazuhide Higuchi; Ken Haruma; Koji Nakada
Journal:  J Gastroenterol       Date:  2015-04-08       Impact factor: 7.527

4.  Lubiprostone does not influence visceral pain thresholds in patients with irritable bowel syndrome.

Authors:  W E Whitehead; O S Palsson; L Gangarosa; M Turner; J Tucker
Journal:  Neurogastroenterol Motil       Date:  2011-10       Impact factor: 3.598

5.  Linaclotide: promising IBS-C efficacy in an era of provisional study endpoints.

Authors:  Gregory S Sayuk
Journal:  Am J Gastroenterol       Date:  2012-11       Impact factor: 10.864

6.  Confirmatory factor analysis of the Patient Assessment of Constipation-Symptoms (PAC-SYM) among patients with chronic constipation.

Authors:  Luca Neri; Paul Maurice Conway; Guido Basilisco
Journal:  Qual Life Res       Date:  2014-12-19       Impact factor: 4.147

7.  The interpretation of Rome III criteria and method of assessment affect the irritable bowel syndrome classification of children.

Authors:  D I Czyzewski; M M Lane; E M Weidler; A E Williams; P R Swank; R J Shulman
Journal:  Aliment Pharmacol Ther       Date:  2010-12-08       Impact factor: 8.171

8.  Cost Effectiveness of Biomarker Tests for Irritable Bowel Syndrome With Diarrhea: A Framework for Payers.

Authors:  Christopher V Almario; Benjamin D Noah; Alma Jusufagic; Daniel Lew; Brennan M R Spiegel
Journal:  Clin Gastroenterol Hepatol       Date:  2018-03-27       Impact factor: 11.382

9.  Comparison of adequate relief with symptom, global, and responder endpoints in linaclotide phase 3 trials in IBS-C.

Authors:  Michael Camilleri; Anthony J Lembo; Bernard J Lavins; James E MacDougall; Robyn T Carson; Valerie Sl Williams; Lauren M Nelson; Steven J Shiff; Mark G Currie; Caroline B Kurtz; Jeffrey M Johnston
Journal:  United European Gastroenterol J       Date:  2015-02       Impact factor: 4.623

10.  Treatment of Dientamoeba fragilis in patients with irritable bowel syndrome.

Authors:  Anne Line Engsbro; C Rune Stensvold; Henrik V Nielsen; Peter Bytzer
Journal:  Am J Trop Med Hyg       Date:  2012-10-22       Impact factor: 2.345

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