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

ABSTRACT: 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.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.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.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.

SUBMITTER: Spiegel B

PROVIDER: S-EPMC2793276 | biostudies-literature | 2009 Dec

REPOSITORIES: biostudies-literature

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

Spiegel Brennan B Camilleri Michael M Bolus Roger R Andresen Viola V Chey William D WD Fehnel Sheri S Mangel Allen A Talley Nicholas J NJ Whitehead William E WE

Gastroenterology 20090823 6

<h4>Background & aims</h4>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.<h4>Methods</h4>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 sever ...[more]

PMID: 19706292

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Project description:Background & aimsThe existence of postinfection irritable bowel syndrome (PI-IBS) has been substantiated by epidemiology studies conducted in diverse geographic and clinical settings. However, the available evidence has not been well summarized, and there is little guidance for diagnosis and treatment of PI-IBS. The ROME Foundation has produced a working team report to summarize the available evidence on the pathophysiology of PI-IBS and provide guidance for diagnosis and treatment, based on findings reported in the literature and clinical experience.MethodsThe working team conducted an evidence-based review of publication databases for articles describing the clinical features (diagnosis), pathophysiology (intestinal sensorimotor function, microbiota, immune dysregulation, barrier dysfunction, enteroendocrine pathways, and genetics), and animal models of PI-IBS. We used a Delphi-based consensus system to create guidelines for management of PI-IBS and a developed treatment algorithm based on published findings and experiences of team members.ResultsPI-IBS develops in about 10% of patients with infectious enteritis. Risk factors include female sex, younger age, psychological distress during or before acute gastroenteritis, and severity of the acute episode. The pathogenesis of PI-PBS appears to involve changes in the intestinal microbiome as well as epithelial, serotonergic, and immune system factors. However, these mechanisms are incompletely understood. There are no evidence-based, effective pharmacologic strategies for treatment of PI-IBS. We provide a consensus-based treatment algorithm, based on clinical presentation and potential disease mechanisms.ConclusionsBased on a systematic review of the literature and team experience, we summarize the clinical features, pathophysiology (from animal models and human studies), and progression of PI-IBS. Based on these findings, we present an algorithm for diagnosis and treatment of PI-IBS based on team consensus. We also propose areas for future investigation.

Project description:OBJECTIVES: Despite a documented clinical need, no patient reported outcome (PRO) symptom measure meeting current regulatory requirements for clinically relevant end points is available for the evaluation of treatment benefit in diarrhea-predominant IBS (IBS-D). METHODS: Patients (N=113) with IBS-D participated in five study phases: (1) eight concept elicitation focus groups (N=34), from which a 17-item IBS-D Daily Symptom Diary and four-item IBS-D Symptom Event Log (Diary and Event Log) were developed; (2) one-on-one cognitive interviews (N=11) to assess the instrument's comprehensiveness, understandability, appropriateness, and readability; (3) four data triangulation focus groups (N=32) to confirm the concepts elicited; (4) two hybrid (concept elicitation and cognitive interview) focus groups (N=16); and (5) two iterative sets of one-on-one cognitive interviews (N=20) to further clarify the symptoms of IBS-D and debrief a revised seven-item Diary and four-item Event Log. RESULTS: Of thirty-six concepts initially identified, 22 were excluded because they were not saturated, not clinically relevant, not critical symptoms of IBS-D, considered upper GI symptoms, or too broad or vaguely defined. The remaining concepts were diarrhea, immediate need (urgency), bloating/pressure, frequency of bowel movements, cramps, abdominal/stomach pain, gas, completely emptied bowels/incomplete evacuation, accidents, bubbling in intestines (bowel sounds), rectal burning, stool consistency, rectal spasm, and pain while wiping. The final instrument included a daily diary with separate items for abdominal and stomach pain and an event log with four items completed after each bowel movement as follows: (1) a record of the bowel movement/event and an assessment of (2) severity of immediacy of need/bowel urgency, (3) incomplete evacuation, and (4) stool consistency (evaluated using the newly developed Astellas Stool Form Scale). Based on rounds of interviews and clinical input, items considered secondary or nonspecific to IBS-D (rectal burning, bubbling in intestines, spasms, and pain while wiping) were excluded. CONCLUSIONS: The IBS-D Symptom Diary and Event Log represent a rigorously developed PRO instrument for the measurement of the IBS-D symptom experience from the perspective of the patient. Its content validity has been supported, and future work should evaluate the instrument's psychometric properties.

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

Publications

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

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