title: Patient preferences for endpoints in fecal incontinence treatment studies.
abstract: Randomized controlled trials of treatments for fecal incontinence (FI) are difficult to compare because case definitions and study endpoints vary. Our aims were to assess patient perspectives on the case definition for FI and how treatment success should be measured.
  
  
  
  In Phase 1, 28 FI patients participated in anonymous on-line focus groups, and in Phase 2, 186 people with FI, stratified by gender, race, and age completed an online survey.
  
  
  
  Focus group participants described frequency and urgency as the most important characteristics for defining FI. Most (80%) thought staining of underwear constitutes FI, but only 33% thought gas leakage was FI. When asked how the success of treatment should be defined, 77% said by a reduction in frequency or complete cure, but less than half thought a 50% reduction in frequency was enough. When asked how much reduction would be needed, responses averaged 80%. The Phase 2 survey confirmed that frequency, urgency, and intestinal discomfort are the most important characteristics for case definition, and that success should be defined by at least a 75% decrease in frequency. A 50% reduction was an acceptable endpoint for 58% overall but only 26% for those aged ≥65. "Adequate relief" was acceptable to 78%.
  
  
  
  Inclusion criteria for trials should specify a minimum frequency of FI. Most patients would require a ≥75% reduction in FI frequency to call a treatment successful but young adults and those with more severe FI would accept a ≥50% reduction as meaningful.
authors.affiliation: Center for Functional Gastrointestinal and Motility Disorders, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: S
authors.initials: S
authors.lastname: Heymen
authors.affiliation: Center for Functional Gastrointestinal and Motility Disorders, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: O
authors.initials: O
authors.lastname: Palsson
authors.affiliation: Center for Functional Gastrointestinal and Motility Disorders, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.|Department of Internal Medicine & Clinical Nutrition, Institute of Medicine, Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.
authors.forename: M
authors.initials: M
authors.lastname: Simren
authors.affiliation: Center for Functional Gastrointestinal and Motility Disorders, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: W E
authors.identifier: 0000-0001-6587-7602
authors.initials: WE
authors.lastname: Whitehead
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002986:Clinical Trials as Topic; D023381:Endpoint Determination; D005242:Fecal Incontinence / Q000628:therapy*; D017144:Focus Groups; D006801:Humans; D008297:Male; D008875:Middle Aged; D063868:Patient Outcome Assessment; D011795:Surveys and Questionnaires; D055815:Young Adult
keywords: accidental bowel leakage; adequate relief; focus group; inclusion criteria; outcome measure; patient reported outcome