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Validating endpoints for therapeutic trials in fecal incontinence
Author(s) -
Noelting J.,
Zinsmeister A. R.,
Bharucha A. E.
Publication year - 2016
Publication title -
neurogastroenterology and motility
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.489
H-Index - 105
eISSN - 1365-2982
pISSN - 1350-1925
DOI - 10.1111/nmo.12809
Subject(s) - medicine , minimal clinically important difference , fecal incontinence , physical therapy , placebo , randomization , quality of life (healthcare) , rating scale , depression (economics) , anxiety , clinical endpoint , defecation , randomized controlled trial , surgery , psychology , psychiatry , alternative medicine , developmental psychology , nursing , pathology , economics , macroeconomics
Background A 50% or greater reduction in the frequency of fecal incontinence ( FI ) recorded with daily bowel diaries is the primary endpoint in clinical trials of FI . Whether this difference is clinically important is unknown. The relationship between FI symptoms recorded with daily and weekly instruments is unknown. The contribution of psychological factors to quality of life ( QOL ) in FI is unclear. Methods Fecal incontinence severity was assessed with daily bowel diaries and periodic questionnaires (fecal incontinence severity score [ FISS ], FIQOL , 36‐Item Short Form Health Survey [ SF ‐36], and hospital anxiety and depression scales) for 4 weeks before and during double‐blind randomization to placebo or clonidine in 44 women with FI . The reduction in FI frequency was compared to the minimal clinically important difference ( MCID ) computed from the FISS . Endpoints of FI were compared between daily and weekly diaries. Key Results The FISS exceeded the MCID in 75% and 83% of patients in whom the FI frequency declined by 50–74% and ≥75% respectively. Parameters of FI measured with daily and weekly instruments were significantly correlated. The daily parameters explained 71% of the inter‐patient variation in the FISS . The SF ‐36 health scores, rather than the FISS rating, explained a majority of the inter‐subject variation in FIQOL . Conclusions & Inferences Most patients who report a ≥50% reduction in FI frequency experience a clinically important improvement. Weekly questionnaires accurately assess the severity of FI . Self‐reported physical and mental health explained a greater proportion of the variance in FIQOL than FI symptom severity.