title: Converging goals of treatment of inflammatory bowel disease from clinical trials and practice.
abstract: It is important to have clear goals for treating inflammatory bowel disease in clinical practice and in research. Conventional end points for trials in ulcerative colitis and Crohn's disease have been based on composite indices, such as the Mayo Clinic Score and the Crohn's Disease Activity Index; these indices incorporate symptoms, signs, and findings from laboratory tests and sometimes endoscopic assessments. Although definitions of clinical response and remission have been based on these indices for regulatory purposes, they are difficult to apply to practice because they are complex and not intuitive to clinicians. This has caused a disconnect between clinical trials and practice. Recently, the use of composite indices in trials has been reevaluated in Food and Drug Administration-sponsored Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics workshops due to concerns about the validity of the indices. Alternative measures of outcome and definitions of response are being developed. Patient-reported outcomes are psychometric instruments created and defined by patients to quantify symptoms. A combination of end points, comprising patient-reported outcomes and objective evaluation of inflammation by endoscopy, offers a clinically meaningful and scientifically valid alternative to existing composite indices. Unlike composite indices, response definitions based on endoscopy and patient-reported outcomes can be readily applied in practice. This convergence of outcome assessment in clinical trials and practice could expedite implementation of "treat-to-target" algorithms, in which therapy is progressively intensified until a specific treatment goal is reached. This approach could improve patient care by reducing rates of disease-related complications, surgery, and hospitalization.
authors.affiliation: Division of Gastroenterology, University of California San Diego, La Jolla, California.
authors.forename: Barrett G
authors.initials: BG
authors.lastname: Levesque
authors.affiliation: Division of Gastroenterology, University of California San Diego, La Jolla, California. Electronic address: wsandborn@ucsd.edu.
authors.forename: William J
authors.initials: WJ
authors.lastname: Sandborn
authors.affiliation: Dr Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York.
authors.forename: Joannie
authors.initials: J
authors.lastname: Ruel
authors.affiliation: Robarts Clinical Trials, Robarts Research Institute, Department of Medicine, Western University, London, Ontario, Canada.
authors.forename: Brian G
authors.initials: BG
authors.lastname: Feagan
authors.affiliation: Dr Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York.
authors.forename: Bruce E
authors.initials: BE
authors.lastname: Sands
authors.affiliation: Dr Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York. Electronic address: jean-frederic.colombel@mssm.edu.
authors.forename: Jean-Frederic
authors.initials: JF
authors.lastname: Colombel
mesh_terms: D000465:Algorithms; D002986:Clinical Trials as Topic; D003093:Colitis, Ulcerative / Q000175:diagnosis* / Q000628:therapy*; D019091:Critical Pathways; D003424:Crohn Disease / Q000175:diagnosis* / Q000628:therapy*; D016099:Endoscopy, Gastrointestinal; D023381:Endpoint Determination; D019317:Evidence-Based Medicine; D006801:Humans; D010347:Patient Care Planning; D011237:Predictive Value of Tests; D012074:Remission Induction; D057566:Self Report; D012720:Severity of Illness Index; D016896:Treatment Outcome
keywords: CDAI; GREAT; IBD Therapy; Outcome Measure