Background & aims: Clinical trials evaluating biologics and small molecules in patients with ulcerative colitis are predominantly excluding ulcerative proctitis. The objective of the Definition and endpoints for ulcerative PROCtitis in clinical TRIALs initiative was to develop consensus statements for definitions, inclusion criteria, and endpoints for the evaluation of ulcerative proctitis in adults.
Methods: Thirty-five international experts held a consensus meeting to define ulcerative proctitis, and the endpoints to use in clinical trials. Based on a systematic review of the literature, statements were generated, discussed, and approved by the working group participants using a modified Delphi method. Consensus was defined as at least 75% agreement among voters.
Results: The group agreed that the diagnosis of ulcerative proctitis should be made by ileocolonoscopy and confirmed by histopathology, with the exclusion of infections, drug-induced causes, radiation, trauma, and Crohn's disease. Ulcerative proctitis was defined as macroscopic extent of lesions limited to 15 cm distance from the anal verge in adults. Primary and secondary endpoints were identified to capture response of ulcerative proctitis to therapy. A combined clinical and endoscopic primary endpoint for the evaluation of ulcerative proctitis disease activity was proposed. Secondary endpoints that should be evaluated include endoscopic remission, histologic remission, mucosal healing, histologic endoscopic mucosal improvement, disability, fecal incontinence, urgency, constipation, and health-related quality of life.
Conclusions: In response to the need for guidance on the design of clinical trials in patients with ulcerative proctitis, the Definition and end points for ulcerative PROCtitis in clinical TRIALs consensus provides recommendations on the definition and endpoints for ulcerative proctitis clinical trials.
Bénédicte Caron, Maria T Abreu, Corey A Siegel, Remo Panaccione, Bruce E Sands, Axel Dignass, Dan Turner, Iris Dotan, Ailsa L Hart, Vineet Ahuja, Matthieu Allez, Ashwin N Ananthakrishnan, Subrata Ghosh, Anne M Griffiths, Jonas Halfvarson, Arthur Kaser, Paulo G Kotze, Ioannis E Koutroubakis, Peter L Lakatos, Arie Levine, James D Lewis, Fernando Magro, Gerassimos J Mantzaris, Colm O'Morain, Zhihua Ran, Walter Reinisch, Gerhard Rogler, David B Sachar, Britta Siegmund, Mark S Silverberg, Ajit Sood, Antonino Spinelli, Flavio Steinwurz, Curt Tysk, Jesus K Yamamoto-Furusho, Stefan Schreiber, David T Rubin, William J Sandborn, Silvio Danese, Laurent Peyrin-Biroulet
Disease Category: Gastroenterology
Disease Name: Ulcerative Proctitis
Age Range: 18
Sex: Either
Nature of Intervention: Advice
- Clinical experts
- Recommendations made
- Consensus meeting
- Systematic review
Based on the needs identified in our previously published systematic review, we organized a meeting to achieve international expert consensus on the definition and endpoints to be used in future UP trials.