TY - JOUR
T1 - IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis
T2 - The PROCTRIAL Consensus
AU - Caron, Bénédicte
AU - Abreu, Maria T.
AU - Siegel, Corey A.
AU - Panaccione, Remo
AU - Sands, Bruce E.
AU - Dignass, Axel
AU - Turner, Dan
AU - Dotan, Iris
AU - Hart, Ailsa L.
AU - Ahuja, Vineet
AU - Allez, Matthieu
AU - Ananthakrishnan, Ashwin N.
AU - Ghosh, Subrata
AU - Griffiths, Anne M.
AU - Halfvarson, Jonas
AU - Kaser, Arthur
AU - Kotze, Paulo G.
AU - Koutroubakis, Ioannis E.
AU - Lakatos, Peter L.
AU - Levine, Arie
AU - Lewis, James D.
AU - Magro, Fernando
AU - Mantzaris, Gerassimos J.
AU - O'Morain, Colm
AU - Ran, Zhihua
AU - Reinisch, Walter
AU - Rogler, Gerhard
AU - Sachar, David B.
AU - Siegmund, Britta
AU - Silverberg, Mark S.
AU - Sood, Ajit
AU - Spinelli, Antonino
AU - Steinwurz, Flavio
AU - Tysk, Curt
AU - Yamamoto-Furusho, Jesus K.
AU - Schreiber, Stefan
AU - Rubin, David T.
AU - Sandborn, William J.
AU - Danese, Silvio
AU - Peyrin-Biroulet, Laurent
N1 - Publisher Copyright:
© 2022 AGA Institute
PY - 2022/11
Y1 - 2022/11
N2 - 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.
AB - 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.
KW - Consensus
KW - Definition
KW - Endpoint
KW - Ulcerative Proctitis
UR - http://www.scopus.com/inward/record.url?scp=85132674590&partnerID=8YFLogxK
U2 - 10.1016/j.cgh.2022.02.032
DO - 10.1016/j.cgh.2022.02.032
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C2 - 35189386
AN - SCOPUS:85132674590
SN - 1542-3565
VL - 20
SP - 2619-2627.e1
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
IS - 11
ER -