IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus

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

doi:10.1016/j.cgh.2022.02.032

IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus

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

CHU de Nancy
University of Miami
Dartmouth Geisel School of Medicine and Dartmouth-Hitchcock Medical Center
University of Calgary
Dr. Henry D. Janowitz Division of Gastroenterology
Goethe University Frankfurt
Hebrew University of Jerusalem
Tel Aviv University
London North West University Healthcare NHS Trust
All India Institute of Medical Sciences, New Delhi
Université Paris Cité
Harvard University
University of Toronto
Örebro University
University of Cambridge
Pontifícia Universidade Católica do Paraná
Heraklion University Hospital
Semmelweis University
Edith Wolfson Medical Center Israel
University of Pennsylvania
University of Porto
Centro Hospitalar Universitário de São João
Center for Health Technology and Services Research (CINTESIS)
Athens General Hospital
Trinity College Dublin
Shanghai Jiao Tong University
Medical University of Vienna
University of Zurich
Icahn School of Medicine at Mount Sinai
Berlin Institute of Health
Dayanand Medical College & Hospital
Humanitas University
Hospital Israelita Albert Einstein
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
University Hospital Schleswig-Holstein
The University of Chicago
University of California at San Diego
Vita-Salute San Raffaele University

Research output: Contribution to journal › Article › peer-review

Abstract

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.

Original language	English
Pages (from-to)	2619-2627.e1
Journal	Clinical Gastroenterology and Hepatology
Volume	20
Issue number	11
DOIs	https://doi.org/10.1016/j.cgh.2022.02.032
Publication status	Published - Nov 2022

Keywords

Consensus
Definition
Endpoint
Ulcerative Proctitis

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10.1016/j.cgh.2022.02.032

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Caron, B., Abreu, M. T., Siegel, C. A., Panaccione, R., Sands, B. E., Dignass, A., Turner, D., Dotan, I., Hart, A. L., Ahuja, V., Allez, M., Ananthakrishnan, A. N., Ghosh, S., Griffiths, A. M., Halfvarson, J., Kaser, A., Kotze, P. G., Koutroubakis, I. E., Lakatos, P. L., ... Peyrin-Biroulet, L. (2022). IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus. Clinical Gastroenterology and Hepatology, 20(11), 2619-2627.e1. https://doi.org/10.1016/j.cgh.2022.02.032

@article{494c2c9ed2fa429dbe102374ba9c88e4,

title = "IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus",

abstract = "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.",

keywords = "Consensus, Definition, Endpoint, Ulcerative Proctitis",

author = "B{\'e}n{\'e}dicte Caron and Abreu, \{Maria T.\} and Siegel, \{Corey A.\} and Remo Panaccione and Sands, \{Bruce E.\} and Axel Dignass and Dan Turner and Iris Dotan and Hart, \{Ailsa L.\} and Vineet Ahuja and Matthieu Allez and Ananthakrishnan, \{Ashwin N.\} and Subrata Ghosh and Griffiths, \{Anne M.\} and Jonas Halfvarson and Arthur Kaser and Kotze, \{Paulo G.\} and Koutroubakis, \{Ioannis E.\} and Lakatos, \{Peter L.\} and Arie Levine and Lewis, \{James D.\} and Fernando Magro and Mantzaris, \{Gerassimos J.\} and Colm O'Morain and Zhihua Ran and Walter Reinisch and Gerhard Rogler and Sachar, \{David B.\} and Britta Siegmund and Silverberg, \{Mark S.\} and Ajit Sood and Antonino Spinelli and Flavio Steinwurz and Curt Tysk and Yamamoto-Furusho, \{Jesus K.\} and Stefan Schreiber and Rubin, \{David T.\} and Sandborn, \{William J.\} and Silvio Danese and Laurent Peyrin-Biroulet",

note = "Publisher Copyright: {\textcopyright} 2022 AGA Institute",

year = "2022",

month = nov,

doi = "10.1016/j.cgh.2022.02.032",

language = "English",

volume = "20",

pages = "2619--2627.e1",

journal = "Clinical Gastroenterology and Hepatology",

issn = "1542-3565",

publisher = "W.B. Saunders",

number = "11",

}

Caron, B, Abreu, MT, Siegel, CA, Panaccione, R, Sands, BE, Dignass, A, Turner, D, Dotan, I, Hart, AL, Ahuja, V, Allez, M, Ananthakrishnan, AN, Ghosh, S, Griffiths, AM, Halfvarson, J, Kaser, A, Kotze, PG, Koutroubakis, IE, Lakatos, PL, Levine, A, Lewis, JD, Magro, F, Mantzaris, GJ, O'Morain, C, Ran, Z, Reinisch, W, Rogler, G, Sachar, DB, Siegmund, B, Silverberg, MS, Sood, A, Spinelli, A, Steinwurz, F, Tysk, C, Yamamoto-Furusho, JK, Schreiber, S, Rubin, DT, Sandborn, WJ, Danese, S & Peyrin-Biroulet, L 2022, 'IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus', Clinical Gastroenterology and Hepatology, vol. 20, no. 11, pp. 2619-2627.e1. https://doi.org/10.1016/j.cgh.2022.02.032

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

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 - https://www.scopus.com/pages/publications/85132674590

U2 - 10.1016/j.cgh.2022.02.032

DO - 10.1016/j.cgh.2022.02.032

M3 - Article

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 -

IOIBD Recommendations for Clinical Trials in Ulcerative Proctitis: The PROCTRIAL Consensus

Abstract

Keywords

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