J Crohns Colitis. 2024 Jan 27;18(1):91-105. doi: 10.1093/ecco-jcc/jjad130.

Stefan Schreiber ¹, Silvio Danese ², Axel Dignass ³, Eugeni Domènech ^4 5, Massimo C Fantini ⁶, Marc Ferrante ⁷, Jonas Halfvarson ⁸, Ailsa Hart ⁹, Fernando Magro ¹⁰, Charlie W Lees ¹¹, Salvo Leone ¹², Marieke J Pierik ¹³, Michele Peters ¹⁴, Polly Field ¹⁵, Helen Fishpool ¹⁵, Laurent Peyrin-Biroulet ^{16 17 18 19 20 21}

Author information

¹University Hospital Schleswig-Holstein, Department of Internal Medicine I, Kiel, Germany.

²Gastroenterology and Endoscopy, IRCCS Ospedale San Raffaele and Vita-Salute San Raffaele University, Milan, Italy.

³Department of Medicine I, Agaplesion Markus Hospital, Goethe University, Frankfurt, Germany.

⁴Department of Gastroenterology, Hospital Universitari Germans Trias i Pujol and CIBEREHD, Badalona, Spain.

⁵Departament de Medicina, Universitat Autònoma de Barcelona, Barcelona, Spain.

⁶Department of Medical Science and Public Health, University of Cagliari, Cagliari, Italy.

⁷Department of Gastroenterology and Hepatology, University Hospitals Leuven, KU Leuven, Leuven, Belgium.

⁸Department of Gastroenterology, Faculty of Medicine and Health, Örebro University, Örebro, Sweden.

⁹IBD Unit, St. Mark's Hospital, London, UK.

¹⁰CINTESIS@RISE, Faculty of Medicine of the University of Porto, Porto, Portugal.

¹¹Edinburgh Inflammatory Bowel Disease Unit, Western General Hospital, Edinburgh, UK.

¹²European Federation of Crohn's & Ulcerative Colitis Associations [EFCCA], Brussels, Belgium.

¹³Division Gastroenterology and Hepatology, Maastricht University Medical Centre, Maastricht, The Netherlands.

¹⁴Nuffield Department of Population Health, University of Oxford, Oxford, UK.

¹⁵Oxford PharmaGenesis, Oxford, UK.

¹⁶Department of Gastroenterology, Nancy University Hospital, Vandœuvre-lès-Nancy, France.

¹⁷Inserm, NGERE, University of Lorraine, Nancy, France.

¹⁸INFINY Institute, Nancy University Hospital, Vandœuvre-lès-Nancy, France.

¹⁹FHU-CURE, Nancy University Hospital, Vandœuvre-lès-Nancy, France.

²⁰Groupe Hospitalier privé Ambroise Paré - Hartmann, Paris IBD Center, Neuilly sur Seine, France.

²¹Division of Gastroenterology and Hepatology, McGill University Health Centre, Montreal, QC, Canada.

Abstract

Background and aims: Treatment of ulcerative colitis [UC] requires a patient-centric definition of comprehensive disease control that considers improvements in aspects not typically captured by classical landmark trial endpoints. In an international initiative, we reviewed aspects of UC that affect patients and/or indicate mucosal inflammation, to achieve consensus on which aspects to combine in a definition of comprehensive disease control, using a modified Delphi process.

Methods: The Delphi panel comprised 12 gastroenterologists and one patient advocate. Two gastroenterologists were elected as chairs and did not vote. To inform statements, we asked 18 patients and the panel members about their experiences of remission and reviewed published literature. Panel members voted on statements anonymously in three rounds, with a live discussion before Round 3. Consensus was met if ≥67% of the panel agreed. Statements without consensus in Rounds 1 and 2 were revised or discarded after Round 3.

Results: The panel agreed to measure individual patient benefit using a definition of comprehensive disease control that combines aspects currently measured in trials [rectal bleeding, stool frequency, disease-related quality of life, endoscopy, histological inflammatory activity, inflammatory biomarkers, and corticosteroid use] with additional patient-reported symptoms [bowel urgency, abdominal pain, extraintestinal manifestations, fatigue, and sleep disturbance]. The panel agreed on scoring systems and thresholds for many aspects.

Conclusions: Using a robust methodology, we defined comprehensive disease control in UC. Next, we will combine the measurement and scoring of these aspects into a multicomponent tool and will adopt comprehensive disease control as a treatment target in clinical practice and trials.