Objective Lack of standardised outcomes hampers effective analysis and comparison of data when comparing treatments in fistulising perianal Crohn’s disease (pCD). Development of a standardised set of outcomes would resolve these issues. This study provides the definitive core outcome set (COS) for fistulising pCD.
Design Candidate outcomes were generated through a systematic review and patient interviews. Consensus was established via a three-round Delphi process using a 9-point Likert scale based on how important they felt it was in determining treatment success culminating in a final consensus meeting. Stakeholders were recruited nationally and grouped into three panels (surgeons and radiologists, gastroenterologists and IBD specialist nurses, and patients). Participants received feedback from their panel (in the second round) and all participants (in the third round) to allow refinement of their scores.
Results A total of 295 outcomes were identified from systematic reviews and interviews that were categorised into 92 domains. 187 stakeholders (response rate 78.5%) prioritised 49 outcomes through a three-round Delphi study.
The final consensus meeting of 41 experts and patients generated agreement on an eight domain COS. The COS comprised three patient-reported outcome domains (quality of life, incontinence and a combined score of patient priorities) and five clinician-reported outcome domains (perianal disease activity, development of new perianal abscess/sepsis, new/recurrent fistula, unplanned surgery and faecal diversion).
Conclusion A fistulising pCD COS has been produced by all key stakeholders. Application of the COS will reduce heterogeneity in outcome reporting, thereby facilitating more meaningful comparisons between treatments, data synthesis and ultimately benefit patient care.
Kapil Sahnan1,2, Phil J Tozer1,2, Samuel O Adegbola1,2, Matthew J Lee3, Nick Heywood4, Angus G K McNair5, Daniel Hind6, Nuha Yassin1, Alan J Lobo3, Steven R Brown3, Shaji Sebastian7, Robin K S Phillips1,2, Phillip F C Lung1,2, Omar D Faiz1,2, Kay Crook1, Sue Blackwell8, Azmina Verjee8, Ailsa L Hart1,2, Nicola S Fearnhead9 the ENiGMA collaborators
Author affiliations
1 Fistula Research Unit, St Mark’s Hospital and Academic Institute, London, UK
2 Department of Surgery and Cancer, Imperial College, St Mary’s Hospital, London, UK
3 Department of Colorectal Surgery, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
4 Department of Colorectal Surgery, University Hospitals of South Manchester, Manchester, UK
5 School of Social and Community Medicine, University of Bristol, Bristol, UK
6 Clinical Trials Research Unit, School of Health and Related Research, Sheffield, UK
7 Department of Gastroenterology, Hull and East Yorkshire NHS trust, Hull, UK
8 Patient Representative, London, UK
9 Department of Colorectal Surgery, Cambridge University Hospitals, Cambridge, UK
Disease Category: Gastroenterology
Disease Name: Perianal Crohn's disease, Crohn's disease
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Drug, Surgery
- Clinical experts
- Consumers (patients)
- COS for clinical trials or clinical research
- Consensus meeting
- Delphi process
- Systematic review
In phase 1, a long list of candidate outcomes that could be measured in fistulising pCD trials was identified, and outcomes were categorised into domains.
In phase 2, outcome domains were presented via a web-based Delphi system that was used to assess key stakeholders’ views on the importance of each domain.
In phase 3, a consensus meeting with all stakeholders was used to finalise and ratify the COS.