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Headline
Findings that probability of remission and likelihood of side-effects influenced treatment decisions with therapeutic benefits prioritised by professionals and time to improvement by patients can help treatment options discussions.
Abstract
Background:
Corticosteroids are a mainstay of the treatment of moderately severe relapses of ulcerative colitis, yet almost 50% of patients do not respond fully to these and risk prolonged steroid use and side effects. There is a lack of clarity about the definitions of steroid resistance, the optimum choice of treatment, and patient and health-care professional treatment preferences.
Objectives:
The overall aim of this research was to understand how steroid-resistant ulcerative colitis is managed in adult secondary care and how current practice compares with patient and health-care professional preferences.
Design:
A mixed-methods study, including an online survey, qualitative interviews and discrete choice experiments.
Setting:
NHS inflammatory bowel disease services in the UK.
Participants:
Adults with ulcerative colitis and health-care professionals treating inflammatory bowel disease.
Results:
We carried out a survey of health-care professionals (n = 168), qualitative interviews with health-care professionals (n = 20) and patients (n = 33), discrete choice experiments with health-care professionals (n = 116) and patients (n = 115), and a multistakeholder workshop (n = 9). The interviews with and survey of health-care professionals showed that most health-care professionals define steroid resistance as an incomplete response to 40 mg per day of prednisolone after 2 weeks. The survey also found that anti-tumour necrosis factor drugs (particularly infliximab) are the most frequently offered drugs across most steroid-resistant (and steroid-dependent) patient scenarios, but they are less frequently offered to thiopurine-naive patients. Patient interviews identified several factors influencing their treatment choices, including effectiveness of treatment, recommendations from health-care professionals, route of administration and side effects. Over time, depending on the severity and duration of symptoms and, crucially, as medical treatment options become exhausted, patients are willing to try alternative treatments and, eventually, to undergo surgery. The discrete choice experiments found that the probability of remission and of side effects strongly influences the treatment choices of both patients and health-care professionals. Patients are less likely to choose a treatment that takes longer to improve symptoms. Health-care professionals are willing to make difficult compromises by tolerating greater safety risks in exchange for therapeutic benefits. The treatments ranked most positively by patients were infliximab and tofacitinib (each preferred by 38% of patients), and the predicted probability of uptake by health-care professionals was greatest for infliximab (62%).
Limitations:
The survey and the discrete choice experiments with patients and health-care professionals are limited by their relatively small sample sizes. The qualitative studies are subject to selection bias. The timing of the different substudies, both before and during the COVID-19 pandemic, is a potential limitation.
Conclusions:
We have identified factors influencing treatment decisions for steroid-resistant ulcerative colitis and the characteristics to consider when choosing treatments to evaluate in future randomised controlled trials. The findings may be used to improve discussions between patients and health-care professionals when they review treatment options for steroid-resistant ulcerative colitis.
Future work:
This research highlights the need for consensus work to establish an agreed definition of steroid resistance in ulcerative colitis and a greater understanding of the optimal use of tofacitinib and surgery for this patient group. A randomised controlled trial comparing infliximab with tofacitinib is also recommended.
Funding:
This project was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 26, No. 41. See the NIHR Journals Library website for further project information.
Contents
- Plain English summary
- Scientific summary
- Chapter 1. Introduction
- Chapter 2. Methods
- Chapter 3. Results from the health-care professional survey
- Chapter 4. Results from qualitative interviews with health-care professionals
- Chapter 5. Results from qualitative interviews with patients
- Chapter 6. Results from the health-care professional discrete choice experiment
- Chapter 7. Results from the patient discrete choice experiment
- Chapter 8. Results from the multistakeholder workshop
- Chapter 9. Discussion and conclusion
- Acknowledgements
- References
- Appendix 1. Protocol version history
- Appendix 2. Supporting information for the health-care professional survey results
- Appendix 3. Supporting information for the health-care professional qualitative interviews
- Appendix 4. Supporting information for the patient qualitative interviews
- Appendix 5. Supporting information for the discrete choice experiment with health-care professionals
- Appendix 6. Supporting information for the discrete choice experiment with patients
- Appendix 7. Supporting information for the multistakeholder workshop
- List of abbreviations
- List of supplementary material
About the Series
Declared competing interests of authors: Matthew Lee was a member of the medical research panel for Crohn’s & Colitis UK (Hatfield, UK) and reports funding from Crohn’s & Colitis UK (Development and pilot testing of a decision aid to support patients with ulcerative colitis to choose between surgery and ongoing medical therapy). Daniel Hind is a member of the National Institute for Health and Care Research Health Technology Assessment Clinical Evaluation and Trials Committee (2019–present). Christopher Probert reports non-financial support from Vifor Pharma UK Ltd (Staines-upon-Thames, UK), and personal fees from Dr Falk Pharma (Bourne End, UK), Celltrion Healthcare UK Ltd (Slough, UK), Galapagos UK (Uxbridge, UK) and Janssen Pharmaceuticals (High Wycombe, UK). Shaji Sebastian reports grants from Takeda UK Ltd (London, UK), Amgen Inc. (Cambridge, UK), Pfizer Inc. (Pfizer Inc., New York, NY, USA), AbbVie (Maidenhead, UK) and Tillott Pharma UK Ltd (Wellingore, UK), and personal fees from AbbVie, Cellgene Healthcare UK Ltd, Takeda UK Ltd, Janssen Pharmaceuticals, Tillots Pharma UK Ltd, Dr Falk Pharma, Amgen Inc. and Pharmacosmos (Reading, UK). Alan Lobo reports personal fees from, and Advisory Board membership of, Takeda UK Ltd, personal fees from Janssen Pharmaceuticals, Advisory Board membership of Vifor Pharma UK Ltd, Celltrion Healthcare UK Ltd and Medtronic (Watford, UK), and grants from Crohn’s & Colitis UK (SP2018/2) and The Health Foundation (London, UK; FR-000002444).
Article history
The research reported in this issue of the journal was funded by the HTA programme as project number 17/72/02. The contractual start date was in November 2018. The draft report began editorial review in May 2021 and was accepted for publication in March 2022. The authors have been wholly responsible for all data collection, analysis and interpretation, and for writing up their work. The HTA editors and publisher have tried to ensure the accuracy of the authors’ report and would like to thank the reviewers for their constructive comments on the draft document. However, they do not accept liability for damages or losses arising from material published in this report.
Disclaimer
This report contains transcripts of interviews conducted in the course of the research and contains language that may offend some readers.
Last reviewed: May 2021; Accepted: March 2022.
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