Title: PoPSTER: Patient preferences and current Practice for adults with STERoid resistant ulcerative colitis
Funder: Department of Health and Social Care

Abstract: Background Ulcerative Colitis (UC) is a lifelong Inflammatory Bowel Disease (IBD) where people develop ulcers in the lining of the colon (also known as the large bowel). UC has many painful symptoms and has a big impact on quality of life. People with this disease may have long periods of remission (good health) but many will have relapses where their symptoms get worse. Steroids are one of the potential treatments for UC, but this is only effective for about half of the people with this condition, the other half can be described as ‘steroid resistant’. A number of other treatments are recommended for use in this country, but the supporting research evidence is limited. For example, there are no direct ‘head-to-head’ comparisons and it is often not clear whether the people who took part in these studies were resistant to, or dependent on steroids. To confuse matters, there is no agreed definition of steroid resistance in this patient group. Aims To design better research in the future about the best treatment for people with UC that does not respond to steroids, we need to find out: - What different treatments are offered in UK hospitals? - How do specialist IBD healthcare professionals define steroid resistance? - What are the factors influencing the offer of treatment options? - What do patients and healthcare professionals think of the different treatment options? Methods We plan to answer these questions by: 1. Conducting a questionnaire study of IBD healthcare professionals from across the UK. This will describe their current clinical practice in treating people with UC, definitions of steroid resistance and the factors that influence the treatments that are offered. 2. Interviewing healthcare professionals to find out about their clinical practice. 3. Interviewing patients about their views on different treatment options and how they make decisions about those. 4. Carrying out a questionnaire study of patients and healthcare professionals to work out their preferences for treatment options. 5. Bringing together healthcare professionals and patients to review the findings from our study, to help develop ideas for future research and treatments. The outputs of our study can be used to design a large scale randomised controlled trial for steroid resistant UC. This future trial will identify the most cost-effective way of improving quality of life and reducing symptoms, the need to be admitted to hospital or need for surgery. Public and patient involvement We have worked on the plans for this research with a group of patient representatives. Their feedback led to a number of improvements to the study. During the study, we will have three patient co-applicants who will be members of the study management group steering the delivery of the research. We will also establish a wider patient advisory group. Through these mechanisms, patients will help us by reviewing the content and accessibility of patient information, help us to understand and interpret the study results, and make recommendations. Dissemination We will publish in academic journals and do presentations at conferences, local and national IBD meetings. Summaries of findings will be shared with everyone who takes part in the research, and relevant policy and commissioning groups. We will also promote the study via social media channels.

Lay abstract: Background UC runs a relapsing/remitting course, with debilitating symptoms, impaired quality of life and severe attacks requiring hospitalisation. One of the treatment options for UC is corticosteroids, however, just under 50% of patients do not respond fully and relapse on dose reduction, leading to prolonged use and debilitating side-effects. A number of other treatments are recommended for use in the UK, but the research evidence is limited. To confuse matters, there is no agreed definition of steroid resistance in this patient group. There is a pressing need for a trial with a clearly defined population of steroid resistant patients to evaluate clinical and cost-effectiveness. In order to develop such a trial, detailed understanding of how best to describe steroid resistance, and of patients and clinicians’ views of treatment options and treatment objectives is required. This will allow appropriate identification of equipoise and acceptable invention and comparator arms in a trial. Aims and objectives 1. Describe current practice in the management of adults with steroid resistant UC and how medical resistance is defined. 2. Understand how treatment pathways and definitions of steroid resistance are operationalized in practice. 3. Understand patient experiences of different treatment options and approaches to decision making. 4. Estimate the relative utility of different treatment options; elicit patient and clinician preferences for these and their willingness to trade between them. 5. Make recommendations about future research and treatment options. Methods This mixed methods study is composed of five concurrent work packages, which correspond with the five objectives: WP1 – Cross-sectional online survey of 700 UK IBD healthcare professionals WP2 – Qualitative interviews with 25 UK IBD healthcare professionals WP3 – Qualitative interviews with 35 patients with ulcerative colitis from three NHS IBD centres to explore the complexity of patient decision making in steroid resistant UC WP4 – Discrete choice experiment with 180 patients from three NHS IBD centres and 200 UK IBD healthcare professionals WP5 – Multi-stakeholder workshop to generate recommendations about future research and optimum treatment for this patient group Timetable We are proposing a 24-month project with current work packages, planned to commence November 2018: (WP1): Survey design and piloting (M1-4); survey recruitment and data collection (M5-6); survey data analysis (M7); (WP2-3): Design and set up of qualitative studies including NHS ethics and HRA approvals (M1-7); recruitment (M7-9) interview data collection (M8-10) and analysis (M11-17); (WP4): DCE design and piloting (M13-14); recruitment and data collection (M15-17); analysis (M18-19); (WP5): Triangulation of findings from WP1-4 (M18-21); multi-stakeholder workshop (M22); reporting and wider dissemination activity (M23-24). Anticipated impact and dissemination Specific outputs will include conventional academic outputs (abstracts and conference presentations, journal publications), and presentations at local and national meetings for IBD. All study participants will receive a copy of the research findings, and the study will be promoted via social media channels throughout. We will work collaboratively with people with UC throughout the study and develop dissemination materials that are accessible and meaningful to both patients and healthcare professionals.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: ORAL AND GASTROINTESTINAL