Clinical Burden of Refractory Proctitis

Study code
NBR130

Lead researcher
Dr Gordon W Moran

Study type
Online

Institution or company
Nottingham University Hospitals NHS Trust

Researcher type
Academic

Speciality area
Gastroenterology

Recruitment Site
National

Summary

Refractory proctitis is a prevalent problem and a large unmet need in Inflammatory Bowel Disease (IBD). In patients whose disease does not progress and remains confined to the rectum, ulcerative proctitis is still often responsible for distressing symptoms such as urgency, incontinence, and constipation and rectal bleeding, leading to a reduced quality of life.

Refractory disease is defined as active proctitis which fails rectal and oral therapy. Treatment of refractory proctitis remains challenging because patients with the condition are systematically excluded from randomized controlled trials with drugs with new modes of action. Without this data, recommendations for the management of ulcerative proctitis are therefore often extrapolated from studies involved more in ulcerative colitis or from small real-world evidence studies.

We aim to use the patient and clinical experience of managing ulcerative proctitis to identify the clinical and research needs of this patient group. We will use this information to inform future research and clinical guidance on this condition.

Organisation: This research is organised by Dr Gordon Moran from Nottingham University Hospitals NHS Trust.

Participation: 6 volunteers from the IBD BioResource took part in an online questionnaire for this research study.

Study Outcomes and Publications

Please see here for link to the published article in the BMJ. A summary of the findings is given below:

Refractory ulcerative proctitis presents a huge clinical challenge not only for the patients living with this chronic, progressive condition but also for the professionals who care for them. Currently there is limited research and evidence-based guidance resulting in many patients living with the symptomatic burden of disease and reduced quality of life. The aim of this study was to establish a consensus on the thoughts and opinions related to refractory proctitis disease burden and best practice for management. We conducted a three-round Delphi consensus survey inviting patients living with refractory proctitis and healthcare experts with knowledge on this disease from the UK. There were three rounds of Delphi survey in which participants were asked to rank the importance of the statements and provide any additional comments or clarifications. Following completion of three Delphi survey rounds, fourteen statements reached consensus opinion of the participants. This represents the first step towards developing clinical research data and ultimately the evidence needed for best practice management guidance of this condition. The fourteen statements are:

	Final Statements
1	In patients who are compliant to both rectal and oral therapy over an 8-week period, mesalazine-refractory proctitis is still an existing clinical problem.
2	Present patient reported outcomes do not capture the symptom burden appropriately in proctitis. Disability, faecal incontinence, urgency, constipation, and health-related quality of life are not captured.
3	Constipation is a common problem in symptomatic refractory proctitis, and efforts should be made to treat it independently of inflammatory disease.
4	A multi-disciplinary team (MDT) approach should be highly considered and at the appropriate time, a surgical option should also be considered in refractory inflammatory disease, though the type of surgical intervention is as yet unclear.
5	Patient age and co-morbidities should be factored into the decision-making process for therapies in refractory proctitis.
6	Drug costs should not play a major role in the decision-making process regarding therapies for refractory proctitis.
7	In the treatment of refractory inflammatory disease, patients prefer oral or systemic therapies rather than topical therapy.
8	Research investigating the role of thiopurines to treat inflammatory disease in mesalazine-refractory proctitis is limited.
9	Low-dose topical or oral steroid therapy (5mg prednisolone tablets or suppositories, or budesonide) may be considered to treat symptoms from inflammatory disease in select situations.
10	Present evidence does not provide any clarity regarding the use and sequencing of biological agents and small molecules to treat inflammatory disease from refractory proctitis.
11	Combination treatment with immunomodulators and biological agents should be considered to treat refractory inflammatory disease in proctitis.
12	After excluding other differential diagnoses inflammatory disease may be treated with 2nd or 3rd line biological treatments and small molecules.
13	The role of off-licence topical therapies like acetarsol or tacrolimus is unclear in the treatment of active inflammatory disease. More research is needed.
14	Further research should be focused on refractory proctitis.

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