Development of the Escalation of Therapy or Intervention (ETI) Calculator for Patients with Ulcerative Colitis Using ePROMs.

BACKGROUND AND AIMS: Digital collection of patient-reported outcome measures [PROMs] is largely unexplored as a basis for follow-up for patients with ulcerative colitis [UC]. Our aim was to develop a model to predict the likelihood of escalation of therapy or intervention at an outpatient appointment that may be used to rationalize follow-up. METHODS: TrueColours-IBD is a web-based, real-time, remote monitoring software that allows longitudinal collection of ePROMs. Data for prediction modelling were derived from a Development Cohort, guided by the TRIPOD statement. Logistic regression modelling used ten candidate items to predict escalation of therapy or intervention. An Escalation of Therapy or Intervention [ETI] calculator was developed, and applied in a Validation Cohort at the same centre. RESULTS: The Development Cohort [n = 66] was recruited in 2016 and followed for 6 months [208 appointments]. From ten items, four significant predictors of ETI were identified: SCCAI, IBD Control-8, faecal calprotectin, and platelets. For practicality, a model with only SCCAI and IBD Control-8, both entered remotely by the patient, without the need for faecal calprotectin or blood tests was selected. Between 2018 and 2020, a Validation Cohort of 538 patients [1188 appointments] was examined. A 5% threshold on the ETI calculator correctly identified 343/388 [88%] escalations and 274/484 [57%] non-escalations. CONCLUSIONS: A calculator based on digital, patient-entered data on symptoms and quality of life can predict whether a patient with UC requires escalation of therapy or intervention at an outpatient appointment. This may be used to streamline outpatient appointments for patients with UC.

Ustekinumab for the treatment of moderate to severe ulcerative colitis: a multicentre UK cohort study.

Objective: Ustekinumab is an interleukin-12/interleukin-23 receptor antagonist licensed for the treatment of ulcerative colitis (UC). Clinical trial data were promising; however, real-world data are limited. We assessed the safety and effectiveness of ustekinumab in UC in a real-world setting. Design/method: This was a multicentre, retrospective, observational cohort study between February 2020 and January 2022. Disease activity was assessed using the Simple Clinical Colitis Activity Index (SCCAI). Clinical remission was defined as a SCCAI≤2. The primary endpoints were rates of corticosteroid-free remission (CSFR) at week 16 and at week 26. Objective outcomes, including faecal calprotectin (FCAL), were also collected. Results: 110 patients with UC (65% male; median age 40 (IQR range 29-59); 96% with prior biologic and/or tofacitinib exposure) had a median follow-up of 28 weeks (IQR 17-47). CSFR was 36% (18/50) at week 16% and 33% (13/39) at week 26, corresponding with a significant fall in SCCAI from 6 (IQR 4-8) at baseline to 3 (IQR 0-5) at week 26, p<0.001. By week 16, there was improvement of median FCAL measurements, which fell from a baseline of 610 µg/g (IQR 333-1100) to 102 µg/g (IQR 54-674) at week 16. At the end of follow-up, 15% (17/110) had discontinued treatment; 13 patients due to primary non-response or loss of response, and 1 patient for family planning. Treatment was discontinued in three patients due to adverse events. Conclusion: In the largest real-world study to date, ustekinumab was effective with a reassuring safety profile in a refractory cohort of patients.

Patient-reported Outcomes: the ICHOM Standard Set for Inflammatory Bowel Disease in Real-life Practice Helps Quantify Deficits in Current Care.

BACKGROUND: Patient-reported outcome measures [PROMs] are key to documenting outcomes that matter most to patients and are increasingly important to commissioners of health care seeking value. We report the first series of the ICHOM Standard Set for Inflammatory Bowel Disease [IBD]. METHODS: Patients treated for ulcerative colitis [UC] or Crohn's disease [CD] in our centre were offered enrolment into the web-based TrueColours-IBD programme. Through this programme, e-mail prompts linking to validated questionnaires were sent for symptoms, quality of life, and ICHOM IBD outcomes. RESULTS: The first 1299 consecutive patients enrolled [779 UC, 520 CD] were studied with median 270 days of follow-up (interquartile range [IQR] 116, 504). 671 [52%] were female, mean age 42 years (standard deviation [sd] 16), mean body mass index [BMI] 26 [sd 5.3]. At registration, 483 [37%] were using advanced therapies. Median adherence to fortnightly quality of life reporting and quarterly outcomes was 100% [IQR 48, 100%] and 100% [IQR 75, 100%], respectively. In the previous 12 months, prednisolone use was reported by 229 [29%] patients with UC vs 81 [16%] with CD, p <0.001; 202 [16%] for <3 months; and 108 [8%] for >3 months. An IBD-related intervention was reported by 174 [13%] patients, and 80 [6%] reported an unplanned hospital admission. There were high rates of fatigue [50%] and mood disturbance [23%]. CONCLUSIONS: Outcomes reported by patients illustrate the scale of the therapeutic deficit in current care. Proof of principle is demonstrated that PROM data can be collected continuously with little burden on health care professionals. This may become a metric for quality improvement programmes or to compare outcomes.

Early development of a novel scale to measure adaptation in people diagnosed with inflammatory bowel disease - the A-inflammatory bowel disease.

Objective: To undergo the preliminary development of a new measure of patient adaptation to Inflammatory Bowel Disease (IBD): A-IBD. Design: Based on a prior conceptualisation of adaptation, a 40-item scale was generated and completed by 304 people diagnosed with IBD. Main outcome measures: Psychometric analysis of the measure. Association with the Brief Illness Perception Questionnaire (Brief IPQ) and the Inflammatory Bowel Disease Questionnaire (IBDQ). Results: The 18-item scale consisted of four subscales (patient identity, person identity, acceptance, expectations). Weak to moderate correlations were found between subscales of the A-IBD and the Brief IPQ and IBDQ. Conclusion: The A-IBD shows potential for assessing adaptation. Further analysis could confirm its usefulness.

Using item response theory (IRT) to improve the efficiency of the Simple Clinical Colitis Activity Index (SCCAI) for patients with ulcerative colitis.

BACKGROUND: The SCCAI was designed to facilitate assessment of disease activity in ulcerative colitis (UC). We aimed to interrogate the metric properties of individual items of the SCCAI using item response theory (IRT) analysis, to simplify and improve its performance. METHODS: The original 9-item SCCAI was collected through TrueColours, a real-time software platform which allows remote entry and monitoring of patients with UC. Data were securely uploaded onto Dementias Platform UK Data Portal, where they were analysed in Stata 16.1 SE. A 2-parameter (2-PL) logistic IRT model was estimated to evaluate each item of the SCCAI for its informativeness (discrimination). A revised scale was generated and re-assessed following systematic removal of items. RESULTS: SCCAI data for 516 UC patients (41 years, SD = 15) treated in Oxford were examined. After initial item deletion (Erythema nodosum, Pyoderma gangrenosum), a 7-item scale was estimated. Discrimination values (information) ranged from 0.41 to 2.52 indicating selected item inefficiency with three items < 1.70 which is a suggested discriminatory value for optimal efficiency. Systematic item deletions found that a 4-item scale (bowel frequency day; bowel frequency nocturnal; urgency to defaecation; rectal bleeding) was more informative and discriminatory of trait severity (discrimination values of 1.50 to 2.78). The 4-item scale possesses higher scalability and unidimensionality, suggesting that the responses to items are either direct endorsement (patient selection by symptom) or non-endorsement of the trait (disease activity). CONCLUSION: Reduction of the SCCAI from the original 9-item scale to a 4-item scale provides optimum trait information that will minimise response burden. This new 4-item scale needs validation against other measures of disease activity such as faecal calprotectin, endoscopy and histopathology.

Real-time data monitoring for ulcerative colitis: patient perception and qualitative analysis.

BACKGROUND/AIMS: TrueColours ulcerative colitis (TCUC) is a comprehensive web-based program that functions through email, providing direct links to questionnaires. Several similar programs are available, however patient perspectives are unexplored. METHODS: A pilot study was conducted to determine feasibility, usability and patient perceptions of real-time data collection (daily symptoms, fortnightly quality of life, 3 monthly outcomes). TCUC was adapted from a web-based program for patients with relapsing-remitting bipolar disorder, using validated UC indices. A semi-structured interview was developed and audio-recorded face-to-face interviews were conducted after 6 months of interaction with TCUC. Transcripts were coded in NVivo11, a qualitative data analysis software package. An inductive approach and thematic analysis was conducted. RESULTS: TCUC was piloted in 66 patients for 6 months. Qualitative analysis currently defies statistical appraisal beyond "data saturation," even if it has more influence on clinical practice than quantitative data. A total of 28 face-to-face interviews were conducted. Six core themes emerged: awareness, control, decision-making, reassurance, communication and burden of treatment. There was a transcending overarching theme of patient empowerment, which cut across all aspects of the TCUC experience. CONCLUSIONS: Patient perception of the impact of real-time data collection was extremely positive. Patients felt empowered as a product of the self-monitoring format of TCUC, which may be a way of improving self-management of UC whilst also decreasing the burden on the individual and healthcare services.

A qualitative study of patients' experience of living with inflammatory bowel disease: A preliminary focus on the notion of adaptation.

This study investigates the lived experience of inflammatory bowel disease with the aim of examining the process of adapting to life with inflammatory bowel disease. Adaptation is often referred to as the desirable outcome in chronic illnesses such as inflammatory bowel disease; yet little is known as to how this is achieved. A total of 10 Crohn's and 12 ulcerative colitis patients recruited from online support networks participated in individual, semi-structured interviews. The study has identified the notion of a 'new normal' as central to adaptation, whereby patients seek to recover a sense of normality by finding an equilibrium between their lives before and after diagnosis.