CARDS-CF
Research type
Research Study
Full title
A Comprehensive Approach To Relief Of Digestive Symptoms In Cystic Fibrosis: CARDS-CF
IRAS ID
304643
Contact name
Alan Smyth
Contact email
Sponsor organisation
Nottingham University Hospitals NHS Trust
Duration of Study in the UK
2 years, 2 months, 31 days
Research summary
Gut symptoms such as bloating, pain and sickness are common for many people with Cystic Fibrosis (pwCF). These symptoms can be embarrassing, have an effect on body image and interfere with quality of life. Two in every three pwCF will miss either school or work because of gut symptoms and these also affect their ability to exercise or attend social events. We want to better understand how these symptoms disrupt or impact on the daily lives of pwCF.
Despite these pervasive symptoms, there is no validated patient reported outcome measurement (PROM) for measuring the burden of GI symptoms for pwCF. The pattern of GI symptoms in CF is similar to functional gastrointestinal (GI) disorders such as irritable bowel syndrome which requires a daily assessment of symptoms. Therefore, the development of a CF specific PROM for GI symptoms is essential and should include a daily assessment of symptom burden. To develop this we will conduct a series of focus groups and interviews with pwCF to understand their experiences and create a list of possible items to be used in our measurement. These will then be included in a survey of 100 people to find out people’s opinions on these. Following this, a prototype of the PROM will be developed and then tested by 100 people in an app over a 2 week period.
PwCF 12 years and over are eligible to take part in the study. We will primarily focus on people from the UK, but pwCF from other countries who are able to speak English will also be included. PwCF will be recruited from 6 UK CF centres as well as via social media. The PROM is being developed for use within research trials. However in the future it may also be used in clinical practice.
Summary of Results:
This study aimed to create a patient reported outcome measure (PROM) which is a questionnaire that people with cystic fibrosis (CF) can use to track daily gastrointestinal (GI) symptoms. The aim was to develop this PROM to be electronic to sit on a smartphone app.We had planned to achieve this through the following objectives
• To develop and refine a conceptual framework of the factors contributing to GI symptom burden. This defines the scope of what we want the PROM to measure.• To create a primary list of item questions, to address the conceptual framework.
• To refine the relevance, comprehensiveness, and understandability of the items in the primary list through think aloud interviews and a patient survey.
• To pilot the resulting items for the PROM over a two week period to explore the patient responses when using the PROM to assess content validity.
Through this study we have achieved the above objectives to develop a CF specific patient reported outcome measure, CF Tummy Tracker, to understand daily gastrointestinal symptom burden for pwCF.
The study was completed in five stages, with guidance from an expert panel that included CF researchers, doctors, people with CF, and parents. Recruitment was international, promoted via online platforms and CF organisations (e.g. CF Europe, US CF Foundation) alongside UK NHS CF care centres. Ethical approval was obtained, and the study was registered on clinicaltrials.gov.
Stage 1: Defining the scope of the PROM with the conceptual framework.
The conceptual framework for the PROM focused on five areas: GI symptom severity, emotional well-being, daily living, treatment burden, and overall acceptability. We help a focus group consisting of seven pwCF, aged 14-59. The most disruptive GI symptoms identified by the group included bloating; stool frequency, urgency, and consistency; and the impact symptoms had on sleep and social situations. Participants broadly accepted the framework although suggested refining some terms, and the framework was used as the basis for the development of the items for the PROM.Stage 2: Question development
The expert panel created 93 potential questions for the PROM, which were condensed into 30 questions using a 5 point Likert scale for the answers. The questions targeted the five key areas from Stage 1.Stage 3: Question refinement
This stage refined the questions through a series of cognitive interviews with pwCF using the "think-aloud" method. The interviews were completed in rounds, with items adapted between rounds if any problems were found with the questions. The subsequent interviews focused on whether the changes had resolved the initial problems. Eleven participants took part in think-aloud interviews across four rounds. Feedback including splitting complex questions where two aspects of symptom burden were incorporated into one question, removing redundant ones, and trialling different wording of the recall periods. This led to 19 questions being finalised for further testing in stage 4.Stage 4: reducing the number of questions An online survey helped to finalise the final questions for the PROM. People with CF rated items based on their experience 1) in the last 24 hours 2) last year and 3) importance. In total, 180 participants from 15 countries consented to take part in the survey. Seventeen items firstly underwent impact analysis to identify items with the greatest impact. All items scored above the pre-defined threshold of two for inclusion. Eight items had an impact score of three or higher.
A lasso regression model (a type of statistical test) identified the best-performing questions to include in the PROM, whilst preventing too many questions being used. This reduced the final number of items to nine. Following expert panel discussion, an additional question about the enjoyment of eating was added into the model as it was felt to be important but underrepresented by the model. The final PROM included 10 questions addresses GI symptoms and associated burden.Stage 5: testing the PROM on the smartphone app This involved tested the final PROM called CF Tummy Tracker in a smartphone app. A total of 151 people consented to take part in the study, 145 participants submitting at least one day of PROM data. Most participants (94%) identified as white, with an average age of 35.8 years. A small number (12%) of participants were involved in the previous study stages. The median number of days people completed the app was 10 out of 14 days although this was higher in people who were recruited from one of the UK CF centres compared to those recruited online. The PROM showed good reliability when being with stable symptoms gave their scores over 2 separate days. While the study initially planned to compare results to an existing CF symptom tracker, permission to use this was withdrawn, so alternative analysis was used which showed strong reliability in tracking symptoms over time.
User feedback at the end of the was positive, with most participants who completed the feedback finding the questions relevant and understandable. The majority (73%) reported that the app improved their awareness of GI symptoms, and 85% found it helpful for tracking their symptoms. Many participants (69%) indicated they would continue using the app to monitor their symptoms in the future.In summary, this study has developed a CF-specific PROM, CF Tummy Tracker, to measure daily GI symptom burden for people with CF. Future work will involve testing it further to validate it for use in clinical trials, trying it in different formats (such a paper versions) and translating it to other languages to improve its accessibility.
REC name
North West - Preston Research Ethics Committee
REC reference
21/NW/0345
Date of REC Opinion
17 Jan 2022
REC opinion
Further Information Favourable Opinion