CF Tummy Tracker: A Cystic Fibrosis-Specific Patient-Reported Outcome Measure for Daily Gastrointestinal Symptom
Rebecca J Calthorpe1, Hisham A Saumtally2, Laura M Howells3
1School of Medicine and NIHR Nottingham Biomedical Research Centre, Nottingham, United Kingdom.
Insights
A new tool, the CF Tummy Tracker, measures daily gastrointestinal symptom burden in people with cystic fibrosis (pwCF). This validated patient-reported outcome measure (PROM) addresses a critical need for CF gastrointestinal symptom assessment.
Area of Science:
- Gastroenterology
- Pulmonology
- Medical Device Development
Background:
- Gastrointestinal (GI) symptoms significantly impact the quality of life for individuals with cystic fibrosis (CF).
- A lack of validated, CF-specific patient-reported outcome measures (PROMs) for daily GI symptom burden hinders effective clinical management and research.
- Existing outcome measures do not adequately capture the daily nuances of GI distress experienced by people with CF (pwCF).
Purpose of the Study:
- To develop and validate a novel, CF-specific PROM designed for daily assessment of gastrointestinal symptom burden.
- To address the unmet need for a reliable tool to measure the daily impact of GI symptoms in pwCF aged 12 and older.
- To create a patient-centered outcome measure that can be easily integrated into clinical practice and research.
Main Methods:
- A 5-stage development process adhering to regulatory guidance, including conceptual framework development, item generation, refinement, reduction, and initial testing.
- A mixed-methods approach incorporating expert panels, focus groups (n=7 pwCF), interviews (n=11 pwCF), and an online survey (n=180 pwCF) for comprehensive input.
- Initial validation involved 151 pwCF completing the CF Tummy Tracker daily for 14 days via a smartphone application.
Main Results:
- The CF Tummy Tracker, a 10-item PROM, was developed with significant input from the CF community.
- The PROM demonstrated no floor or ceiling effects, indicating a comprehensive measurement range.
- High test-retest reliability (ICC=0.94) and strong correlation with an anchor question confirm the PROM's stability and validity.
Conclusions:
- The CF Tummy Tracker effectively addresses the gap for a validated, CF-specific PROM for daily GI symptom assessment.
- Further psychometric validation in a new patient cohort is planned to support its use in clinical trials.
- The PROM is intended for use in both electronic and paper formats to enhance accessibility for pwCF.
Objective:
To develop a cystic fibrosis (CF)-specific patient-reported outcome measure (PROM) to measure the daily burden of gastrointestinal symptoms for people with cystic fibrosis (pwCF) aged 12 years and older and address the lack of validated outcome measures for gastrointestinal symptoms in CF.
Patients And Methods:
CF Tummy Tracker was developed through a 5-stage approach in accordance with regulatory guidance. This included development and refinement of a conceptual framework; item generation; refinement; reduction; selection; and initial PROM testing. A mixed-methods approach, consisting of expert panel discussions, a focus group, interviews, and an online survey, was used. In initial testing, participants completed the PROM daily for 14 days via a smartphone application. This study was performed from March 14, 2022, December 12, 2023.
Results:
The CF community were involved throughout the development via a focus group (n=7 pwCF), interviews (n=11 pwCF), and an online survey (n=180 pwCF). A formative model was confirmed for the PROM. The final PROM, CF Tummy Tracker, consists of 10 items capturing gastrointestinal symptom burden, tested in 151 pwCF. The PROM reported no floor or ceiling effects, high test-retest reliability (intra-class correlation coefficient=0.94), and strong correlation with the anchor question.
Conclusion:
CF Tummy Tracker aims to address the gap in validated CF-specific PROMs for daily completion. Further testing of the psychometric properties of the PROM are planned in a new patient cohort to validate its use in clinical trials and support its use in both electronic and paper formats to increase accessibility.
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