CF Tummy Tracker: A Cystic Fibrosis-Specific Patient-Reported Outcome Measure for Daily Gastrointestinal Symptom...

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.