BACKGROUND A critical limitation in outcome assessment for celiac disease (CeD) is the lack of a pediatric-specific patient-reported outcome (PRO) instrument to capture disease- and treatment-related outcomes and experiences of health and health care from the patient perspective.
OBJECTIVE This protocol describes phase 1 of a multiphase mixed methods study to develop the CELIAC-Q Kids, a cross-cultural, internationally applicable PRO instrument for children and youth aged 8 to 18 years with CeD.
METHODS The CELIAC-Q Kids will be developed using a multistage mixed methods approach consistent with best practices for PRO instrument development. First, a scoping review identifying concepts previously measured in individuals with CeD informed the development of a preliminary conceptual framework. Second, concept elicitation interviews with children and youth aged 8 to 18 years with CeD and their caregivers will be conducted to identify concepts of importance to patients. Qualitative data will be analyzed using interpretive description to refine the conceptual framework and generate an item pool and preliminary scales. Third, cognitive debriefing interviews with children and youth, together with multidisciplinary expert clinician input from participating international centers, will be used to iteratively refine items and scales. Finally, the field-test version of the CELIAC-Q Kids will be translated using rigorous, standardized methods in preparation for an international field-test study. Ethics approval has been obtained from the research ethics boards at all participating sites.
RESULTS The study was first funded in 2020, with additional funding received in 2025. Recruitment for the concept elicitation interviews began in July 2020, and data collection was completed in June 2024 with 25 participants. As of September 2026, cognitive debriefing interviews are ongoing. The field-test version of the CELIAC-Q Kids is expected to be complete by December 2026, translation and cultural adaptation are planned for 2027, and phase 1 results are expected to be published in 2027
CONCLUSIONS At the end of this phase, a conceptual framework and set of scales capturing disease- and treatment-related outcomes and experiences relevant to pediatric CeD will be developed and ready for field testing. An integrated knowledge translation approach will engage patients, families, and clinicians throughout the study, and findings will be disseminated through peer-reviewed publications and scientific conferences.
CLINICALTRIAL ClinicalTrials.gov NCT05548166
