Validation of the Novel Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire-Caregiver Version in EoE KIDS

Background: The Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire for Caregivers (PESQ-C) is a novel clinical outcome assessment that was administered daily in EoE KIDS to assess signs (caregiver-observed eosinophilic esophagitis [EoE] symptoms) in patients aged 1 to 11 years. Objective: This study aimed to validate this novel instrument. Methods: Blinded baseline and week 16 data from the randomized, interventional, phase 3 EoE KIDS study (NCT04394351) were analyzed to evaluate the measurement properties of the PESQ-C, including reliability, construct and known-groups validity, responsiveness, and interpretation of change. Exit interviews at week 16 elicited caregivers’ perspectives on patient experience before and after treatment to ascertain the level of change in PESQ-C scores that was considered meaningful. Results: Caregivers completed the PESQ-C for 99 patients (median age, 8 years; 75.8% male; 82.8% White). Test-retest reliability scores (intraclass correlation coefficient, 0.94) exceeded the acceptable threshold for reliability (>0.70). As hypothesized, construct validity correlations with other clinical assessments measuring concepts similar to those of the PESQ-C were moderate at baseline and week 16, and PESQ-C scores discriminated among patient groups defined by EoE severity. Insufficient correlation with anchor measures precluded quantitative determination of meaningful change. In qualitative exit interviews performed with 69 caregivers, 83% felt that the patient experienced meaningful change; for patients with 2 or more days’ improvements, 38% of caregivers said that even a 1-day improvement is meaningful. Conclusion: This analysis confirmed the reliability and validity of the PESQ-C for evaluating caregiver-observed EoE signs in pediatric patients within a clinical study context. Exit interviews provided evidence supporting meaningful changes in EoE signs.