Validation of the novel Eosinophilic Esophagitis Symptom Questionnaire

Objective: The novel Eosinophilic Esophagitis Symptom Questionnaire (EoE-SQ) is a patient-reported outcome measure that assesses symptoms beyond dysphagia in adults and adolescents with eosinophilic esophagitis (EoE). Methods: The EoE-SQ was administered in the phase 3 R668-EE-1774 trial (NCT03633617), which assessed the efficacy and safety of dupilumab in adults and adolescents with EoE. Baseline and follow-up data were used to estimate its psychometric properties and to determine meaningful score change. Results: The sample included 239 patients (median [range] age, 24 [12-68] years). The EoE-SQ frequency/severity scores showed internal consistency and test–retest reliability. Construct validity was demonstrated through moderate-to-strong correlations between EoE-SQ frequency/severity and other EoE-specific patient-reported outcome measures at baseline (frequency, r = 0.49-0.84; severity, r = 0.46-0.84) and at week 24 (frequency, r = 0.55-0.83; severity, r = 0.46-0.83). In known-groups analyses, the EoE-SQ frequency/severity scores discriminated between patient groups at varying EoE severity levels (defined by the Patient Global Impression of Severity [PGIS]). The EoE-SQ had good responsiveness, with the highest reductions in EoE-SQ frequency/severity scores (indicating greater symptomatic improvement) observed for patients with improved global assessment scores; ANOVA tests were statistically significant for PGIS and Patient Global Impression of Change (P ≤ .0001). A minimum 3.7-point and 5.3-point reduction (representing symptom improvement) in EoE-SQ frequency and severity scores, respectively, were considered clinically meaningful by patients. Conclusions: The EoE-SQ showed good reliability, construct validity, and responsiveness for the assessment of EoE symptoms beyond dysphagia and can be used in combination with existing patient-reported outcome questionnaires to evaluate the full breadth of symptoms experienced by adult and adolescent patients with EoE.