Background
EoE
INFLAMMATORY DISEASE, EOE, EOSINOPHILIC ESOPHAGITIS
Eosinophilic esophagitis (EoE) is a chronic, progressive, type 2 inflammatory disease that has a substantial impact on health-related quality of life (HRQOL) and that is increasing in incidence [It is important to understand the impact of EoE symptoms on HRQOL when evaluating new therapeutics. The United States Food and Drug Administration recommends the use of patient-reported outcome (PRO) measures in clinical trials as a way to measure the impact of treatment on symptoms and HRQOL [The initial development of the EoE-IQ was informed by a literature review and advice meetings with EoE experts. The findings of this research led to the selection of 11 HRQOL-related impact concepts for inclusion in the draft questionnaire. An initial 11-item draft of the EoE-IQ was tested in a set of 23 cognitive debriefing interviews. These interviews were designed to assess patients’ ability to understand the content of the EoE-IQ and assess the relevance and comprehensiveness of the concepts measured by the questionnaire. During 11 of the interviews, the EoE-IQ was debriefed in full with a combination of adult (n = 5) and adolescent (n = 6) participants, while the remaining 12 interviews were conducted with adult participants who were asked to read the EoE-IQ and provide high-level feedback on the questionnaire. Findings from these interviews indicated that the EoE-IQ was well understood and comprehensively measured concepts relevant to the experience of patients with EoE (Additional file Subsequent to these interviews, the EoE-IQ was implemented in a randomized, placebo-controlled trial investigating the efficacy and safety of dupilumab for adult and adolescent patients with EoE (R668-EE-1774; NCT03633617) [Before a new PRO can be considered valid and reliable for use in a target population, it is important to assess its measurement properties, including reliability, construct validity, and responsiveness. The aim of this analysis was to evaluate the validity of the EoE-IQ for measuring HRQOL in adults and adolescents with EoE in a clinical trial. The objectives were to provide evidence of the measurement properties of the EoE-IQ and to determine a threshold for meaningful change using questionnaire responses gathered in the R668-EE-1774 study.