Novel Questionnaires for Assessing Signs and Symptoms of Eosinophilic Esophagitis in Children

Siddhesh Kamat, Andrew Yaworsky, Isabelle Guillemin, Meaghan Krohe, Leighann Litcher-Kelly, Megan McLafferty, Roger E. Lamoureux, Caitlyn Lowe, Mirna Chehade, Jonathan M. Spergel, Kevin Weinfurt, Diane M. Turner-Bowker

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

Background: Pediatric patients with eosinophilic esophagitis (EoE) experience heterogeneous symptoms and the patient's age may preclude reliable self-report of symptoms. Objective: The goal of this study was to develop a patient-reported outcome and an observer-reported outcome questionnaire to evaluate the signs and symptoms of EoE in pediatric patients (≥1 to <12 y of age) in a clinical trial setting. Methods: A concept-focused literature review, expert advice meetings, and concept elicitation interviews with pediatric EoE patients and their caregivers were conducted to identify disease-related signs and symptoms. Instructions, items, and response options were drafted. Cognitive debriefing interviews were conducted to evaluate children's and caregivers’ ability to understand and respond to the questionnaires and to evaluate the comprehensiveness of the concepts measured. Results: Results from the literature review, expert advice meetings (n = 6), and concept elicitation interviews (n = 24) informed the development of the Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire intended for use by patients (PESQ-P) with EoE 8 years or older to younger than 12 years and an observer-reported outcome questionnaire planned for use by caregivers of patients (PESQ-C) 1 year old or older to younger than 12 years. Both questionnaires measure the same concepts; the PESQ-P assesses the frequency, duration, and/or severity of symptoms and the PESQ-C assesses the presence/absence of the signs/symptoms. The cognitive debriefing interviews (n = 17) demonstrated that participants were able to comprehend and complete the questionnaires as intended. Conclusions: This study provides evidence of the content validity of 2 novel questionnaires, PESQ-P and PESQ-C, designed to evaluate the symptom experience of pediatric EoE patients in a clinical trial setting.

Original languageEnglish
Pages (from-to)1856-1863.e3
JournalJournal of Allergy and Clinical Immunology: In Practice
Volume10
Issue number7
DOIs
StatePublished - Jul 2022

Keywords

  • Allergy
  • Clinical outcome assessment
  • EoE
  • Eosinophilic esophagitis
  • Observer-reported outcome
  • Patient-reported outcome
  • Pediatric
  • Quality of life
  • Symptoms

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