TY - JOUR
T1 - Novel Questionnaires for Assessing Signs and Symptoms of Eosinophilic Esophagitis in Children
AU - Kamat, Siddhesh
AU - Yaworsky, Andrew
AU - Guillemin, Isabelle
AU - Krohe, Meaghan
AU - Litcher-Kelly, Leighann
AU - McLafferty, Megan
AU - Lamoureux, Roger E.
AU - Lowe, Caitlyn
AU - Chehade, Mirna
AU - Spergel, Jonathan M.
AU - Weinfurt, Kevin
AU - Turner-Bowker, Diane M.
N1 - Funding Information:
This work was supported by Regeneron Pharmaceuticals, Inc. , and Sanofi .
Funding Information:
This work was supported by Regeneron Pharmaceuticals, Inc., and Sanofi.Conflicts of interest: S. Kamat is an employee of Regeneron Pharmaceuticals, Inc. A. Yaworsky, L. Litcher-Kelly, M. McLafftery, R. E. Lamoureux, and C. Lowe are employees of Adelphi Values. I. Guillemin was an employee of Sanofi at the time of the research and is now an employee of IQVIA. M. Krohe and D.M. Turner-Bowker were employees of Adelphi Values at the time of the research. M. Chehade is an employee of the Icahn School of Medicine at Mount Sinai; provided expert clinical advice during all research steps related to questionnaire development; received consulting fees from Regeneron, Allakos, Adare/Ellodi, Shire/Takeda, AstraZeneca, Sanofi, Bristol Myers Squibb, Phathom; and research funding from Regeneron, Allakos, Shire/Takeda, AstraZeneca, Adare/Ellodi, and Danone. J. M. Spergel is an employee of The Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania; and provided expert clinical advice during all research steps related to questionnaire development. K. Weinfurt is an employee of the Duke University School of Medicine; and served as a paid consultant to provide input on approaches for outcomes assessment during questionnaire development.All authors contributed to the development of the publication. Adelphi Values conducted the qualitative research activities, sponsored by Regeneron Pharmaceuticals, Inc. United States and Sanofi, France.
Publisher Copyright:
© 2022 The Authors
PY - 2022/7
Y1 - 2022/7
N2 - 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.
AB - 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.
KW - Allergy
KW - Clinical outcome assessment
KW - EoE
KW - Eosinophilic esophagitis
KW - Observer-reported outcome
KW - Patient-reported outcome
KW - Pediatric
KW - Quality of life
KW - Symptoms
UR - http://www.scopus.com/inward/record.url?scp=85129525502&partnerID=8YFLogxK
U2 - 10.1016/j.jaip.2022.02.049
DO - 10.1016/j.jaip.2022.02.049
M3 - Article
C2 - 35398552
AN - SCOPUS:85129525502
SN - 2213-2198
VL - 10
SP - 1856-1863.e3
JO - Journal of Allergy and Clinical Immunology: In Practice
JF - Journal of Allergy and Clinical Immunology: In Practice
IS - 7
ER -