Dupilumab improves clinical outcome assessments in children with eosinophilic esophagitis in a randomized phase 3 study.
8 affiliations
- 1Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
- 2Division of Allergy and Immunology, Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at University of Pennsylvania, Philadelphia, Pennsylvania, USA.
- 3Pediatric Digestive Endoscopy, Pediatric Gastroenterology and Liver Unit, Maternal and Child Health Department, University Hospital Umberto, Sapienza University of Rome, Rome, Italy.
- 4University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
- 5Division of Pediatric Gastroenterology and Hepatology, Department of Pediatrics, Children's Hospital, London Health Sciences Centre, Western University, London, Ontario, Canada.
- 6Division of Allergy and Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.
- 7Regeneron Pharmaceuticals Inc., Tarrytown, New York, USA.
- 8Sanofi, Cambridge, Massachusetts, USA.
Abstract
Background
Eosinophilic esophagitis (EoE) is a chronic, progressive disease impacting patient quality of life (QoL). Assessment of pediatric EoE symptoms and their impact is challenging. This post hoc analysis of the phase 3 EoE KIDS trial (NCT04394351) evaluated a range of clinician-, caregiver-, and patient-reported outcomes (ClinROs/ObsROs/PROs) in pediatric patients aged 1-11 years with active EoE.Methods
EoE KIDS evaluated the efficacy and safety of dupilumab in a 16-week, placebo-controlled, double-blind period (Part A). ClinRO/ObsRO/PRO measures included the Pediatric EoE Sign/Symptom Questionnaire caregiver (PESQ-C) and patient (PESQ-P) versions, Pediatric EoE Symptom Score version 2.0 (PEESS v2.0-Caregiver version), Global Impression of Severity (GIS), Global Impression of Change (GIC), and Pediatric EoE Impact Scale (PEIS). EoE KIDS was not powered to detect significant differences in the main symptom measure, PESQ-C.Results
In Part A, 37 patients were assigned to higher-exposure dupilumab and 34 to placebo. The percentage reduction (improvement) from baseline to week 16 (least-squares mean [95% confidence interval]) in proportion of days with ≥1 EoE sign assessed by PESQ-C was numerically greater with dupilumab (-48.5% [-73.4, -23.6]) than placebo (-19.65% [-45.3, 6.0]). Changes in symptoms and QoL assessed by PEESS v2.0-Caregiver version, GIS, GIC, and PEIS instruments also generally favored dupilumab vs. placebo. High concordance was observed between individual signs of EoE (symptoms observed by caregivers) and corresponding patient-reported symptoms using PESQ-C and PESQ-P.Conclusions
Improvements with dupilumab vs. placebo were observed in most ClinROs and ObsROs, but these improvements were not consistent within certain PROs.Trial registration
ClinicalTrials.gov ID NCT04394351; Trial registered on May 7th, 2020.
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BibTeX
@article{chehade2026dupilumab,
title = {{Dupilumab improves clinical outcome assessments in children with eosinophilic esophagitis in a randomized phase 3 study.}},
author = {Mirna Chehade and Jonathan M Spergel and Salvatore Oliva and Evan S Dellon and Dhandapani Ashok and Marc E Rothenberg and Ruiqi Liu and Margee Louisias and Allen Radin and Sarette T Tilton and Eilish McCann},
journal = {BMC gastroenterology},
year = {2026},
month = jul,
doi = {10.1186/s12876-026-05017-8},
url = {https://europepmc.org/article/MED/42928144},
note = {CC BY},
}
RIS
TY - JOUR
TI - Dupilumab improves clinical outcome assessments in children with eosinophilic esophagitis in a randomized phase 3 study.
AU - Mirna Chehade
AU - Jonathan M Spergel
AU - Salvatore Oliva
AU - Evan S Dellon
AU - Dhandapani Ashok
AU - Marc E Rothenberg
AU - Ruiqi Liu
AU - Margee Louisias
AU - Allen Radin
AU - Sarette T Tilton
AU - Eilish McCann
PY - 2026
DA - 2026/07/29
JO - BMC gastroenterology
DO - 10.1186/s12876-026-05017-8
UR - https://europepmc.org/article/MED/42928144
AB - Background Eosinophilic esophagitis (EoE) is a chronic, progressive disease impacting patient quality of life (QoL). Assessment of pediatric EoE symptoms and their impact is challenging. This post hoc analysis of the phase 3 EoE KIDS trial (NCT04394351) evaluated a range of clinician-, caregiver-, and patient-reported outcomes (ClinROs/ObsROs/PROs) in pediatric patients aged 1-11 years with active EoE.Methods EoE KIDS evaluated the efficacy and safety of dupilumab in a 16-week, placebo-controlled, double-blind period (Part A). ClinRO/ObsRO/PRO measures included the Pediatric EoE Sign/Symptom Questionnaire caregiver (PESQ-C) and patient (PESQ-P) versions, Pediatric EoE Symptom Score version 2.0 (PEESS v2.0-Caregiver version), Global Impression of Severity (GIS), Global Impression of Change (GIC), and Pediatric EoE Impact Scale (PEIS). EoE KIDS was not powered to detect significant differences in the main symptom measure, PESQ-C.Results In Part A, 37 patients were assigned to higher-exposure dupilumab and 34 to placebo. The percentage reduction (improvement) from baseline to week 16 (least-squares mean [95% confidence interval]) in proportion of days with ≥1 EoE sign assessed by PESQ-C was numerically greater with dupilumab (-48.5% [-73.4, -23.6]) than placebo (-19.65% [-45.3, 6.0]). Changes in symptoms and QoL assessed by PEESS v2.0-Caregiver version, GIS, GIC, and PEIS instruments also generally favored dupilumab vs. placebo. High concordance was observed between individual signs of EoE (symptoms observed by caregivers) and corresponding patient-reported symptoms using PESQ-C and PESQ-P.Conclusions Improvements with dupilumab vs. placebo were observed in most ClinROs and ObsROs, but these improvements were not consistent within certain PROs.Trial registration ClinicalTrials.gov ID NCT04394351; Trial registered on May 7th, 2020.
SN - 1471-230X
ER -