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Background <p>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.</p> Methods <p>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.</p> Results <p>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.</p> Conclusions <p>Improvements with dupilumab vs. placebo were observed in most ClinROs and ObsROs, but these improvements were not consistent within certain PROs.</p> Trial registration <p>ClinicalTrials.gov ID NCT04394351; Trial registered on May 7th, 2020.</p> Graphical Abstract <p></p>

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Dupilumab improves clinical outcome assessments in children with eosinophilic esophagitis in a randomized phase 3 study

  • 期刊论文

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  • 发表日期：

    2026年7月29日

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  • Mirna Chehade,
  • Jonathan M. Spergel,
  • Salvatore Oliva,
  • Evan S. Dellon,
  • Dhandapani Ashok,
  • Marc E. Rothenberg,
  • Ruiqi Liu,
  • Margee Louisias,
  • Allen Radin,
  • Sarette T. Tilton,
  • Eilish McCann

摘要

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.

Graphical Abstract