Dupilumab in Pediatric Eosinophilic Esophagitis

BACKGROUND AND OBJECTIVES: Dupilumab is an approved biologic therapy for eosinophilic esophagitis (EoE); however, real-world pediatric data remain limited. The aim of this study is to evaluate the effectiveness and safety of dupilumab in children with EoE enrolled in the international European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN)-funded pEEr registry, with a focus on combined remission, treatment deescalation, and predictors of sustained response. METHODS: DU-PED is a multicenter, retrospective cohort study including children with confirmed EoE treated with dupilumab in routine clinical practice. The primary outcome was combined remission at 6 and 12 months, defined as simultaneous clinical (≥75% Pediatric Eosinophilic Esophagitis Symptom Score [PEESS] v2.0 reduction or symptom resolution), endoscopic (EoE Endoscopic Reference Score [EREFS] ≤2), and histologic remission (<15 eos/high-power field [HPF]). Secondary outcomes included longitudinal changes in disease domains, treatment deescalation, predictors of remission, and safety. Multivariable logistic regression and ROC analysis were performed. RESULTS: A total of 109 patients from 22 centers were included (median age 16 years; 75% male). Combined remission was achieved in 53% at 6 months and 86% at 12 months. PEESS v2.0, EREFS, and peak eosinophil counts significantly improved over time (P < 0.001). At 12 months, 81% of patients were receiving dupilumab monotherapy, and 94% reduced concomitant EoE-directed therapy. Higher baseline proximal eosinophil density was independently associated with a lower likelihood of sustained combined remission at 12 months (odds ratio 0.98, 95% CI 0.96-0.99). Dupilumab was well tolerated, with no treatment discontinuations. CONCLUSIONS: In a large international registry-based pediatric cohort, dupilumab achieved high and durable rates of combined remission and enabled substantial treatment simplification, supporting its role as an effective long-term treatment strategy for pediatric EoE.

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Journal
PEDIATRICS
Published
2026-11-03
DOI
https://doi.org/10.1542/peds.2026-076556
Primary Topic
Eosinophilic Esophagitis
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article
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article

Dupilumab in Pediatric Eosinophilic Esophagitis

Saskia Vande Velde, Eleftheria S. Roma, Sara Renzo, Léa Chantal Tran et al.
6 links
PEDIATRICS
Eosinophilic Esophagitis
article

Dupilumab in Pediatric Eosinophilic Esophagitis

Saskia Vande Velde, Eleftheria S. Roma, Sara Renzo, Léa Chantal Tran, Caterina Strisciuglio, Jan Melek, Ali Alsarhan, Noam Zevit, Danila Volpe, Giovanna Alfano, Erasmo Miele, Lorenzo Norsa, Nicolas Kalach, Mordechai Slae, Salvatore Oliva, Christos Tzivinikos, Víctor Vila-Miravet, Matjaž Homan, Carolina Gutiérrez‐Junquera, Johanna H. Oudshoorn, Vasiliki Spyropoulou, Giusy Russo, Alexandra Papadopoulou, Gloria Domínguez Ortega, Francesca Rea, Cosimo Ruggiero, Sraya Greenberger, Sonia Fernández Fernández
article en

Abstract

BACKGROUND AND OBJECTIVES: Dupilumab is an approved biologic therapy for eosinophilic esophagitis (EoE); however, real-world pediatric data remain limited. The aim of this study is to evaluate the effectiveness and safety of dupilumab in children with EoE enrolled in the international European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN)-funded pEEr registry, with a focus on combined remission, treatment deescalation, and predictors of sustained response. METHODS: DU-PED is a multicenter, retrospective cohort study including children with confirmed EoE treated with dupilumab in routine clinical practice. The primary outcome was combined remission at 6 and 12 months, defined as simultaneous clinical (≥75% Pediatric Eosinophilic Esophagitis Symptom Score [PEESS] v2.0 reduction or symptom resolution), endoscopic (EoE Endoscopic Reference Score [EREFS] ≤2), and histologic remission (<15 eos/high-power field [HPF]). Secondary outcomes included longitudinal changes in disease domains, treatment deescalation, predictors of remission, and safety. Multivariable logistic regression and ROC analysis were performed. RESULTS: A total of 109 patients from 22 centers were included (median age 16 years; 75% male). Combined remission was achieved in 53% at 6 months and 86% at 12 months. PEESS v2.0, EREFS, and peak eosinophil counts significantly improved over time (P < 0.001). At 12 months, 81% of patients were receiving dupilumab monotherapy, and 94% reduced concomitant EoE-directed therapy. Higher baseline proximal eosinophil density was independently associated with a lower likelihood of sustained combined remission at 12 months (odds ratio 0.98, 95% CI 0.96-0.99). Dupilumab was well tolerated, with no treatment discontinuations. CONCLUSIONS: In a large international registry-based pediatric cohort, dupilumab achieved high and durable rates of combined remission and enabled substantial treatment simplification, supporting its role as an effective long-term treatment strategy for pediatric EoE.

PEDIATRICS
Dubai Health Authority (AE), Hospital Sant Joan de Déu Barcelona (ES), University of Ljubljana (SI), Inserm (FR), Tel Aviv University (IL), Université Catholique de Lille (FR), University of Milan (IT), Hebrew University of Jerusalem (IL), University of Campania "Luigi Vanvitelli" (IT), National and Kapodistrian University of Athens (GR), Hadassah Medical Center (IL), Great Ormond Street Hospital (GB), Gelre Hospitals (NL), Ghent University Hospital (BE), Centre Hospitalier Universitaire de Lille (FR), Hôpital Jeanne de Flandre (FR), Hospital Universitario Puerta de Hierro Majadahonda (ES), Schneider Children's Medical Center (IL), Hospital Infantil Universitario Niño Jesús (ES), Meyer Children's Hospital (IT), Children's Hospital Agia Sophia (GR), Bambino Gesù Children's Hospital (IT), University Children's Hospital Zurich (CH), University Hospital Hradec Králové (CZ), Groupe Hospitalier de l'Institut Catholique de Lille (FR), Ospedale dei Bambini Vittore Buzzi (IT), Hospital Universitario Severo Ochoa (ES), Institute for Translational Research in Inflammation (FR), Universidad Autónoma de Madrid (ES), University of Naples Federico II (IT), Sapienza University of Rome (IT)
Good health and well-being
Openalex Percentile: Top 11%
Eosinophilic Esophagitis
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