Dupilumab in Pediatric Eosinophilic Esophagitis
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.
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Authors: Giusy Russo, Ali Alsarhan, Mordechai Slae, Sraya Greenberger, Carolina Gutiérrez‐Junquera, Nicolas Kalach, Cosimo Ruggiero, Lorenzo Norsa, Francesca Rea, Víctor Vila-Miravet, Sara Renzo, Danila Volpe, Matjaž Homan, Vasiliki Spyropoulou, Johanna H. Oudshoorn, Eleftheria S. Roma, Gloria Domínguez Ortega, Sonia Fernández Fernández, Saskia Vande Velde, Giovanna Alfano, Alexandra Papadopoulou, Jan Melek, Erasmo Miele, Léa Chantal Tran, Caterina Strisciuglio, Noam Zevit, Christos Tzivinikos, Salvatore Oliva
Institutions: Inserm, Hospital Universitario Puerta de Hierro Majadahonda, Ghent University Hospital, Universidad Autónoma de Madrid, Great Ormond Street Hospital, Hebrew University of Jerusalem, Tel Aviv University, Sapienza University of Rome, University of Campania "Luigi Vanvitelli", University of Naples Federico II, University of Milan, University of Ljubljana, Centre Hospitalier Universitaire de Lille, National and Kapodistrian University of Athens, Meyer Children's Hospital, Schneider Children's Medical Center, Hadassah Medical Center, Ospedale dei Bambini Vittore Buzzi, Bambino Gesù Children's Hospital, Hospital Universitario Severo Ochoa, Dubai Health Authority, Hôpital Jeanne de Flandre, Children's Hospital Agia Sophia, Hospital Infantil Universitario Niño Jesús, Hospital Sant Joan de Déu Barcelona, University Children's Hospital Zurich, University Hospital Hradec Králové, Université Catholique de Lille, Groupe Hospitalier de l'Institut Catholique de Lille, Gelre Hospitals, Institute for Translational Research in Inflammation