Dupilumab in Pediatric Eosinophilic Esophagitis

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.

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.

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.

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.