Dupilumab in Eosinophilic Gastrointestinal Disorders With Extraesophageal Involvement: A Pediatric Case Series and...

Dupilumab has proven effective in the treatment of eosinophilic esophagitis (EoE) in randomized controlled trials. Its efficacy in other, less common and generally more severe eosinophilic gastrointestinal disorders (EGIDs) is scientifically plausible, although it has not been studied to date. Our objective was to describe published and our experience on the empirical use of dupilumab in patients affected by EGIDs with extraesophageal involvement. We retrospectively analyzed the medical charts of children diagnosed with extraesophageal EGIDs treated with dupilumab at our tertiary medical center. The Medline, Embase, and Cochrane databases were searched up to January 2025 for articles describing the use of dupilumab in patients with diagnosed or suspected extraesophageal EGID. Our cohort included 8 patients with a clinical and histological diagnosis of EGID and extraesophageal involvement. All of them had recurrent gastrointestinal symptoms refractory to standard treatments. Three patients had growth retardation. In all patients, symptoms and macroscopic and histological abnormalities, including eosinophilic infiltration, quickly improved after initiation of dupilumab. The systematic review identified 11 case reports (n=29 patients) of pediatric and adult patients with extraesophageal EGIDs treated with dupilumab. All 11 reports described significant clinical and histological improvement following therapy. Clinical experience suggests that dupilumab is effective in treating pediatric EGIDs with extraesophageal features. Given the rarity and high morbidity of these disorders, dupilumab could be considered a reasonable option while waiting for high-quality evidence from ongoing randomized controlled trials.