Researchers Publish Final Results of Key Clinical Trial for Monoclonal Antibody in Young Children with Eosinophilic Esophagitis

An international team of researchers including several at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial that led to the approval of the monoclonal antibody dupilumab for the treatment of pediatric patients between the ages of 1 and 11 years old with eosinophilic esophagitis (EoE) by the U.S. Food and Drug Administration earlier this year.

The findings were recently published in the New England Journal of Medicine, building upon prior work that examined the safety and efficacy of dupilumab for adolescents and young adults with EoE.

EoE a chronic allergic inflammatory disease of the esophagus, and during an allergic reaction, various cells congregate and cause symptoms like redness, swelling and itchiness. Traditional treatments for EoE include food elimination diets, topical glucocorticoids, proton-pump inhibitors and esophageal dilation. However, about a third of patients do not respond to these first-line treatments, and some patients experience negative side effects with them.

Researchers began investigating dupilumab as a potential treatment for EoE after growing evidence suggested type 2 cytokines play a key role in EoE. Dupilumab blocks interleukin-4 and interleukin-13 - two key drivers of inflammation in patients with EoE.

In this study, histologic remission - meaning no inflammation was present - occurred in 25 of 37 (68%) patients who received higher-exposure dupilumab and 18 of 31 (58%) patients receiving lower-exposure dupilumab. Higher-exposure dupilumab significantly improved several clinical outcomes. COVID-19, nausea, injection site pain, and headache occurred with ≥10% higher incidence in patients receiving dupilumab versus placebo.

"This manuscript and the related FDA approval finally provides a treatment option for our patients," said study co-author Jonathan Spergel, MD, PhD, Chief of the Allergy Program at CHOP. "We are excited for our patients and new potential benefits for them."

This study was supported by Sanofi and Regeneron Pharmaceuticals Inc.

Chehade et al, "Dupilumab for Eosinophilic Esophagitis in Patients Aged 1 to 11 Years." N Engl J Med. Online June 27, 2024. DOI: TK.