Children achieve remission in eosinophilic esophagitis with dupilumab

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Key takeaways:

  • The FDA approved dupilumab for eosinophilic esophagitis in children in January.
  • Patients achieved improvements in histologic, endoscopic and transcriptomic measures.
  • Adverse events were mild to moderate.

There were significant differences in percentages of children with eosinophilic esophagitis who experienced histologic remission with dupilumab compared with placebo, according to a study published in The New England Journal of Medicine.

Children treated with dupilumab also experienced improvements in seven other endpoints, Mirna Chehade, MD, MPH, professor of pediatrics and medicine at Icahn School of Medicine at Mount Sinai and founding director of the Mount Sinai Center for Eosinophilic Disorders, and colleagues wrote.

Data were derived from Chehade M, et al. NJEM. 2024;doi:10.1056/NEJMoa2312282.

EoE is a chronic disease of the esophagus associated with a type 2 allergic inflammation, Chehade told Healio.

Mirna Chehade

“Children with EoE can suffer from multiple symptoms including abdominal pain, vomiting, regurgitation, trouble swallowing and food impaction,” she said. “In addition, when EoE affects children at a young age, it can severely impact their ability to eat and can cause failure to thrive at a critical time of their growth and development.”

In January, the FDA approved dupilumab (Dupixent; Sanofi, Regeneron) to treat children with EoE aged 1 to 11 years who weigh at least 15 kg. As a fully human monoclonal antibody, dupilumab blocks IL-4 and IL-13 pathways to inhibit type 2 inflammation.

“Before dupilumab, there were no treatments approved specifically for EoE in this age group,” Chehade said. “Now, with dupilumab, parents/caregivers have an FDA-approved biologic treatment option that targets key drivers of the type 2 allergic inflammation that contributes to this disease.”

Study design, results

The study included 102 children aged 1 to 11 years (mean age, 7.1 years; 76% boys; 82% white) with active EoE, defined as a peak esophageal intraepithelial eosinophil count of 15 or more per high-power field in at least two of three esophageal regions, along with a lack of response to proton-pump inhibitors.

The three-part trial began with part A, which included 37 patients assigned to higher exposures of dupilumab (mean age, 6.8 years; 76% boys; 86% white), 31 assigned to lower exposures (mean age, 7.2 years; 81% boys; 71% white) and 34 assigned to placebo (mean age, 7.2 years; 74% boys; 84% white) for 16 weeks.

In part B, which ran from week 17 to week 52, the 37 patients (100%) on higher exposure in part A continued this regimen, and 29 patients (94%) who were in the lower exposure group in part A continued with that regimen. Also, 18 patients (53%) in the placebo group switched to a higher exposure regimen, and 14 (41%) switched to lower exposure.

“We were able to demonstrate not only reduction of esophageal eosinophil counts when using dupilumab, but also significant improvements in histologic, endoscopic, and transcriptomic measures as compared with placebo,” Chehade said.

At week 16, 68% (n = 25) of the higher-exposure group, 58% (n = 18) of the lower-exposure group and 3% (n = 1) of the placebo group experienced histologic remission (P < .001), defined as a peak esophageal intraepithelial eosinophil count of 6 or less per high-power field.

There also were significant differences between the higher-exposure and placebo groups at week 16 in seven secondary endpoints across histologic (peak esophageal intraepithelial eosinophil count and EoE histology scoring system grade and stage scores), transcriptomic (type 2 inflammation and EoE diagnostic panel gene signatures) and endoscopic (total EoE Reference Score) categories.

“These improvements were maintained when children were evaluated after a full year of receiving dupilumab,” Chehade said.

Patients who continued the same dupilumab treatment from part A into part B experienced improvements in histologic and endoscopic measures that were similar to those seen between baseline and week 16, the researchers said.

The patients who switched from placebo to dupilumab between parts A and B saw improvements that were similar to the improvements experienced by the dupilumab groups between baseline and week 16.

Compared with the placebo group, esophageal gene-expression profiles of two gene sets that are associated with active EoE — the type 2 inflammation set and the EoE diagnostic panel set — also saw significant improvement among patients in the higher-exposure group between baseline and week 16, the researchers said.

Specifically, the higher-exposure group had significantly lower normalized enrichment scores for the relative change between baseline and week 16 in the gene-expression signatures of these sets than the placebo group.

The higher-exposure group had mean serum concentrations of dupilumab at weeks 16 and 52 that were approximately twice as high as those of the lower-exposure group, the researchers said.

Also, one patient each in the higher-exposure group and placebo group had antibodies to dupilumab in part A. The patient from the higher-exposure group had relatively low levels of antibodies, the researchers said, with no apparent effect on the biologic’s efficacy. No antibodies to dupilumab were detected in any other patients.

Adverse event incidence ranged from 73% to 100% across the study groups and parts. Most were mild or moderate. Three patients discontinued treatment because of adverse events, but these events were not considered serious.

Nine patients reported serious events in parts A and B, but none of them were related to dupilumab or placebo, and none led to death.


Overall, the researchers said, these findings indicate significantly higher rates of remission with dupilumab among children aged 1 to 11 years through 52 weeks compared with placebo, as well as other histologic, endoscopic and transcriptomic improvements with a tolerable safety profile.

Chehade also said that these results led to the FDA’s approval for the use of dupilumab to treat EoE in this age group.

“I am excited because dupilumab has the potential to transform the standard of care for many young children living with EoE,” she said.

By demonstrating that dupilumab can significantly reduce esophageal inflammation and possibly improve symptoms, Chehade said that these findings also bring hope to children with EoE and their caregivers at a critical time of their growth and development.

“Controlling the inflammation in EoE also has the potential to reduce the chance of long-term complications that patients can develop over time if their EoE is left untreated or inadequately treated,” Chehade said.

The open-label extension of the ongoing trial will run through 2 years to enable the researchers to evaluate longer-term outcomes in children with EoE, Chehade said.

For more information:

Mirna Chehade, MD, MPH, can be reached at

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