Dupilumab Demonstrates Benefit in Pediatric Eosinophilic Esophagitis Trial

Positive results were announced from a phase 3 study evaluating the efficacy and safety of dupilumab in patients 1 to 11 years of age with eosinophilic esophagitis (EoE).

Dupilumab, an interleukin-4 receptor alpha antagonist, is currently marketed under the brand name Dupixent^® and is indicated for the treatment of eosinophilic esophagitis in patients 12 years of age and older weighing at least 40kg; for the treatment of moderate to severe atopic dermatitis in patients 6 years of age and older; for maintenance treatment of severe asthma in patients 12 years of age and older; and for chronic rhinosinusitis with nasal polyps in patients 18 years of age and older.

The randomized, double-blind, placebo-controlled EoE KIDS study (ClinicalTrials.gov Identifier: NCT04394351) included 102 patients 1 to 11 years of age with active EoE based on histologic improvement meeting validated histologic criteria. Patients were randomly assigned to receive dupilumab, at either a higher dose (n=37) or lower dose (n=31) based on body weight, or placebo (n=34). The primary endpoint was histological disease remission, defined as the proportion of patients achieving peak esophageal intraepithelial eosinophil count ≤6 eosinophils [eos]/high power field [hpf]) at week 16.

Findings showed that 68% and 58% of patients treated with the higher and lower dose of dupilumab, respectively, achieved significant histological disease remission at week 16 compared with 3% of those who received placebo (P <.0001 for both). Moreover, patients treated with the higher dose of dupilumab achieved the following at week 16:

A 86% reduction in peak esophageal intraepithelial eosinophil count from baseline vs a 21% increase for placebo (P <.0001).
A 0.88 and 0.84 reduction from baseline in disease severity and extent, respectively, as measured at the microscopic level in biopsy specimens vs a 0.02 and 0.05 increase for placebo (both P <.0001).
A 3.5-point reduction in abnormal endoscopic findings from baseline vs a 0.3-point increase for placebo (P <.0001).

The safety profile of dupilumab in patients 1 to 11 years of age was generally consistent with that seen in patients aged 12 years and older. Adverse events more commonly observed with dupilumab compared with placebo included COVID-19 (21% dupilumab, 0% placebo; all cases were mild or moderate), rash (9% dupilumab, 6% placebo), headache (8% dupilumab, 3% placebo), viral gastroenteritis (6% dupilumab, 3% placebo), diarrhea (6% dupilumab, 3% placebo), and nausea (6% dupilumab, 0% placebo).

Want to read more?