Budesonide Oral Suspension Improves Outcomes in Patients With Eosinophilic Esophagitis: Results from a Phase 3 Trial.

BACKGROUND & AIMS: Eosinophilic esophagitis (EoE) is a chronic, immune-mediated disease for which there is currently no pharmacologic therapy approved by the US Food and Drug Administration.

METHODS: In this double-blind, placebo-controlled, phase 3 trial, patients aged 11-55 years with EoE and dysphagia were randomized 2:1 to receive budesonide oral suspension (BOS) 2.0 mg b.i.d. or placebo for 12 weeks at academic/community care practices. Co-primary endpoints were the proportion of stringent histologic responders (≤6 eosinophils/high-power field) or dysphagia symptom responders (≥30% reduction in Dysphagia Symptom Questionnaire [DSQ] score) over 12 weeks. Changes in DSQ score (key secondary endpoint) and EoE Endoscopic Reference Score (EREFS) (secondary endpoint) from baseline to week 12, and safety parameters were examined.

RESULTS: Overall, 318 patients (BOS, n=213; placebo, n=105) were randomized and received ≥1 dose of study treatment. More BOS-treated than placebo-treated patients achieved a stringent histologic response (53.1% vs 1.0%; Δ52% [95% confidence interval (CI), 43.3%-59.1%];P<.001) or symptom response (52.6% vs 39.1%; Δ13% [95% CI, 1.6%-24.3%];P=.024) over 12 weeks. BOS-treated patients also had greater improvements in least-squares mean (standard error of the mean) DSQ scores and EREFS over 12 weeks than placebo-treated patients: DSQ, -13.0 (1.2) vs -9.1 (1.5); Δ-3.9 (95% CI, -7.1 to -0.8);P=.015; EREFS, -4.0 (0.3) vs -2.2 (0.4); Δ-1.8 (95% CI, -2.6 to -1.1);P<.001. BOS was well tolerated; most adverse events were mild/moderate in severity.

CONCLUSIONS: In patients with EoE, BOS 2.0 mg b.i.d. was superior to placebo in improving histologic, symptomatic, and endoscopic outcomes over 12 weeks. BOS 2.0 mg b.i.d. was well tolerated. ClinicalTrials.gov number: NCT02605837.