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

Abstract

BACKGROUND & AIMS:Eosinophilic esophagitis (EoE) is a chronic, immune-mediated disease for which there is currently no pharmacologic therapy approved by the U.S. Food and Drug Administration. METHODS:In this double-blind, placebo-controlled, phase 3 trial, patients 11-55 years of age with EoE and dysphagia were randomized 2:1 to receive budesonide oral suspension (BOS) 2.0 mg twice daily or placebo for 12 weeks at academic or 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%; D52% [95% confidence interval (CI), 43.3%-59.1%]; P < .001) or symptom response (52.6% vs 39.1%; D13% [95% CI, 1.6%-24.3%]; P [ .024) over 12 weeks. BOS-treated patients also had greater improvements in least-squares mean DSQ scores

CD73+ Epithelial Progenitor Cells That Contribute to Homeostasis and Renewal Are Depleted in Eosinophilic Esophagitis

“…In our recent report, 3 25% of inactive patients have endoscopic findings, 29.1% had basal zone hyperplasia, and 73.6% had spongiosis, suggesting that there are ongoing and significant epithelial changes while under the current definition of fewer than 15 eos/hpf. There has been some movement in the field to consider pushing down the eosinophil count in this population, and recent clinical trials 45 , 46 are using fewer than 6 eos/hpf in their analysis to account for this.…”

Budesonide Oral Suspension Improves Outcomes in Patients With Eosinophilic Esophagitis

“…Arandomized controlled trial recently compared the clinical efficacy of budesonide oral suspension (BOS) and placebo in patients with eosinophilic esophagitis (EoE). 1 In this study of 318 participants, the authors found that after 12 weeks of treatment, BOS significantly improved histology, symptoms, and endoscopic outcomes in patients with EoE. We sincerely congratulate the authors for their great efforts to provide more evidence for the treatment of patients with EoE.…”

Budesonide orodispersible tablets for induction of remission in patients with active eosinophilic oesophagitis: A 6‐week open‐label trial of the EOS‐2 Programme

“…The particular mode of drug delivery with BOT might explain the high histological remissions rates in contrast to other corticosteroids suspensions which are usually ingested via a single swallow of a relatively large volume. For example, in the recent ORBIT trial, a phase 3 randomized placebo‐controlled trial including 318 adolescent and adult EoE patients, a 12‐week treatment with 2 mg BID of a budesonide oral suspension (10 ml at a concentration of 0.2 mg/ml), specifically developed for treatment of EoE, resulted in a histological remission rate (≤6 eos/hpf) of 53.1% 25 …”

“…For example, in the recent ORBIT trial, a phase 3 randomized placebo-controlled trial including 318 adolescent and adult EoE patients, a 12-week treatment with 2 mg BID of a budesonide oral suspension (10 ml at a concentration of 0.2 mg/ml), specifically developed for treatment of EoE, resulted in a histological remission rate (≤6 eos/hpf) of 53.1%. 25 Obviously, comparison of such trials conducted in different patient populations are not permitted and head-to-head comparisons among different topical corticosteroids are lacking. However based on the available evidence, the latest network meta-analysis 26 concluded that BOT currently appears to be the most effective drug therapy for EoE in adults.…”