Esophagus to Small Intestine
Clin Gastroenterol Hepatol. 2022;20(3):525–34.e10
Budesonide oral suspension improves outcomes in patients with eosinophilic esophagitis: Results from a phase 3 trial
Background and 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 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 end points 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 end point) and EoE Endoscopic Reference Score (EREFS) (secondary end point) 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 < 0.001) or symptom response (52.6% vs. 39.1%; Δ13% [95% CI: 1.6–24.3%]; p = 0.024) over 12 weeks. BOS-treated patients also had greater improvements in least-squares mean DSQ scores and EREFS over 12 weeks than placebo-treated patients: DSQ, -13.0 (standard error of the mean [SEM] 1.2) versus -9.1 (SEM 1.5) (Δ-3.9 [95% CI: -7.1 to -0.8]; p = 0.015); EREFS, -4.0 (SEM 0.3) versus -2.2 (SEM 0.4) (Δ-1.8 [95% CI: -2.6 to -1.1]; p < 0.001). BOS was well tolerated; most adverse events were mild or moderate in severity.
Conclusions: In patients with eosinophilic esophagitis, budesonide oral suspension (BOS) 2.0 mg twice daily was superior to placebo in improving histologic, symptomatic, and endoscopic outcomes over 12 weeks. BOS 2.0 mg twice daily was well tolerated.