Efficacy and Safety of Budesonide Orodispersible Tablets for Eosinophilic Esophagitis up to 3 Years: an Open-Label Extension Study.

Abstract

Background and aims: Budesonide orodispersible tablets (BOT) have been shown to be safe and effective in phase 3 double-blind trials of induction and 48-week maintenance therapy of eosinophilic esophagitis (EoE). We now analyzed the long-term efficacy and safety of BOT in a 96-week open-label extension (OLE) study.

Methods: All EoE patients in the 48-week double-blind maintenance study were eligible to receive BOT treatment for up to 96 weeks. Dosage was 0.5 or 1.0 mg BOT, twice daily, at investigator's discretion. Clinical, histologic, endoscopic, quality of life, and safety measures were assessed.

Results: A total of 186 patients participated in the OLE up to 96 weeks. At week 96, 81.9% of patients had clinical remission, defined as an EoE Symptom Activity Index (EEsAI) score of ≤ 20, versus 77.7% at OLE baseline. A further 80.1% of patients were in histologic remission, defined as peak eosinophils per high-power field of <5, at week 96 versus 91.8% at OLE baseline. Mean EoE endoscopic reference scores (EREFS) were 1 at all time points measured. Mean EoE Quality of Life (EoE-QoL-A) Scale scores improved from 3.3 at OLE baseline to 3.5 at week 96. No new safety concerns were observed across 96 weeks of treatment. Suspected symptomatic candidiasis occurred at similar rates to prior BOT studies, and was predominantly mild and resolved with treatment.

Conclusions: Clinical and histological remission of EoE could be maintained with BOT in a large majority of patients for up to 96 weeks, and for up to 144 weeks in patients with uninterrupted BOT therapy across all trials. No additional safety concerns were identified with long-term BOT treatment. (ClinicalTrials.gov, Number: NCT02493335).