Efficacy and safety of long-term treatment with budesonide orodispersible tablets in patients with EoE: real-life data from a single-centre experience

Aims Swallowed topical steroids (STCs), particularly budesonide orodispersible tablets (BOT), are a cornerstone in treating eosinophilic esophagitis (EoE). While clinical trials have shown high efficacy, real-world data on the long-term effectiveness and safety of BOT remain limited. This single-center study aims to evaluate the efficacy and safety of BOT during induction and long-term maintenance therapy in EoE patients.

Methods We prospectively enrolled EoE patients who started induction tratment with BOT (2 mg) between 2021-2024 and completed at least 1 year of maintenance therapy (1 mg). Histological remission, defined as an eosinophil count of<15 per high-power field (HPF) in esophageal biopsies, was assessed at the end of induction (T1) and after 1 year of maintenance (T2). BOT-related adverse events were also documented at T1 and T2.

Results 126 EoE patients are currently receiving BOT treatment at our center. Of these, 56 completed at least 1 year of follow-up on maintenance therapy and were therefore included in this analysis. The median age at diagnosis was 29 years, 85.7% of patients were male. Patients presented with dysphagia (89.3%) and bolus impaction (62.5%). At diagnosis, 53.6% had inflammatory and 46.4% fibrostenotic disease phenotypes. 55% of patients had a history of gastroesophageal reflux disease (GERD) symptoms at diagnosis and 1.7% had evidence of erosive esophagitis (EE) prior to starting BOT therapy. 12.5% of patients started BOT as first-line therapy, 30.3% after switching from off-label STCs and 57.1% after failure of previous therapy with proton pump inhibitors (PPIs). 39.2% of patients started BOT as monotherapy, 60.7% started combined BOT and PPI therapy. At T1, 80.4% of patients presented histological remission; at T2, 25% of patients lost histological response to BOT therapy. The prevalence of EE was 17% at T1 and 15.4% at T2. The lack of histological response at T1 and loss of histological remission at T2 was tendentially associated with the presence of esophageal stenosis (p=0.067 and p=0.067, respectively). Failure to induction treatment with BOT (T1) was significantly associated with the presence of EE (p=0.021) and BOT monotherapy (p=0.027). Similarly, loss of response at T2 was significantly associated with BOT monotherapy (p=0.012). Adverse events during induction were described in 28.6% of patients, including oral (10.7%) and esophageal candidiasis (10.7%), oral burning (3.7%) and dysgeusia (1.8%). During maintenance, 16.1% patients had adverse events. Recurrence of adverse events led to discontinuation of BOT in 3.6% of patients during the induction and maintenance phases, but was not associated with treatment failure at either T1 or T2.

Conclusions BOT therapy effectively induces and maintains histological remission in EoE,. Our results suggest a possible association between the occurrence of erosive esophagitis and the lack of response to induction with BOT. Therefore, we highlight the potential role of BOT-PPIs combination therapy to achieve a higher histological response rate during induction and a better long-term efficacy in those patients at high risk of developing erosive esophagitis.

Publication History

Article published online:
27 March 2025