Real-world-data on efficacy and safety of topical budesonide therapy (orodispersible tablet) in induction and maintenance therapy in adult patients with active eosinophilic esophagitis

Background: Eosinophilic esophagitis (EoE) is a chronic, progressive, immune-mediated disease of the esophagus. Esophageal dysfunction in solid food dysphagia combined with eosinophil-dominant inflammation (⩾15 eos/high-power-field (hpf)) serves as diagnostic criteria. Treatment objectives for active EoE include induction and maintenance of clinical-histological remission. Topical budesonide therapy in the form of an orodispersible tablet (BUD-ODT) has shown effectiveness in both scenarios. Objectives: To examine the efficacy and safety of BUD-ODT therapy in adults with active EoE in a Real-World-Setting. Design: This retrospective study from two German EoE centers examined the efficacy and safety of BUD-ODT therapy in adults with active EoE between June 2018 and August 2022. Methods: Patient demographics, clinical characteristics, and EoE history were extracted from medical records. Evaluations included clinical (Straumann Dysphagia Index, range: 0–9, remission: ⩽3 points), histological (remission: ⩽15 eos/hpf), and endoscopic assessment (endoscopic reference scoring system (EREFS) score, range: 0–9, remission: ⩽2 points). Three periods were analyzed: Induction phase (induction therapy (IT): 1 mg BID, 6–12 weeks; n = 201), maintenance phase I (RM1: 0.5 mg BID or 1 mg QD, 14–24 weeks; n = 109), and maintenance phase II (RM2: same dosage as RM1, 52–104 weeks; n = 72). Results: A total of 221 adults with clinical-histologically proven active EoE were included. Clinical-histological remission was achieved in 75.1% (151/201) of patients after IT, maintained in 73.4% (80/109) after RM1, and in 76.4% (55/72) after RM2. The relapse rate was 26.6% (29/109) after RM1 and 23.6% (17/72) after RM2. Those patients underwent re-induction therapy for 6–8 weeks and were re-evaluated separately (remission: 36.0% after RM1 and 46.2% after RM2). Endoscopic remission was demonstrated in 88.1% (177/201) after IT. The most prevalent side effect was endoscopically detected combined oral-esophageal candidiasis. Conclusion: BUD-ODT demonstrated high effectiveness in inducing and maintaining remission in a real-world setting. The spectrum of side effects did not reveal any new aspects compared to known clinical data.