Long-Term Efficacy and Safety of Budesonide Orodispersible Tablets in Eosinophilic Esophagitis

The long-term efficacy and safety of budesonide orodispersible tablets (BOT) in treating eosinophilic esophagitis (EoE) have been evaluated in a comprehensive study, extending up to 96 weeks after an initial 48-week double-blind trial. Below is a detailed summary of the findings:

### **1. Chronic Nature of EoE and Need for Long-Term Treatment**

Eosinophilic esophagitis is a chronic, immune-mediated disease that requires sustained maintenance therapy to prevent relapse. Previous clinical trials for EoE were generally limited to one year or less, leaving uncertainty about the long-term effectiveness and safety of treatments. This study provides critical insights into the prolonged use of BOT in managing EoE.

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### **2. Study Design: Open-Label Extension**

• **Participants:** 186 adults with EoE who had completed the initial 48-week trial were enrolled in the open-label extension phase.
• **Duration:** The extension phase lasted for an additional 96 weeks, providing a total treatment period of up to 144 weeks.
• **Dosing Strategy:** Patients received either 0.5 mg or 1.0 mg of BOT twice daily, with dosing tailored based on clinical judgment, mimicking real-world practice.

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### **3. Long-Term Efficacy**

#### **Clinical Remission:**

• Sustained clinical remission was observed in over 80% of patients throughout the 96-week open-label treatment phase.
• Dysphagia (difficulty swallowing) and odynophagia (painful swallowing) scores remained consistently near zero across all follow-up visits, indicating stable symptom control.

#### **Histologic Remission:**

• Approximately 80% of patients achieved histologic remission at week 96, defined as ≤6 eosinophils per high-power field (hpf) in esophageal biopsies.
• Nearly four-fifths of patients demonstrated a deep histologic response, with complete absence of eosinophils in their biopsies.

#### **Endoscopic Stability:**

• Endoscopic Reference Scores remained minimal over the long-term follow-up, showing persistent control of inflammatory and fibrotic features in the esophagus.

#### **Clinico-Histologic Remission:**

• More than three-quarters of patients achieved combined clinical and histologic remission by the end of the 96-week extension phase.

#### **Quality of Life Improvement:**

• EoE-specific quality-of-life scores improved steadily over time with continued BOT therapy, reflecting enhanced patient well-being.

#### **Low Relapse Rates:**

• Symptomatic and histologic relapse occurred infrequently and was typically transient.
• Patients who experienced relapse during the earlier blinded phase of the trial frequently regained remission during the open-label extension with reinduction therapy.

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### **4. Safety Profile**

#### **Adverse Events:**

• The safety profile of BOT remained consistent throughout the study, with no evidence of cumulative toxicity.
• The most common adverse events were oral and esophageal candidiasis, which were generally mild and successfully treated with antifungal medications.

#### **Adrenal Function:**

• Morning cortisol levels remained stable, and no clinically significant adrenal insufficiency was observed, indicating that BOT does not cause adrenal suppression.

#### **No Progression to Fibrostenotic Disease:**

• Long-term BOT therapy prevented the progression of EoE to fibrostenotic disease, eliminating the need for endoscopic dilation procedures.

#### **Patient Satisfaction:**

• Nearly all patients reported being satisfied or extremely satisfied with their long-term treatment experience using BOT.

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### **5. Disease-Modifying Potential**

The study findings suggest that budesonide orodispersible tablets may have disease-modifying potential in EoE. Sustained clinical, histologic, and endoscopic control over nearly two years indicates that BOT can effectively manage the disease course and prevent long-term complications.

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### **6. Conclusion**

The long-term use of budesonide orodispersible tablets demonstrates robust efficacy and safety in managing eosinophilic esophagitis. The treatment maintained clinical remission, histologic response, and endoscopic stability while improving patient quality of life and minimizing relapse rates. The consistent safety profile, including the absence of adrenal suppression or progression to fibrostenotic disease, underscores the suitability of BOT as a long-term maintenance therapy for EoE.