Efficacy And Safety Of Budesonide Oral Suspension In A Pediatric Population: Pooled Data From A Phase 2 And Phase 3 Trial In Patients With Eosinophilic Esophagitis

To evaluate efficacy and safety of budesonide oral suspension (BOS) in pediatric patients with eosinophilic esophagitis (EoE). We used pooled data from two 12-week, randomized, double-blind, placebo-controlled trials of BOS 2.0 mg b.i.d. (phase 2 [MPI 101-06/NCT01642212] and phase 3 [ORBIT1/SHP621-301/NCT02605837]) in patients aged 11–17 years with EoE and dysphagia. Peak eosinophil counts, the Dysphagia Symptom Questionnaire (DSQ) and EoE Endoscopic Reference Score (EREFS) assessed histologic, symptomatic and endoscopic outcomes, respectively. Overall, 76 patients received ≥ 1 dose of study drug (BOS 2.0 mg b.i.d. [n = 45] or placebo [n = 31]). Significantly more BOS- than placebo-treated patients had histologic responses (≤ 6 eosinophils/high-power field [eos/hpf], 46.7% vs 6.5%; < 15 eos/hpf, 53.3% vs 9.7%; ≤ 1 eos/hpf, 42.2% vs 0.0%; all, p < 0.001) after 12 weeks of therapy. More BOS- than placebo-treated patients had symptom responses (≥ 30% reduction in DSQ score: 68.9% vs 58.1%; p = 0.314) after 12 weeks of therapy. Significantly more BOS- than placebo-treated patients had a combined response (≤ 6 eos/hpf and ≥ 30% reduction in DSQ score: 31.1% vs 3.2%; p = 0.003) after 12 weeks of therapy. BOS-treated patients had significantly greater reductions in least-squares mean EREFS (−4.1 vs −2.1; p = 0.021) from baseline to week 12 than placebo. BOS was generally well tolerated, with no significant differences in adverse events versus placebo. BOS significantly improved histologic, endoscopic and combined (histologic and symptomatic) outcomes in pediatric patients with EoE versus placebo.