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