TY - JOUR
T1 - Budesonide Oral Suspension Improves Outcomes in Patients With Eosinophilic Esophagitis
T2 - Results from a Phase 3 Trial
AU - ORBIT1/SHP621-301 Investigators
AU - Hirano, Ikuo
AU - Collins, Margaret H.
AU - Katzka, David A.
AU - Mukkada, Vincent A.
AU - Falk, Gary W.
AU - Morey, Robin
AU - Desai, Nirav K.
AU - Lan, Lan
AU - Williams, James
AU - Dellon, Evan S.
N1 - Publisher Copyright:
© 2022 The Authors
PY - 2022/3
Y1 - 2022/3
N2 - Background & Aims: Eosinophilic esophagitis (EoE) is a chronic, immune-mediated disease for which there is currently no pharmacologic therapy approved by the US Food and Drug Administration. Methods: In this double-blind, placebo-controlled, phase 3 trial, patients 11–55 years of age with EoE and dysphagia were randomized 2:1 to receive budesonide oral suspension (BOS) 2.0 mg twice daily or placebo for 12 weeks at academic or community care practices. Co-primary endpoints were the proportion of stringent histologic responders (≤6 eosinophils/high-power field) or dysphagia symptom responders (≥30% reduction in Dysphagia Symptom Questionnaire [DSQ] score) over 12 weeks. Changes in DSQ score (key secondary endpoint) and EoE Endoscopic Reference Score (EREFS) (secondary endpoint) from baseline to week 12, and safety parameters were examined. Results: Overall, 318 patients (BOS, n = 213; placebo, n = 105) were randomized and received ≥1 dose of study treatment. More BOS-treated than placebo-treated patients achieved a stringent histologic response (53.1% vs 1.0%; Δ52% [95% confidence interval (CI), 43.3%–59.1%]; P <.001) or symptom response (52.6% vs 39.1%; Δ13% [95% CI, 1.6%–24.3%]; P =.024) over 12 weeks. BOS-treated patients also had greater improvements in least-squares mean DSQ scores and EREFS over 12 weeks than placebo-treated patients: DSQ, –13.0 (SEM 1.2) vs –9.1 (SEM 1.5) (Δ–3.9 [95% CI, –7.1 to –0.8]; P =.015); EREFS, –4.0 (SEM 0.3) vs –2.2 (SEM 0.4) (Δ–1.8 [95% CI, –2.6 to –1.1]; P <.001). BOS was well tolerated; most adverse events were mild or moderate in severity. Conclusions: In patients with EoE, BOS 2.0 mg twice daily was superior to placebo in improving histologic, symptomatic, and endoscopic outcomes over 12 weeks. BOS 2.0 mg twice daily was well tolerated. ClinicalTrials.gov number: NCT02605837.
AB - Background & Aims: Eosinophilic esophagitis (EoE) is a chronic, immune-mediated disease for which there is currently no pharmacologic therapy approved by the US Food and Drug Administration. Methods: In this double-blind, placebo-controlled, phase 3 trial, patients 11–55 years of age with EoE and dysphagia were randomized 2:1 to receive budesonide oral suspension (BOS) 2.0 mg twice daily or placebo for 12 weeks at academic or community care practices. Co-primary endpoints were the proportion of stringent histologic responders (≤6 eosinophils/high-power field) or dysphagia symptom responders (≥30% reduction in Dysphagia Symptom Questionnaire [DSQ] score) over 12 weeks. Changes in DSQ score (key secondary endpoint) and EoE Endoscopic Reference Score (EREFS) (secondary endpoint) from baseline to week 12, and safety parameters were examined. Results: Overall, 318 patients (BOS, n = 213; placebo, n = 105) were randomized and received ≥1 dose of study treatment. More BOS-treated than placebo-treated patients achieved a stringent histologic response (53.1% vs 1.0%; Δ52% [95% confidence interval (CI), 43.3%–59.1%]; P <.001) or symptom response (52.6% vs 39.1%; Δ13% [95% CI, 1.6%–24.3%]; P =.024) over 12 weeks. BOS-treated patients also had greater improvements in least-squares mean DSQ scores and EREFS over 12 weeks than placebo-treated patients: DSQ, –13.0 (SEM 1.2) vs –9.1 (SEM 1.5) (Δ–3.9 [95% CI, –7.1 to –0.8]; P =.015); EREFS, –4.0 (SEM 0.3) vs –2.2 (SEM 0.4) (Δ–1.8 [95% CI, –2.6 to –1.1]; P <.001). BOS was well tolerated; most adverse events were mild or moderate in severity. Conclusions: In patients with EoE, BOS 2.0 mg twice daily was superior to placebo in improving histologic, symptomatic, and endoscopic outcomes over 12 weeks. BOS 2.0 mg twice daily was well tolerated. ClinicalTrials.gov number: NCT02605837.
KW - Dysphagia
KW - Esophageal Eosinophilia
KW - Placebo-Controlled Trial
KW - Topical Corticosteroid
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UR - http://www.scopus.com/inward/citedby.url?scp=85108948957&partnerID=8YFLogxK
U2 - 10.1016/j.cgh.2021.04.022
DO - 10.1016/j.cgh.2021.04.022
M3 - Article
C2 - 33887475
AN - SCOPUS:85108948957
SN - 1542-3565
VL - 20
SP - 525-534.e10
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
IS - 3
ER -