Division of Gastroenterology and Hepatology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio.
Clin Gastroenterol Hepatol. 2022 Mar;20(3):525-534.e10. doi: 10.1016/j.cgh.2021.04.022. Epub 2021 Apr 19.
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.
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.
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.
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.
嗜酸性食管炎（EoE）是一种慢性、免疫介导的疾病，目前尚无获得美国食品和药物管理局批准的药物治疗方法。
在这项双盲、安慰剂对照、3 期临床试验中，年龄在 11-55 岁之间、患有 EoE 和吞咽困难的患者以 2:1 的比例随机分配，接受布地奈德混悬液（BOS）2.0mg，每日两次或安慰剂，治疗 12 周，在学术或社区护理机构进行。主要共同终点是在 12 周时严格组织学应答者（≤6 个嗜酸性粒细胞/高倍视野）或吞咽困难症状应答者（Dysphagia Symptom Questionnaire [DSQ] 评分至少降低 30%）的比例。从基线到第 12 周，检查 DSQ 评分（关键次要终点）和 EoE 内镜参考评分（EREFS）（次要终点）的变化以及安全性参数。
总体而言，318 名患者（BOS，n=213；安慰剂，n=105）被随机分配并接受了至少一剂研究治疗。与安慰剂治疗相比，接受 BOS 治疗的患者更能达到严格的组织学应答（53.1%比 1.0%；Δ52%[95%置信区间（CI），43.3%-59.1%]；P<.001）或症状应答（52.6%比 39.1%；Δ13%[95% CI，1.6%-24.3%]；P=0.024）在 12 周内。与安慰剂治疗相比，BOS 治疗的患者在 12 周内的 DSQ 评分和 EREFS 也有更大的改善：DSQ，-13.0（SEM 1.2）比-9.1（SEM 1.5）（Δ-3.9[95% CI，-7.1 至-0.8]；P=.015）；EREFS，-4.0（SEM 0.3）比-2.2（SEM 0.4）（Δ-1.8[95% CI，-2.6 至-1.1]；P<.001）。BOS 耐受性良好；大多数不良事件的严重程度为轻度或中度。
在 EoE 患者中，BOS 2.0mg，每日两次，在改善组织学、症状和内镜结果方面优于安慰剂，持续 12 周。BOS 2.0mg，每日两次，耐受性良好。临床试验注册号：NCT02605837。
