Center for Esophageal Diseases and Swallowing, Division of Gastroenterology and Hepatology, Department of Medicine, University of North Carolina, Chapel Hill, North Carolina.
Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, Ohio.
Clin Gastroenterol Hepatol. 2021 Apr;19(4):699-706.e4. doi: 10.1016/j.cgh.2020.03.060. Epub 2020 Apr 6.
BACKGROUND & AIMS: Quantification of eosinophilic esophagitis (EoE) symptoms is crucial for assessing treatment outcomes. We aimed to explore the effect of budesonide oral suspension (BOS) on dysphagia and pain with swallowing.
We performed a secondary analysis of data from a phase 2 multicenter, double-blind, trial (conducted from July 2012 through October 2014) of patients with EoE, 11-40 y old, who were randomly assigned to groups given placebo or BOS (2.0 mg twice daily) for 12 weeks. Symptoms were quantified using the Dysphagia Symptom Questionnaire (DSQ) from baseline to week 12 of therapy.
Overall, 93 patients were randomly assigned to groups; the prespecified modified intention-to-treat analysis set comprised 87 patients (38 from the placebo group and 49 from the BOS group). Improvements from baseline in least-squares mean (standard error) DSQ (Q2+Q3) scores were observed. The difference between groups was statistically significant only at week 12 (placebo vs BOS: week 4, -4.9 [1.7] vs -7.4 [1.5]; P = .265; week 8, -7.4 [2.1] vs -10.3 [1.8]; P = .288; week 12, -7.5 [1.9] vs -14.3 [1.7]; P = .01). Similar findings were observed for pain (Q4) scores (placebo vs BOS: week 4, -2.5 [0.8] vs -3.3 [0.7]; P = .484; week 8, -3.0 [0.8] vs -4.9 [0.7]; P = .066; week 12, -3.1 [0.8] vs -4.9 [0.7]; P = .109). More severe DSQ and DSQ+pain scores were associated with presence of other symptoms (such as regurgitation) and physician-rated severity. Improvements in DSQ and DSQ+pain scores were greater in patients with either a histologic or endoscopic response than in patients without a response.
In a secondary analysis of data from a phase 2 trial of patients with EoE, we found evidence for improvements in dysphagia and pain scores in patients who received BOS (2.0 mg twice daily) vs placebo. Pain with swallowing should be considered in the clinical assessment of patients with EoE. ClinicalTrials.gov no: NCT01642212.
量化嗜酸性食管炎（EoE）的症状对于评估治疗结果至关重要。我们旨在探讨布地奈德混悬液（BOS）对吞咽困难和吞咽疼痛的影响。
我们对一项为期 12 周、多中心、双盲、2 期临床试验（2012 年 7 月至 2014 年 10 月进行）的数据进行了二次分析，纳入了年龄为 11-40 岁、患有 EoE 的患者，这些患者被随机分为安慰剂组或 BOS（2.0 mg，每日两次）组。使用吞咽困难症状问卷（DSQ）从基线到治疗第 12 周来量化症状。
总体而言，93 例患者被随机分配到各个组；预设的改良意向治疗分析集包括 87 例患者（安慰剂组 38 例，BOS 组 49 例）。从基线开始，最小二乘均值（标准误差）DSQ（Q2+Q3）评分均有所改善。仅在第 12 周时观察到组间差异具有统计学意义（安慰剂 vs BOS：第 4 周，-4.9[1.7] vs-7.4[1.5]；P=0.265；第 8 周，-7.4[2.1] vs-10.3[1.8]；P=0.288；第 12 周，-7.5[1.9] vs-14.3[1.7]；P=0.01）。疼痛（Q4）评分也存在类似的发现（安慰剂 vs BOS：第 4 周，-2.5[0.8] vs-3.3[0.7]；P=0.484；第 8 周，-3.0[0.8] vs-4.9[0.7]；P=0.066；第 12 周，-3.1[0.8] vs-4.9[0.7]；P=0.109）。DSQ 和 DSQ+疼痛评分更严重与其他症状（如反流）的存在和医生评估的严重程度有关。与无反应的患者相比，在有组织学或内镜反应的患者中，DSQ 和 DSQ+疼痛评分的改善更大。
在对 EoE 患者 2 期试验数据的二次分析中，我们发现接受 BOS（2.0 mg，每日两次）治疗的患者的吞咽困难和疼痛评分有改善，而接受安慰剂治疗的患者则没有改善。吞咽疼痛应在 EoE 患者的临床评估中考虑。临床试验编号：NCT01642212。
