Background & Aims

Topical steroids, proton pump inhibitors (PPIs), and dietary interventions are recommended first- and second-line therapies for eosinophilic esophagitis (EoE). We investigated differences in their effectiveness in a real-world, clinical practice cohort of patients with EoE.

Methods

We collected data on the efficacy of different therapies for EoE (ability to induce clinical and histologic remission) from the multicenter EoE CONNECT database—a database of patients with a confirmed diagnosis of EoE in Europe that began in 2016. We obtained data from 589 patients, treated at 11 centers, on sex, age, time of diagnosis, starting date of any therapy, response to therapy, treatment end dates, alternative treatments, and findings from endoscopy. The baseline endoscopy was used for diagnosis of EoE; second endoscopy was performed to evaluate response to first-line therapies. After changes in treatment, generally because lack of efficacy, a last endoscopy was performed. The time elapsed between endoscopies depended on the criteria of attending physicians. Clinical remission was defined by a decrease of more than 50% in Dysphagia Symptom Score; improvement in symptoms by less than 50% from baseline was considered as clinical response. Histologic remission was defined as a peak eosinophil count below 5 eosinophils/hpf. A peak eosinophil count between 5 and 14 eosinophils/hpf was considered histologic response. We identified factors associated with therapy selection and effectiveness using χ2 and multinomial logistic regression analyses

Results

PPIs were the first-line treatment for 76.4% of patients, followed by topical steroids (for 10.5%) and elimination diets (for 7.8%). Topical steroids were most effective in inducing clinical and histologic remission or response (in 67.7% of patients), followed by empiric elimination diets (in 52.0%), and PPIs (in 50.2%). Among the 344 patients who switched to a second-line therapy, dietary interventions were selected for 47.1% of patients, followed by PPIs (for 29.1%) and topical steroids (for 18.6%). Clinical and histologic remission or response was achieved by 80.7% of patients treated with topical steroids, 69.2% of patients given PPIs, and 41.7% of patients on empiric elimination diets. Multivariate analyses found the stricturing phenotype of EoE to be associated with selection of topical steroids over PPIs as the first-line therapy; lack of fibrotic features at initial endoscopy was associated with selection of elimination diets over topical steroids as a second-line therapy. The recruiting center was significantly associated with therapy choice; second-line treatment with topical steroids or PPIs were the only variables associated with clinical and histologic remission.

Conclusions

In an analysis of data from a large cohort of patients with EoE in Europe, we found topical steroids to be the most effective at inducing clinical and histologic remission, but PPIs to be the most frequently prescribed. Treatment approaches vary with institution and presence of fibrosis or strictures.

中文翻译：

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现实世界实践中嗜酸性粒细胞性食管炎的治疗效果。

背景与目标

外用类固醇、质子泵抑制剂 (PPI) 和饮食干预被推荐为嗜酸性粒细胞性食管炎 (EoE) 的一线和二线治疗。我们在现实世界的 EoE 临床实践队列中研究了它们的有效性差异。

方法

我们从多中心 EoE CONNECT 数据库中收集了不同疗法对 EoE（诱导临床和组织学缓解的能力）疗效的数据，该数据库是 2016 年开始的欧洲确诊 EoE 患者数据库。我们获得了 589 名患者的数据，在 11 个中心接受治疗，涉及性别、年龄、诊断时间、任何治疗的开始日期、对治疗的反应、治疗结束日期、替代治疗和内窥镜检查的结果。基线内窥镜检查用于诊断 EoE；进行第二次内窥镜检查以评估对一线治疗的反应。改变治疗后，一般是因为缺乏疗效，最后进行了一次内窥镜检查。内窥镜检查之间的时间间隔取决于主治医师的标准。临床缓解定义为吞咽困难症状评分下降 50% 以上；症状比基线改善不到 50% 被认为是临床反应。组织学缓解定义为峰值嗜酸性粒细胞计数低于 5 个嗜酸性粒细胞/hpf。嗜酸性粒细胞计数峰值在 5 到 14 个嗜酸性粒细胞/hpf 之间被认为是组织学反应。我们使用 χ2 和多项逻辑回归分析确定了与治疗选择和有效性相关的因素

结果

PPI 是 76.4% 患者的一线治疗，其次是局部类固醇（10.5%）和消除饮食（7.8%）。局部类固醇在诱导临床和组织学缓解或反应方面最有效（67.7% 的患者），其次是经验性消除饮食（52.0%）和 PPI（50.2%）。在转为二线治疗的 344 名患者中，47.1% 的患者选择了饮食干预，其次是 PPI（29.1%）和局部类固醇（18.6%）。80.7% 的局部类固醇治疗患者、69.2% 的 PPI 患者和 41.7% 的经验性消除饮食患者实现了临床和组织学缓解或反应。多变量分析发现 EoE 的狭窄表型与选择外用类固醇而非 PPI 作为一线治疗有关；初始内窥镜检查缺乏纤维化特征与选择消除饮食而不是局部类固醇作为二线治疗有关。招募中心与治疗选择显着相关；外用类固醇或 PPI 的二线治疗是与临床和组织学缓解相关的唯一变量。

结论

在对欧洲大量 EoE 患者数据的分析中，我们发现外用类固醇在诱导临床和组织学缓解方面最有效，但 PPI 是最常用的处方。治疗方法因机构和纤维化或狭窄的存在而异。