Center for Esophageal Diseases and Swallowing, Division of Gastroenterology and Hepatology, Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, North Carolina; Center for Gastrointestinal Biology and Disease, Division of Gastroenterology and Hepatology, Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, North Carolina.
Department of Genetics, University of North Carolina School of Medicine, Chapel Hill, North Carolina.
Clin Gastroenterol Hepatol. 2019 May;17(6):1081-1088.e2. doi: 10.1016/j.cgh.2018.09.005. Epub 2018 Sep 10.
BACKGROUND & AIMS: Few factors have been identified that can be used to predict response of patients with eosinophilic esophagitis (EoE) to topical steroid treatment. We aimed to determine whether baseline clinical, endoscopic, histologic, and molecular features of EoE can be used to predict histologic response.
We collected data from 97 patients with EoE, from 2009 through 2015, treated with a topical steroid for 8 weeks; 59 patients had a histologic response to treatment. Baseline clinicopathologic features and gene expression patterns were compared between patients with a histologic response to treatment (<15 eos/hpf) and non-responders (≥15 eos/hpf). We performed sensitivity analyses for alternative histologic response definitions. Multivariate logistic regression was performed to identify predictive factors associated with response to therapy, which were assessed with area under the receiver operator characteristic (AUROC) curves.
Baseline dilation was the only independent predictor of non-response (odds ratio [OR], 0.30; 95% CI, 0.10-0.89). When an alternate response (<1 eos/hpf) and non-response (<50% decrease in baseline eos/hpf) definition was used, independent predictors of response status were age (OR, 1.08; 95% CI, 1.02-1.14), food allergies (OR, 12.95; 95% CI, 2.20-76.15), baseline dilation (OR, 0.17; 95% CI, 0.03-0.88), edema or decreased vascularity (OR, 0.20; 95% CI, 0.04-1.03), and hiatal hernia (OR, 0.07; 95% CI, 0.01-0.66). Using these 5 factors, we developed a predictive model that discriminated complete responders from non-responders with an AUROC of 0.88. Baseline gene expression patterns were not associated with treatment response and did not change with different histologic response thresholds.
In an analysis of 97 patients with EoE, we found dilation to be the only baseline factor associated with non-response to steroid treatment (<15 eos/hpf). However, a model comprising 5 clinical, endoscopic, and histologic factors identified patients with a complete response (<1 eos/hpf). A baseline gene expression panel was not predictive of treatment response at any threshold.
目前仅有少数因素可用于预测嗜酸细胞性食管炎（EoE）患者对局部皮质类固醇治疗的反应。本研究旨在确定 EoE 的基线临床、内镜、组织学和分子特征是否可用于预测组织学反应。
我们收集了 2009 年至 2015 年间 97 例接受 8 周局部皮质类固醇治疗的 EoE 患者的数据，其中 59 例患者的治疗组织学反应为阳性。对比治疗组织学反应（<15 个 eos/hpf）和无反应（≥15 eos/hpf）患者的基线临床病理特征和基因表达模式。我们对替代组织学反应定义进行了敏感性分析。采用多变量逻辑回归分析与治疗反应相关的预测因素，并通过受试者工作特征曲线（AUROC）评估其曲线下面积（AUROC）。
基线扩张是唯一与无反应相关的独立预测因素（比值比[OR]，0.30；95%CI，0.10-0.89）。当采用替代反应（<1 eos/hpf）和无反应（<50%基线 eos/hpf 减少）定义时，与反应状态相关的独立预测因素为年龄（OR，1.08；95%CI，1.02-1.14）、食物过敏（OR，12.95；95%CI，2.20-76.15）、基线扩张（OR，0.17；95%CI，0.03-0.88）、水肿或血管减少（OR，0.20；95%CI，0.04-1.03）和食管裂孔疝（OR，0.07；95%CI，0.01-0.66）。利用这 5 个因素，我们建立了一个预测模型，该模型可以区分完全缓解者和无缓解者，其 AUROC 为 0.88。基线基因表达模式与治疗反应无关，且不随不同的组织学反应阈值而变化。
在对 97 例 EoE 患者的分析中，我们发现扩张是唯一与皮质类固醇治疗无反应（<15 eos/hpf）相关的基线因素。然而，由 5 个临床、内镜和组织学因素组成的模型可以识别完全缓解者（<1 eos/hpf）。在任何阈值下，基线基因表达谱均不能预测治疗反应。
