Background and Aims

Peroral endoscopic myotomy (POEM) is an effective treatment for esophageal motility disorder. However, some people are poor responders who will probably need retreatments, such as endoscopic pneumatic dilation or re-POEM, and a scoring system for the prediction of poor responders preoperatively has not yet been established. We aimed to develop and validate a preoperative scoring system for predicting poor responders.

Methods

Overall, 244 patients who underwent POEM for esophageal motility disorders in our hospital from April 2015 to March 2019 were retrospectively included in this study. Poor responders were defined as patients with any of following: (1) Eckardt score ≥3 at 1-year follow-up, (2) endoscopic findings of food retention at 1-year follow-up, and (3) retreatments within 1 year after POEM. A risk-scoring system for poor responders was developed based on multiple logistic regression analysis, and its performance was internally validated using bootstrapping.

Results

Forty patients were diagnosed as poor responders at the 1-year follow-up. In the multivariate study, points for risk scores were assigned for 4 independent risk factors as follows: pretreatment Eckardt score (1-point increments), previous treatments (4 points), sigmoid-type esophagus (4 points), and esophageal dilation grade ≥II (4 points). The scoring system could predict an estimated risk for poor responders and provided satisfactory discrimination (area under the receiver operating characteristic curve, 0.78; 95% confidence interval, 0.68-0.88) and calibration (slope = 0.93; 95% confidence interval, 0.62-1.31).

Conclusions

A validated risk-scoring system for predicting poor responders preoperatively was established; this system could be useful for selecting treatment strategies and postoperative surveillance.

中文翻译：

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术前风险评分系统的开发，用于预测经口内镜下肌切开术的不良反应。

背景和目标

经口内镜下肌切开术（POEM）是一种有效的治疗食道动力障碍的方法。但是，有些人是反应较差的人，可能需要进行再治疗，例如内窥镜气管扩张术或re-POEM，并且尚未建立用于预测术前不良反应者的评分系统。我们旨在开发和验证术前评分系统，以预测不良反应者。

方法

总体而言，本研究回顾性分析了2015年4月至2019年3月在我院进行POEM食管运动障碍性疾病的244例患者。反应不良者定义为以下情况之一：（1）随访1年时Eckardt评分≥3；（2）随访1年时内镜检查发现食物retention留；（3）一年内再次治疗在POEM之后。基于多重逻辑回归分析，开发了针对反应较差者的风险评分系统，并使用自举对内部性能进行了验证。

结果

在1年的随访中，有40名患者被诊断为反应不良。在多变量研究中，为以下4个独立的危险因素分配了风险评分点：治疗前的Eckardt评分（1分增量），既往治疗（4分），乙状结肠食道（4分）和食管扩张度≥ II（4分）。评分系统可以预测不良响应者的估计风险，并提供令人满意的区分度（接收器工作特性曲线下的面积，0.78； 95％置信区间，0.68-0.88）和校准（斜率= 0.93； 95％置信区间，0.62-1.31） ）。

结论

建立了经过验证的术前预测不良反应者的风险评分系统；该系统可用于选择治疗策略和术后监测。