Clinical Trial Endpoints for Perianal Fistulizing Crohn's Disease.

The lack of high-quality randomized controlled trials (RCTs) with primary endpoints specifically validated for pCD has contributed to suboptimal treatment recommendations.

缺乏针对pCD主要终点进行验证的高质量随机对照试验（RCTs），导致了治疗建议的不理想。

This review discusses considerations for pCD RCTs and provides expert opinion on current endpoints in the context of guidance from regulatory authorities.

本综述探讨了pCD RCTs（患者报告的结果临床试验）的考量因素，并在监管机构指导的背景下提供了关于当前终点的专家意见。

A challenge designing pCD RCTs is that placebo response rates and treatment effect sizes can change over time and are influenced by surgical and medical interventions that are often required pre-enrollment.

设计pCD RCTs的一个挑战是安慰剂反应率和治疗效果大小会随时间变化，并且受到通常在入组前需要的外科和医疗干预的影响。

Analysis of the main endpoint in 10 pCD phase III/IV RCTs and post hoc analyses highlighted the lack of universal definitions for clinical fistula response/remission.

在10项pCD III/IV期随机对照试验的主要终点分析和事后分析中，突显了缺乏对临床瘘管反应/缓解的普遍定义。

Most trials assessed clinical response based on a physical examination, which has not been formally validated, and one used a combined clinical and radiological response endpoint.

大多数试验基于体检评估临床反应，这尚未经过正式验证，而有一项试验使用了结合临床和放射学反应终点。

Five endpoints evaluating fistula closure/healing, clinical remission, and patient-reported outcomes were deemed to reflect clinically meaningful changes for patients.

评估瘘管闭合/愈合、临床缓解和患者报告结果的五个终点被认为反映了对患者具有临床意义的变化。

However, none were considered to be reproducible or show a reasonable effect size with low placebo background noise or response rates.

然而，这些终点均被认为无法重复，或在低安慰剂背景噪声或反应率的情况下显示出合理的效果大小。

Based on our evaluation, future trials should use coprimary clinical and radiologic assessments to capture both how the patient feels and an objective measure of disease activity.

根据我们的评估，未来的试验应使用共同的主要临床和放射学评估，以全面捕捉患者的感受以及疾病的客观活动度测量。

These findings reinforce the need for clearly defined, objective, and standardized endpoints for pCD clinical trials that will enable evaluation of novel therapies to improve patient outcomes.

这些发现强化了为pCD临床试验明确界定、客观和标准化终点的必要性，这将使评估新疗法以改善患者预后成为可能。