Focal Segmental Glomerulosclerosis (FSGS) is a characteristic histopathological lesion that is indicative of underlying glomerular dysfunction. It is not a single disease entity but rather a heterogeneous disorder that is an important cause of nephrotic syndrome and kidney failure in children and adults. The aim of this Kidney Health Initiative project was to evaluate potential endpoints for clinical trials in FSGS. This paper focuses on the data supporting proteinuria as a surrogate endpoint. Available data support the use of complete remission of proteinuria in patients with heavy proteinuria as a surrogate endpoint for progression to kidney failure. While substantial treatment effects on proteinuria that are short of a complete remission may also predict the effect of a treatment on progression to kidney failure, further work is needed to determine how such an endpoint should be defined. Fortunately, efforts are underway to bring together patient-level data from randomized controlled trials, observational studies, and registries to address this issue.

中文翻译：

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蛋白尿作为局灶节段性肾小球硬化临床试验的终点。


局灶节段性肾小球硬化 （FSGS） 是一种特征性组织病理学病变，提示潜在的肾小球功能障碍。它不是一个单一的疾病实体，而是一种异质性疾病，是儿童和成人肾病综合征和肾衰竭的重要原因。该肾脏健康倡议项目的目的是评估 FSGS 临床试验的潜在终点。本文重点介绍支持蛋白尿作为替代终点的数据。现有数据支持将重度蛋白尿患者的蛋白尿完全缓解作为进展为肾衰竭的替代终点。虽然对蛋白尿的实质性治疗效果未完全缓解也可能预测治疗对进展为肾衰竭的影响，但需要进一步的工作来确定应如何定义此类终点。幸运的是，正在努力整合来自随机对照试验、观察性研究和登记处的患者水平数据来解决这个问题。