Department of Gastroenterology & Hepatology, Academic Medical Center, Amsterdam, The Netherlands.
College of Health Solutions, Arizona State University, Phoenix, AZ.
Hepatology. 2018 Sep;68(3):1174-1188. doi: 10.1002/hep.29882. Epub 2018 Aug 31.
Primary sclerosing cholangitis (PSC) is a rare and chronic liver disease for which there is no effective therapy. Interest has grown in developing treatments for this condition, with several agents proposed as potential therapies. However, there is a lack of clarity about how to measure clinical benefit in trials involving patients with this complex and rare disease. This article reviews regulatory information, the available literature on natural history, as well as potential candidate clinical and surrogate endpoints for PSC. (Hepatology 2018; 00:000-000).
原发性硬化性胆管炎（PSC）是一种罕见的慢性肝病，目前尚无有效的治疗方法。人们对开发这种疾病的治疗方法越来越感兴趣，有几种药物被提议作为潜在的治疗方法。然而，在涉及这种复杂和罕见疾病的患者的试验中，如何衡量临床获益尚不清楚。本文综述了监管信息、关于自然史的现有文献，以及 PSC 的潜在候选临床和替代终点。（《肝脏病学》2018 年；00:000-000）。
