Tabibian James H, Ali Ahmad H, Lindor Keith D
Dr Tabibian is an associate professor in the Geffen School of Medicine at UCLA in Los Angeles, California and director of endoscopy in the Department of Medicine at Olive View-UCLA Medical Center in Sylmar, California. Dr Ali is a research fellow in the Division of Gastroenterology and Hepatology at Mayo Clinic in Phoenix, Arizona. Dr Lindor is a professor of medicine in the Division of Gastroenterology and Hepatology at Mayo Clinic and senior advisor to the provost at Arizona State University in Phoenix, Arizona.
Gastroenterol Hepatol (N Y). 2018 May;14(5):293-304.
Primary sclerosing cholangitis (PSC) is a chronic, idiopathic cholangiopathy that can progress to cirrhosis, end-stage liver disease, hepatobiliary cancer, and/or colorectal cancer. The course of PSC is often complicated by portal hypertension, symptoms of cholestasis, and recurrent bacterial cholangitis, among other conditions, with a consequent decrease in survival (median, approximately 20 years) and quality of life. The etiopathogenesis of PSC remains poorly understood, and, as such, pharmacotherapy has yet to be definitively established. Despite its rarity, PSC is the fifth leading indication for liver transplantation (LT) in the United States. Although the only intervention known to extend survival of patients with PSC, LT is costly and invasive, and recurrent PSC affects approximately 30% of LT recipients. Over the past several years, owing in part to progress in the understanding of PSC, novel pharmacotherapeutics have been developed, some of which are currently in the PSC clinical trial pipeline. Here, in the first of a 2-part series, we provide a review and update of the epidemiology, etiopathogenesis, clinical features, and treatment of PSC. The second part of the series will focus on cancer risk, prevention, and surveillance of PSC.
原发性硬化性胆管炎（PSC）是一种慢性特发性胆管病，可进展为肝硬化、终末期肝病、肝胆癌和/或结直肠癌。PSC的病程常伴有门静脉高压、胆汁淤积症状和复发性细菌性胆管炎等情况，从而导致生存率（中位数约为20年）和生活质量下降。PSC的病因发病机制仍知之甚少，因此药物治疗尚未明确确立。尽管PSC较为罕见，但它是美国肝移植（LT）的第五大主要适应证。虽然LT是已知能延长PSC患者生存期的唯一干预措施，但成本高昂且具有侵入性，复发性PSC影响约30%的LT受者。在过去几年中，部分由于对PSC认识的进展，已开发出新型药物疗法，其中一些目前正处于PSC临床试验阶段。在此，在这个分两部分的系列文章的第一篇中，我们对PSC的流行病学、病因发病机制、临床特征和治疗进行综述与更新。该系列文章的第二篇将聚焦于PSC的癌症风险、预防和监测。
