Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

被引：51

作者：

Ponsioen, Cyriel Y. ^{[1
]}

Lindor, Keith D. ^{[2
]}

Mehta, Ruby ^{[3
]}

Dimick-Santos, Lara ^{[3
]}

机构：

[1] Acad Med Ctr, Dept Gastroenterol Hepatol, POB 22700, NL-1100 DE Amsterdam, Netherlands

[2] Arizona State Univ, Coll Hlth Solut, Phoenix, AZ USA

[3] US FDA, Div Gastroenterol & Inborn Errors Prod, Silver Spring, MD USA

来源：

HEPATOLOGY | 2018年 / 68卷 / 03期

关键词：

DOSE URSODEOXYCHOLIC ACID; PRIMARY BILIARY-CIRRHOSIS; HISTOLOGIC SCORING SYSTEMS; NATURAL-HISTORY; ALKALINE-PHOSPHATASE; TRANSIENT ELASTOGRAPHY; PROGNOSTIC VALUE; ULCERATIVE-COLITIS; GRADING SYSTEM; LIVER-FUNCTION;

D O I：

10.1002/hep.29882

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

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).

引用

页码：1174 / 1188

页数：15

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