A review of the medical treatment of primary sclerosing cholangitis in the 21st century

Therapeutic Advances in Chronic Disease
Elizabeth C Goode, Simon M Rushbrook

Abstract

Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease that progresses to end-stage liver disease and cirrhosis. Recurrent biliary inflammation is thought to lead to dysplasia, and as such PSC confers a high risk of cholangiocarcinoma. PSC accounts for 10% of all UK liver transplants, although transplantation does not guarantee a cure with 20% recurrence in the graft. At present there are no effective medical treatment options for PSC, and trials of novel therapeutic agents are limited by the time taken to reach clinically significant endpoints with no well defined early surrogate markers for disease outcome. Moreover, PSC appears to be a heterogeneous disease with regards to disease distribution, associated inflammatory bowel disease and subsequent disease outcome, further compounding the issue. Thus existing trials have taken place in heterogeneous groups, are likely to be underpowered to detect any individual subgroups effect. The current mainstay of medical treatment is still with ursodeoxycholic acid, although there is no evidence that it alters long-term outcome. Small pilot studies of immunosuppressive agents have taken place, but despite evidence that may support studies in larger groups, these have...Continue Reading

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Citations

Aug 16, 2016·Journal of Biochemical and Molecular Toxicology·Natalia Nuño-LámbarriNorberto C Chávez-Tapia
Nov 19, 2017·Hepatology Research : the Official Journal of the Japan Society of Hepatology·Tadashi Ikegami, Akira Honda
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Apr 25, 2021·Prilozi·Gregor KrstevskiVladimir Serafimovski
Aug 17, 2021·Journal of Transplantation·John Paul NsubugaAlan Bonder

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Methods Mentioned

BETA
biopsy
lavage

Clinical Trials Mentioned

NCT01802073
NCT02239211
NCT02177136
NCT01755507
NCT01672853
NCT01456468
NCT02061540

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