Cholestatic liver diseases: new targets, new therapies

Therapeutic Advances in Gastroenterology
Priscila SantiagoCynthia Levy

Abstract

Cholestatic liver diseases result from gradual destruction of bile ducts, accumulation of bile acids and self-perpetuation of the inflammatory process leading to damage to cholangiocytes and hepatocytes. If left untreated, cholestasis will lead to fibrosis, biliary cirrhosis, and ultimately end-stage liver disease. Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are the two most common chronic cholestatic liver diseases affecting adults, and their etiologies remain puzzling. While treatment with ursodeoxycholic acid (UDCA) has significantly improved outcomes and prolonged transplant-free survival for patients with PBC, treatment options for UDCA nonresponders remain limited. Furthermore, there is no available medical therapy for PSC. With recent advances in molecular biochemistry specifically related to bile acid regulation and understanding of immunologic pathways, novel pharmacologic treatments have emerged. In this review, we discuss the standard of care and emphasize the various emerging treatments for PBC and PSC.

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Methods Mentioned

BETA
biopsies

Clinical Trials Mentioned

NCT01473524
NCT00570765
NCT02177136
NCT01456468
NCT02026401
NCT02704364
NCT01654731
NCT00575042
NCT01142323
NCT02609048

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