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Trials of ursodeoxycholic acid for the treatment of primary biliary cholangitis (primary biliary cirrhosis)

Authors
Steven Flamm, MD
Raoul Poupon, MD
Section Editor
Keith D Lindor, MD
Deputy Editor
Anne C Travis, MD, MSc, FACG, AGAF

INTRODUCTION

Ursodeoxycholic acid (UDCA) was proposed as a potential therapy for cholestatic liver disease based on the following rationales: accumulation of toxic bile acids might be at least partially responsible for liver injury in chronic cholestasis, and replacement of endogenous bile acids by a non-toxic bile acid (UDCA) might protect the liver and retard the progression of these disorders. In preliminary studies of patients with primary biliary cholangitis (PBC; previously referred to as primary biliary cirrhosis) [1], UDCA was found to provide marked improvement in liver tests, leading to randomized trials and meta-analyses that also found a benefit. UDCA is generally well tolerated. Side effects may include headache, weight gain [2], constipation, and diarrhea.

Experimental and clinical evidence suggest that at least four mechanisms of action are involved in the beneficial therapeutic effects of UDCA in cholestatic disorders [3]:

An increased hydrophilicity index of the circulating bile acid pool

Stimulation of hepatocellular and ductular secretions

Cytoprotection against bile acid- and cytokine-induced injury

     

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Literature review current through: Nov 2016. | This topic last updated: Thu Oct 15 00:00:00 GMT+00:00 2015.
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References
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