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Primary Biliary Cholangitis

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Diseases of the Liver and Biliary Tree

Abstract

Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease of autoimmune etiology characterized by inflammation and progressive destruction of the small intrahepatic bile ducts. The disease develops due a combination of a genetic predisposition and environmental and possibly infectious triggers. The diagnosis is generally confirmed in case of cholestatic abnormalities, a positivity for antimitochondrial antibody or antinuclear-specific antibodies (sp-100, gp-210). Liver biopsy is helpful in case of suspicion of an overlap syndrome with autoimmune hepatitis or association with a nonalcoholic steatohepatitis. The annual incidence ranges between 0.7 and 49 cases per million; it predominantly affects women around menopause age. Nowadays, more cases in males are diagnosed. Male gender in PBC is a very important issue for many reasons: (a) administrative data show a decrease in F:M ratio in many countries; (b) a particular risk for liver malignancy for males with PBC has been observed. During the recent decades PBC has changed its natural history because more asymptomatic cases are diagnosed than in the past. There is a need for risk stratification for disease progression through clinical tools. Biochemical response to ursodeoxycholic acid (UDCA) is a strong predictor of long-term outcome. Approximately, 40% of patients fail to respond to UDCA and require a second-line treatment. Obeticholic acid, a farnesoid X receptor (FXR) agonist, demonstrates a biochemical efficacy in 50% of patients when added to UDCA in case of inadequate response to standard therapy. Fibrates may be beneficial for patients with PBC and incomplete response to UDCA, in particular, for cases with hypercholesterolemia and atherosclerotic profile; moreover, fibrates might be useful in case of pruritus. Budesonide might be beneficial for some patients with PBC with precirrhotic stage but should be avoided in cirrhosis for the possibility of risk of portal vein thrombosis. Novel therapies, including different FXR-agonists, PPRA agonists, rituximab, biologic agents, antifibrotics, are currently under investigation.

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Authors and Affiliations

  1. Scientific Consultant, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS) Negrar, Verona, Italy

    Annarosa Floreani

  2. Senior Scholar University of Padova, Padova, Italy

    Annarosa Floreani

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  1. Annarosa Floreani
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Correspondence to Annarosa Floreani .

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  1. Scientific Consultant, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS) Negrar, Verona, Italy

    Annarosa Floreani

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Floreani, A. (2021). Primary Biliary Cholangitis. In: Floreani, A. (eds) Diseases of the Liver and Biliary Tree. Springer, Cham. https://doi.org/10.1007/978-3-030-65908-0_8

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  • DOI: https://doi.org/10.1007/978-3-030-65908-0_8

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