Author information

• ¹Division of Gastroenterology and Liver Unit, University of Alberta, Edmonton, Alberta, Canada.
• ²Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, Saint-Antoine Hospital, Assistance Publique-Hôpitaux de Paris, European Reference Network Rare-Liver, Saint-Antoine Research Center, Sorbonne University, Paris, France.

Abstract

Primary biliary cholangitis (PBC) is an immune-mediated chronic liver disease characterized by biliary epithelial injury, cholestasis, and progressive fibrosis that can lead to cirrhosis and requirement for liver transplantation. All patients with PBC should receive initial treatment with ursodeoxycholic acid (UDCA), and odds for response are based on characteristics at baseline. It is important to have clear definitions of patients at risk for a poor response to therapy, of biochemical markers of an incomplete response, and standardized management. Patients typically are assessed after 12 months of treatment with UDCA for biochemical markers of response. However, evaluation at 6 months has been proposed for patients with more severe disease or symptoms (such as pruritus or fatigue). Markers of response to therapy include reduced serum levels of alkaline phosphatase and bilirubin (Paris-2, Toronto, GLOBE, and so forth); patients with high levels of total and conjugated bilirubin or levels of alkaline phosphatase more than 1.5-fold the upper limit of normal should be considered for second-line therapy. Patients with adequate biochemical responses can continue UDCA monotherapy. Incomplete responders should be considered for second-line therapies with obeticholic acid (licensed) or fibrates (unlicensed) in addition to continued treatment with UDCA. Patients with PBC should be followed up for life.