Last update:
25-Nov-2018

Primary biliary cholangitis (PBC), which is the commonest autoimmune cholestatic liver disease in adults, may lead to increasingly severe ductopenia and liver fibrosis. The cause of PBC is unknown, but a genetic predisposition in synergy with an environmental factor(s) is probably implicated. Biochemical criteria for expected non-response to treatment with ursodeoxycholic acid (UDCA), after 6 to 24 months, have been proposed in the last decade and the value of prognostic indicators in PBC has been assessed, including the patient's age, blood levels of bilirubin, albumin and alkaline phosphatase, and platelet count (Globe score). Despite better understanding of prognostic indicators and the response criteria to UDCA therapy in PBC, upward trends are being noted in the mortality rate and the need for liver transplantation. Patients who fail to achieve a satisfactory biochemical response to UDCA, who constitute about one third, are in need of second line therapies, either alone or in combination with UDCA, because this population shows a distinct risk of progression to cirrhosis and the complications of end-stage liver disease, including liver failure, portal hypertension, and hepatocellular carcinoma. A currently licensed, second-line therapeutic agent for PBC is obeticholic acid (OCA), which has been approved by EMA and FDA. Other novel therapies under development aim not only at preventing the onset of end-stage liver disease, but also at ameliorating the symptoms of PBC.

Key words: Antimitochondrial antibodies, Farnesoid X receptor, Fibroblast growth factor-19, Obeticholic acid, Peroxisome proliferatoractivated receptor alpha, Primary biliary cholangitis, Ursodeoxycholic acid.