Primary biliary cholangitis

Primary biliary cholangitis (PBC), formally known as primary biliary cirrhosis, is a chronic cholestatic liver disease that mainly affects middle-aged women. PBC, if untreated, can progress to liver failure and death; liver transplantation is the only curative therapeutic option. Ursodeoxycholic acid (UDCA) has been proven to improve the long-term prognosis of patients with PBC and is approved as the first-line treatment for PBC in clinical practice, while almost 30% of the patients are refractory to UDCA treatment and can progress to cirrhosis in the absence of appropriate second-line treatment. Recently obeticholic acid (OCA), a farnesoid X receptor agonist, was approved for UDCA-refractory or UDCA-intolerant patients. However, the efficacy of OCA is still suboptimal and more new therapeutic approaches are seriously awaited. In this regard, we need to know more about the etiology of PBC, to identify more appropriate and specific biomarkers that predict the clinical course and also to know which therapies are applicable at different stages, since treatment for PBC should be individualized based on stratification of the risk for progression.