Improving prognosis in primary biliary cholangitis – Therapeutic options and strategy

Overall survival in primary biliary cholangitis is diminished. As patients are often asymptomatic, the disease may silently progress towards cirrhosis and liver failure. Timely diagnosis and effective treatment options are of vital importance to improve the prognosis of affected patients. Ursodeoxycholic acid is the standard of care first-line therapy and is associated with a reduced risk of liver transplantation and death. Treatment with UDCA is relevant for all patients, irrespective of disease stage or biochemical response. In case of incomplete biochemical response according to internationally accepted criteria, second-line treatment should be considered to improve long-term prognosis. Ursodeoxycholic acid has been the only accepted treatment for PBC during the last decades. Recent research, however, has identified a number of new therapeutic targets and agents, including obeticholic acid, fibrates and budesonide. While these agents all qualify as potentially beneficial second-line treatment, obeticholic acid is currently the only drug specifically approved for the treatment of PBC. Although long-term follow-up studies for these agents are mostly lacking, improvement of biochemical surrogate markers of clinical outcome induced by these drugs suggests a therapeutic benefit. The authors of this review aim to provide a summary of the results of previous and current studies evaluating medical treatments, and propose a treatment strategy based on the evidence available today.