Evolving Concepts of Treatment Targets for PBC: A Global Perspective

Primary biliary cholangitis (PBC) is a chronic cholestatic disease that if left untreated can progress to cirrhosis, end-stage liver disease, liver transplantation and, ultimately, death. PBC occurs worldwide, with rising prevalence in the North American, European, and Asia-Pacific regions. Ursodeoxycholic acid (UDCA) is the globally recommended first-line treatment, but up to half of UDCA-treated patients do not respond adequately. Fortunately, the PBC treatment landscape has evolved, and new treatments with novel mechanisms of action are available for patients with inadequate response to UDCA, but questions remain. Different treatment strategies have emerged in recent years from major societies across the globe regarding treatment targets, timing of treatment response assessment, management of hepatic and extra-hepatic manifestations, and choice of second-line therapies. Drug availability also has global variation. Nine international PBC experts, representing the United States, Canada, Europe, Asia, Africa, and Central/South America, collaborated to formulate opinions about the unanswered questions related to treatment targets in PBC. The result of this expert review is a global perspective on terminology related to the definition of responses to treatment, risk stratification, and treatment targets that can guide clinicians to optimize PBC treatment worldwide.