Treatment in primary biliary cholangitis: Beyond ursodeoxycholic acid

Area of Science:

• Hepatology
• Immunology
• Gastroenterology

Background:

• Primary biliary cholangitis (PBC) is a rare immune-mediated liver disease with variable progression to cirrhosis and associated complications.
• Ursodeoxycholic acid (UDCA) is the first-line treatment, improving surrogate markers and transplant-free survival, but many patients still progress.
• Risk stratification is crucial to identify patients needing additional therapy beyond UDCA.

Purpose of the Study:

• To review current insights into UDCA therapy for PBC.
• To discuss patient selection for intensified treatment based on residual risk.
• To summarize the efficacy of second-line treatment options in PBC.

Main Methods:

• Review of recent literature on PBC treatment and outcomes.
• Analysis of multifactor-based risk stratification strategies.
• Evaluation of randomized controlled trials for second-line therapies.

Main Results:

• UDCA offers survival benefits, but residual risk necessitates further management.
• Stricter biochemical goals may be achievable with add-on therapies.
• Farnesoid X receptor agonists, PPAR agonists, and corticosteroids show promise in select patients.

Conclusions:

• Personalized risk stratification is key in managing PBC.
• Optimizing treatment response with second-line agents can improve clinical outcomes.
• The evolving treatment landscape offers more options for achieving better patient outcomes in PBC.