Primary biliary cholangitis: a summary of pathogenesis and therapies

Abstract

Primary biliary cholangitis (PBC) is a progressive autoimmune liver disease characterized by chronic inflammation and destruction of interlobular bile ducts. Its pathogenesis involves a complex interplay of genetic predisposition, environmental triggers, and immune-mediated mechanisms, particularly T-helper cell activity, leading to bile duct damage. First-line therapy includes ursodeoxycholic acid (UDCA), which improves liver biochemistry and slows disease progression, with obeticholic acid (OCA) as an option for non-responders. Double and/or triple therapy, including UDCA, OCA, and fibrates, appears to be superior in achieving therapeutic benefits in UDCA-nonresponsive PBC patients. Emerging therapies, such as peroxisome proliferator-activated receptor-α agonists, biologics such as dacetuzumab and rituximab, and experimental approaches such as stem-cell therapy, offer promising advances in managing PBC. Liver transplantation remains a final treatment option for advanced cases.

Keywords Primary biliary cholangitis, pathogenesis, therapy, peroxisome proliferator-activated receptor agonists, ileal bile acid transporter inhibitors

Ann Gastroenterol 2025; 38 (2): 121-132