Primary biliary cholangitis (PBC) is a chronic, female-predominant cholestatic liver disease characterized by autoimmune-mediated destruction of small- to medium-sized intrahepatic bile ducts, elevated alkaline phosphatase levels, and the presence of high-titer anti-mitochondrial autoantibodies (AMA). Anti-gp210, anti-Sp100, anti-Kelch, and anti-hexokinase can also be detected in both AMA positive and AMA negative PBC patients. The laboratory and serological markers as well as criteria for the clinical diagnosis of PBC have been well established. However, the clinical course of PBC is heterogeneous with variable response to drug therapy. Ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) are currently employed as first- and second-line treatments, respectively. Here, we discuss the key criteria for clinicians in diagnosing PBC, focusing on clinical and biochemical abnormalities, immunological parameters, liver pathology, and the importance of distinguishing PBC from other causes of cholestatic disease. Finally, we discuss current approaches to preventing disease progression, treatment, and highlight recent advances in mechanism-based target therapies for PBC, as well as potential therapeutic strategies to enhance the efficacy of existing treatments.
Leung, P., Shao, T., Wang, L., Tsuneyama, K., Invernizzi, P., Timilsina, S., et al. (2025). Primary Biliary Cholangitis. In Y. Shoenfeld, R. Cervera, G. Espinosa, M.E. Gershwin (a cura di), Autoimmune Disease Diagnosis: Systemic and Organ-specific Diseases, Second Edition (pp. 427-432). Springer Nature [10.1007/978-3-031-69895-8_60].
Primary Biliary Cholangitis
Invernizzi P.;
2025
Abstract
Primary biliary cholangitis (PBC) is a chronic, female-predominant cholestatic liver disease characterized by autoimmune-mediated destruction of small- to medium-sized intrahepatic bile ducts, elevated alkaline phosphatase levels, and the presence of high-titer anti-mitochondrial autoantibodies (AMA). Anti-gp210, anti-Sp100, anti-Kelch, and anti-hexokinase can also be detected in both AMA positive and AMA negative PBC patients. The laboratory and serological markers as well as criteria for the clinical diagnosis of PBC have been well established. However, the clinical course of PBC is heterogeneous with variable response to drug therapy. Ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) are currently employed as first- and second-line treatments, respectively. Here, we discuss the key criteria for clinicians in diagnosing PBC, focusing on clinical and biochemical abnormalities, immunological parameters, liver pathology, and the importance of distinguishing PBC from other causes of cholestatic disease. Finally, we discuss current approaches to preventing disease progression, treatment, and highlight recent advances in mechanism-based target therapies for PBC, as well as potential therapeutic strategies to enhance the efficacy of existing treatments.
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