A Practical Review of Primary Biliary Cholangitis for the Gastroenterologist

Primary biliary cholangitis (PBC) is an autoimmune chronic cholestatic liver disease characterized by biliary destruction and progressive intrahepatic cholestasis. PBC primarily affects women in their fifth or sixth decade of life. Although many patients are asymptomatic at presentation, fatigue, pruritus, sicca syndrome, and upper abdominal discomfort are common symptom manifestations. The etiology of PBC is thought to be related to interactions between underlying genetic predisposition and microbial and xenobiotic environmental triggers. The diagnosis is established in the setting of biochemical cholestasis and antimitochondrial or disease-specific antinuclear antibodies, with histologic evidence of nonsuppurative granulomatous cholangitis being supportive, but not required, to confirm disease. Care of patients with PBC encompasses therapies to slow disease progression, manage symptoms associated with cholestasis, and treat complications of advanced liver disease. Risk stratification based on simple clinical and laboratory parameters, either as binary response criteria and/or continuous models, helps identify the patients at greatest risk of poor outcome. First-line therapy to slow disease progression is ursodeoxycholic acid (UDCA), which is the mainstay of pharmacologic therapy for all patients with PBC. The only currently approved second-line option for patients who do not achieve adequate biochemical response or are intolerant to UDCA is the novel farnesoid X receptor agonist obeticholic acid. Off-label use of peroxisome proliferator-activated receptor agonists, including the fibrate class of drugs where available, is also recognized as an option for patients.