Primär biliäre Cholangitis: Geänderter Name, neues Glück?

Primary biliary cholangitis (PBC, formerly primary biliary cirrhosis) is the most common immune-mediated cholestatic hepatobiliary disease. Middle-aged women are commonly affected. Patients often present clinically with fatigue, pruritus, and sicca syndrome. The diagnosis of PBC is based on increased serum markers of cholestasis and serum antimitochondrial or PBC-specific antinuclear antibodies. Liver biopsy is not required for diagnosis in most cases. Without medical treatment, PBC patients will progress to liver cirrhosis and may require liver transplantation. For several decades ursodeoxyholic acid (UDCA) was the only approved therapy for PBC. UDCA improves serum liver tests including surrogate markers of long-term prognosis, histological features, and prognosis of patients. Still, in a substantial percentage of patients (35–40%), the therapeutic response is insufficient. Despite appropriate dosage of UDCA, they are at risk for disease progression. For these patients with inadequate response to UDCA alone, obeticholic acid has been licensed as second-line therapy, which has synergistic anticholestatic effects to UDCA. Bezafibrate and budesonide have also shown promising effects as second-line therapies, but await publication and are prescribed in off-label use. Additional therapeutic approaches are currently being investigated in clinical trials. Despite novel therapeutic options, many patients suffer from extrahepatic manifestations which may dramatically reduce the quality of life. These symptoms need adequate attention and symptomatic treatment whenever possible.