New developments in the treatment of primary biliary cholangitis

Abstract

Primary biliary cholangitis is a chronic, immunologically mediated, progressive liver disease characterized by progressive destruction of small bile ducts, resulting in cholestasis, liver fibrosis with the development of liver cirrhosis and all of its complications. These ultimately lead to liver failure, resulting in death or the need for a liver transplant. Early and effective treatment is a key factor in influencing the prognosis. The disease mainly affects women, accounting for 90% of cases. Diagnosis is possible based on an elevation of serum alkaline phosphatase (ALP) above 1.5-times the normal level for 6 months and in combination with the presence of antimitochondrial antibodies (AMA) ≥ 1: 40 or specific antinuclear antibodies (ANA) sp100, gp210, or a combination of elevated serum ALP with typical histological findings found during liver biopsy. Although ursodeoxycholic acid (UDCA) remains the treatment of choice, unfortunately some patients do not tolerate this treatment or do not respond adequately to it. Second-line treatment is currently available – peroxisome proliferator-activated receptor (PPAR) agonists, delta (PPAR-) or dual agonists (PPAR-/). Seladelpar (PPAR-) and elafibranor (PPAR-/) are promising treatment alternatives due to their anti- -inflammatory and antifibrotic properties.