Breaking Grounds: A Comprehensive Analysis of Cutting-Edge Treatments for Primary Biliary Cirrhosis/Primary Biliary...

Primary biliary cholangitis (PBC) is an autoimmune disorder characterized by biliary destruction leading to intrahepatic biliary cholestasis. It predominantly affects women during the fifth and sixth decades. Treatment options have progressed from ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) to liver and stem cell transplant. The objectives include summarizing established and new diagnostic approaches for PBC along with reviewing efficacy treatments, their side effects, and future directions. The treatment of PBC is based on risk stratification, including assessment of the patient's age, sex, clinical pattern, biochemical and antibody profile, histology, and markers of fibrosis. UDCA and OCA are Food and Drug Administration (FDA) approved first-line and second-line agents. Elafibranor, a recently FDA-approved agent based on its efficacy, was shown in the ELATIVE trial. Seladelpar, currently under FDA review in the ENHANCE III trial, is also used in PBC. Fibrates, a third-line treatment, are found efficacious in different trials. Other treatment options are in phase II/III clinical trials. The question of whether we use immunotherapy has been answered in the NCT02376335 and NCT00746486 trials, stating that rituximab and budesonide cannot be used as no clinical significance is observed. The emergence of new therapies and the potential of combination treatments offer hope for improving outcomes for all patients with PBC. Personalized treatment strategies, continuous monitoring, and a comprehensive approach to symptom management are key to optimizing care and enhancing the quality of life for individuals affected by this chronic liver disease.