Breaking grounds: a comprehensive analysis of cutting-edge treatments for primary biliary cirrhosis/primary biliary cholangitis with futuristic treatments
Khan, Asad Ali Ul Haq, Furqan Wahab, Qazi Muhammad Farooq Aslam, Taimur Khalid, Azeem Ali, Asad
Khan, Asad Ali
Ul Haq, Furqan
Wahab, Qazi Muhammad Farooq
Aslam, Taimur
Khalid, Azeem
Ali, Asad
Affiliation
University Hospitals Birmingham NHS Foundation Trust; Shifa International Hospitals Limited; Hayatabad Medical Complex; Staten Island University Hospital; Aiken Regional Medical Centers; State University of New York Upstate Medical University Hospital
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Publication date
2025-02-24
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Abstract
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.
Citation
Khan AA, Ul Haq F, Wahab QMF, Aslam T, Khalid A, Ali A. Breaking Grounds: A Comprehensive Analysis of Cutting-Edge Treatments for Primary Biliary Cirrhosis/Primary Biliary Cholangitis With Futuristic Treatments. Cureus. 2025 Feb 24;17(2):e79582. doi: 10.7759/cureus.79582.
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Article