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Primary biliary cholangitis. Treatment options in 2025. A narrative review
Maria Angelara1, Klairi Papachristou2, Margarita Papatheodoridi3
1First Department of Internal Medicine, Propaedeutic Clinic, "Laiko" Hospital, National and Kapodistrian University of Athens, Athens, Greece.
Primary biliary cholangitis (PBC) treatments are evolving. While ursodeoxycholic acid (UDCA) is standard, new therapies like PPAR agonists show promise for non-responders, though further options are needed.
Area of Science:
- Hepatology
- Immunology
- Pharmacology
Background:
- Primary biliary cholangitis (PBC) is a chronic, autoimmune liver disease predominantly affecting women.
- Its pathogenesis involves genetic, epigenetic, and environmental factors leading to biliary epithelial cell apoptosis.
- Current treatments like ursodeoxycholic acid (UDCA) are insufficient for 15-40% of patients.
Purpose of the Study:
- To review current and emerging treatments for PBC.
- To elucidate the pathogenetic pathways targeted by various therapies.
- To highlight unmet needs in PBC management.
Main Methods:
- Review of existing literature on PBC treatments.
- Analysis of clinical trial data for novel therapeutics.
- Discussion of drug mechanisms of action.
Main Results:
- Ursodeoxycholic acid (UDCA) remains a first-line therapy but lacks efficacy in a significant patient subset.
- Peroxisome proliferator-activated receptor (PPAR) agonists demonstrate biochemical improvements and pruritus relief in non-responders.
- Several novel agents targeting different pathways are in development.
Conclusions:
- PBC treatment landscape is expanding beyond UDCA.
- PPAR agonists offer a new option for non-responsive patients.
- Continued research is crucial to develop therapies achieving complete biochemical normalization.
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