Mitchell NE, Chan SY, Jerez Diaz D, Ansari N, Lee J, Twohig P. Evolving therapeutic landscape of primary biliary cholangitis: A review. World J Hepatol 2025; 17(7): 107223 [DOI: 10.4254/wjh.v17.i7.107223]
Corresponding Author of This Article
Patrick Twohig, Assistant Professor, FRCPC, Department of Gastroenterology & Hepatology, University of Rochester Medical Center, 601 Elmwood Avenue, Rochester, NY 14682, United States. patrick_twohig@urmc.rochester.edu
Research Domain of This Article
Gastroenterology & Hepatology
Article-Type of This Article
Minireviews
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
World J Hepatol. Jul 27, 2025; 17(7): 107223 Published online Jul 27, 2025. doi: 10.4254/wjh.v17.i7.107223
Evolving therapeutic landscape of primary biliary cholangitis: A review
Natalie E Mitchell, Shu-Yen Chan, David Jerez Diaz, Nida Ansari, Junseo Lee, Patrick Twohig
Natalie E Mitchell, Junseo Lee, Patrick Twohig, Department of Gastroenterology & Hepatology, University of Rochester Medical Center, Rochester, NY 14682, United States
Shu-Yen Chan, Department of Internal Medicine, Weiss Memorial Hospital, Chicago, IL 60630, United States
David Jerez Diaz, Department of Internal Medicine, Florida State University, Sarasota Memorial Hospital, Sarasota, FL 34239, United States
Nida Ansari, Department of Internal Medicine, St. Joseph’s University Medical Center, Paterson, NJ 07504, United States
Author contributions: Mitchell NE, Chan SY, Jerez Diaz D, Ansari N, Lee J, and Twohig P contributed equally to this work; Twohig P designed the overall concept and outline of the manuscript; Mitchell NE, Chan SY, Jerez Diaz D, Ansari N, Lee J conducted the literature review and drafted the manuscript; Mitchell NE and Twohig P made critical revisions; all authors prepared the draft and approved the submitted version.
Conflict-of-interest statement: All authors declare no conflict of interests for this article.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Patrick Twohig, Assistant Professor, FRCPC, Department of Gastroenterology & Hepatology, University of Rochester Medical Center, 601 Elmwood Avenue, Rochester, NY 14682, United States. patrick_twohig@urmc.rochester.edu
Received: March 19, 2025 Revised: April 23, 2025 Accepted: June 9, 2025 Published online: July 27, 2025 Processing time: 129 Days and 10.1 Hours
Abstract
Primary biliary cholangitis (PBC) is a chronic autoimmune cholestatic liver disease characterized by progressive bile duct destruction, leading to fibrosis, cirrhosis, and eventual liver failure. Over the past two decades, significant advancements have paved the way for novel therapeutic strategies. Ursodeoxycholic acid (UDCA) has been the cornerstone of PBC management, improving survival and delaying disease progression in most patients. However, up to 40% of patients demonstrate an inadequate response to UDCA, necessitating additional treatment options. Obeticholic acid (OCA), a farnesoid X receptor agonist, has emerged as a second-line therapy, showing efficacy in reducing alkaline phosphatase levels and improving liver biochemistry. Beyond UDCA and OCA, a new wave of therapeutic agents are reshaping the PBC landscape. These include fibrates, peroxisome proliferator-activated receptor agonists and novel immunomodulatory drugs aimed at reducing autoimmune-mediated liver injury. Bile acid transport inhibitors, anti-fibrotic agents, and gut microbiome-targeted therapies are also under investigation, offering hope for personalized treatment approaches. This review highlights the evolution of PBC therapy, emphasizing the unmet needs of patients with refractory disease and the potential of emerging therapies to improve outcomes. As the therapeutic landscape continues to expand, optimizing treatment strategies through precision medicine holds the promise of transforming the management of PBC.
Core Tip: Primary biliary cholangitis (PBC) is a chronic, progressive, cholestatic autoimmune liver disease predominantly affecting middle-aged women. Novel therapeutic strategies have emerged in recent years, providing promising treatment options for more refractory disease. This comprehensive review provides an overview of existing and new treatment therapies for PBC.
