Journal article 7 views
Up-to-Date Snapshot of Current and Emerging Medical Therapies in Primary Biliary Cholangitis
,
Nikhil Anand ,
Husam Al Maliki,
Shuell De Souza ,
Arya Kamyab ,
Amin Al Hadad,
Laith Alrubaiy
Journal of Personalized Medicine, Volume: 14, Issue: 12, Start page: 1133
Swansea University Author:
Laith Alrubaiy
Full text not available from this repository: check for access using links below.
DOI (Published version): 10.3390/jpm14121133
Abstract
Background/Objectives: Primary biliary cholangitis (PBC) is an autoimmune chronic cholestatic disease of the liver that symptomatically can present with pruritus and fatigue. Its established first- and second-line therapies are ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) although they pro...
| Published in: | Journal of Personalized Medicine |
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| ISSN: | 2075-4426 |
| Published: |
MDPI AG
2024
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| Online Access: |
Check full text
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| URI: | https://cronfa.swan.ac.uk/Record/cronfa68568 |
| Abstract: |
Background/Objectives: Primary biliary cholangitis (PBC) is an autoimmune chronic cholestatic disease of the liver that symptomatically can present with pruritus and fatigue. Its established first- and second-line therapies are ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) although they provide limited symptom management. Liver transplantation offers a potentially curative therapeutic option in refractory cases progressing to cirrhosis. Novel research published after the current guidelines highlights the importance of providing an up-to-date analysis of treatment options available. Methods: In this study, we conducted a literature search using Pubmed, Ovid Medline, and SCOPUS to provide a narrative review of first-line, second-line, and emerging therapies in PBC. Results: UDCA has been well established as a long-term, safe therapy within the literature although it is possible that treatment dosage can be further optimised in refractory patients. It has a favourable side effect profile. Despite improving biochemical markers, histopathological profile, and overall outcomes, up to 30–40% of patients are refractory to it. Age and sex are highlighted as independent indicators of non-responsiveness. This necessitates effective second-line therapies. Future trials could aim to investigate UDCA as a co-first-line therapy. Further supporting results for OCA were found in the interim extension trial of the seminal POISE study. The long-term phase 4 COBOLT trial is still awaiting results to further assess the complications, adherence, and potential adverse effects. It is a viable option in UDCA-refractory patients. The high incidence rate of dose-related pruritis indicates that alternative second-line options are needed. Bezafibrate is an off-label antilipemic agent that shows promise as a prospective second-line therapy option. The landmark BEZURSO trial alleviated some efficacy and safety concerns, but it remains associated with elevated serum creatinine; thus, it should be considered with caution. Other prospective second-line therapies are budesonide, triple therapy, and novel agents such as seladelpar and elafibranor. Conclusions: UDCA should remain the treatment of choice for PBC, though perhaps not as monotherapy. With further investigation, BF shows promise as a new second-line therapy alongside OCA, which it may outperform. |
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| Keywords: |
Primary biliary cholangitis; primary biliary cirrhosis; PBC; ursodeoxycholic acid; obeticholic acid; bezafibrate; fenofibrate |
| College: |
Faculty of Medicine, Health and Life Sciences |
| Funders: |
This research received no external funding. |
| Issue: |
12 |
| Start Page: |
1133 |