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Old and new treatments for primary biliary cholangitis
David Chascsa1, Elizabeth J Carey1, Keith D Lindor1,2
1Division of Gastroenterology and Hepatology, Mayo Clinic, Phoenix, AZ, USA.
Summary
Ursodeoxycholic acid (UDCA) is the standard treatment for primary biliary cholangitis, but many patients lack adequate response. Obeticholic acid offers a new option for refractory cases, improving biochemical profiles.
Area of Science:
- Hepatology and Gastroenterology
- Pharmacology and Therapeutics
Background:
- Primary biliary cholangitis (PBC) is a rare, progressive cholestatic liver disease characterized by elevated alkaline phosphatase and anti-mitochondrial antibodies.
- Ursodeoxycholic acid (UDCA) has been the sole therapeutic agent since 1998, improving outcomes but failing to adequately treat 25-40% of patients.
Purpose of the Study:
- To review the benefits of UDCA in managing primary biliary cholangitis.
- To discuss the efficacy and application of obeticholic acid as a novel treatment.
- To summarize ongoing research and therapeutic targets for PBC.
Main Methods:
- Review of existing literature on ursodeoxycholic acid (UDCA) therapy for primary biliary cholangitis.
- Analysis of clinical trial data for obeticholic acid, including its use in combination with UDCA and as monotherapy.
- Compilation of information on current and future therapeutic strategies under investigation.
Main Results:
- UDCA improves biochemical markers, histology, and delays liver transplantation in PBC patients.
- Obeticholic acid demonstrates significant and sustained reductions in alkaline phosphatase when used with UDCA or as monotherapy in intolerant patients.
- A substantial unmet need exists for patients with suboptimal response to UDCA.
Conclusions:
- Obeticholic acid represents a significant advancement in treating primary biliary cholangitis, particularly for patients unresponsive to UDCA.
- Ongoing research continues to explore new targets and treatments to improve management and achieve a cure for PBC.
- A comprehensive understanding of current and emerging therapies is crucial for optimizing patient care.