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Update in the Care and Management of Patients with Primary Sclerosing Cholangitis
1Department of Internal Medicine, University of Miami/Jackson Memorial Hospital, Miami, FL, USA.
Purpose Of Review:
Primary sclerosing cholangitis (PSC) is a progressive cholestatic liver disease for which specific medical therapy is not available. The goals of treatment are primarily early detection and management of complications. In this review, we discuss novel therapies under evaluation and provide the foundation for surveillance strategies.
Recent Findings:
Drugs under investigation include norursodeoxycholic acid, nuclear receptor agonists, anti-fibrotics, antibiotics, and anti-inflammatory drugs. Endoscopic therapy is indicated for symptomatic dominant strictures and in the work-up of malignancies. Recently, the use of stents was associated with an increased rate of complications compared to balloon dilatation; and long-term stenting should be avoided. Malignancies currently account for most of the PSC-related mortality. Many drugs are emerging for the treatment of PSC but liver transplantation is the only treatment modality shown to prolong survival. PSC recurrence occurs in up to 35% of transplanted allografts within a median of 5 years. Surveillance for hepatobiliary and colorectal malignancies is indicated.
Insights
Primary sclerosing cholangitis (PSC) lacks specific medical treatments. Research explores new drugs and endoscopic therapies, but liver transplantation remains the only survival-extending option for this progressive liver disease.
Area of Science:
- Hepatology
- Gastroenterology
- Internal Medicine
Background:
- Primary sclerosing cholangitis (PSC) is a chronic, progressive cholestatic liver disease.
- Currently, no specific medical therapies exist for PSC, with treatment focusing on complication management.
Purpose of the Study:
- To review novel therapies under investigation for PSC.
- To provide a foundation for surveillance strategies in PSC patients.
Main Methods:
- Literature review of emerging drugs and endoscopic interventions for PSC.
- Analysis of current treatment outcomes and recurrence rates post-transplantation.
Main Results:
- Investigational drugs include norursodeoxycholic acid, nuclear receptor agonists, anti-fibrotics, antibiotics, and anti-inflammatory agents.
- Endoscopic therapy is recommended for dominant strictures; stenting may increase complications compared to balloon dilatation.
- Malignancies are a leading cause of PSC mortality, and PSC recurrence affects up to 35% of liver transplant recipients.
Conclusions:
- Liver transplantation is the only current treatment to prolong survival in PSC.
- Ongoing research into novel pharmacotherapies and improved endoscopic techniques is crucial.
- Regular surveillance for malignancies and monitoring for PSC recurrence post-transplant are essential.
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