# Treatment options for primary biliary cholangitis
The treatment of primary biliary cholangitis aims to slow liver damage and relieve symptoms. The most commonly used medications work through different routes: some suppress the autoimmune reaction, others improve bile fluid drainage or protect liver tissue. What is applied in your case depends on the stage of your disease, how quickly it progresses, and how you respond to previous medications.
Ursodeoxycholic acid (UDCA)
ProveniIncluded in official guidelines, or approved by EMA or FDA
This medication is used as a first-line agent. It is a natural salt found in bile that helps slow liver damage by improving bile fluid drainage through the bile ducts and reducing inflammatory substances in the liver. UDCA also protects liver tissue against toxic substances from bile.
Since the 1990s, it has been shown that UDCA leads to slowing of disease progression in approximately seven out of ten patients. It is recommended as standard treatment in all major guidelines (including the European and American). The effectiveness can be monitored through regular blood tests; if no improvement occurs after one year, a second medication is usually added.
Known side effects are mild: mainly diarrhea and abdominal complaints, especially initially. These usually resolve on their own.
Fibrates
ProveniIncluded in official guidelines, or approved by EMA or FDA
(fenofibrate in combination; specific dosing experimental)
Fibrates are used as additional treatment alongside UDCA, especially if UDCA alone is insufficiently effective. They reduce inflammatory substances in the liver and improve bile drainage. They also help lower certain fat levels in the blood, which can provide additional protection.
Fenofibrate in combination with UDCA shows in studies that it can slow the appearance of liver damage. Its use is growing especially in specialized centers.
Known side effects are usually mild: muscle complaints, elevated uric acid levels, and elevated liver enzymes in blood can occur. The medication requires regular blood monitoring.
Second-line immunosuppressants (azathioprine, mycophenolate mofetil, bosentan)
ResearchediPositive results in clinical studies, not yet standard treatment
In patients who do not respond well to UDCA, medications can be used that more broadly suppress the immune system. These work by inhibiting the attack of immune cells on the bile ducts.
Azathioprine and mycophenolate mofetil have been studied longest in PBC, with mixed results: some studies show benefit, others do not. In practice they are used especially when worsening threatens and other options are limited.
Bosentan (a medication that promotes vasodilation) shows benefit in smaller studies for pruritus, but the evidence for slowing liver damage is limited.
Side effects vary: azathioprine can cause infections and bone marrow problems; mycophenolate mofetil often leads to abdominal complaints; bosentan can cause rash and headache.
Obeticholic acid (OCA)
ProveniIncluded in official guidelines, or approved by EMA or FDA
OCA is a more recent addition, working on bile acid receptors in the liver and immune cells. It inhibits inflammatory reactions and bile-related symptoms.
A large study (POISE) showed that OCA, added to UDCA, leads to normalization of certain liver values and reduction of bile-related symptoms in more than half of patients. OCA has been approved by the EMA as an additional medication for patients who respond insufficiently to UDCA.
A common side effect is pruritus, especially in the first weeks; furthermore borborygmus, elevated cholesterol, and weight changes can occur.
Elafibranor
ResearchediPositive results in clinical studies, not yet standard treatment
Elafibranor activates certain receptors (PPARα/δ) whereby inflammatory and fibrosis-causing processes in the liver are inhibited. Various studies (including ELATIVE) show that it can improve liver markers and relieve bile-related symptoms.
It is currently under intensive review for official approval; in Europe this is still ongoing. In several countries it is already being used in specialist settings based on research results.
Side effects appear mild to moderate: fatigue, muscle complaints and abdominal complaints have been reported.
Bezlotoxumab and anti-inflammatory agents (under investigation)
ExperimentaliOngoing in study setting, outcome still unknown
Several newer approaches are currently being investigated. Agents that block certain inflammatory mediators (for example against interleukins) or restore immune balance show early promise in small studies and in cell research.
These have not yet been recommended as standard treatment and are available only in an experimental setting.
Symptomatic treatment
ProveniIncluded in official guidelines, or approved by EMA or FDA
Alongside agents against disease progression, symptoms are treated:
- **Itching (pruritus):** antihistamines, rifampicin, naltrexone and certain resins help many patients. These provide targeted relief without directly acting on the underlying disease.
- **Fatigue:** there is no targeted medication for this, but adjustments to activity patterns help many.
- **Bone demineralization (osteoporosis):** preferably through diet (calcium, vitamin D); calcium supplements and bisphosphonates on indication.
- **Fat and fat-soluble vitamin deficiencies:** vitamins A, D, E and K may be needed if diarrhea occurs or bile duct complications are severe.
Liver transplantation
ProveniIncluded in official guidelines, or approved by EMA or FDA
This is not a medication but a procedural treatment, reserved for patients with severe cirrhosis (advanced liver scarring) or acute liver failure. The goal is to replace the diseased liver with a healthy one from a donor.
Transplantation is the only cure, but PBC returns after transplantation in about thirty percent of cases in the donor organ (recurrent PBC), although this usually follows a milder course. Studies show that preparation with certain training programs (prehabilitation with muscle and exercise training) improves outcome and recovery.
Future research underway
Currently, investigations are underway into fenofibrate in larger cohorts, saroglitazar (another type of fibrate), and gengravingassay to detect early who will deteriorate most rapidly. These may in the future refine treatment choice further, but are not yet ready for routine care.
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_This information never replaces a doctor's judgment. Always discuss your situation with your own healthcare provider._