The goal of treatment in PBC is to prolong life (through reducing the risk of progression to cirrhosis and, if present, reducing the risks associated with cirrhosis) and to increase quality of life through reduction of symptoms.
The mainstay of risk reducing therapy is ursodeoxycholic acid (UDCA) which is recommended for use in all patients with PBC. It reduces risk and rate of progression to cirrhosis.
In the UK 80% of patients respond to UDCA and have normal or near-normal life expectancy. The approach to treatment of the 20% who don’t respond sufficiently is an area where research is needed. This is the focus of the UK-PBC Stratified Medicine Project.
Where patients have progressed to cirrhosis with complications liver transplantation can be a highly effective treatment. Where cirrhosis is present, but transplantation is not indicated, risk reduction through screening for and treatment of portal hypertension is recommended.
UK-PBC has developed the UK-PBC Varices Prediction score which uses simple blood test values to identify patients who might be at risk of having oesophageal or gastric varices.
Effective treatments are available for itch, and response to therapy can be monitored using the PBC-40, a validated quality of life measure.
Fatigue is currently more difficult to treat although reducing associated symptoms such as daytime somnolence and autonomic dysfunction can be helpful. Rituximab therapy is undergoing clinical evaluation as a treatment for fatigue in PBC in the RITPBC trial which is linked to UK-PBC.
Osteoporosis is seen at increased levels in PBC patients and proactive screening and treatment can be useful in reducing fracture risk.