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 first line treatment for PBC is ursodeoxycholic acid (UDCA) which is recommended for use in all patients. It can reduce risk and rate of progression to cirrhosis, however not all patients respond to it and the importance of second-line treatments in high risk patients is now appreciated. In the UK c60% of patients respond to UDCA and have normal or near-normal life expectancy.
The approach to treatment of the 40% who don’t respond sufficiently is an area where research is needed. The major focus of UK-PBC is on understanding why some patients do not respond to UDCA and identifying and evaluating better approaches to their treatment.
UK-PBC has developed two clinical tools to help clinicians manage their PBC patients. The UK-PBC risk score allows accurate prediction of risk to life from PBC once UDCA treatment is established. The UDCA Response Score (URS) allows the prediction of the likelihood of someone responding to UDCA and is designed for use in patients presenting with PBC and before they start UDCA. These clinical tools are at the heart of the current model for personalising treatment in PBC.
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. We have also developed and validated a shortened version of the PBC-40 (the PBC-10) which is suitable for use in the clinic to screen PBC patients for symptoms.
Fatigue is currently more difficult to treat although reducing associated symptoms such as daytime somnolence and autonomic dysfunction can be helpful. Trials of therapy for fatigue and its associated symptoms in PBC are likely to be an area of progress in the next few years.
Osteoporosis is seen at increased levels in PBC patients and proactive screening and treatment can be useful in reducing fracture risk.