Last edited 07/2018 and last reviewed 03/2020

Primary biliary cholangitis (previously known as primary bilary cirrhosis) cirrhosis is an autoimmune disease characterised by chronic, progressive, destruction of intrahepatic bile ducts, resulting in chronic cholestasis, portal inflammation, and fibrosis which will eventually lead to cirrhosis and liver failure (1).

  • a highly specific auto antibody - anti-mitochondrial autoantibodies (AMA) seen in 90-95% of patients is the characteristic serologic hallmark of the disease
  • it predominantly affects women typically in their fifth and sixth decades (1,3)
    • ratio of affected females: males is as high as 10:1 (1)

The condition is uncommon with a reported prevalence ranging between 19 and 402 cases per million

  • highest incidence and prevalence rates are reported from the northern hemisphere – the UK, Scandinavia, Canada, and the USA while lowest rates were seen in Australia (4)
  • prevalence and incidence of PBC is thought to be increasing due to easier recognition of the disease and improved case finding strategies (1)
    • in the UK, annual incidence rates increased from 5.8 to 20.5 cases per million population  between 1980 and 1999 among residents of Sheffield and in Newcastle-upon-Tyne  from 11 to 32 cases per million population between 1976 and 1994
    • this increase was paralleled by prevalence reaching more than 200 cases per million in the middle to late 1990s (1)
  • diagnosis should be suspected in a patient – particularly a woman – who presents with fatigue, itching, jaundice or right upper quadrant discomfort (1)

Management (5):

  • until recently, the only effective treatment was ursodeoxycholic acid (UDCA)
    • note though that up to 40% of PBC patients have an inadequate response to UDCA and may continue to have disease progression
  • several models have been developed, including the UK-PBC and GLOBE scores, to assist in identifying patients who may benefit from second-line therapies, such as the farnesoid X receptor (FXR) agonist obeticholic acid (OCA)
    • the addition of OCA can significantly improve serum alkaline phosphatase and total bilirubin, which are strong surrogate markers of clinical outcomes in PBC
  • other alternatives, including the peroxisome proliferator-activated receptor (PPAR)-alpha agonists fenofibrate and bezafibrate, may also improve liver biochemistries in PBC patients with an inadequate response to UDCA, but further study is needed to demonstrate their safety and long-term efficacy