Primary biliary cholangitis* (PBC)Effectively manage PBC as early as possible in the course of the disease
Overview of PBC
- PBC is a liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids (BAs) out of the liver, resulting in cholestasis
- As PBC progresses, persistent toxic buildup of BAs causes progressive liver damage
- If left untreated, or if a patient does not adequately respond to treatment, chronic inflammation and fibrosis can advance to cirrhosis
- Disease progression in PBC is usually relatively slow, but can vary significantly
- Data from published long-term studies demonstrate that a significant portion of patients with advancing disease progress to liver failure, transplant, or death within 5 to 10 years
Natural history of PBC
- In the silent preclinical phase, patients may be completely free of symptoms; however, in one study, despite otherwise normal serum biochemistry, patients with PBC tested positive for antimitochondrial antibodies (AMAs)
- In the asymptomatic phase, patients may be diagnosed at an older age; length of survival can be shorter than for the general population
- In the symptomatic phase, patients show more rapid progression to end-stage liver disease and have a worse prognosis than asymptomatic patients
- In the preterminal phase, serum bilirubin increases and complications such as portal hypertension, ascites, and hepatic encephalopathy may develop as liver failure approaches
- Falling rates of liver transplants for PBC since ursodeoxycholic acid (UDCA) became widely used suggest a true change in natural history
Epidemiology of PBC
While PBC is rare, it is the most common cholestatic liver disease.
- Incidence and prevalence vary geographically, ranging from 0.7 to 49 and 6.7 to 402 per million, respectively
- In the United States, PBC is 1 of the top 10 most common causes of liver transplant
- Since 1988, PBC has been the second leading overall cause of liver transplant in women in the United States, behind hepatitis C
- PBC accounts for up to 2% of deaths from cirrhosis
Identifying patients with PBC
- Approximate age of diagnosis is generally in the fifth decade of life
- The presence of a concomitant autoimmune condition can signal increased risk for PBC, which should be evaluated and, if diagnosed, promptly treated with effective therapy
- An estimated 90% of patients are women
Symptoms of PBC
A majority of patients with PBC are asymptomatic at the time of initial diagnosis, but most develop symptoms over time.
- Initiation of symptoms such as fatigue accompanying PBC may not correlate with disease severity
- Fatigue and pruritus are by far the most common symptoms accompanying PBC
- Less common signs and symptoms include dry eyes and mouth as well as jaundice, which can be seen in more advanced disease
Potential complications of PBC
- Hypercholesterolemia
- Hepatocellular carcinoma
- Esophageal varices
- Jaundice
Diagnosis of PBC
- Based on American Association for the Study of Liver Diseases (AASLD) and European Association for the Study of the Liver (EASL) guidelines, clinical diagnosis of PBC is established based on 2 of 3 assessments:
- A positive test for AMAs, seen in 95% of patients with PBC
- Elevated serum levels of ALP
- Liver biopsy, which may be used to confirm staging during the diagnosis of PBC
PBC resources for in-depth clinical information
- AASLD Practice Guidelines: Primary Biliary Cirrhosis (Lindor et al, Hepatology, July 2009)
- AASLD Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis (Silveira et al, Hepatology, March 2010)
- Risk factors and prediction of long-term outcome in primary biliary cirrhosis (Ishibashi et al, Internal Medicine, Jan 2011)
- Farnesoid X receptor agonists for primary biliary cirrhosis. (Lindor, Current Opinions in Gastroenterology, May 2011)
- Primary biliary cirrhosis and bile acids (Corpechot, Clinics and Research in Hepatology and Gastroenterology, September 2012)
- The immunobiology and pathophysiology of primary biliary cirrhosis (Hirschfield et al, Annual Review of Pathology. January 2013)
*Formerly known as primary biliary cirrhosis.