What is primary biliary cholangitis (PBC)?

From the Guidelines

Primary biliary cholangitis (PBC) is an autoimmune liver disease characterized by the presence of antimitochondrial antibodies (AMA) in over 90% of patients, leading to progressive destruction of bile ducts, cholestasis, and eventual cirrhosis, primarily affecting middle-aged women 1.

Key Characteristics of PBC

• The disease is often asymptomatic at diagnosis, but patients may present with symptoms such as fatigue, pruritus, and/or jaundice 1.
• A diagnosis of PBC is made with confidence on a combination of abnormal serum liver tests and presence of AMA in serum 1.
• The diagnosis is confirmed by disclosing characteristic histological features of florid bile duct lesions 1.

Management and Treatment

• The first-line treatment is ursodeoxycholic acid (UDCA) at a dose of 13-15 mg/kg/day, which should be started immediately upon diagnosis to slow disease progression 1.
• For patients who don't respond adequately to UDCA, obeticholic acid (5-10 mg daily) may be added as second-line therapy 1.
• Symptom management is also important, with cholestyramine (4-16 g/day) for pruritus and calcium/vitamin D supplementation to prevent metabolic bone disease 1.

Lifestyle and Complications

• Patients with PBC can take exercise, and in fact, there is some pilot trial evidence that exercise therapy is helpful for the treatment of fatigue 1.
• There is no evidence to support an association between either the development of PBC or disease severity and alcohol consumption, but patients with advanced liver disease are advised to abstain from alcohol 1.
• Patients with PBC do not have an increased cardiac atherosclerotic risk, despite the elevations in cholesterol seen in the disease, but appropriate cardiac preventative screening and intervention is recommended 1.

Quality of Life and Support

• Fatigue has been shown to be the symptom with the biggest impact on patients, and patients with PBC who report high levels of fatigue are more vulnerable to emotional distress and are more likely to perceive that their quality of life has been negatively affected 1.
• Psychological approaches, such as cognitive behavioral therapy, may be used to support patients with PBC and manage distress resulting from debilitating symptoms 1.

From the FDA Drug Label

OCALIVA is a prescription medicine used to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have not responded well enough to UDCA, or alone in adults who cannot tolerate UDCA. The answer to what primary biliary cholangitis (PBC) is, is not directly stated in the provided drug labels.

• Definition of PBC is not provided in the labels.
• The labels only mention that OCALIVA is used to treat PBC. 2

From the Research

Definition and Characteristics of Primary Biliary Cholangitis (PBC)

• Primary biliary cholangitis (PBC) is a chronic, cholestatic liver disease characterized by lymphocytic cholangitis and intralobular bile duct destruction 3.
• It is an autoimmune disease that leads to the slow destruction of small intrahepatic bile ducts, impaired biliary secretion, and stasis of toxic endogenous bile acids within the liver, resulting in liver fibrosis and cirrhosis 4.
• PBC is a progressive disorder associated with increased mortality and decreased quality of life, related to hepatic fibrosis, troublesome symptoms such as fatigue and pruritus, and ultimately end-stage cirrhosis 3.

Clinical Features and Diagnosis

• The disease typically affects adults, with a higher prevalence in women than men, at a ratio of 10:1 4.
• Most patients are asymptomatic at diagnosis, but some may present with symptoms such as fatigue, pruritus, ascites, hepatic encephalopathy, and/or esophageal variceal hemorrhage 5.
• Diagnosis is based on a combination of clinical features, abnormal liver biochemical pattern in a cholestatic picture, and the presence of detectable antimitochondrial antibodies (AMA) in serum 5, 6.

Treatment and Management

• Ursodeoxycholic acid (UDCA) is the only approved pharmacotherapy for PBC, which can slow disease progression and improve survival, even in patients with advanced liver disease 3, 4, 5, 6.
• However, up to 40% of patients do not respond satisfactorily to UDCA therapy and may require adjunctive therapies 4.
• Other treatment options, such as obeticholic acid (OCA), are being investigated, and liver transplantation may be considered for patients with liver failure 7.