A Guide to Primary Biliary Cholangitis and Clinical Trials


Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease characterized by the inflammation of the small bile ducts within the liver. These ducts degrade over time, resulting in a detrimental bile buildup. 

Bile ducts play a crucial role in transporting bile out of the liver and into the small intestine. In cases of PBC, the immune system mistakenly targets and damages these bile ducts, leading to impaired bile flow, accumulation of toxic substances in the liver, and liver damage. 

Over time, this damage can result in fibrosis, cirrhosis, and, in severe cases, liver failure.

PBC is most commonly seen in women, particularly those between the ages of 30 and 60, but it can affect individuals of any age or gender. The exact cause of PBC is not well understood, but it is believed to involve a combination of genetic, environmental, and immunological factors. 

While PBC is considered a relatively rare liver disease, its prevalence varies globally. Some regions report higher incidence rates, and it is estimated that around 1 in 1,000 individuals in the United States may be affected. Generally, PBC is considered one of the most common autoimmune diseases.

The danger and concern associated with PBC lie in its potential to progress silently in its early stages, often without noticeable symptoms. As the disease advances, symptoms such as fatigue, itching, and abdominal discomfort may manifest, signaling significant liver damage.

If left untreated, PBC can pose a serious threat to an individual’s health and require more aggressive interventions, such as liver transplantation. 

Early diagnosis, regular monitoring, and appropriate medical management are essential in addressing primary biliary cholangitis and mitigating its potential long-term consequences.


Persistent, unexplained fatigue is a common symptom experienced by individuals with PBC. It can significantly impact daily activities and quality of life.

Itching (Pruritus)

Pruritus is a characteristic symptom of PBC and can be quite bothersome. It often occurs on the palms of the hands and soles of the feet but can affect other areas of the body as well.

Abdominal Discomfort

Some individuals with PBC may experience discomfort or pain in the upper right quadrant of the abdomen, where the liver is located.

Dry Eyes and Mouth

PBC can affect the glands that produce moisture, leading to dry eyes and dry mouth. This is especially common in association with other autoimmune conditions like Sjögren’s syndrome.


In more advanced stages of PBC, jaundice may occur, causing a yellowing of the skin and eyes. This is due to the buildup of bilirubin, a yellow pigment produced during the normal breakdown of red blood cells when the liver is unable to process it effectively.

Dark Urine and Pale Stools

Discoloration of urine to a darker shade and pale-colored stools can occur as a result of impaired bile flow and the accumulation of bilirubin.

Joint Pain and Stiffness

Some individuals with PBC may experience joint pain and stiffness, which can be associated with inflammation and autoimmune activity.

Note: the severity and combination of symptoms can vary among individuals with PBC. 

Additionally, some patients may be diagnosed with PBC based on abnormal liver function tests before experiencing noticeable symptoms. Individuals experiencing any of these symptoms or who are concerned about their liver health should seek medical attention for a proper evaluation and diagnosis.

Progression to Cirrhosis

One of the primary concerns with PBC is the potential progression to cirrhosis, a condition characterized by extensive scarring of the liver tissue. This scarring disrupts the normal structure and function of the liver, impairing its ability to perform essential tasks. 

Researchers estimate that roughly 1 in 400 adults in the United States live with cirrhosis. Although not all are associated with PBC, it is a common contributor to extensive, long-term liver damage.  

Liver Failure

In the advanced stages of cirrhosis, there is an increased risk of liver failure. Liver failure occurs when the liver is no longer able to perform its essential functions, such as detoxification and production of proteins necessary for blood clotting.

When the liver fails, it can lead to a string of life-threatening complications that affect multiple organ systems. As of 2023, liver failure accounts for a staggering 2 million deaths annually, and at least 1 million are due to complications of cirrhosis. 

Complications of Cirrhosis

Liver failure isn’t the only potential complication of cirrhosis resulting from PBC. Another significant problem is portal hypertension, which can cause enlarged veins (varices) in the esophagus and stomach. These veins can rupture and result in life-threatening bleeding.

Hepatocellular Carcinoma (Liver Cancer)

Individuals with advanced cirrhosis, especially in the context of chronic liver diseases like PBC, have an increased risk of developing hepatocellular carcinoma (HCC). This form of liver cancer is the fifth most common cancer in men and the seventh most common in women (globally), and it is known to be highly aggressive. 

Risk of Other Autoimmune Conditions

People with PBC may have an increased susceptibility to other autoimmune conditions. Common associations include conditions like Sjögren’s syndrome (the immune system attacks the moisture glands in the eyes and mouth) and systemic sclerosis (abnormal growth of connective tissue).

Emotional and Psychological Impact

Dealing with a chronic liver disease takes a toll on an individual’s mental and emotional well-being. The uncertainty of the disease’s progression, the impact on daily life, and the potential need for medical interventions, such as liver transplantation, can easily contribute to stress and anxiety.

Individuals with PBC should work closely with their healthcare team to manage the condition. Regular monitoring, lifestyle modifications, and appropriate medical interventions can help mitigate some of these risks and improve overall outcomes.

Why Primary Biliary Cholangitis Is Challenging to Overcome

Primary biliary cholangitis presents unique challenges that make it a complex condition to overcome. As a lifelong condition, it necessitates continuous monitoring and management.

In most cases, the following factors contribute to the intricacy of managing and living with PBC:

PBC often begins silently, with subtle or no symptoms in its early stages. This allows the disease to progress without notice, causing damage to the liver. The longer PBC goes undiagnosed, the more significant the liver damage becomes. 

The symptoms of PBC can vary widely among individuals. Some may experience fatigue, itching, or abdominal discomfort, but others remain asymptomatic. This heterogeneity in symptom presentation complicates diagnosis and disease progression assessment.

Overcoming the challenges associated with primary biliary cholangitis requires a comprehensive and multidisciplinary approach involving close collaboration between patients and healthcare professionals. 

Fortunately, ongoing research and advancements in understanding the disease mechanisms are helping to develop more effective treatments and improve the quality of life for individuals affected by PBC.

While the exact cause of PBC remains unclear, the condition is more commonly observed in certain demographic groups. 

Currently, PBC has a significantly higher prevalence in females. More specifically, women are up to nine times more likely than men to develop PBC. The reason for this gender bias is not fully understood, but hormonal and genetic factors may contribute.

Furthermore, PBC is often diagnosed in middle-aged individuals, typically between the ages of 30 and 60. While it can occur at any age, the risk increases with advancing age.

There is also evidence to suggest a genetic predisposition to PBC. It tends to cluster in families, and individuals with a family history of autoimmune diseases may have a higher risk of developing PBC.

While these factors may increase the likelihood of developing PBC, the disease can still occur in individuals without these predisposing characteristics. The interplay of genetic, hormonal, and environmental factors in the development of PBC is a complex area of research, and ongoing studies aim to deepen our understanding of these relationships.

Match With Primary Biliary Cholangitis Clinical Trials

Ursodeoxycholic Acid (UDCA)

UDCA is the standard first-line treatment for PBC. It is a bile acid that helps improve liver function, reduce inflammation, and slow the progression of the disease. 

UDCA works by increasing the flow of bile and reducing the concentration of toxic bile acids. This helps to alleviate the damage to the bile ducts and liver cells. At the same time, UDCA offers anti-inflammatory benefits, which can control the progression of inflammation. 

Not all individuals respond adequately to UDCA, but it remains a cornerstone in the management of PBC.

Obeticholic Acid (OCA)

In cases where UDCA alone is insufficient in controlling the disease, healthcare providers may consider additional therapies or interventions, such as obeticholic acid. 

OCA acts on FXR, a nuclear receptor that plays a role in regulating bile acid synthesis and transport. By activating FXR, OCA helps improve the flow of bile, reducing the accumulation of toxic bile acids in the liver. This is crucial in PBC, where the immune system attacks the bile ducts, leading to inflammation and impaired bile flow.

Symptom Management

In addition to UDCA and Obeticholic Acid OCA, several other medications may be considered, depending on the individual’s response to initial therapies or the stage of the disease.

Fibrates, such as fenofibrate, are sometimes used in combination with UDCA to address elevated cholesterol levels and improve liver function in individuals with PBC. These work by reducing the production of cholesterol and triglycerides, thereby helping to manage the metabolic aspects associated with PBC.

Budesonide, a corticosteroid, may also be prescribed to alleviate symptoms of fatigue and pruritus. While corticosteroids have anti-inflammatory properties, their use is carefully monitored due to potential side effects, and they are typically reserved for specific situations where other treatments are not effective.

Ultimately, the choice of medication depends heavily on individual factors such as disease severity, response to previous treatments, and the presence of other health conditions. 

Liver Transplant 

In cases where PBC progresses to advanced stages with cirrhosis and liver failure, liver transplantation may be considered. This involves replacing the damaged liver with a healthy liver from a deceased or living donor.

Individuals with PBC need to work closely with their healthcare team to develop a personalized treatment plan. Additionally, ongoing communication with healthcare providers ensures treatment plans are properly adjusted as needed to optimize outcomes.

Match With Primary Biliary Cholangitis Clinical Trials

Have you been diagnosed with primary biliary cholangitis? Do you have health conditions that are related to or triggered by PBC? 

If so, you may qualify for clinical studies to help researchers learn more about PBC and how it affects our long-term health. Scientists are studying many aspects of the condition and the health concerns it can cause, such as cirrhosis

The purpose of these studies may include researching:

  • The relationship between genetics, environmental factors, and PBC
  • Risk factors for PBC and other autoimmune conditions 
  • New treatments for PBC

Participating in these studies benefits both you and future PBC patients. If you’re interested in participating in a trial, reach out to us today to learn more about opportunities near you. 

If you have a loved one struggling with PBC, you may be wondering how you can support them throughout their experience. There are many ways you can help a friend or family member, including:

Medical care

Helping Them Find Medical Care

Finding the right medical care can be a challenge, especially during the initial stages of PBC. If your friend or family member has expressed difficulty finding a doctor, insurance, or medication, take time to help them with the necessary research. 

You can also help by accompanying your friend or family member to appointments and providing care during any procedures if that is something they would appreciate.

Empathetic listener

Being an Empathetic Listener

Living with primary biliary cholangitis can be very stressful at times, especially if it results in complications such as cirrhosis. Lend an ear when you can, and give your loved one plenty of opportunities to vent about their experience. 

When speaking to your loved one, let them guide the conversation and avoid sharing advice unless it’s requested. Sometimes, they’re not looking for input – just a person to hear them out.

Educate yourself

Educating Yourself 

Unless you’ve experienced PBC yourself, you may not have a concrete understanding of what people go through. To support your loved one better, do some homework and educate yourself on the causes and symptoms of primary biliary cholangitis. 

Celebrate achievements

Celebrating Achievements

Although there is no cure for primary biliary cholangitis, it’s still important to acknowledge and celebrate accomplishments throughout your loved one’s treatment journey. Even small daily wins can boost morale and help your friend or family member feel supported. 

Match With Primary Biliary Cholangitis Clinical Trials