Primary Biliary Cholangitis: Treatment Approaches and Options

Ah, the liver. It’s an organ that tends to get overlooked in terms of how crucial its functionality is to our bodies (hint: it does a lot more than process alcohol). So, what exactly does it do?

The liver is an important organ and gland usually located underneath the ribs on the right side of the abdomen. While the average liver performs hundreds of jobs, two of its most important roles are filtering your blood and producing bile — a fluid that aids in digestion.

Your bile ducts are small tubes that move — you guessed it — bile from your liver to your gallbladder for storage, and from your liver and gallbladder to your duodenum, the first part of the small intestine. Once in the small intestine, bile aids digestion by breaking down the fats in the foods we eat.

What is primary biliary cholangitis (PBC)?

Bile duct system inflammation — called “cholangitis,” from the Greek roots “chol-,” meaning “bile,” “ang-,” meaning “vessel,” and “-itis,” meaning swelling or inflammation — can cause liver scarring and permanent damage, also known as cirrhosis. One illness that causes bile duct inflammation is primary biliary cholangitis (PBC), a chronic autoimmune disease that causes a person’s immune system to attack the healthy bile duct cells in their liver. PBC specifically involves the intrahepatic bile ducts, the bile ducts within the liver itself, and leads to the eventual destruction of them. Without functioning bile ducts, bile can’t be transported out of the liver. This buildup of bile is what leads to liver damage as scar tissue replaces healthy tissue and the liver is no longer able to function properly.

What are the symptoms of PBC?

PBC typically progresses slowly, and people with the condition may not have any symptoms in the early stages of the illness. When people do develop symptoms of PBC, they usually experience fatigue and itchy skin — called pruritus — first. In addition to those more common symptoms, other signs and symptoms of PBC can include the following:

Pain on the upper right side of the abdomen
Joint and muscle pain
Symptoms of other autoimmune diseases, such as dry eyes and mouth caused by Sjögren’s syndrome
Darkening skin color and/or jaundice, a yellow discoloration of the skin and eyes, due to worsening liver damage
Edema, which refers to swelling in the legs and feet, and ascites, extra fluid accumulation in the abdomen, can also indicate disease progression

How is PBC diagnosed?

Due to a lack of initial symptoms, PBC is often diagnosed based on incidental findings, meaning that the disease is found by accident. PBC may be suspected after someone receives abnormal results on a routine blood test evaluating liver function, or while a person is undergoing tests or scans for another condition entirely.

If PBC is suspected, further tests will be conducted to confirm the diagnosis, rule out other conditions, and determine disease severity. These tests usually start with an antimitochondrial antibody (AMA) blood test. Next up is a liver biopsy followed by imaging studies, such as ultrasounds.

Who treats PBC?

Gastroenterologists, who specialize in treating disorders of the gastrointestinal tract, and hepatologists, who focus specifically on conditions affecting the liver, can both treat PBC. If you or someone you care about is in the market for a new specialist, we’re passing along this evergreen reminder: When it comes to putting together a health care team for a chronic condition, it’s important to find providers who are knowledgeable about your condition — while also willing to learn more — and show you respect as both a person and a patient.

How is PBC treated?

As of now, PBC is incurable. The first line of treatment for PBC is typically ursodiol (ursodeoxycholic acid), a medication that falls under the class of medications called “gallstone dissolution agents.” This medication helps move bile from the liver to the small intestine, thereby reducing the toxic levels of bile that accumulate in the liver of someone with PBC. In some cases, where ursodiol isn’t effective enough, additional medications, such as obeticholic acid or elafibranor, can be prescribed.

Other treatments — including many options that can be purchased over the counter — may be used to manage symptoms of PBC. These include antihistamines for pruritus, artificial tears for dry eyes, and sucking candy or artificial saliva for dry mouth.

Lifestyle changes also play an important role in disease management and the prevention of further complications — as well as improve someone’s chances of making it on the liver transplant waiting list, should the need arise. Doctors typically recommend that people with PBC quit smoking, abstain from drinking alcohol, maintain a body weight that is healthy for them, and follow their health care provider’s instructions, which include attending regular check-ups and follow-up appointments and tests.

If someone with PBC develops complications of the disease, there are medications, supplements, procedures, and lifestyle changes that their health care team may prescribe, perform, or suggest in addition to their treatment for PBC:

A person with high cholesterol may be placed on statins and begin a diet and exercise regimen to lower their levels.
If someone develops Sjögren’s syndrome, then they’ll likely be referred for additional eye and dental exams.
Due to an increased risk of osteoporosis in people with PBC, the managing health care provider may run regular bone density tests and, if bone loss is found, prescribe medications that slow or stop the progression of osteoporosis. Some folks may be told to start taking calcium and vitamin D supplements regularly to prevent osteoporosis.
An impaired liver may lead to deficiencies in vitamins A, D, E, and K. A build-up of scar tissue and inflammation in the liver affects the organ’s ability to absorb vitamins and nutrients from the food we eat. Doctors may recommend dietary supplements for their patients with PBC.
Cirrhosis can also lead to portal hypertension, which occurs when scar tissue in the liver builds up to the point that blood cannot flow at a normal rate. This causes hypertension — aka high blood pressure — in the portal vein, the blood vessel that transports blood from several organs to the liver. Depending on the severity of the portal hypertension and if any complications have arisen, the first treatment step may be medication to lower blood pressure.
An important part of PBC management is monitoring disease progression, which includes regular screenings for liver cancer, especially if a person with PBC has developed cirrhosis. These screenings typically involve blood tests, ultrasounds, and CT scans.

If PBC causes severe enough cirrhosis or progresses to liver failure, then a person with PBC may be placed on the waiting list for a liver transplant. People with PBC usually do well after receiving a liver transplant, although there is a chance that the PBC will return, which is not unheard of in autoimmune diseases. That being said, PBC after a liver transplant tends to progress more slowly than the initial disease course.

Of course, no two people with PBC are the same, so be sure to work with your health care team to find a treatment plan that works for you!

What’s next for patients living with PBC?

While there are currently only three FDA-approved medications for PBC, there are many clinical studies examining medical interventions for PBC. Who knows what the treatment landscape for PBC will look like even two years from now?