Primary biliary cholangitis: What does successful treatment look like?

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Adjusting to life with an autoimmune condition often comes with a learning curve — and primary biliary cholangitis (PBC) is no exception.

PBC is an autoimmune condition that impacts small bile ducts in the liver. As with all autoimmune diseases, PBC happens when the immune system “misfires” and mistakenly attacks the body’s own cells and tissues as if they were the harmful pathogens or cancer cells it usually targets. As a result, PBC can inflame and destroy the liver’s bile ducts. Over time, chronic inflammation can cause severe scarring of the liver, referred to as cirrhosis. PBC-related cirrhosis can lead to liver failure.

For many people, a PBC diagnosis may come as a surprise. Not only does PBC progress slowly over many years but more than half of people diagnosed with PBC don’t report any symptoms. And for those that do experience symptoms, they may be mild, such as fatigue, itchiness or mental fogginess. Dry eyes and dry mouth can occur in people with overlapping autoimmune conditions such as Sjogren syndrome. As PBC progresses, however, symptoms may become varied and more severe.

Although scientists don’t yet understand why PBC develops or how to cure the condition, there are effective treatment options that can delay progression of the disease, according to Mayo Clinic expert and liver specialist Kymberly D. Watt, M.D.

Understanding what successful treatment looks like — and how long it takes — can help you take a more active role in your care and keep your health on track.

Defining successful PBC treatment

There are now a number of treatment options for PBC, according to Dr. Watt. Treatment choices depend on various factors, including how you respond to initial therapy, as well as the symptoms and severity of your PBC. For most people, a treatment plan involves symptom management, lifestyle changes, medication, and monitoring for or treating potential complications.

According to Dr. Watt, successful treatment is generally measured three ways — through clinical, biochemical and histological improvement. “Clinical improvement is defined by the resolution of symptoms, so with PBC that would include things like reducing the severity of itching, fatigue or body aches,” says Dr Watt. Biochemical and histological improvements, meanwhile, simply mean changes that can be measured through blood test results or observed directly under a microscope of a liver biopsy, respectively.

Your healthcare team will likely take regular blood tests to monitor certain biomarkers and determine a treatment’s efficacy. Dr. Watt says levels of alkaline phosphatase, a biomarker associated with liver inflammation, is often used to assess how treatment is going.

“In general, although normalization of alkaline phosphatase is ideal, a reduction to less than roughly 1.5 times the upper limit of normal is considered an acceptable response,” says Dr. Watt. “These numbers are generally associated with slowed progression of disease.”

However, effective treatment isn’t defined solely through test results or crunching numbers — it’s also about feeling good, maintaining your quality of life and staying motivated to follow your treatment plan.

“Since there is no cure for PBC, it’s important to make the most of the options available and stick to your regimen. It can be easy to slip out of the habit of taking your medication, skip talking to your doctor, or miss exams here and there since many people with PBC do not have symptoms to remind them,” says Dr. Watt. But early, consistent treatment is critical to slowing the progression of PBC — and for living as well as possible for as long as possible. With proper treatment, many people can effectively manage PBC for years or even decades.

How long does it take to see treatment results with PBC?

Your exact timeline depends on the medication and how your body responds to treatment. But in general, Dr. Watt says it takes several months to see improvement.

Ursodeoxycholic acid (UDCA) is typically the first line treatment. UDCA is a medication that can help improve liver function, reduce scarring and move bile through the liver. “It can take several months before a biochemical response is seen in the body,” says Dr. Watt. “After 6 to 12 months, if your treatment goals aren’t met with UDCA, your doctor may consider adding additional medications. Options can include one or more of these Food and Drug Administration (FDA)-approved drugs: obeticholic acid (Ocaliva), seladelpar (Livdelzi) and elafibranor (Iqirvo). Fenofibrate (Lipofen, Triglide) also may be used off-label, meaning that they’re not FDA approved for PBC specifically but studies show improvement in PBC.” Again, it can take several months to see improvements. 

As medications take effect, Dr. Watt says many people may notice their physical symptoms slowly getting better as well. “As with any medication, symptoms tend to improve when the underlying disease improves. In the case of PBC, the main symptoms tend to be fatigue and itch,” says Dr. Watt. “Fatigue, in particular, is exceedingly hard to treat other than treating the underlying disease, though maintaining an active lifestyle, healthy diet, and mindful sleep schedule can all help manage tiredness.”

For some people, itching can be a persistent issue and challenging to manage even when the disease itself is being treated effectively. If itching does not improve — or simply doesn’t improve enough — Dr. Watt says there are other medications that may be used. Likewise, for people experiencing chronic dry eyes or dry mouth, there are several nonprescription and prescription options to help alleviate these symptoms.

Key steps for managing PBC

When it comes to effective, successful PBC treatment, there are two main takeaways.

First, it’s critical to be an active, committed participant in your PBC treatment plan by communicating with your healthcare team, following up with exams and taking all medications as prescribed — even when you don’t have symptoms.

Second, it may take some time before you see — or feel — the results of your treatment. Try to stay patient as you and your healthcare team learn what’s most effective to manage your PBC.

If you have questions about your PBC treatment goals, talk with your healthcare team about your options.

Mayo Clinic does not endorse companies or products. Advertising revenue supports our not-for-profit mission.

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