Overview of Primary Biliary Cholangitis (PBC)

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: Hello, and welcome to this Peer Exchange titled, “Advances in Treatment of Primary Biliary Cholangitis.” I am Dr Kris Kowdley. I am the director of the Liver Institute Northwest and am a professor at Elson S. Floyd College of Medicine, Washington State University. Joining me today in this virtual discussion are 4 of my colleagues. Dr Steven Flamm is a professor of medicine at RUSH Medical College and a transplant hepatologist. Dr Sonal Kumar is an assistant professor of medicine and director of clinical hepatology at Weill Cornell Medical College, New York City. Dr Ed Mena is a hepatologist and medical director of the Pasadena Liver Center. He is also president and CEO of California Liver Research Institute in Pasadena, California. Dr David Victor is a specialist in transplant hepatology at Houston Methodist Hospital in Texas.

Today we are going to discuss primary biliary cholangitis [PBC]. We will discuss recent updates in PBC diagnosis, treatment, and monitoring. We will review recently published real-world data and share our approach to personalizing treatment and discuss what is on the horizon for PBC. Let’s get started on our first topic. Dr Kumar, can you provide an overview of PBC, trends and its prevalence, and signs and symptoms of PBC, both hepatic and extrahepatic?

Sonal Kumar, MD, MPH: Sure. PBC is an autoimmune disease, where you get destruction of the small intrahepatic bile ducts of the liver. That leads to cholestasis, which can lead to liver damage, and ultimately, cirrhosis. If you look at the prevalence of PBC, it’s a rare disease, but the prevalence over the last decade or so has increased, while the incidence has pretty much stayed the same, maybe a slight increase. It’s really the prevalence that has gone up, and we think that’s due to more disease awareness, which leads to more diagnosis, especially in earlier stages of disease.

So, as we’re learning more about the disease and there’s more disease recognition, gastroenterologists, hepatologists, and primary care providers are all testing for it, we’re diagnosing these patients when they are in an early stage. On top of that, we think the prevalence is increasing because, with ursodeoxycholic acid and obeticholic acid providing pretty good treatment options for these patients, we’ve had better disease control, so people are living longer.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: That’s a great summary and introduction. From your perspective as a hepatologist, what are some of the signs and symptoms of PBC, both hepatic and extrahepatic, you commonly see in your practice?

Sonal Kumar, MD, MPH: I think a lot of people can be asymptomatic from PBC, but it’s one of the liver diseases that commonly comes with symptoms. I would say the most common symptoms I will see in my practice are pruritus and fatigue. Then, we have to remember that PBC is associated with other autoimmune conditions as well. Sometimes you’ll get the symptoms from those other diseases. Sometimes you’ll see joint pain, sicca complex, dry eyes, or dry mouth. Those are probably the major ones that I’ll see.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: Anybody want to comment about whether that’s similar to your experience? I would say, in my experience, about 50% to 60% of patients have no symptoms. The ones who have symptoms, they are frequently nonspecific. It’s difficult to identify the symptoms as being necessarily related to liver disease. Ed, any comments on that?

Edward Mena, MD: I agree. I think most of our patients come for a consultation usually because of abnormal liver enzymes. Then for those who have symptoms, I agree, it’s usually fatigue and itching. The itching they describe it as a total body itching. Usually, it can keep them up at night and can cause difficulty with sleep.

David Victor III, MD: I agree. I think insomnia is one of the most prominent symptoms that impacts the quality of life of these patients. I do agree that a lot of times they are presenting based on laboratory abnormalities, but when asked, their symptomatic effects of the disease are that they overall generally don’t feel well in comparison to how they would like to feel. A lot of that does stem from the insomnia associated with their itching or just generalized fatigue.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: David, that’s a great point. One of the things I’ve noticed is that patients will frequently underreport their symptoms, because they’ve gone to their primary care doctor, reported fatigue, and been diagnosed with depression. If they talked about pruritus, somebody maybe gave them a referral to a dermatologist. What do you do, Steve, to illicit those symptoms from the patient? Because they may get tired of reporting those symptoms and having them be dismissed.

Steven Flamm, MD, FAASLD, FACG: Interestingly, in my experience, a lot of the patients don’t even report the symptoms. They’ve been fatigued for so long that for them it’s just a normal aspect of life. If they’ve had pruritus, they’ve had it for so long they don’t even report it until you actually ask them, and it can be very severe. Sometimes I see excoriations on patients who don’t tell me they have pruritus when I go through the whole list of symptoms. This is until I actually ask them if I have pruritus. They don’t always relate these long-standing symptoms to chronic liver disease, so you need to ask, because some of these symptoms, such as pruritus, have effective therapies, which I think we’re going to touch on shortly.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: That’s a great point.

Transcript edited for clarity