Recent advances in primary biliary cholangitis (PBC) care have resulted in a new focus on personalized care and symptom management. Tune in to learn from patient advocate, Maria G. Morais, RN, how you can integrate patient insights and priorities into treatment plans for PBC.
Recent advances in primary biliary cholangitis (PBC) care have resulted in a new focus on personalized care and symptom management. Tune in to learn from patient advocate, Maria G. Morais, RN, how you can integrate patient insights and priorities into treatment plans for PBC. Topics covered include:
Presenters:
Maria G. Morais, RN
PBC Transplant Patient
VP Patient Advocacy
Canadian PBC Society
Toronto, Canada
Link to full program:
https://bit.ly/44ySoL3
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This transcript was automatically generated from the audio recording and may contain inaccuracies, including errors or typographical mistakes.
Zachary Schwartz (DCE): Hello, and welcome to the Clinical Care Options podcast. I'm your host, Zachary Schwartz. In today's episode, we hear from a PBC patient advocate, Maria Morais, who is also a registered nurse. We'll hear her perspectives on how you can integrate patient insights and priorities into treatment plans for people with PBC.
To follow along with the slides, visit the show notes for this episode. Now let's get started.
Maria Morais (Canadian PBC Society): In PBC, we have two treatment goals. The first one is to prevent poor clinical outcomes, and the second is to improve overall quality of life. We want to prevent cirrhosis. We want to prevent hepatic decompensation, liver transplantation and eventual liver-related death. We also want to improve quality of life through a decreased itch and fatigue, recognizing that all patient journeys are different and experience PBC differently.
Let's look at the Canadian PBC Society survey that was done over a period of time in 2024, with a membership survey of 377 patients out of 875 that were sent out the survey opportunity. And it was to assess how patients with PBC are dealing with physical emotional challenges, how they're dealing with symptoms, where they're seeking support, and how they're dealing with uncertainty about their future.
We see that people living with PBC have other chronic diseases. So not only PBC. Of significance, PBC patients reported that they have one or more chronic conditions. We also recognize that there are greater than one - in this study survey, we had 197 patients, or 52% that had more than one. So obviously, we can be sure that PBC patients are dealing with many more complicated chronic conditions that also impact their quality of life, their - and their emotional stages, looking at osteoporosis, digestive disorders, thyroid disease, anxiety and depression as the major reported ones in this survey.
In addition, the comorbidities were also reported, where patients reported 62 other chronic health conditions. And these looked at things like cancer, arthritis, heart disease, chronic kidney disease, CREST, and just to name a few. In addition, we looked at what are patients that are experiencing PBC, what do they have to say? What are their quality of life statements and how is it affecting from itch, fatigue, the brain fog, some social isolation and anxiety?
So looking at some of the patient voices around itch, let's hear from a patient where they say, “Before diagnosis, I was extremely itchy to the point of being suicidal. Nightmare. Itching is horrid. Sitting around with ice packs on your body is embarrassing, and to shower is the same. And having severe itch is depressing. Looking at isolation, it's terrible. The fatigue and brain fog. Joint pain is like nothing I can describe and people just don't understand when you just can't function. At times I feel like a hypochondriac. I can't imagine living with this forever.”
So there are a number of patient voices that really describe what it's like to live with PBC from a symptom perspective.
We also asked, what improvements would you like to see for new treatment? Now, thinking about the landscape with new treatments on the horizon, some are already available to some jurisdictions. It is very consistent with what patient voices had to say around what they want to see for new treatments, and those being symptom management around the fatigue and itch and improvements in actual medication coverage and treatment or reduction of adverse effects.
We also saw that of those that were participating in the survey, 93% were not currently participating in clinical trial. So it's good news that the landscape of new developments in treatment certainly matches the expectation of patient population.
Now, when looking at defining PBC care regulate biochemistry, PBC treatment aims to reduce or normalize alkaline phosphatase and bilirubin, which we know is associated with positive long-term outcomes. We want to emphasize the prompt diagnosis of PBC personalized care so that we can prevent end-stage liver disease and target a normal biochemistry, managing those symptoms and looking at equity issues for access to treatment so that we can pursue in excellence of care.
How to achieve this - these goals really is from short to long term efforts. Firstly, to obviously treat the disease by stratifying risk and beginning the treatment as soon as possible and setting those goals for that unique patient. From there, we can improve symptoms and liver biochemistry through looking at the bloodwork, assessing and considering the patient's quality of life, and looking for opportunities to implement second-line treatment for higher risk patients if first-line is ineffective, to then normalize ALP and bilirubin so that the patient can then stabilize and respond appropriately, so that we can have improved outcomes of liver and assess any additional therapies that are required. And obviously, listening to the patient to look at quality of life.
Now looking at life before treatment. So historically, we know that ursodeoxycholic acid was the primary treatment for PBC, and we do recognize that some patients don't respond adequately to ursodiol, which could mean that ALP does not normalize in the liver. And so obeticholic acid then became available as a second-line therapy. But we also know that there's limitations on using this treatment when cirrhosis is present.
Historically, patients have feared disease progression that didn't have a lot of options of treatment. They feared worsening of symptoms and eventually cirrhosis and requiring a transplant. There's been limited awareness of this rare liver disease among healthcare providers that are often have resulted in delayed diagnoses.
However, we know that recent advances in PBC have improved diagnostic tools. And with that, we've seen earlier diagnoses. So we want to look at that continued trend. We know that there's a new focus on personalized care and symptom management. So as I indicated, PBC patients experience PBC disease in a variety of different ways. However, we know that the most common are itching and fatigue, and we really want to look at reducing the burden of these symptoms on patients. We want to also increase emphasis on patient-reported outcomes and mitigating impact of quality of life with these new advancements.
Now looking at some of the newer agents on the scene, they are approved as combination therapy with UDCA or as monotherapy if patients are unable to tolerate UDCA. So the first category is FXR agonists, and OCA being one in that category that has been used as a second-line therapy, which we know tends to reduce ALP and reduces bile production, improving bile removal from the system. However, it does not seem to improve pruritus.
The second category of PPAR agonist, elafibranor and seladelpar have been on the market in some jurisdictions, and they have been both proven in - in research study to decrease ALP and some potential to improve pruritus. Certainly these new treatments have impacts on our patient population, where we're seeing a greater sense of empowerment and feeling of hope on the horizon.
So, for many, many years, new treatments have not been in development. So it's an exciting time to see so many new medications and treatments and studies come out. Patients have the opportunity now to be more proactive when they're engaging with their healthcare team, to be able to ask questions about potential entry into additional therapies. We still know that early diagnosis with our earlier treatment initiation can certainly lead to improved biomarkers and better patient-reported outcomes. And we know that better management of symptoms like fatigue and itching are the drivers of some of the pharmaceuticals to manage the patient's symptoms.
Patient-centered care is an important aspect of the new area of development, where there's more attention to patient - listening to patient voices and patient needs and fulfilling those unmet needs. And ultimately, a quality of life that is high will impact on reducing emotional and physical ailments and improve the overall health and well-being of PBC patients.
However, there still remains a few challenges. We know that inequities in access to treatment still persist across healthcare systems, depending where you are. And as a patient accessing healthcare, patients’ access to care and coverage for new treatment options is certainly not universal.
Increased education and knowledge of current and new treatments among healthcare providers must remain a priority. We need to have healthcare providers continue to learn about PBC and treatment and diagnosis as - as early as possible.
Healthcare providers must also continue to improve the access to new treatments and be knowledgeable about what's out there so that they can prescribe.
However, ultimately, despite the new developments in treatments and managing PBC, we still would like to have our ultimate goal remain as a cure for PBC.
Zachary Schwartz: Thank you to our faculty, and many thanks to you, our listeners, for joining us. To learn more from this program on PBC, click on the link in the show notes. And to get access to all of our new podcasts, subscribe to the CCO podcast channel on your favorite player. Thank you, and we'll see you on the next podcast soon.