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Dr. Hirschfield:
Hello. This is CME on ReachMD, and I'm Dr. Gideon Hirschfeld. I am a hepatologist based in Toronto, and I look after over 500 patients with PBC. Our mini lecture today will focus on the risk stratification of PBC.
What I'd like to get across to you is the real importance of making a clear and confident diagnosis of PBC so that all patients can be offered first-line therapy with ursodeoxycholic acid. To make that diagnosis, you can look at the patient's blood tests, symptoms, and autoimmune markers. And a patient with cholestasis and antimitochondrial antibodies can be diagnosed confidently and clearly with primary biliary cholangitis. There may be times when other tests are needed, but generally speaking, the diagnosis is straightforward, and importantly, with a straightforward and clear diagnosis, you are in a position to start your patient's journey on effective treatment. All patients diagnosed with PBC should be offered ursodeoxycholic acid, because that is the first-line treatment.
However, not everyone benefits from urso completely, and we need to be cognizant of making sure that we are preventing end-stage liver disease in all our patients. To do that, we really need to understand risk stratification. We need to think about our patients as being different and understand whether they've got low-risk disease or high-risk disease. And from a patient perspective, that includes the risk of developing end-stage liver disease, but also the symptoms that they live with, including pruritus.
So when I see a patient with PBC, I'm thinking to myself, not only have I made a clear diagnosis, not only have I made sure that I will offer them treatment with ursodeoxycholic acid, but do I understand this patient's PBC journey for the future? Are they symptomatic? Do I need to worry about itch and how I'm going to manage that? What are their risk factors for progressing to cirrhosis? How are they starting their disease? Do they have early disease, or do they already have evidence of fibrosis?
So for my patients, I'm looking at pretreatment disease stage and risk stratification as being very important. But of course, I will be optimizing my risk stratification later on after they've been on ursodeoxycholic acid for some time. I know that a patient who is under the age of 50 has a 50% chance of not responding adequately to UDCA. I know that patients who have advanced fibrosis, as measured, for example, by elastography or sometimes liver biopsy, will not respond to UDCA as effectively. I know that patients who are very itchy and have a high alkaline phosphatase are also patients who may not get the most benefit from UDCA and who may need better drugs in due course.
Therefore, when I see a patient, I need to look at the patient in front of me, their symptoms, their blood tests, their imaging, such as an ultrasound looking at their liver morphology, their spleen size, and usually something like a FibroScan which gives me some information on the degree of fibrosis.
With that information I already have, at the very beginning of their journey, risk stratification to make sure that I’m always considering, is my patient on the best treatment to prevent end-stage liver disease? And is my patient on the best treatment to manage their quality of life?
Therefore, today, my key takeaways of this important part of the journey of PBC are, please diagnose PBC with confidence because this opens up the opportunities for your patients to get the best treatment. Offer UDCA to all your patients in a weight-based manner, because UDCA works; it doesn't work for everybody.
And think about what is the chance that your patient won't be a sufficient responder to UDCA and/or is symptomatic, and that may impact the choice of second-line treatments, which are all about preventing disease progression and maintaining quality of life. And we know that some of these risk factors are easily measured and easily acted upon.
Thank you for tuning in to this brief discussion. I hope it will be helpful in your practice.
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