Personalizing MPN Disease Management and Monitoring

After initial diagnosis of a myeloproliferative neoplasm, such as essential thrombocytopenia (ET) or polycythemia vera (PV), patients will undergo an intensive period of monitoring to understand the behavior of their individual disease, said Jennifer Vaughn, MD, hematologist-oncologist and assistant professor in the Division of Hematology at The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute.

Transcript

Since MPNs are a chronic, lifelong disease, what is the importance of patient monitoring and follow-up in managing MPNs? What does that involve?

I think it's very important that patients understand there are really 2 things that we're working on, especially in in the conditions like ET and PV, where a lot of times patients may not be symptomatic and may feel really well and feel like they're going about their daily lives without the disease really causing a lot of issues. One of the things we're trying to do is mitigate their immediate risk of blood clotting, but we're monitoring them long term to see if there's any risk or we're seeing any signs of progression to a more serious condition.

I always tell my patients at the beginning, once they're first diagnosed, it's going to be a little bit of an intensive follow up at the beginning, because part of that is also understanding the behavior of their individual disease. You'll have some patients with PV, for example, who start on phlebotomy, they achieve that perfect balance of iron deficiency and then won't need phlebotomies more than every month or 2 months even 3 months. But then you'll have some patients who remain very proliferative, even despite iron deficiency and aggressive treatment. Those patients may really benefit more from initiation of some other cytoreductive therapy.

To kind of summarize, I tell them at the beginning: expect a little bit more intensive follow up, maybe every few weeks or so until we get an idea of how their disease is going to behave, and we can figure out what it's going to take to be able to achieve optimal count control for them. And then long term, as things sort of even out, and we know what they're going to need, we will see them every 3 months to every 6 months, depending on the behavior of their disease and how well controlled it is on their current therapies.

How are MPNs usually diagnosed and what is the typical time to diagnosis from symptom onset? Do these patients experience delays?

It depends, really, on the type of disease, exactly as we were talking about. Many of the diseases will present in an asymptomatic way, particularly ET and PV—ET probably being the most likely to be diagnosed sort of incidentally I would think. I actually have a clinic that focuses on diagnosis of these conditions, where we will see patients who are referred from the primary care setting for elevated platelet counts, elevated red blood cell counts, and really go through and do an assessment of whether there are other factors that could be causing this versus whether they need to undergo a molecular assessment to see if they've acquired a mutation that that would categorize them as having an MPN.

For those patients, I would say there is a lag time often. I'll see anything from a patient who's had an elevated platelet count for years that's never really gotten high enough to attract the attention of their primary care provider to a patient who has a more aggressive sort of phenotype, where they'll present with very high white blood cell count that triggers the provider to make the referral or to start the workup for an MPN fairly quickly. Then the other subset, unfortunately, are the patients who do present with a thrombotic event, which is, certainly, the less-ideal situation. But there is a subset who will present with sort of unusual thromboses or arterial events at a young age—things like that, that fall outside of the standard presentation of these types of disorders and end up leading to a diagnosis as well.

I think there's a lot more awareness now in terms of looking for these disorders, both in the area of patients presenting at a young age or with unusual sites of thromboses, to be looking for MPNs. But also, I think, among providers as the WHO [World Health Organization] reduced the sort of cut off, cut offs for ET for platelet counts and for red blood cell counts a few years ago, back in 2016, I think there is a lower threshold in the primary care community to refer for evaluation if there's not an obvious other cause of these sort of cytoses, as we call them.

Then another interesting phenomenon that that cannot be discounted is patient access to their own records, and patients taking charge of their own care and being advocates if they see laboratory abnormalities that are persisting. I have had several patients come to me because they were the ones that noticed the abnormal lab value in their chart and brought it to the attention of their primary care provider for referral. That's been a refreshing outcome of these new laws that that make medical charts readily available to the patients, how they can kind of take control and get these things worked up themselves.