Educated Patient® MPN Summit Essential Thrombocythemia Panel: May 20, 2023


Watch Dr. Lucia Masarova, Dr. Laura C. Michaelis and Celia Militz answer questions about essential thrombocythemia during the CURE® Educated Patient® MPN Summit.

This panel was moderated by Brielle Benyon and included Dr. Lucia Masarova, from MD Anderson Cancer Center, Dr. Laura C. Michaelis, from the Medical College of Wisconsin, and Celia Miltz, from the MPN Research Foundation.

Benyon: So to start, Dr. Masarova, can you distinguish between (Essential thrombocythemia [ET])-related migraines and simply a continuation of migraines and a patient who has a history of them?

Masarova: That's a very good point. So surely, we will have patients that had migraines preceding their diagnosis. We have to make sure that we gather all the information about the migraines from the past, especially making sure that there is no worsened, frequency type side control with the medications before, I'm pretty positive, that neurologists would like to have some scan done to make sure it could be lots of ocular migraines associated with the different events in life and stuff like that. So that is important to know.

To me, what would be interesting at the diagnosis is, has anything changed? Have the migraines differed from what (they were) before? And how does it respond? Has it really changed with aspirin? Does it really respond to something else? Do we dare to change, to do aspirin more? So those kinds of questions are relevant and very important.

Other than that, I don't really think we have actually a tool. So we can imagine or look and then say, “Hey, this is from our ET, we just take aspirin and we’re fine.” Versus we actually blame it on one or the other. It certainly could be that it is aggravated by the diagnosis. I had, unfortunately, lots of patients that have migraines at baseline. And they've kind of complained…But I have also viewed it that the symptoms got better and stabilized. But unfortunately, a few of my patients have just intractable migrants, and they have to take a lot of anti-migraine medication from neurologists, even though the disease with ET is perfectly controlled. However, there is not a direct correlation.

I can tell as we work from the past, if the disease is controlled, there is no symptoms. That's not true. We have seen patients that have completely normal blood count and are healthy, and have still miserable symptoms. So I cannot really 100% tell you, I know how to distinguish them. But what I will be really, really looking at would be whether something's changed in the character and behavior and the response, and whether we must establish treatment for the ET and how everything else it produces look like. For example, there's only migraine that sustained, consistent and intractable and not controlled, but other symptoms disappeared. Well, it's unlikely that the disease is the primary driver of the migraine. But at the same time, it is the driver that happens every time of the year, every time on some event. And we know the triggers as well. It could be triggered by something else, maybe by the underlying bathymetric changes in the disease. So we would maximize treatment for making sure everything else is controlled as much as we can, and then work together with a neurologist to support. But to really tell you which, I don't think we (know) right now.

Benyon: Thank you so much. And my next question is for Celia. So we talked a bit on the importance of clinician communication. Can you speak to the importance of communication between patients and caregivers and their clinicians, especially when it comes to questions that they have side effects they're experiencing and things like that?

Militz: I think patients should realize that the MPN Research Foundation website,, If you go on to that there are a couple of tabs that are really important for patients to look at. And one of the tabs is understanding MPNs and that has a list of ET, PV (polycythemia vera) and MF (myelofibrosis). And the second tab is called Living with Impedance. And then there is also a guideline that has been published, which is also on the website from the National Comprehensive Cancer Network that gives you guidelines for treatment strategies. So if the patient is aware of what the treatment strategies might be, and how it impacts their disease, then they can go to their doctors ask the right questions. In my case, I was the caregiver for my daughter who was diagnosed at the age of 16. Many years ago, I had to be her best advocate, so I had to learn. So the caregivers should also learn to ask the right questions and be their own best advocates for the patients. The patients should become their own best advocates. And there's just a wealth of information on the MPN Research Foundation website for patients, caregivers and clinicians.

Benyon: Fantastic, thank you so much. My next question is for Dr. Michaelis. Is there a time of day that is best to take Hydrea (hydroxyurea) – maybe late in the afternoon or earlier in the day? Does this timing of the drug matter here?

Michaelis: No, the timing doesn't matter. I can't remember exactly if it's required for food or non-food. But usually I tell people, especially initially, they might get a little bit of nausea when they take it. And so sometimes I start it in the evening time and tell them to because that way they could sleep through the symptoms. I do think it's important for whenever you take a new medicine, whether or not it's interferon, Hydrea, any new medication in the first week or so that you take it, just write down any symptoms that you might think are new or associated. Because then you can, first off, understand what's related or not. And sometimes it's good to write down those symptoms before you take it and then afterwards, so “Oh, yeah, I had headaches before I took this medicine, it's not just related to it.” But also, then you can juggle the timing. I do have some people who take it in the morning, and most people, especially with hydroxyurea, a lot of (patients) don't mention a lot of immediate side effects to it.

Benyon: Great, thank you. And this one, I guess I'll throw it out to anybody. Is there any research yet on COVID and long-COVID In patients with ET, and if it increases any risk factors or symptoms?

Michaelis: Well, I can speak a little bit to that. So the the world of long-term COVID research, I think, is only just beginning, I think there is some role for understanding the sense of the inflammation that goes along with COVID and how that causes the body to release additional cytokines or become a little bit more sensitive to the cytokines that are released, meaning that their cytokine receptors, the things that tell you that you're tired, the things that tell you that you're worn out, the things that give you night sweats, you might be more likely to feel those if you went through COVID and had long-COVID, for example. But I think what (that we’re) learning from COVID. And that those features (from) COVID, that lasts a long time, we've only just begun to understand. Most of my patients who had long-COVID got better, did recover. After about six to eight months of feeling that persistent fatigue, almost like people that had a bad mononucleosis, might feel fatigued for a long period of time. But interesting, I think that the science that develops from studying long-COVID may, in the future, be applicable to our understanding of the symptoms related to MPNs. And then maybe some other diseases that are marked by fatigue and a sense of frustration and inability to do things.

Benyon: Thank you so much. Our next question is for Dr. Masarova. Is there an association between mutated allele burden and survival rates? And on that note, is there any benefit of having hematologists and clinicians measure allele burden periodically, say every few years?

Masarova: Very, very good point. I do not think we will be able to measure that around the clock because (they are) expensive tests. We do have some experiences, for example, as Dr. Michaelis said, from interferon and I published that from our own on data where we had few patients with EEG, where our burden completely disappeared. Well, then we were actually able to eradicate a disease. Well, we had a follow up on any coding, they're very deep assessments of bone marrow biopsies and we did indeed have some patients that got cured, so everything's improved. But we did have also patients that had the treatment (and then their disease) returned. So on that note, of course, we always would like to have lower allele burden and lower disease scores than have been shown. Also, as Dr. Michaelis said, in the major PV study where we have other forms of interferon and working up with the allele burden and I'm getting the lower-dose patient had the longest benefits, the best survival, the lower risk of progression of the disease. So definitely, that implies the consequences were the lower we get, the better we can expect.

I think the major point would be easy, doable to achieve and stay. I think that would become a very interesting treatment point, to lead our future to really guide our decisions based on where we are, how the disease looks like, there are different aspects to it as well, in terms of other molecular backgrounds, where we know that we have drugs such as interferon that does target more than active mutated clones. However, it has been also tried in patients with (inaudible), but then we get some studies that suggested that it's not so effective. Regardless, I have patients that are completely clear (after treatment with) interferon. So both are possible.

And the other side, we may have cortical and directed monoclonal antibody, which Dr. Michaelis nicely showed where we actually can be targeting and (more importantly) eliminating the clone. And (does) actually mean we cure completely the disease and (it) worked? That's going to be the case, but I think it is relevant, and it's going to be more and more relevant to us to shoot deeper and cleaner and then get rid of it. But I think we (are all going to learn what kind of implications for the future it is going to have in terms not only the one driver, but the whole conglomerates of the disease backgrounds. So if there are other comutations, other abnormalities that we want to clean? Are we going to have effective agents to do so? And then what is really the directionality to do the cleaning to do right, so we measure those, how often what does that tell us if we decrease it? Are we going to change our approach? Are we going to add on something? I'm hoping we're going to be in the era where we have more effective therapies, very tolerable therapies, as we currently have much more movements in the myelofibrosis field where we add on, if we see it's not ideal responding, and we're going to be able to act on it based on if the algorithm doesn't go the right direction. I hope so. But I'm really going to be looking forward to the future and to see what it stands for us and tells us, but based on other diseases that we had, for example, in CML, we've seen the Philadelphia chromosome, we eliminate that people can get cured, so-called cured, right? And (we) stop the medicines completely, where we eliminate what's driving the disease, and it automatically dies off and people don't need the medicine? Is it going to be that easy in this disease, which is more complex? And we don't have one driver giving us the same disease, when you're going to soon hear about PV and myelofibrosis? Well, JAK3, could lead to all three, why? Why one patient is acting, he didn't have very simple ET, one has a very complicated model of a process. That's something we have to really bebe more alert of and put all of those things into context to see. We kind of imply and hope that the longer the disease burden, the better. And that's what we've seen. However, the long-term implications are still to be learned.

Benyon: Great, thank you. And my final question is for Celia. What is your advice for patients and their loved ones, their caregivers, who are newly diagnosed, and maybe they're not sure where to go? They're unfamiliar with the disease? What's a good first step for these patients?

Militz: That's a very good question. Back when my daughter was diagnosed, there wasn't much on the internet, and a lot (of what) was on the internet was quite frightening. But once again, I suggest that patients and their caregivers try to educate themselves on the MPN Research Foundation, (it) is probably the best site in order to get a better understanding about what the disease is, what it means to have it, how it may be treated. And again, learn about it, educate yourself about it. And then you have the tools with which to talk to your doctor about your disease and whether or not you should be treated. And, if so, what treatment seems to be best. Again, being your own best advocate is my suggestion. And the best way to do that is to go to the MPN Research Foundation website and learn about it and then take your questions to your doctor being an educated patient. That's the most important thing

Transcription edited for clarity.

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