Discussing MPN Treatment Options


CURE spoke with Dr. Gabriela Hobbs, on behalf of the MPN Research Foundation, about currently available treatment options, as well as those under clinical investigation, for patients with myeloproliferative neoplasms.

Kristie L. Kahl: To start with MPN, when should patients consider treatment versus monitoring?

Dr. Gabriela Hobbs: That's a great question. So the first thing is that not all MPs are the same. And so some patients with MPN may require treatment right away, whereas some patients with MPN may never require treatment. And so I think the two main distinctions that I'd like to make is between patients that have polycythemia vera, or PV, and essential thrombocytosis, or ET, and patients with myelofibrosis, and the treatments really are very different between PV and myelofibrosis. So generally speaking, for patients that have PV and ET, we consider starting treatment for a few different things, the most common reason for starting treatment is not abnormal blood counts as much as it is risk of having a blood clot. So I think the thing that's most important to remember is that we are giving medications to normalize blood counts in order to prevent patients from having issues with blood clots, and less commonly, but also important with bleeding. So patients that are considered at high risk for having a blood clot are generally considered candidates, or it's generally recommended that those patients take medications to improve their blood counts to prevent them from having a blood clot. Now, there's other types of treatments that don't, that aren't medications to normalize blood counts that are also an important part of the care of patients with PV and ET. And those include aspirin for most patients, except for some very low risk patients with knee pain. And then for patients that have place I think they're basically any patient that has PV should also get phlebotomy. And phlebotomy is the medical word for removal of blood basically. So it's like getting a donation of a blood. So giving up giving a donation of blood. And that is to normalize blood counts, especially the hematocrit, which is a red blood cell measure. And, and that's regardless of whether or not your high risk or low risk or your age or anything like that patients with PV are recommended to have to buy and so that kind of comes with the diagnosis, if you will. And that's usually you know, the hardest part about that is that you have to go somewhere and get a needle and get blood removed. And so sometimes patients feel a little dizzy, after you know, getting rid of that unit of blood, and then for PV and ET outside of considering medications to reduce the risk of having blood clots, some patients also have a lot of symptoms. And so talking to your doctor about symptoms is really important. Because sometimes it's just symptoms alone are a reason why some patients need to have medications to normalize blood counts. One handy tool if you're not sure if your symptoms are related to the MPN is to look online for the MPN staff or myeloproliferative neoplasms symptom assessment form. It's a super easy 10-point questionnaire that asks about common symptoms that patients with MPNs may have. And so that's something that you can talk to your doctor about, if you feel like a lot of the symptoms that you have are related to the MPN. And then there's myelofibrosis, which is really a little different than then PV and et. And so for myelofibrosis, we use several calculators to determine if a patient is in the lower risk category like lower risk of having any complications from their myelofibrosis, or in the higher risk category.

And, and in general, if patients are kind of low risk, and they don't have symptoms from all fibrosis, and their blood counts are okay, and their spleen doesn't bother them. Those patients can really just be observed and they can be observed for many, many years. So starting treatment just because a patient has a diagnosis of myelofibrosis is not the right thing to do. If patients have symptoms from their myelofibrosis, such as fevers, night sweats, itching, unintentional weight loss, and then pain or discomfort from having a large spleen, and those patients should consider talking to their doctor about starting a medication to improve those and for that we have to put medications that are approved, that are what are called JAK inhibitors, and one is fedratinib and the other one is ruxolitinib. So those two are what are called JAK inhibitors, and they help with that. Okay, and then the next thing to consider is if patients are in the higher risk category where they have more likelihood of having complications from their disease, then a bone marrow transplant is really something that should be considered. And this consideration and although it's scary should be considered early in a patient's treatment course as opposed to Later. So that's something that I think is really important to consider early. And then lastly, if patients have issues with low blood counts, then they may consider medications to help boost those blood counts with medications such as erythropoietin, which is a hormone that is that can be given to patients and among others, other treatments that don't necessarily always work that well. So those are kind of the broad broad categories.

Kristie L. Kahl: So with that, what are the currently approved treatment options that are available to patients with MPNs?

Dr. Gabriela Hobbs: PV and ET kind of are more similar and their management and also in the medications that are approved for them. And myelofibrosis patients are kind of a little bit in a different category. It's all start with PV and ET. So for a PV patient or ET patient that needs to be on medications to lower their blood counts, also known as cytoreduction meetings have reduced the amount of cells that you have in your body. There are basically two main treatment options that are considered frontline. One of those is a pill called hydroxyurea, that pill has been around for decades. It's usually pretty easy to tolerate. And, and that's one of the options that we have. The other option is a group of medicines called interferons. Long time ago, we used to have interferon that was that was really difficult to tolerate. Now we have a better interferons, one that is called Pegasus or pegylated interferon. And the nice thing about those is that you only have to take those once a week, and they're actually much better tolerated. And then there's a new interferon that hopefully will be approved in the United States in the next year or so called rogue interferon is already approved in Europe. And so for frontline, either hydroxyurea or interferons are acceptable treatment options. And that's something that I spend a lot of time talking about, and something that we in the MPN world are really interested in. And there's really been a lot of interest in knowing you know, who should get pegylated interferon in general. So there's a lot of interest in the field related to interferon, especially now that we have robotic interferon, as some of the studies show that maybe over time, the level of the mutations that patients have maybe decreases over time in these patients. So that kind of makes us think a little bit like should we be using these medications earlier, and in patients that we used to think were lower risk, maybe they need to be treated with medication earlier. So that's something that's evolving, that would say, so, so those are kind of the two medicines that are available upfront. And then for patients that have polycythemia vera, that don't do well with hydroxyurea, or interferon, there's also ruxolitinib, or Jakafi, which is a JAK inhibitor, it's approved specifically for patients that don't tolerate hydroxyurea, or they don't have their symptoms controlled of hydroxyurea. And so, geography is a wonderful option, especially for those PV patients that have a lot of itching. Itching is a really difficult symptom to control. And Jakafi really works very well for those patients. And so those are kind of like the general treatment options that are available for ET and PV. For myelofibrosis, we have, like I mentioned previously, to JAK inhibitors that are approved fedratinib and rouxel Platinum. And those are both approved to be used in the upfront setting. And the reason those medications should be given is that the patient has splenomegaly, or a big spleen, and symptoms of their disease. And then the other treatment that we use a lot is a not a lot but should be used more frequently as a bone marrow transplant or stem cell transplant. We also have some medications like I was mentioning before to improve red cells such as erythropoietin stimulating agents, it was appointed as a hormone that's normally made by our kidneys, and we can just kind of give extra doses to boost the production of red cells. And there's a few other agents that are like medications and mimic testosterone that can sometimes help improve the red cell, or medication called the lid, amide or lenalidomide, that can sometimes be given to help improve what counts. Outside of these medications. There are a ton of new medications that are being investigated. So I think it's actually a really exciting time in the world of MPN right now, because there's tons of clinical trials with a lot of new and exciting drugs. And a lot of these drugs are actually in like a more advanced clinical trials. And the reason why that's exciting is because that means that they're closer to getting approved. So there's two JAK inhibitors that will hopefully be approved soon. One called Mama latinum that helps to improve red blood cells, which is really interesting because you know, if a patient has mild fibrosis and they also have anemia, then then they have a medicine that can both help with the symptoms of mild fibrosis, but also the anemia. So that's really exciting. And then there's another JAK inhibitor called crithmum that hopefully will be approved soon as well. It's specifically being studied in patients that have their Low platelets, which is also important because the other medicines are usually not given to patients that have very low platelets, outside of the medicines that are called jak inhibitors. There are a few other medications that are being studied in combination with jak inhibitors. So there, there are several, several different drugs that are undergoing trials. And so that's something to look forward to for sure.

Kristie L. Kahl: Why is it important for patients to make sure that they're learning more about their treatment options? And how can patients learn more so that wa, they're the most knowledgeable and can participate in that treatment decision?

Dr. Gabriela Hobbs: That's really important. So I think, as a patient with a diagnosis, it's rare, I think it's really difficult to feel comfortable with their diagnosis and to feel in control. You know, I think the when, when folks get diagnosed, there's a sense of loss of control where like something happened, that was, you know, not something that anybody asks for. So I think a part of gaining some of that control is inflammation, I really am a big believer and patients being informed patients understanding what their disease is having a name, like, you know, I've met so many patients that are like, Well, you know, I know that I have these weird platelets, but nobody's ever given it a name. And so I think even being educated in terms, like knowing what the name of the disease is, right, is really important. And just facing it by calling it what it is, you know. And so, thankfully, in the MPN world, there's lots of great foundations like this one that can help patients, you know, get reliable information. And I think that's one of the things that's really difficult about this, the world we live in, where there's so much information out there. And it's hard to sometimes get reliable information. So I would say, you know, going to the NPN Research Foundation, the National Cancer Care Network guidelines, American Cancer Society guidelines, a reputable sources like that, the MPN advocacy or International Foundation, etc. Like going to sources where they are written by reputable people I think is really, really important to learn about what the diseases are, what trials or treatments exist, what potential complications are, etc, is really very important. You know, watching videos like this, I think is important also. And so once you're an informed patient, you can go to your doctor. And remember, you know, these are not the most common diseases. So if you're able to go to the physician or nurse practitioner, or nurse or whoever is treating you with a set of questions and things that you've learned online, then I think that it makes the visit much more productive. Even something as simple as saying, you know, I found my, the MPN symptom assessment form online, and I have the symptoms, what do you think about that? Am I a candidate for treatment for that? I think that that's really important. But but but on the flip side of that, I also think that we need to be very careful with what we find online. You know, some of my patients definitely spend a lot of time on social media. And though I'm, obviously somebody that really likes social media, for so many reasons, that makes us all connected. Social media can sometimes also have, you know, lots of groups where a lot of information is shared that anecdotal, meaning is the experience of only one patient. And although that can certainly bring community to some degree. And sometimes the information that's shared in these groups, because it's just one person's experience doesn't necessarily reflect what a group of large patients may experience. And so sometimes I spend really a lot of time talking to my patients about, well, just because this person had a bad side effect, or etc, doesn't mean that that's what's kind of happened to us. So I think we have to be really, really careful with the information that we get, both in social media and just online in general, make sure that it's from a reputable source.

Kristie L. Kahl: Why should patients be hopeful looking towards the future of their treatment options?

Dr. Gabriela Hobbs: Now is definitely a time to be hopeful. And I would say, you know, one of the things that I sort of say, almost joking to my patients, but the reason why I was hired to work where I work, is because there's so much understanding about MPN now that they can hire a person that just treats MPNs, you know, whereas before, that would have been funny, right? Like, how do you how do you hire somebody to treat diseases that don't have any treatment? And so, there's more of me elsewhere, you know, I'm not the only one and, and I think that that's just one sign of like the changing times. And so there was lots of clinical trials now with a lot of medications or an advanced stages that will hopefully get approved in the next couple of years. And, and I feel optimistic about that, because I'll be able to offer more treatments to my patients. And I think another thing to remember is it really in the last decade, our understanding of how these diseases start and progress or evolve over time, has really changed exponentially. And so the amount of people that are doing research on these diseases has really increased significantly. And nothing is more objective proof of that, than the fact that there are really dozens of clinical trials across the country, testing a variety of different medications to treat the treat NPN. And so even though, when a patient has an NPN, they may feel like they're the only ones that have it. Remember that you're not the only one that has it. And a lot of people are really thinking about these diseases and trying to improve treatment and improve quality of life for these patients.

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