Living Strong with MPN - Replay Live!
Navigating Everything from Treatments to Clinical Trials with Resilience
Part 1 Transcript
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Tiffany Drummond 00:00
Music, hello everyone. How are you? Can everyone hear me? I want to make sure. Great, great. Thank you all so much for joining us this evening, for our Living Strong with MPN program, I am so happy to see this full house this evening. It really does mean a lot to me to see you all here. My name is Tiffany. I lead community programming and partnerships with The Patient Story, and you’ll learn more about us in a little bit. But you know, I know that MPN can be a challenging journey for our patients and our care funds, and so I want to start this program today by introducing you to a fellow MPN survivor. His name is Andrew Schorr. He is a myelofibrosis survivor, and so he [couldn’t be] here in person, but he did [want to] share [with] us. And so I want to make sure that we all take a look at our screen right here. And if you’re online, make sure that you follow our video as we introduce Andrew Schorr.
Andrew Schorr 03:23
Hello, Saint Louis, or wherever you may be watching from. I’m Andrew shore. I’m joining you from Del Mar California, which is in San Diego County. And I’ve been living with an MPN since 2011 and actually, I have a doctor’s appointment today with my MPN specialist, and we’re going to be going over my situation. I am taking one of the jack inhibitors. Is it still working for me? A few weeks ago, I had a bone marrow biopsy, so they’re looking at all the genomics of my cancer and seeing if things are progressing. And so I wanted to talk to you for just a few minutes about living with an MPN, whether it’s ET or PV or for me, starting as myelofibrosis, but I’m grateful that I’ve been living with myelofibrosis since 2011 so when I say grateful, because I’ve been living pretty well and treatments have been working now we’re at an exciting time, and Dr. Oh, and the team are available to talk to you about all of that, because things are changing. When I was first diagnosed, the first JAK inhibitor had just been approved, and so there was a new option before that. They weren’t great at all. And what’s happened now is a progression of science understanding what. What we’ve got, what could be possibly affecting progression from et to PV to MF, what’s driving it, and also knowing that we’re different and so what are different situations for different patients, different medicines and timing of where you are in your journey with one of these MPNs. And I guess where I’m coming from is I’m really encouraged. I mean, when I was first diagnosed, I didn’t know how long I’d lived with it. I knew was a really serious, rare condition. I also knew that it was really important to connect with a specialist, Dr. Oh and his colleagues are great examples of that, because we’re talking about more rare conditions. Not every doctor knows about it, not every doctor sees it very often, and they may not be up on all the latest options to evaluate and then treat it for you. So I’m always saying, go to a specialist now that opens up something else as well. Depending upon your situation, it may be appropriate to be in a clinical trial. What’s a clinical trial. A clinical trial is how we get new medicines and see would it be effective? Years ago, for another condition I have, I was in a clinical trial, and it led to much better care, and in that case, really lasting benefit. So I’m a big fan of clinical trials. Also, they monitor you like crazy, and so you feel like kind of a VIP. So that’s a good thing. You may need some extra tests, but it’s worth it to really have them look with a magnifying glass at you and your situation and maybe give you tomorrow’s medicine today. So I’m a big fan about clinical trials, but overall, whether you’re in a clinical trial or not living with an MPN now, we have an increasing number of options that are approved by the FDA and others that may well be likely, and research continues. So we’re on a journey right with the researchers, with the doctors and with other patients, so that we can get the best care for us. So you got to be informed, and that’s what you’re doing today. You are learning what the latest is, and then you’ll discuss it with your healthcare team as to what applies to you. Could be a new treatment, could be a clinical trial, could be just monitoring what your treatment plan is now, now a little bit about et and PV and MF differently, so they’re different, but they’re related conditions, and they can progress from one to another, and that’s where monitoring is so important. Okay, but fortunately, we do have treatment options, and as I said, others are coming. So what’s the bottom line? I’m going to my doctor today. I’m going to have a frank discussion. We’re going to go over all the data that she has from a bone marrow biopsy, and I get a blood test just before I see her. And what is all this mean? Where are we now, and where maybe are we headed? Is there a change to my treatment plan? And if so, what could it involve a clinical trial? This is my ongoing dialog with my MPN specialist, so make sure you check in with an MPN specialist. Make sure that’s a recurring conversation, as your situation may change, and don’t be afraid of clinical trials. So there you go. So that’s my point of view, from living with myelofibrosis since 2011 and having the benefit of meeting Dr. Oh, and other renowned specialists, and I’m really excited for you to get the information that you will get this evening. Thanks for watching. And again, I’m Andrew Schorr, and I like to say that knowledge can be the best medicine of all.
Tiffany Drummond 09:39
That was amazing, right? Yes, please give it up for Andrew Schorr, I hope that that video resonates with you, and I do believe, personally that sharing our experiences has major impact for others out there, and that’s something that the patient story is actually committed to. So again, thank you so much, Andrew, for sharing your story with us. And so now to kick off the program, I would like to introduce our moderator for the evening, Josh Bollam, who is the Director of Patient and community outreach in the St Louis area for The Leukemia and Lymphoma Society better known as The LLS. So I know you’re tired of me talking, so it’s okay. So I am going to turn this over to Josh. Josh, take it away. Thank you so much.
Josh Bollam (The LLS) 10:26
It’s my pleasure to be here. As Tiffany said, my name is Josh Bollam. I’ve been with The LLS for eight years. Nine years ago, I started my blood cancer journey as a joint caregiver with my mother to my uncle, who was diagnosed with non Hodgkin’s lymphoma. He lived in Las Vegas. My mother moved out there to give her a little bit of reprieve. I would bounce back and forth so she could have some time off. We wound up having to put him into a clinical trial at City of Hope. I got to see that wonderful institution and a side of LA that I hadn’t seen before. And when I was there at City of Hope, I started to look on the LLS website, and I saw a little line at the bottom that said, work with us. Clicked it, and eight years later, here I am so very, very excited to be here and very excited to talk with everybody in the room, as well as those online. LLS is here to support our blood cancer patients. We have a mission that begins with cure. Our goal is to cure blood cancers, and until that time comes, support patients along every stage of their journey. We know that cancer affects patients, both physically, mentally and financially, and we’re there to help every stage of that. This evening’s program is going to be is produced by the patient story. It’s an organization dedicated to building community for patients and families through education and content. I encourage you to check out their site, where you can find hundreds of patient stories. The Patient Story would like to thank their collaborators for this evening’s program, The Leukemia and Lymphoma Society and Imerman Angels. Putting together a program is truly a team effort, and we want to thank the cancer support community and MPN Research Foundation in their efforts to promote and share this program to the members and supporters. We also want to thank our sponsors, Sobi, Karyopharm, GSK, and Novartis, for their support. As with all our programs, the sponsors have no editorial content or control over the content. Also, while we hope you learn a lot today, our discussions are not a substitute for seeking medical advice or care from your own doctor. Please speak with your own doctor and about what’s most appropriate for you. Now, if you’re watching this program virtually, we’d like to encourage you to send in a question by emailing us at community, at the patient story.com, our producers will review and send questions to me. We will include as many as we have time for. Remember nothing too personal as we can’t provide specific medical advice tonight. Also, we would love your feedback on whether this program is helpful and what topics you’d like for us to focus on in future programs. So please fill out the survey. For those tuning in virtually, it will come to you when the program ends, and for those of you here in person, the survey is on the colored paper in your folders. So let’s meet our panel. Joining us tonight, we have two special guests who many of you in the audience probably already know very well. Dr Steven Oh is an Associate Professor of Medicine and Co-Chief of the Division of Hematology at Washington University School of Medicine. His clinical practice and research efforts are focused on myeloproliferative neoplasms, MPNS. So welcome. Dr. Oh, next we have Dr Amy Zhou, also an Associate Professor of Medicine in the Division of Hematology at Washington University in St Louis. She sees patients with a variety of hematological disorders, and her research focuses on developing new therapies and improving the care of MPN patients. Thank you, Dr Zhou, so let’s go ahead and get started. We have so much to cover. Tonight, we’re going to cover the three MPN types, Polycythemia, Vera, Essential. Excuse me, I’m going to stumble through some of these words tonight. I’ve rehearsed them many times, but it’s still going to stumble essential thrombocythemia (ET) and myelofibrosis, MF, and the related molecular testing of each one of them we’re going to talk about. Monitor. And managing symptoms and side effects, and we’re going to talk about the latest treatments, including clinical trials. So let’s do a big picture overview here. Dr. Oh would you describe briefly the differences in similarities between the three types of MPNs, and we have to recognize that myelofibrosis can be primary or secondary. And just talk a little bit about that. Sure, absolutely.
Stephen Oh, MD, PhD 15:25
So, you know, Andrew, in his introduction, mentioned that there are many similarities between these three types of MPNs. That’s that’s absolutely the case. There are absolutely distinctions that separate them as well. So let’s talk a little bit about some of those specifics. So you know, first, these three diseases are grouped together for a number of reasons, one of which is that they all have some in some fashion, result in abnormalities of the blood counts. So for instance, in polycythemia Vera, typically, we see an elevation in the hemoglobin or hermatocrit Too many red blood cells. That’s the primary feature of polycythemia Vera, or PV. It is the case that in patients with PV, the other blood counts may be elevated as well. So some patients have elevation in the white blood cell count. Some patients have elevation in the platelet count, but at the minimum elevation of the hemoglobin and hematocrit is a defining feature of PV. ET is very analogous. So we see in the essential thrombocythemia, or ET, that the platelet count is elevated. That can be in conjunction with the abnormalities of the other blood counts as well. But the primary feature is elevation in the platelet count. Now myelofibrosis is, in some ways, shares some of those features, but is more so typically associated with a decrease in the hemoglobin anemia. So to varying extents, it can be mild, it can be more severe, but typically patients have some degree of anemia, and patients with myelofibrosis, not always, but often have more prominent symptoms, including fatigue, night sweats, sometimes poor appetite, sometimes varying degrees of weight loss. And the spleens can become enlarged. We do see in PV and et that the spleens can become enlarged as well, but in particular in myelofibrosis, that tends to be more commonly a prominent feature. And when the spleen becomes very enlarged, that can cause symptoms, it can cause abdominal discomfort, it can affect the appetite, that alone can lead to weight loss. So those are some of the primary features of these diseases. And as you mentioned, Josh, myelofibrosis can occur as a primary myelofibrosis, meaning that there was no prior history of a blood disorder. But in many cases, that can also be a subsequent development after having a history of polycythemia Vera or essential thrombocythemia. Patients with PV or et may live for many years, decades, and the disease may never change. It may never progress to myelofibrosis in some patients, eventually, that may occur, so that would be post PV or post et myelofibrosis, or maybe referred to often as secondary myelofibrosis. Now the other aspect of why we think of these diseases together is that they share in common some of the similar genetic or molecular abnormalities. So the most common genetic change or mutation in these diseases is a mutation the JAK2 gene, J-A-K-2 many of you are familiar with this. This JAK2v617F mutation, so it is present in the vast majority of patients with polycythemia Vera. It’s present in about 50 to 60% of patients with ET and also about 50 to 60% of patients with myelofibrosis. And for those patients who do not have this, JAK2v617F mutation, the second most common mutation is in a gene called CALR, or Cal reticulum, and that mutation is present in about 20 to 30% of patients with ET and a similar percentage of patients with myelofibrosis. And then less commonly are mutations in nipple MPL, which are present in a small fraction of patients with et and myelofibrosis. So we think of JAK2, CALR and MPL, as we call the three primary driver mutations. So not everybody, but most patients with this disease, with these diseases, have one of these three mutations.
Josh Bollam (The LLS) 19:36
Factors out what is the role of molecular testing in the management of MPNs, especially thinking in terms of a more accurate diagnosis or a risk that the person’s MPN might progress and possibly affect your decision about earlier treatments.
Amy W. Zhou, MD 19:52
Yeah, so I would say, you know, to follow up on what Dr. Oh was talking about in regards to, you know, the mutation. So. I don’t know what I but with the three main driver mutations, you and I would say, from a diagnostic standpoint, it’s very helpful, because the expectation is that you know, if you do have an MPN, you would have one of these three mutations. But there is a small percentage of patients who may not have these three mutations, and then we can do more extended mutational testing with something called Next Generation influencing where we have these panels where they include a lot more mutations that aren’t necessarily specific for npns, but can help establish a diagnosis that there is a blood cancer present, because that’s really, you know, I would say an important piece of information in addition to, you know, let’s say the blood count abnormalities or bone marrow biopsy. So all of that information is very helpful in terms of diagnosis. But also, what we’re learning more now is that these different mutations also may convey different prognostic significance, and so it’s also very helpful for your provider. In regards to having that information, it’s not, you know, I would say entirely, oh, I’m sorry, yeah, hopefully you guys heard all of that from before, but yeah, so you know that additional information also does have prognostic value. And so there are different, you know, kind of these mutations are being incorporated into the risk stratification models for these diseases, where, based on, you know, blood counts and and various features, and also incorporating these mutations, we can try to predict how, try to better predict how patients are going to do, but certainly I would say, you know, it’s not as personalized as we would like it to be. But you know, ultimately, that’s the goal is, as we understand these diseases more that, you know, kind of the prognostication and risk stratification does become more personalized to the individual.
Josh Bollam (The LLS) 22:05
Thank you. It wouldn’t be a patient education program without a few AV challenges, of course. So thanks for rolling with us. How do how do those molecular tests influence your treatment decisions for patients?
Amy W. Zhou, MD 22:19
So that’s a good question. I would say right now, in general, the molecular mutations may not necessarily impact the choice of treatment that’s currently available, but that may change in the future. I think we’ll talk a little bit more about clinical trials, but it may be that in the future, the mutations that you have may impact, you know, kind of what’s best treatment? Yeah.
Josh Bollam (The LLS) 22:45
Excellent. Thank you. Dr. Oh, what’s the difference between cytopenic myelofibrosis and proliferative myelofibrosis? And what might you see in a patient in the clinic, in their laboratory results? That would be different. And do these differences impact their prognosis?
Stephen Oh, MD, PhD 23:01
Sure. So these terms, sometimes they can be confusing and sound very technical, but let’s start with proliferative. So we know MPN, myeloproliferative, right? So proliferation, things are growing or proliferating. We know that’s a feature, underlying feature of these diseases. The most straightforward way to think of that is in the case of pvet, you have high blood counts, your bone marrow is producing too many blood cells, right? So that’s proliferation, hyper proliferation, when we think of that in terms of cytopenic and proliferative, that is mostly referring to patients with myelofibrosis, and when patients with myelofibrosis, again, it can be a little bit confusing, because in that setting, in some patients, one or more of the blood counts can be high, and one or more of the blood counts can be low, and sometimes you have a combination of both. For instance, a patient with myelofibrosis may have anemia, so hemoglobin is low, but the white blood cell count may be high, so they’re kind of the opposite. But when we think of those terms, cytopenic and proliferative, as it refers to myelofibrosis, we think of cytopenic means low, essentially, as it refers to the blood counts. So that means that, generally speaking, there’s more prominent anemia. There may be more of an issue with the platelet count being low, so thrombocytopenia, whereas with the proliferative subset or grouping of myelofibrosis, that’s less the case, and that tends to be that more proliferative, MF myelofibrosis, more so for patients who had a prior history of polycythemia Vera, or essential thrombocythemia. So they started out with the higher blood counts. They may be drifting down a little bit, but they’re still higher than patients who are more commonly from primary myelofibrosis, where those blood counts may be. So that’s more so the cytopenic category.
Josh Bollam (The LLS) 25:02
Are there unique challenges in treating those two patients?
Stephen Oh, MD, PhD 25:06
Absolutely. So, you know, we’re going to talk in more detail about some of the available therapies for these diseases, in particular when we’re thinking about patients with myelofibrosis, and many of the treatments that we use, those treatments can lower the blood counts, and if they’re already low, and you’re thinking about treatments that may further lower the blood counts, that can become limiting, right? That can limit the dosage of the treatment you can administer, it can cause other issues when the blood counts get low, you can think of, for instance, if the platelet count is very low that can put patients at higher risk for bleeding complications, et cetera. So we have to absolutely factor those things in, and it has relevance to which of the available treatment options we might recommend, excellent.
Josh Bollam (The LLS) 25:53
Thank you. So question for both. We know that clinical assessment forms are used in clinics to track and monitor symptoms. But How can patients monitor their symptoms at home even more closely? What tools are out there? What do you tell your patients about the importance of recognizing their symptoms and how might they change over time? I’ll start Sure.
Amy W. Zhou, MD 26:16
So I would say, as far as you know. So the symptom assessment forms that we give in clinic, they are available online, but also, I Google searched this a while back. But there are, like, MPN symptom trackers or apps that are available for specifically patients to use, and you can download it on your phone, or, you know, kind of use, like their online form, and that could be a way for you to follow your own symptoms at home, like in between your visits. But I would say, you know, generally, for the symptoms that we want our patients to let us know about would be things like, you know, persistent night sweats or bone pain or weight loss or itching. Those are, those are symptoms that, if they’re getting worse, we want to know about earlier, because that could be a sign that, you know, maybe the your your disease might be changing a little bit, and that may, you know, be something that might prompt a change in treatments.
Josh Bollam (The LLS) 27:19
So excellent. Yeah, thank you both. We just in recognition of the Leukemia and Lymphoma Society and you mentioned it, we have our Health Manager app, a free to download in all the app stores or wherever you get your apps on your phone. I think that’s how you’re supposed to say it in in terms of a moderator, let’s, let’s go ahead and switch gears a little bit here to the treatments associated in managing MPNs. Dr. Zhou, can you provide an overview of the current treatments for PV and ET, and what’s your experience with their effectiveness in managing those diseases?
Amy W. Zhou, MD 27:59
Yes, absolutely. So for so starting with PV. So we do risk stratify patients into like a higher risk group and a lower risk group, and that risk specifically is the risk for blood clots. Because, as Dr. Oh mentioned, you know, we the expectation is that for our PV patients and also our et patients, the prognosis is very favorable, and we do expect that most patients will live a long time with these diseases. But one of the biggest complications, and you all are probably aware of this, is there is a higher risk of blood clots associated with these MPNS in general. And so a lot of the therapies are meant to decrease the risk for blood clots. And so for how we risk stratify patients is really based on two factors, age and also whether or not you’ve had a prior blood clot. And blood clots could be things like heart attack, stroke, so those are types of blood clots in the arteries, or they could be blood clots in the vein, so like a deep vein clot in the leg, or that goes along those types of things. And so if you’re over age 60 or you have had a history of blood clots, that does put you at a higher risk category for getting blood clots. And so for our higher risk patients, in addition to aspirin or blood thinner, we do also recommend taking a medication to lower the hematocrit. So for our PV patients, our goal is to keep your hematocrit, which is a blood number that that looks at the red blood cell number below 45 that’s the recommendation, and which is a little bit different for our lower risk patients, where, if you don’t have either of those risk factors, then generally it’s aspirin. And also what we call therapeutic phlebotomy, so that’s removing blood periodically to maintain that hematocrit at that goal that we want. Months. And so then for our higher risk patients, as far as the treatment options go, there’s a couple of different ones to choose from. The most common one that I prescribe, and I think most people prescribe, is something called hydroxyurea. So that’s a pill medication that’s taken, and the dose is dependent on how high a dose you need to maintain that hematocrit goal. So we use that most often because it is very well tolerated and it’s easy to take because it’s a pill. Other options, so interferon is is another option for our PV patients. It’s an injection I won’t get into, you know, kind of the side effects of these therapies too much, but the side effect profiles do differ, and so that that that is a difference in regards to some of these treatments and Ruxolitinib, so that’s a Jak inhibitor that’s also used in myelofibrosis, that is also FDA approved in the second line setting for PV. And what we mean by second line is it may not be the first option you start with, but for patients who may not be able to tolerate hydroxyurea or are not getting the response that they should from the hydroxyurea, then that Ruxolitinib or Jakafi is the commercial name, may be a good option to consider. So those are some of the more common treatments we’ll use for PV and then for ET. So similar idea in regards to risk stratification. So age and history of blood clots are still kind of the main factors. We do also incorporate mutations to some degree, because, as Dr o mentioned with PV patients, the majority of patients are JAK2 mutated. But it’s a little bit different for ET and MF patients, where even though the like 60% of patients have a jack two mutation, there’s a percentage of patients who have a mutation in CALR and so there is a little bit of a difference in blood clotting risk between having these different mutations, and so that that gets included in the Risk ratification. But overall, it’s still going to be age and history of blood clots that determines if somebody is going to be at higher risk and may need to take a medication in this situation to lower the platelet count to a more normal level. Because, you know, again, with et generally, it’s the platelets that are high and not the red blood cell count. And so as far as kind of treatment options go, so there is some overlap. So again, hydroxyurea, we use fairly commonly for ET as well. There’s another medication called anagrelide, which is another pill medication that’s used for to lower the platelet counts. We do generally use that in more of the second line setting. So again, if somebody does not do well or is needing a very high dose of hydria to keep their platelets at the goal that we want. Interferon also can be used in ET and so I will say I have also used Ruxolitinib in my ET patients, but because it’s not FDA approved for that indication, sometimes it can be challenging for insurance to cover medications that are not approved, FDA approved for specific indication, but we have been able to get it off label for some patients.
Josh Bollam (The LLS) 33:40
Thank you. Dr. Oh, can youprovide an overview of myelofibrosis, including low risk media, intermediate risk and high risk patients
Stephen Oh, MD, PhD 33:51
Sure. So, you know, I could go on for probably a couple of hours about that, but I’ll give the short summary for you all. So you know, we analogous to PV and ET, we have a variety of different prognostic calculators that can be used to give us an understanding of the overall outlook for the disease. That relates, of course, to looking ahead in terms of projected survival or lifespan after diagnosis. It relates to the possibility that the disease, over time, could progress to a more advanced form, in the most advanced case, to acute leukemia. And that is where we write at which, with whichever prognostic calculator we’re using, it generally will result in something like four or five different categories, ranging from lower risk to higher risk categories. So that gives us information about the overall outlook. It does also guide, to some extent, what kinds of treatments we might recommend. So for example, if a patient is in the highest risk category and they are overall relatively healthy, you. We might actually suggest to confer with our transplantation specialists, or bone marrow transplant specialists, and consider whether that is a recommended course of action, that is a treatment that is pretty intense, intensive, but has the payoff of potentially putting the disease into remission. It is not a treatment that is undertaken by the majority of patients for a variety of reasons, including that many patients are older andor have other medical problems that preclude that as an option, even if they are in the higher risk categories. But for some, that would be a recommended path for those that are not pursuing transplantation, then we think about, particularly when we’re talking about the intermediate and higher risk patients, what other available treatments are there that can be helpful? And fortunately, today, we have four FDA approved oral medications to that are can be used for patients with myelofibrosis, they are all in this class of drugs called JAK inhibitors. So you heard about Ruxolitinib as the as one example, Ruxolitinib or Jakafi was the first FDA approved treatment for myelofibrosis, but today we have three others. So you have Ruxolitinib or Jakafi. Fedratinib or Inrebic, we have Pacritinib or Vonjo and good. They’re on the screen there. And then you have, most recently, Momelotinib or Ojjaara. So don’t ask me why or how those names were devised, but those are the names, and they’re all options for patients with myelofibrosis. I don’t know if you want me to drill down on that now or no. Well, yeah, we’ll cover that in just this Okay, so, but as a class, these drugs are capable of providing benefit in various ways, in particular for symptoms. So you know, we talked a little bit earlier about how patients with myelofibrosis to varying extents, may have symptoms such as fatigue, night sweats and large spleens that are causing symptoms, and these treatments tend to be helpful for those kinds of symptoms, and in some cases, in the best cases, they can result in dramatic improvement in symptoms. So those you know, we increasingly, we’ve used those treatments over the years because they provide that kind of helpful benefit.
Josh Bollam (The LLS) 37:24
Yeah, thank you. I’m pretty sure it’s a poker game where they come up with these names. Everybody throws their name in a pod. But in terms of symptoms associated with MPNs, you mentioned fatigue, especially with myelofibrosis. What are the most effective strategies for managing fatigue. And are there any specific medications or lifestyle modifications that you both would recommend?
Stephen Oh, MD, PhD 37:48
Yeah, so I can start, and then Dr Zhou can chime in as well. So you know, as I mentioned, these drugs, they tend to help quite a bit with fatigue and energy level. In some cases, again, really market improvements. We also have to keep in mind that fatigue can be what we call multifactorial. So there can be multiple causes for that. It can be the disease itself. And this can be an issue, not just in myelofibrosis, but in patients with PV and et for sure as well. And it can be in particular in the setting of my process, it can be the disease and it can be because of anemia. You know, I think it’s obvious that if your hemoglobin is lowering, your anemic that’s going to create some degree of fatigue. And so those two can be big contributing factors. And then there could be other reasons to have fatigue as well. So we have to be cognizant that there may be other factors playing in. But as far as lifestyle modifications, you know, we certainly encourage patients to try to be as active as possible. Of course, that can be difficult to a certain extent, if you’re very anemic, if the disease is creating the severe fatigue. So, you know, there’s a limit, and every every individual sort of has to determine whether there’s those limits and how much you can push in that regard, but staying as active as possible certainly is something that we encourage. Excellent
Josh Bollam (The LLS) 39:04
Dr. Zhou, anything to add.
Amy W. Zhou, MD 39:06
I agree with everything you just said. I have nothing to add.
Josh Bollam (The LLS) 39:09
I love it. I love it.
Josh Bollam (The LLS) 39:12
So another question to both of you, I will let Dr Zhou run with this one outside of anemia, because I have a follow up question for that. What other common symptoms do MPN patients present with, and how are they managed?
Amy W. Zhou, MD 39:26
So I think it depends on which MPN so, you know, and I think we’ve discussed this a little bit already, you know, with myelofibrosis, patients can have in a large spleen, in addition to the anemia. And then also, you know what we call these constitutional symptoms, so the fatigue, some patients may have issues with itching and that that can be a symptom that’s noticed. For our ET and PV patients, there may not be, I’d say, in general, we wouldn’t expect to see anemia. You know, for our ET and PV patients and and really, having an enlarged Lean is not very common in those diseases, but certainly, I would say, you know, some of those, again, constitutional symptoms, like the itching, you know, could be a symptom that some patients experience.
Josh Bollam (The LLS) 40:16
Excellent. Thank you. And Dr. Oh, we’ll focus a little bit on anemia. Now, it’s significant in our patient population. We’ve mentioned it a couple times. How does this condition impact treatment approaches? Are there clinical signs of the symptom that should prompt a more frequent evaluation or maybe just try a different approach to the problem? Yeah,
Stephen Oh, MD, PhD 40:35
Absolutely. So anemia certainly is a can be a big problem, of course, in particular, with patients with myelofibrosis, who may start out with relatively mild ademia, and over the course of the disease, that and not always, but in some cases, can become more severe and then ultimately result in requirement of red blood cell transfusions, either every once in a while or on a more frequent basis. So that, again, is a prominent future. I will sort of make it refer to an anecdote that the anemia can be from other causes as well. Just saw a patient this morning who has had a couple instances where she’s become very anemic, and that was due to iron deficiency related to GI bleeding. So it’s just a reminder that we need to be always looking out for other causes that may contribute to the anemia as well. But when we’re talking about anemia from the disease and the problems that can occur, it is a certainly a significant problem from a variety of standpoints. One is we know in patients with myelofibrosis that anemia is a significant risk factor as it relates to the overall course of the disease. If you know we mentioned these prognostic scoring systems, they all include anemia as a big factor. So that is essentially a marker of more severe disease. And so we know that, we know that the anemia itself, in real time, is a problem. It contributes to the fatigue. And we know that the as I mentioned earlier, some of these treatments, they can actually lower the blood counts further and make the anemia worse. So in the case of Ruxolitinib or Jakafi, we we see that the hemoglobin will drop, and we accept that, because the patient generally will say they feel better overall, even if the anemia gets worse. And that’s great. We’ll take that. We’re happy with that. That’s the great, great sort of choice, or course of action for many patients, but it nonetheless remains a problem, and that is something that we’re, you know, I think continuously striving for ways to address that better. Some of the newer therapies have differing effects as far as anemia is concerned, and then there are emerging strategies to more directly target or improve that anemia.
Part 2 Transcript
Click to View Part 2 Transcript
Josh Bollam (The LLS) 00:20
We know that clinical trials can be overwhelming. They can be confusing. It’s sometimes hard to understand what’s going on, but that’s why we at LLS have created a dedicated service to answer your questions and help guide you through the process, to be an extension of your healthcare team to learn more, we’ll now hear from Tiffany, who’s the manager, manager of the community programming at the patient story, as she interviews one of LLS nurse navigators, Melanie Fife,
Tiffany Drummond 00:52
(recorded video) Welcome everyone, and thank you for joining us for our living strong with MPN Program. My name is Tiffany. I manage community programming at the patient story, where we are committed to bringing human answers to your cancer questions, which is why we are grateful for our partners, like the Leukemia and Lymphoma Society, who offer a wide range of support and resources. Today, we’re going to discuss a specific service that helps bring innovative and novel therapies to blood cancer patients the clinical trial Support Center. I have the pleasure of speaking with LLS clinical trial nurse navigator, Melanie Fife, who will provide more in depth information about this great resource. Welcome Melanie. Thank you so much for joining us for our program, living strong with MPN. And first of all, how are you doing?
Melanie Fife (LLS Nurse Navigator) 01:41
Doing great. I’m glad to be here today. Thank you so much. So let’s just kind of kick it off. You know, this is such a great resource, but can you really explain to me in the audience exactly what clinical trial nurse navigation is, especially given the complexities in the healthcare system dealing with clinical trials? Of course, I’d be happy to clinical trial. Nurse navigation is technically a service that helps patients and families navigate the clinical trial process, so patients and families work with trained registered nurses who act as a guide to kind of help them understand clinical trials, search for clinical trials, and also help overcome any barriers to participating in a potential clinical trial. And these navigators also help provide one on one support that’s personalized, and they can serve as a resource for questions and education during the patient cancers journey.
Tiffany Drummond 02:35
So let me ask you this follow up question. So for the clinical trial nurse navigators, are are they all dedicated registered nurses? Or are there a variety in terms of education credentials?
Melanie Fife (LLS Nurse Navigator) 02:47
I think there’s, there’s a variety. Everybody’s a registered nurse, but there’s a variety of credentials that we all have. You know, some people have their masters. There’s certain ones that have been nurse practitioners in the past, or clinical nurse specialists specific to LLS. What can you tell me about the clinical trial Support Center? So the LLS, or Leukemia and Lymphoma Society, clinical trial Support Center, is committed to providing resources to help patients actually access clinical trials. So we have a free personalized service that includes several things. So it includes personalized clinical trial matching. So we work one on one with patients to help identify trials that match their specific diagnosis, stage of disease and many other factors. We also provide education about clinical trials, including risks and benefits you know, and can answer any questions patients and families have about that. We help patients identify potential treatment options, including those clinical trials, obviously, and connect them with clinical trial site staff, and we can help overcome those barriers I mentioned, with a clinical trial, we also provide ongoing support, including identifying and connecting them with LLS resources, such as our Information Resource Center financial assistance programs, and ultimately, we’re here to just build that web of support and make sure we’re supporting patients through their cancer journey, providing guidance and just answering questions as needed.
Tiffany Drummond 04:19
You know, there are so many you know, the internet is just full of information that can be overwhelming, especially for a patient that may be considered a clinical trial so, you know, especially for even clinical trial support resources. So what sets the LLS apart from other resources out there that offer clinical trial support?
Melanie Fife (LLS Nurse Navigator) 04:36
Well, Tiffany, I’m really glad you asked this question, because I can’t wait to tell you how awesome we are. So first of all, we are a team of 12 highly trained nurse navigators, and as I mentioned, with different varying degrees, and we’re with expertise in both pediatric and adult blood cancer. So we are a completely free service where any patient, family or even healthcare professional. Can reach out to us. We’re here to really be an extension of the patient’s healthcare team, really, and to ultimately educate, support and empower patients to be active participants in their care. So it’s this unique relationship between the patient family and our nurse navigators that really sets us apart and enables us to provide personalized like ongoing support, instead of just simply providing patients with a long list of clinical trials. The clinical trial nurse navigators at LLS take the time to really get to know who the patient is, what their goals of care are, and really meet them where they’re at we provide a paired down clinical trial report that takes into consideration the unique patient characteristics, such as their past medical history, previous treatment responses, ability to travel, types of insurance, you know, any of those other life circumstances that can really impact their ability to participate in a clinical trial, and we also do an enormous amount of outreach on the patient’s behalf, including talking to our pharmaceutical partners, to clinical trial site staff, including principal investigators and coordinators, to provide patients and their families with the most up to date information so they can make that those informed decisions.
Tiffany Drummond 06:22
Thank you so much for saying that. So that was a breadth of information, but I heard some words that really resonate with me personally as a former caregiver, which is being an active participant in your care, and that you are able to help people achieve that goal, because a lot of people can’t do that. And also personalized care, what you all do in terms of getting to know the patient and their past medical history, I think that’s just such an amazing service, and so I want the audience to be able to take advantage of that. So how can they get in contact with the LLS Clinical Trial Support Center?
Melanie Fife (LLS Nurse Navigator) 06:55
Good question, good question, so patients and even healthcare providers can contact us at the LLS clinical trial, the sorry Support Center through either our Information Resource Center by calling 1-800-955-4572, or by visiting our website@www.lls.org and they can also email us directly with any questions at ctsc@lls.org
Tiffany Drummond 07:25
Well, Melanie, in this short amount of time, you have given us so much information. Again, I want to thank you so much for taking the time to participate in our Living Strong MPN program. And thank you so much. Any closing remarks,
Melanie Fife (LLS Nurse Navigator) 07:41
Just thank you so much to you and the patient story for having us, and I hope everybody knows we’re here to help.
Tiffany Drummond 07:46
Thank you so much. Wow. What a short but impactful conversation. Melanie’s insights and expertise have been invaluable. I’m sure you heard her email notifications going off during our conversation. I’m going to wager that those are patients just like you requesting clinical trial support, I look forward to seeing LLS continue impact on the lives of blood cancer patients. If you are interested in participating in a clinical trial, please take advantage of this service. And finally, on behalf of the patient story, I want to give a special thank you to our audience for attending this evening’s program in St Louis, and to all of you tuning in virtually enjoy the rest of the event.
Josh Bollam (The LLS) 08:25
Thanks to both Tiffany and millet. Excuse me, Melanie, for highlighting that awesome service. The Leukemia and Lymphoma Society’s clinical trial support center isn’t is an amazing resource for patients and their families navigating clinical trials, the dedicated team of nurse navigators provides personalized service, helps match patients with suitable trials and assists with the enrollment process. If you or someone you know is considering a clinical trial, please don’t hesitate to reach out to the LLS clinical trial Support Center, just as a quick note to our friends who are watching online, there seems to be a little bit of an audio issue with it appearing are coming out low. Been recommended to try to put headphones or connect headphones to your computer to hear a little bit better. Taking a step back, looking at some of the active clinical trials that are going on, and what that means for patients. And just a little bit of an update, I’m gonna stumble again through these drug names, but I hope Dr. Oh will correct me, tell me about the phase three Manifest2 study of pelabresib. Yeah, see, I knew it. Yeah,
Stephen Oh, MD, PhD 09:42
Yeah. So, thank you, Josh. So all of these drugs, you know, you’re probably seven different ways you can pronounce them. The way that I understand to pronounce that one is pelabresib, or some people say pelabresib.
Josh Bollam (The LLS) 09:54
That sounds better than what I said.
Stephen Oh, MD, PhD 09:58
Don’t worry about that. Right? The Manifest2 Study, I know some of you are familiar with it, is a phase three study for patients with myelofibrosis, in which patients were randomized into two groups, which is the way these phase three studies usually are done. So you have two groups. One group receives Ruxolitinib as the most common treatment for patients with myelofibrosis, and the other group receives Ruxolitinib plus pelabresib. And because of the nature of the way these studies need to be done, the group that receives Ruxolitinib alone also receives a placebo, so sort of a sugar pill, that instead of the pelabresib, and nobody knows initially which which of those groups the patient is put into that’s the by design so that we can determine if the collaborative is actually adding something beneficial. So it’s a very common, typical phase three study design. Let me back up and say phase three is the most sort of advanced development within clinical trials that typically would be before FDA approval. So you have phase one, which is the first phase, where that’s usually a small, smaller study. The drug is administered. The study drug is administered, often at very varying doses, and the primary objective is to determine is this actually just a safe and tolerable drug? And you know, of course, that’s critical to establish that. And then you move on to phase two, and then to phase three to learn if the drug is effective. Okay, so phase three manifests to collaborative The question is, adding that to Ruxolitinib, does it provide additional benefit that study that we participated in, and we have been participating in the last year’s ASH meeting the American Society of Hematology. Some of you know that there’s a big national or international meeting the ASH meeting every December, and at that meeting last December, the results from the initial results from that Manifest2 study were reported, and the summary is that the patients who received the two drugs together, clearly there was superior benefit in terms of spleen reduction. So we know, as we’ve talked about, that Ruxolitinib can reduce the size of the spleen, while the two drugs together shrunk the spleen even more. There was no question that the two together were more effective in that regard. And that is really for for most studies in the myelofibrosis field, is what we call the primary endpoint, is the most important metric or readout as to whether a particular treatment is effective. It’s something that we can as some of you know, you can image with various modalities. In the study of clinical trials, typically either MRI or CT scan, you can get, actually, the exact volume of the spleen. And then you can, you know, calculate how much change there’s been over time with that particular treatment. So the with that study, clearly, the combination treatment was superior. The secondary, or key secondary endpoint is about symptoms. And again, we know that Ruxolitinib was helpful for symptoms, so the question was, adding pelabresib does it provide additional benefit for symptoms? And there, the answer was kind of plus minus. It wasn’t clear that the combination treatment was definitely better. There were some aspects that suggested a little bit better, but it wasn’t a slam dunk, and that study continues to is continuing to to progress, but that is the initial readout from that study, and further data is required to understand if this really makes sense as an available treatment option, And as to whether that will lead to FDA approval. FDA approval, I want to take just a quick step back, because we have a lot of different patients in the room, and a lot of them online, and we don’t necessarily know where they are in their journey, especially their treatment journey, and if they’ve been introduced to clinical trials. And one of my goals in the work that I do is to break down the myths of clinical trials. And you mentioned a word that sometimes strike fear into patients, you said the word placebo. Can you take a step back and just talk about MPN clinical trials, what that means for a patient, and how standard of care will always be a part of it?
Stephen Oh, MD, PhD 14:21
Absolutely. So, you know, I think, you know, there’s a couple of different perspectives on this kind of thing. So on the one hand, if you look at it from a patient perspective, and think about a study in which, participating in a study, you might get placebo, you’re not getting the active treatments, you might question, why would I want to do that? Why? Why would I take that chance of committing to this extra time, involvement, extra monitoring, hassle, of of that kind of thing is, you know, when I don’t even know what I’m getting right? So that is a real and legitimate concern. It’s a question and a reason why many choose not to participate in clinical trials. That’s perfectly understandable and appropriate. But the to understand if a new treatment is effective, that is really necessary, because otherwise you really have no indication there. The earlier phases of the study, there’s not typically a placebo. Every patient gets a study treatment. That’s attractive from a certain standpoint, because you know you’re getting the study treatment, you know you’re getting the active treatment, that’s great. And is, you know, again, definitely a reason why some might favor participating in the study at that point. But the placebo is necessary, or some comparison is necessary. But as Josh was saying, typically, as in the Manifest2 study, patients are, at a minimum, getting the standard of care. So they’re getting Ruxolitinib. Now, there’s, of course, four Jak inhibitors that are now available. But particularly at the time that that study started, Ruxolitinib was the predominant JAK inhibitor that was used. So you could say, Well, every patient gets a good treatment to begin with. You know you’re getting that, and then plus or minus the study treatment. So that’s that’s how I would phrase it.
Josh Bollam (The LLS) 16:06
Yeah, absolutely. Thank you so much. I just want to make sure that we’re clarifying that for everybody who’s with us tonight, I want to jump back into some of these other clinical trials. Can you talk about a status of the BCL, BCL2 inhibitors for MF.
Stephen Oh, MD, PhD 16:22
Do you want me to take this one?
Amy W. Zhou, MD 16:24
I mean, I can start, but I would say you could add to it, in case I lied to everybody here. So with the BCL-2 inhibitors. So as Dr Oh mentioned, you know, we have this annual meeting, the ASH meeting, where a lot of you know, data is presented regarding the results of some of these clinical trials. And so navitoclax was a medication that is, is a BCL-2 inhibitor. So it works in a way, basically where you know same i the study that they presented at last year’s Ash was also a phase three study, very similar in design to the study that we just talked about with Pelabresib, where the question was, if you add this other medication on, you know, to standard of care, Ruxolitinib, do you get a better response, you know, in regards to spleen improvement or symptom improvement. And I think, similar to the Pelabresib study with that drug, there was a bigger spleen response. With the combination versus Ruxolitinib alone, they didn’t see a big difference in the symptoms either. And then, you know, I would say, as far as the other aspect of things, which is the safety of adding another medication on top of, you know, an exist, an existing medication, you know, whether or not you know, some of the side effects could be, could be worse, and so the combination did have, like, lower blood counts. So, you know, I would say that that is, is a concern in regards to whether or not you know the the combination, you know, kind of is the best strategy, versus, you know, doing that Ruxolitinib in it by itself. So I think, as far as you know, FDA approval goes, because that’s really the next step after, you know, they do these big trials, and they have, you know, the results is whether or not, you know, from a safety standpoint and what the drug does, whether the FDA thinks that it would be something that they would approve for everybody to use. So I haven’t gotten any updates regarding FDA approval for for that medication. So I’m not sure you know kind of what the result of that will be.
Josh Bollam (The LLS) 18:47
Thank you, either one the century study for select I’m not even going to try, please, selinexor. Selinexor. Thank you.
Stephen Oh, MD, PhD 18:57
Yeah, so I can comment on that. So selinexor is, is a treatment that is currently FDA approved for some subset of patients with multiple myeloma. So it’s somewhat different disease, but is being explored for patients with myelofibrosis. It is a little bit different than the way it’s being the way it’s approved for patients with myeloma, in that lower doses are being used, there has been some concern with side effects with that drug in the myeloma setting, with the lower dose that’s being used in the myelofibrosis studies, perhaps that’s less of a concern. There is this ongoing, or relatively newly started, phase three study now with selinexor in myelofibrosis based on earlier phase studies showing promising activities. So that’s that is of interest for sure to us into the field as a new sort of strategy, and that is in combination with Ruxolitinib, a very analogous to the studies we’ve been discussing with Pelabresib and navitoclax. So we’ll see how that progresses and. We’re not currently as a site participating in that study, but we’re certainly interested to see how that progresses.
Josh Bollam (The LLS) 20:05
Um, and the last one developments regarding navtemadlin (KRT-232)
Amy W. Zhou, MD 20:11
Yeah, I think you Yeah, as I’m not, I would say I’m not as familiar with
Stephen Oh, MD, PhD 20:16
Sure. So navtemadlin (KRT-232) also known as navtemadlin, yeah, so
Josh Bollam (The LLS) 20:22
I’m glad they didn’t write that one down.
Stephen Oh, MD, PhD 20:24
So that drug is also being explored in myelofibrosis, and has gone through various phases of studies, and is now in a phase three study as well. There is some data that will be presented at ASH from some of the earlier phase studies with navtemadlin, suggesting that it may have some ability to not just improve spleen size or symptoms, but improve some other underlying aspects of the disease. So that is appealing. From that standpoint, I think you know, it is again of interest. We’ll see how the phase three study progresses. But I think you know, more broadly speaking. And I hope you know, for those of you that are more focused on PV and ET, it’s not, you know, this is not all about myelofibrosis, but this is certainly just another example where there are a number of novel agents that are in various phases of development that, you know, beyond the currently approved for JAK inhibitors, we have, you know, all these other treatments, and they’re very different from JAK inhibitors, which are progressing through different phases of these clinical trials.
Josh Bollam (The LLS) 21:28
Now, now we get to transition to my favorite part. What does the future look like? What are you excited about in the question becomes, we see our clinical trials now, we see the research going on, and you can theoretically look a year down the road and kind of see what’s coming towards us. Where do you see MPN treatments in five to 10 years? What are the key areas of focus that researchers are looking at now for those five to 10 years?
Josh Bollam (The LLS) 21:56
So I’ll just start off by saying I know Dr Zhou mentioned this earlier, so with the CALR mutation, so patients with ET and myelofibrosis who are positive for this CALR mutation. You know, we think in some ways that the CALR mutation is a little more favorable in terms of risk of blood clots, in terms of sort of lower risk disease overall. But that said, again, we know that these diseases can cause problems, whether it’s in the short term or the long term, and we would love to have more effective therapies for any forms of these diseases. So with the CALR mutation, there are several different strategies which are now going into early phase development, which are very specific for this CALR mutation, so more of that personalized medicine approach. Dr Zhou mentioned this CALR antibody, so we are participating in that study. We just opened it so patients with ET and myelofibrosis who have the calar mutation are eligible for that study if they meet the other specific eligibility criteria. It is a good example of a setting where you might say, Well, if you have a patient with ET or MF who has the CALR mutation, and maybe they’re doing okay overall, you might initially say, why would they want to, you know, pursue a clinical trial and receive an experimental therapy? And again, that’s perfectly reasonable to say, full stop, not interested. But this maybe is a treatment that might be capable of having a profound effect on the disease and be more effective than the other available therapies that we have right now. And so for that reason, it’s, I think, appealing for certain types of patients in certain situations. So we are participating in that study, and there are other specific, personalized treatments for patients with CALR mutations, which are in early phase development as well, and so I think it’s a very exciting time, in that sense, for those types of approaches.
Josh Bollam (The LLS) 23:49
Yeah, excellent. Thank you. Dr Zhou, two phase question here we’ve mentioned the term personalized medicine a couple times. Can you just give us a high level overview of what that is, and then how impactful is the role of personalized medicine in MPN treatment? Specifically, how do you see genetic testing being used to tailor those treatment approaches?
Amy W. Zhou, MD 24:13
So I think, yeah, we definitely touched on this a little bit. You know, I think right now, with with the current therapies, you know, it is more, I would say, you know, something that that is less personalized in the sense that it’s effective for everybody. We don’t necessarily use somebody’s mutational status to decide, like, for example, which JAK inhibitor they could use. And so I think that as we have more treatments available, you know, I do foresee that that that hopefully will be the future, is that, you know, depending on what mutation somebody has, you know, we may have therapies that are more specifically targeted for those specific mutations, but I think that that’s. A work in progress right now? Yeah.
Josh Bollam (The LLS) 25:03
Yeah, excellent. It’s a work in progress across the hematology oncology sphere. But thank you. Question to both of you, MPN, along with a lot of other blood cancers are often seen as a disease of older adults. However, across the board, we’re seeing growing numbers of younger individuals being diagnosed. One, can you talk about what that means? What is a younger individual to you? And two, how might this shift in demographics impact treatment approaches, possibly even clinical trial design and research, and are there unique challenges or even opportunities associated with treating a younger MPM patient?
Stephen Oh, MD, PhD 25:46
Yeah, go first.
Amy W. Zhou, MD 25:48
Sure you can go first.
Stephen Oh, MD, PhD 25:50
So I think this is an important part topic. So yes, we have historically thought of and you know the statistics are that the majority of patients are diagnosed at older age. However you want to define that, I’m still in the young category. I’d like to say, but the reality is that increasingly we are, we are diagnosing patients at younger age, and part of that, of course, is increasing recognition some patients maybe, you know, we may have diagnosed at a certain age, but they maybe they had the disease years or decades prior. There is a lot of research that’s come out in recent years where we have learned that some individuals, maybe we found the JAK2 mutation, let’s just say, for example, at age 60, but they actually had it when they were age 20, or maybe even younger. It’s kind of a crazy concept that we’ve actually now know may be the case. So these things may be sort of brewing or sort of latent, hanging out for a number of years before they’re diagnosed. So we’re realizing, with additional research, with more studies, a better understanding of that concept. When we think about individual patients who are diagnosed at younger age, on the one hand, we say, you know, we know, particularly, let’s say for PV and ET, that patients can live decades with these diseases, and their disease may remain stable for that entire span, 20, 30, 40 plus years. And so you have a long life to live, you’re going to live with this disease. And that may be perfectly fine, but we also have to consider that living with these diseases for that period of time, however long that may be, that any some type of complication could occur at any point in that time, and that over that longer period of time, there is that, that looming possibility that the disease may ultimately progress, and so we have to think of it in terms of being proactive, thinking ahead, being prepared for those possibilities. And it reinforces that, though we want to make sure to reassure patients about the favorable outlook for these diseases overall that we are continually striving for. Can we more effectively treat the diseases? Can we maybe treat them earlier, with safe therapies that will prevent those long term complications from ever occurring?
Josh Bollam (The LLS) 28:14
Excellent. Thank you. Dr Zhou, anything to add?
Amy W. Zhou, MD 28:18
I mean, that was great. And I think as far as you know, the the question about maybe some unique challenges for managing, you know, some of our younger patients. I think something that I you know is becoming more cognizant of is, you know, kind of the fertility aspect and just things that you know may not we, I would say, may not necessarily be a consideration you know, for for older patients, however you define that. And so, you know, we certainly do see some younger patients in my practice and Dr Oh’s clinic, and that is something I’ll be honest. You know, I, I had to, you know, kind of make myself aware of is okay. These are some additional unique challenges. So making sure that they see one of our fertility specialists, for example, because some of the medications that we prescribe, you know, may affect that. And, you know, managing our patients through pregnancy, for example, things like that, which are, you know, unique for younger patients dealing with this disease. Thing, you know, that safety and and, yeah.
Josh Bollam (The LLS) 29:19
Yeah, that’s an incredible insight that somebody might not think about. I really appreciate the fertility aspect of it. Thank you both for your insights. Wonderful conversation. We’re going to have some time now for questions from the audience, both virtually and here in the room. We’re going to try and balance those out as we go around. Remember that doctors Oh and Zhou cannot offer personalized medical advice. We do encourage general questions about symptoms, diagnosis, treatment options, clinical trials. So as we open the floor, we’ll work in the room.
Josh Bollam (The LLS) 29:55
I have an online question first, what causes patients to be anemic and need transfusions.
Amy W. Zhou, MD 30:06
Sure I can take this. So I would say it really depends. But I think specifically in regards to if it is, you know, from, from the disease, you know, I would say it’s, it’s complicated in regards to, you know, of what goes into, you know, kind of development of that, the anemia, but it is a common complication, you know, in myelofibrosis, and I think that you know the mechanisms behind you know what leads to that and what leads to worsening of the the anemia is, I would say there’s many factors that go into that.
Josh Bollam (The LLS) 30:45
Are there ramifications for being transfusion dependent?
Amy W. Zhou, MD 30:49
So, you know, in regards to like prognosis. So transfusion dependence and anemia definitely are considered to be worse prognostic factors. And so those are important factors that are incorporated, you know, into these risk stratification systems that we have for our MF patients, again, in that in regards to trying to estimate their risk for, you know, progression to a more aggressive type of blood cancer like acute leukemia or survival. Yeah.
Josh Bollam (The LLS) 31:20
Yeah, excellent. I did see a hand somewhere in the room. Oh, there we go. Just give us a second while we get the mic back there, sir.
Attendee 31:32
Yes. I was told by Dr.Oh, that I acquired this CalR, it’s got a hereditary thing that correct? Dr, Oh?
Stephen Oh, MD, PhD 31:42
Yeah. So just to repeat the question for everyone in the room, as well as the online participants. So the question was for someone who tests positive for this CALR mutation, are you born with it, or do you acquire it at some time in your lifetime? And my response reply really applies to CALR as well as JAK2 and also MPL. These mutations are generally not something we think of as individuals are born with. And let me just try to be as precise about this as possible, because it can be very confusing. So when we think of mutations as, in some cases, being born with them, we think of diseases or disorders where mutations might be passed down from parent to child. Okay, there, there’s many examples of disorders where that is the case, where a parent has the mutation, they they transmit that to the child, and that’s a sort of a defined genetic disease. These are not those kinds of diseases, or these are not these kinds of mutations, the JAK2, CALR, and MPL mutations, you’re not born with them, and well more specifically, you’re not they’re not passed down from parent to child. Okay, so we think that at some point a blood cell acquires this mutation. Over time, those cells expand that carry that mutation, and then at some point, the disease becomes apparent. I’m speaking I’m choosing my words a little carefully, in part because of some of the research I mentioned a few minutes ago that that has come out in recent years, where we have begun to understand that the exact timing of the mutation acquisition may be years in advance of the disease becoming a parent. But that doesn’t change the fact that we don’t think of this. It’s not something where parent has a mutation and transmits it to a child. So the practical question that commonly arises, of course, is if a person is diagnosed with one of these diseases and is found to be positive for Cal or jak two or mept, the question is, do I need to worry about my kids? Do I need to get them tested for these mutations? Is that possible that they could have it because I have it? The answer is, no, they didn’t. They’re not going to get it from you. That’s not how it works for this these types of mutations, the more complicated answer just, I’ll just kind of take a minute to address this is we are aware that there is a slightly higher risk of family members developing a similar disorder, so what we call first degree relatives, so siblings, children. We see this in some families where multiple family members will have one of these diseases. It may be that one family member has PV and another family member has ET we see that on occasion, it’s not super common, but we know there is a slightly increased risk, that risk is on the order of two to four fold higher. In general, we think of these diseases as approximately one to two out of 100,000 in terms of incidence. So when we multiply that, I’m sorry to get into the mathematics here, but if you multiply one out of 100,000 times four, that’s four out of 100,000 that’s still pretty uncommon. So for that reason, we don’t recommend routine screening of family members. But it is true that there is a slightly increased risk and that there are families where multiple family members are affected. It’s not because the JAK2 mutation, or CALR mutation, is passed down from parent to child. It’s because there’s something else going on, probably some other genetic aspect, that results in some shared increased risk amongst family members, but strictly speaking, the JAK2 CALR mutation, they’re not passed down. You weren’t born with that in that sense. And apologies for the really long response to that question.
Josh Bollam (The LLS) 35:32
No, I think that’s a question that comes up in every blood cancer education program that I do, is, how does this affect my children you know, grandchildren, so thank you for going down that road. Yes, ma’am, right there. We’ll get you a microphone just one second.
Attendee 35:48
I can yell pretty loudly. Thank you. Dr Oh, do we think that any of these are not environmental per se, but influenced by environment.
Stephen Oh, MD, PhD 36:05
So that is a great question. Let me, let me respond in a couple of ways. So on the one hand, we do think, of course, of these diseases as being fundamentally driven by these mutations, JAK2, CALR, MPL being, being the most common, and then a blood cell acquires these mutations, as we just talked about. But as to the role of the environments, and the environment can refer to many, many things. Of course, there is increasing, at least evidence to suggest there is, there is some potential impact and some examples of that. So some of the research that my group has been doing in the laboratory and many others in the field as well has focused on the role of inflammation. And inflammation is a very broad term. Of course, some of you are quite familiar with different types of inflammatory disorders, autoimmune diseases and things like that, but as a concept that what I’m getting at is that we think that inflammation can have an impact on how these diseases develop. Doesn’t necessarily cause these diseases, but if there’s some abnormal inflammation, it may have some bearing on the disease course and the disease progression. That is a very tricky concept, and how that relates to individual patients. You know, may, may vary and may, you know, I think it’s fair to say we don’t fully understand but other aspects of the environment. So when we think about inflammation, in that sense too, you could think about maybe infections. So when, when this concept of these mutations may be initially being acquired years or decades prior to the development of the disease. Some we know, we know that in some cases, the mutation may be at a low level at one point, and then 10 years later, it may still be at a very low level. In other individuals, it becomes much higher. Why is that the case? Why is it different in individual patients? Probably a lot of reasons, but one may be environmental aspects, inflammation, infection. Maybe a severe viral infection could precipitate a change in that regard. This is a lot of speculation and theory, so to speak, as opposed to hard evidence, but I think we’re beginning to better appreciate that that could be a contributing factor.
Stephen Oh, MD, PhD 38:21
Excellent.
Josh Bollam (The LLS) 38:22
Excellent. Thank you. And bouncing back to our online audience question from Gail Dr Zhou, why don’t you take this? What treatments can you suggest that might delay progression, especially for ET or pre MF? We’re thinking about patients with few symptoms, but maybe high platelets.
Amy W. Zhou, MD 38:42
So, you know, I think in regards to progression, I’m assuming they mean, you know, maybe progression to myelofibrosis or acute leukemia. And you know, I would say right now, it’s, it’s not clear whether or not, you know, there really is a therapy that does, you know, decrease somebody’s risk for progression in myelofibrosis, you know, or acute leukemia, you know, Dr. Oh mentioned, there’s a lot of interest, you know, in interferons, in PV and ET and, you know, kind of the disease modifying properties of that, of that drug, but you know, as far as whether or not you know, so not to get too much into it. But I think you know, one of the aspects of that treatment is, they mentioned that it decreases, you know, the amount of mutation that you can detect in the blood. So the JAK2 mutation, for example, the the amount of it might go down over time, and maybe sometimes won’t even be detectable, you know, with that treatment. But whether or not that’s an adequate surrogate or replacement for survival, like actual data to show that okay, if you take this medicine, you will. Longer, or you will, you know, people who take this will have less risk for myelofibrosis. I would say that’s, that’s data that we don’t really have. Because the good thing is, you know, patients with PT do do so well. So if you do do a clinical trial, looking at those, what we call end points, you know, you’d be following patients for a very, very long time. And so that’s why we don’t necessarily have that information. And so that, that’s my long winded answer in regards to, you know, it’s, it’s, it’s a, it’s complicated, but it’s, it’s unclear. And so right now, there’s not necessarily any treatment that we recommend where we say, Yes, you should take this because it will decrease your risk for progression of myelofibrosis or acute leukemia. Yeah.
Josh Bollam (The LLS) 40:50
Thank you. Anybody in the room? Just one second while we get our microphone over there?
Attendee 41:00
Any recommendations? Josh, for lists of email lists where people patients can converse and talk about what they’re experiencing.
Josh Bollam (The LLS) 41:09
Yeah, that’s a great question. So not necessarily an email list, but what I would recommend is LLS’s patient community. It is our social media site or patient forum, and on that site, we have a number of different forums regarding all different blood cancer diagnosis. So MPNS is covered. There are even three sub categories for the major MPN subcategories, as long as well as treatment different psychosocial topics that are also covered on there, as well as being able to connect with patients in other populations around the country that you might relate to, from LGBTQIA to rural Patients, all on the LLS community. The best part about it is that it is monitored every day by one of our information specialists, so one of our nurses or social workers. So if something is posted in error by a patient or might need some clarification, they’re there to add it and provide reliable sources. And it’s just a it’s a good place to go to one connect with patients, know that it’s being monitored for truthful and accurate information, and that way you’re not always constantly asking Dr Google about what’s going on with you, because we know dr Google sometimes lies. So LLS patient community is a great place to start,
Stephen Oh, MD, PhD 42:43
Josh, if it’s okay, I would like to Yeah, please. So some of you are aware there are lots of different sources of information online. So in addition to the great resources, Josh mentioned, there are various websites. There’s MPN Research Foundation has lots of great information. There is MPN advocacy. Some of you are probably familiar with that. If you just Google, use Google, not Dr Google, you’ll find MPN advocacy. So there are email listservs and stuff like that, newsletters that can you can join to receive those updates. I just want to add as well that there are some of you also, I think are familiar. There are various Facebook groups for patients with MPNs. I think some find that extremely helpful. And you know some of you that are here in person, you’re getting to meet others with these diseases for the first time, and that’s meaningful. Online support groups can provide that as well, and again, be really helpful for individual patients to cope with these diseases and diagnoses in certain circumstances that can lead to more distress. So I always caution patients to you, have to make that determination as to whether these kinds of things are helpful, and again, in certain circumstances, it just sort of amplifies the concern and the stress and and so on and so forth. So just be you know, think about that when you explore these different options.
Josh Bollam (The LLS) 44:14
Yeah, thank you, and thank you for asking me a question I wasn’t expecting that. Yes, sir, right there. Hold on one. Oh, I’m sorry, yep.
Attendee 44:24
Is stem cell transplant always a treatment of last resort? Or do you ever recommend it? For instance, with a patient, for a patient with myelofibrosis before it goes into leukemia or something else,
Stephen Oh, MD, PhD 44:41
It’s a great question. I’ll give you my own perspective, and Dr Zhou may may like to offer her thoughts as well. So you know, on the one hand, I would say that, as a non transplant specialist, in some ways, I look at it as we’d like to optimize medical therapies. So. We can avoid the potential risks of transplant because they are substantial. On the other hand, you know, if I may, if I, if I made myself a transplant specialist, what I would probably say is, this is the, currently, the one treatment that has the prospect of long term disease free remission. And, you know, maybe even we can use that word cure in some cases. And in the best cases, patients do really well, and they have, you know, minimal complications, and it’s a great outcome. Okay, so so that that’s, you know, the other sort of side of the coin. And so from to go further along those lines, some might say the goal is to transplant everybody to make that approach safer and more effective, and we should be focusing our efforts on that and make it available to everybody that’s in a very sort of that’s the most extreme perspective along those lines. But I think, you know, there’s a middle ground, and it’s really patient specific, but so certainly not last resort. But at the current time, we choose carefully patients we think are in need of transplant, and who we think are, you know, have a are well enough to withstand the potential risks that go along with that.
Josh Bollam (The LLS) 46:11
I’d like to ask a little bit of a overarching question. It’s something that is near and dear to my heart about equitable access, and a lot of our patients who might be watching online might live in a more rural area or further away from a cancer center. Both of you work at one of our premier comprehensive cancer centers, dr, oh, you and I have talked about how patients drive for hours to see you, and I’m sure Dr. Zhou is the same way, but that’s not always a possibility for our patients, especially those underserved. They might not ever hear about a clinical trial. They’re probably getting treatment in their local community oncology office. What are some ways that we is a society as a world, or what are you doing to address maybe some of these obstacles that patients might face.
Amy W. Zhou, MD 47:04
Sure, you know, I think, and I’ll be honest with you, I don’t have, you know, kind of, I would say, a great solution to that challenge. You know, I think that, that you know, as far as the educational portion of things you know, because I think we recognize, you know, for our community colleagues, where they may be seeing many different types of cancers, and because MPNs are a rare disease, this may not be a part of their clinical practice, you know, compared to lung cancer or breast cancer, or many of these different diseases that they treat. And so, you know, as far as kind of staying up to date with the latest, you know, therapies and things I definitely, I would say for myself and Dr. Oh, you know, we’ve participated in, you know, kind of education that’s geared towards our colleagues as well. And certainly, we’ve always been very, you know, amenable to providing advice. You know, if a patient is not able to come see us, sometimes their provider may reach out and ask, you know, Hey, can I just get some information on that? And so, you know, I would say, sometimes we can provide some some information to local oncologists. If you know, the patient that they’re seeing is not able to travel to St Louis or or an academic center. But I would say, as far as you know, kind of some of these therapies, so the clinical trials themselves, or, you know, transplant, for example, where they they are not offered, you know, at some of some, some other more community based practices. I do think logistically, that’s that’s going to be a challenge, you know, and and in regards to whether or not, because, I think Siteman, you know, does offer, for example, like housing sometimes for our patients if they have to stay overnight, if they have to travel far, but certainly, you know, I think that that resource, you know, providing resources to help support patients who want to be able to, you know, access these treatments, but you know, may be constrained in various ways, yeah, but you know, I would say that that’s definitely a challenge sometimes.
Amy W. Zhou, MD 49:09
No
Josh Bollam (The LLS) 49:09
No thank you for taking a shot at it. It’s a complicated question, and none of us are going to have right answers, but it’s how we approach those underserved patients and how we make access for them. I know Dr Oh’s office is more than willing to help. His nurses, his clinicians, all reach out to patients, and I know your clinic does as well. So like to turn it back to the audience.
Attendee 49:38
On that subject, I’d like to tell you a quick story. I had ET for 10 years, and my provider saw a seminar, a web seminar, with Dr Oh and suggested him and another lady that I drive because I live in a rural area to see Dr. Oh, and I don’t see why anyone can’t drive 2, 3, 4, hours to try to get themselves a diagnosis, because the way Dr Oh was so open, took and Tammy was a great help, to take your files, they analyze them, and then they call you back and they make your appointment. And the only bad part was they took about half a pound, half a liter of blood out of my arm. But then two within the next two to four weeks, I spoke to Dr Oh three times on the telephone, and finally he said, Well, you’re pretty lucky. You got CALR mutation. I said, why is that? He says, Well, of all the people that have ET, the ones with the CALR, have the best longevity. And then I looked up the CALR, and it was discovered 10 years before Dr Oh told me about it. So now I’m going on 13 years. So I think that it should be pushed out there to the rural people that make the sacrifice, if it’s 2, 3, 4, or 5 hours and come to see Dr Oh or anyone in another city, in Cleveland or Pittsburgh, they should feel that they’re not getting
Josh Bollam (The LLS) 51:15
I think your story leads to a great question for those patients who are driving one two hours, how can they optimize the time with their healthcare team for not only their care, but also their quality of life, because they might not be able to make the regularly scheduled appointments that someone who lives In town might be able to attend?
Stephen Oh, MD, PhD 51:41
Yeah, so I can comment on that and definitely appreciate your the positive feedback. I you know, we we recognize that every patient has, you know, maybe have specific circumstances or challenges that you know propose real logistical constraints, no matter how far they might be coming from when they might be in town, and it’s still, you know, there’s still specific challenges. One thing I want to mention is, is we do, do strive for our clinical trials to ensure that there are support for travel. So again, I understand fully that for many patients, that’s just not feasible, partly, in some cases because of the distance, but in many cases, we have mileage reimbursement. We have funds for meals. We’ll have if it’s a long visit and they’re traveling from a distance, we may have support for hotel stays, so we try to make that at least as best as we can, to provide those resources to help patients make it feasible to participate in clinical trials if it’s appropriate. But I think the other thing that I will say I personally learned over time is that we tried to individualize a partnership between the patient and, if it makes sense, a local physician in a way that is productive. So it can be different. It can be that in an individual circumstance, the patient sees a local provider frequently, and comes to see us once a year. And that works well. We have arrangements where we alternate. They see us every six months. They see the other provider every six months, and it becomes every three months they see one of us. We try to support if a patient needs blood count monitoring that that can be done at a local lab that’s close to their home. It doesn’t matter if it’s in our system, as long as we can get the results. So we do our best to make that feasible. And when I say we, I mean our team, Tammy has already highlighted our clinical research team, Nicole and Karen in the back. So you know, the members of our team really make that possible to support patients in whichever way makes sense.
Josh Bollam (The LLS) 53:51
Excellent. Dr Zhou is a follow up. What, when you are first interacting with a newly diagnosed patient, what are some recommendations that you or your team might give them to maximize the time that they have in that clinic, room with you, to ask the right questions, retain the information things along that line.
Amy W. Zhou, MD 54:14
So I think for new visits, I do, I do think that sometimes it can be a little bit overwhelming in regards to because so much is new. You know, you’re getting a new diagnosis. Oftentimes I’m spewing a bunch of information, and it is very hard, I think, to process all of it, you know, in like a 30 minute interval, let’s say, and so oftentimes what I tell patients is, you know, this is a lot of information I’m giving you right now. You know, I understand that for most of us, and for myself included, sometimes you need to just take that information, have time to process it, and then think about, you know, what questions you have, and you don’t have to ask me all of it in that one visit. You know whether, because we you can call us. We have, you have my chart now you know, so you can message us. And so there’s all these different ways to communicate with us where, you know, again, we understand that this is all new, and there’s a lot of information to understand and process. And so you don’t have to feel like you need to get, you know, like everything done in that visit. If you know, afterwards you have questions, you know, certainly I would say for our team, and I think for Dr Oh’s team as well. You know, we’re accessible. In regards to, okay, if you didn’t have this, you didn’t think of this question initially, and you have it now, please message us, and we’re happy to answer that. And you don’t have to, you know, kind of wait until your next visit either. So that that’s generally kind of been my approach, you know, in regards to, yeah, I would say helping to kind of best process and retain that information. But also, I’ve also had patients ask me to write things down, you know, in kind of their little like after visit summary. And I’m also happy to do that too.
Josh Bollam (The LLS) 56:05
You mentioned something that I think is has come about in, I would say, the last 10 years, and it’s something very new to a large part of the population. You called it MyChart. Different hospitals call it many different things, but it’s a way for patients to get a glimpse into their health record that’s kept by their doctor. A lot of times, test results can pop up on there before you, as the doctor has had a chance to go over that with a patient, either one of you, how do you talk through that with your patients? And say, yes, you can look at it or wait till you hear from a member of my team. What is your advice?
Amy W. Zhou, MD 56:47
So generally, I do, you know, my advice is, I would say I don’t expect you to interpret your own test results, you know. So I will, yeah, message you and explain these results, and then we can discuss it together, yeah? But that’s usually my advice is, yeah. Don’t try to interpret it yourself. Don’t go on Dr Google and try to get Dr Google to interpret it. Yeah. You know, I help. Let me provide that information, yeah,
Stephen Oh, MD, PhD 57:16
Just one comment along those lines. So I think it is an evolving dynamic between the provider and the patient. I’ve been on both sides of that equation with myself and family members. Sometimes you can see in my chart, there’s a test result, and, you know, it’s resulted. It tells you the status that’s resulted, but it won’t give you the result, because the doctor has to sign off on it, you know, if it’s a sensitive result for you, but you see that there is a result there, and you want to know what it is, and maybe you might not be able to interpret it, right? So it’s very it can create a lot of stress, right? I understand that it’s, again, it’s a case by case in terms of the dynamic and how best to handle that, but I agree that, you know, it’s the best and most productive way in general, is to talk it through with the patient, if it’s a sort of a complicated result, to best put that into the proper context.
Josh Bollam (The LLS) 58:03
Thank you. And I’m glad that even a doctor looks at that and is like racing, like trying to see the results, so it makes me feel a little bit better about chugging my cholesterol numbers. Uh, anybody else in the room have a question? Yes, ma’am. I
Attendee 58:25
Okay, I have kind of a two part question. I hope I word it right. That makes sense. The big question is, what do you know? What are we doing about a cure? You know, to actually cure this disease, instead of just living with it. And then, along with that, you know, when you’re talking about a cure, are you looking at the allele burden to kind of bring that down to zero, or are you talk is the allele burden irrelevant with that? And I don’t know if I’m phrasing this in a way that makes sense, but hopefully, you know, everything we’ve talked about, talks about living with the disease and symptom management and lowering risk of blood clots, for PV and ET, those things. But what about cures? What about ringing that bell being done? What’s in the what’s in the future for that?
Stephen Oh, MD, PhD 59:16
Yeah, well, so I can take the initial response. So, I think these are great, important fundamental questions for individual patients, for the field, and I hope, hopefully it’s come across that that is something very much on my mind, and for everyone in the field, that, on the one hand, for certain types of these diseases, we do have therapies that can keep things stable. They can help with symptoms. Many patients can live for many years or decades with stable disease, and that’s great, you know, living with the disease, you know, sure that’s that’s great, but we want to reach a higher level. That is definitely a goal. How to achieve that is, is, of course, the question and the challenge. And, you know, just to give an example in the myelofibrosis setting, we say, well, we have these four drugs that are FDA approved in various ways that can help with symptoms and other features the disease. Why are we focusing on those things, continuing to focus on those things? And the answer is, because we need better treatments that are capable of doing more. So we have to have those things at our disposal, or at least with the prospect of that, to be able to really sort of go for that. And so the field is, these fields are evolving very rapidly, but increasingly we are being focusing on that. To get more specific to some of the questions you raised. Yes, in many ways, the goal is to reduce the the mutation burden to zero or undetectable. There are sort of several layers of nuance beyond that, in terms of, what does that mean? Where we that’s a question that has been if you go to a research meeting in this field, there will be hours of discussion about that question with whether it’s with respect to the interferon drugs, which and some patients have that ability to reduce that mutation allele burden. What does it mean? Can we start to use the word cure in some patients? We don’t really know right now so much, but it’s going to take continued effort research. But for sure, it is a goal of the field.
Josh Bollam (The LLS) 1:01:21
I think that that was a great question to end on, and I know we’ve covered a lot of information tonight, but before we end, I want to ask one final question. It’s something that I was talking about with my colleagues a couple days ago, but I would love to hear your perspective. What would you say to your MPN patients now that you would not have been able to say to them 10 years ago.
Stephen Oh, MD, PhD 1:01:49
You want me to go first?
Amy W. Zhou, MD 1:01:52
You can.
Stephen Oh, MD, PhD 1:01:55
Well, I guess you know, 10 years so this is 2024 so 2014 if you if you look back in time, at that time, we had one approved treatment, that was Ruxolitinib (Jakafi) Okay. So we have come a long way. We have four JAK inhibitors approved for myelofibrosis. We’ve had approval of ropeginterferon alfa-2b for polycythemia Vera. And we have more to come in the coming years, I would say, so that’s, you know, those are all really big changes and positive developments in the field. I would also just say, I don’t remember for sure, but I would say back then, I would be giving the same message to all of you, which is, I’m very optimistic, and in the next 10 years, we’re going to be making even more progress. So you know, this is a field of intense focus, research wise, both in the laboratory and in terms of clinical trials. And I fully anticipate that in the next decade, we’re going to have big advancements coming. And so I’m very optimistic.
Josh Bollam (The LLS) 1:02:56
Thank you, Dr Zhou?
Amy W. Zhou, MD 1:02:58
I definitely share that optimism. And, you know, I Yeah, so I definitely agree, you know, kind of with the availability of all of these new therapies that has been within the past 10 years. And also, you know, just again, kind of going back to, you know, what we were talking about in regards to molecular testing and incorporating, you know, these additional mutations, you know, into prognosis. So, you know, thinking back 10 years ago, you know that wasn’t necessarily the those prognostic scoring systems that were incorporating kind of these different mutations that hadn’t yet been developed. And so that is something also that’s come about in these this, these past 10 years. And so, yeah, I’m very excited to see what the next 10 years holds just in regards to what additional therapies and again, kind of more personalized medicine as we understand the disease better?
Date
Held on Nov 14th, 2024
Replay coming soon – sign up to get alerted when it releases.
This program was a live in-person program in St. Louis, MO.
This program is for those affected by Polycythemia vera (PV), Essential thrombocythemia (ET), and myelofibrosis.
MPN Experts from Siteman Cancer Center
Dr. Stephen Oh is Associate Professor of Medicine and Co-Chief of the Division of Hematology and Dr. Amy Zhou is Associate Professor of Medicine in the Division of Hematology. They are both at Washington University School of Medicine. This program covered the following topics:
- Gain practical tools for monitoring symptoms, managing side effects, and improving your day-to-day life.
- Discover the latest treatment breakthroughs, including clinical trials, and what they mean for your future.
- Hear inspiring stories from others living with MPN and receive expert advice on emotional well-being and self-care.
Stephen Oh, MD, PhD
Associate Professor of Medicine
Co-Chief of the Division of Hematology
Washington University School of Medicine
Amy W. Zhou, MD
Associate Professor of Medicine
Division of Hematology
Washington University School of Medicine
Thank you to The Leukemia & Lymphoma Society, Imerman Angels, and the MPN Research Foundation for their partnerships. We are fortunate to work with such amazing partners that are here for you with information about clinical trials, resources, and support.
This program is more than just an information session—it's a vital resource for those affected by myeloproliferative neoplasms (MPNs). Hear from leading experts and patients, and uncover the latest advancements in treatments, including clinical trials and targeted therapies, to help you or your loved one navigate this journey with confidence and hope.
Thank you to GSK, Karyopharm, Novartis, and Sobi for their support of our patient education program! The Patient Story retains full editorial control over all content.
Stacy’s Myelofibrosis Story
Cathy’s Myelofibrosis Story
Natalia’s Myelofibrosis Story
Ruth Fein: Myeloproliferative Neoplasm (MPN) Story
Jeremy’s Myeloproliferative Neoplasm (MPN) Story
