Choosing an MPN Treatment: What Option Is Best for You?

Choosing an MPN Treatment: What Option Is Best for You? from Patient Empowerment Network on Vimeo.

When choosing an MPN treatment, what’s right for you? Dr. Brady Stein from Lurie Cancer Center reviews key decision-making factors, current treatments for ET, PV and MF, and shares advice on advocating for yourself.

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Transcript:

Katherine:                   

Hello, and welcome to the webinar. I’m Katherine Banwell, your host for today’s program. Today, we’ll discuss how you can be proactive in your understanding of MPNs and work with your healthcare team to find the best MPN treatment path for you. Joining me today is Dr. Brady Stein. Thank you for joining us. Would you please introduce yourself?

Dr. Stein:                     

Hi, my name is Brady Stein. I’m a hematologist at Northwestern University in Chicago, and thanks very much for having me.

Katherine:                   

Before we begin, a reminder that this program is not a substitute for seeking medical advice. Please refer to your own healthcare team about what might be best for you. Dr. Stein, let’s start with the basics. The term “myeloproliferative neoplasms,” or MPNs, can be a bit confusing. Would you give us a brief overview of the classic types of MPNs? 

Dr. Stein:                     

Sure. So, there’s three classical types of MPNs that we focus on. These are the BCR-ABL-negative MPNs. So, what we’re referring to in this webinar is essential thrombocythemia, otherwise known as ET, polycythemia vera, often referred to as PV, and myelofibrosis, often referred to as MF, and myelofibrosis can exist on its own.

It can be primary, so a patient can start with that diagnosis, but it’s also important to note that patients who have ET or PV for long periods of time – they can head in the direction of myelofibrosis, so that’s kind of a secondary type of myelofibrosis, sometimes referred to as post-ET MF or post-PV MF. So, those are the three basic subtypes that we’re gonna speak about today.

Katherine:                    

Do symptoms vary for each type – ET, PV, and MF?

Dr. Stein:                     

They do. They vary, but they can overlap. So, oftentimes, ET is thought of as the more indolent of the MPN subtypes, but I think it’s pretty clear that patients with ET can have a similar burden of symptoms compared to patients who have polycythemia vera and myelofibrosis, so they exist on a spectrum, I think most to a degree that symptoms can be the most pronounced in patients with myelofibrosis, but not to undermine the symptom burden that can occur in ET and polycythemia vera.

Katherine:                   

Once a patient is diagnosed with MPN, what sort of testing should take place?

Dr. Stein:                     

So, the test that’s gonna lead to suspicion is gonna be a blood count, and that’s probably gonna be done in the primary care doctor’s office, so that’s gonna be the first suspicious test, and in general, there’s gonna be some abnormality. Myeloproliferative diseases are characterized in general by an overproduction of blood cells, so it’s going to be a higher white count, it’s going to be a high hemoglobin or hematocrit, or a high platelet count, or a combination of the three that’s generally gonna lead to suspicion.

Some patients may have pretty unremarkable blood counts and may present with a blood clot in an unusual location that could ultimately lead to the hematology referral. Some patients might have pretty unremarkable blood counts, but they might have palpation of their spleen, enlargement of their spleen in a physical examination. So, they’re generally the ways that patients are getting to the hematologist.

Katherine:                    

And, what about bone marrow biopsy?

Dr. Stein:                     

So, a bone marrow biopsy is a diagnostic test, and it’s generally recommended for all patients who have a myeloproliferative neoplasm either confirmed or suspected.

It’s advised in WHO criteria – World Health Organization criteria. PV can be made without a bone marrow biopsy – a diagnosis of PV – because it’s the most unique of the MPN subtypes. It’s the one that presents with a high hemoglobin.

So, that diagnosis can be straightforward at times for a hematologist when the setting is right, when there’s a high hemoglobin – or, high enough hemoglobin, I should say, a JAK2 mutation, which all patients with PV have that, or a subnormal erythropoietin level.

Oftentimes, we can make that diagnosis without a bone marrow, and the bone marrow becomes more prognostic. ET, a bone marrow is necessary for diagnosis, and myelofibrosis, you can’t make a diagnosis without it.

Katherine:                   

What do the results of these tests tell us about prognosis and treatment choices?

Dr. Stein:                     

That’s a good question. The bone marrow can be diagnostic in the setting of ET and myelofibrosis. In the setting of polycythemia vera, it can be more prognostic. In general, when a bone marrow is done, 1). To confirm diagnosis, 2). To assess prognosis, what we’re looking for as prognostic features are generally the degree of fibrosis or scarring.

So, each of the MPNs can have that. Of course, MF is characterized by the most pronounced scarring. It can happen to a more subtle degree in ET and PV. That’s gonna be prognostic in the setting of ET or PV. The pathologist will alert us about immature cells called blasts. We basically never see them at diagnosis in patients with ET or PV. We can see them rise in patients with myelofibrosis at diagnosis or through the course of follow-up. So, that’s prognostic.

All bone marrows generally have a chromosome analysis that’s called cytogenetics, and so, if there’s an abnormality, that can help place the patient’s prognosis into different risk categories. And then, nowadays, more so in myelofibrosis than any of the others, there are extended panels done. These are called NGS, or next-generation sequencing, kind of looking at mutations in a greater degree of detail.

So, not just what we call main mutations – JAK2, calreticulin, or MPL. These are looking at additional mutations that basically hold prognostic significance.

These are pretty well defined, and I think more important in MF compared to the other subtypes.

Katherine:                   

Would you explain the driver mutations in MPNs? What are they, and how they – or, what they mean for patients?

Dr. Stein:                     

So, there’s three of what we call driver mutations, and the most common is JAK2V617F, the next most common is calreticulin, and the least common or most rare is a mutation of MPL, the thrombopoietin receptor. So, the driver gene mutations are the three that we assess to help with diagnosis, and the prevalence varies. In ET, about 60% have JAK2, 25% have calreticulin, 5-10% have MPL.

In PV, 99% have some type of JAK2 mutation, and in MF, the situation is a lot like ET – 60% JAK2, 25% calreticulin, about 5-10% MPL. So, the driver mutations – we think of those as the genetic abnormalities that really drive the disease. They’re the main ones we can test for in a diagnostic setting.

I refer to them as the – to a patient, what I’m describing is you have a car, and the driver mutation is the one that’s sort of driving the car, and it’s doing it somewhat recklessly. It’s in the front seat, driving. And, along the way, the driver can pick up hitchhikers, which we should never do. I refer to those other mutations that are found by NGS as hitchhiker mutations that sit in the back seat, cause trouble, and really shouldn’t be there. They’re not the driver, they’re not fully responsible for the disease, but they can make it a bit worse.

Katherine:                   

We have a question from the audience, Dr. Stein. “I was recently diagnosed with MF, and I’ve had a bone marrow biopsy. How often will I need to undergo this procedure?”

Dr. Stein:                     

That’s a really good question. It’s a very important, very common question. So, the first bone marrow biopsy is diagnostic. The second bone marrow biopsy – or third – are generally reactive, meaning we don’t schedule them year to year unless there’s a change. We do them in a reactive way. If there’s something about the condition that’s changing – for example, if we suspect or worry about a progression – that’s when we would do the second bone marrow biopsy. So, we don’t set a determined frequency if the condition or the course is stable.

Katherine:                   

Dr. Stein, I understand that therapy is different for each of the MPNs. What do you take into consideration to help guide the treatment choice?

Dr. Stein:                     

So, that’s a good question. It’s gonna require maybe a little bit of a longer answer. It’s fairly nuanced. The treatment of ET and PV – right now, the goals are – the treatment is largely based on a patient’s vascular risk. That’s largely what influences the choice of therapy. And so if a patient has a perceived high risk for vascular complication in ET or PV, that’s when we’re gonna be a little bit more aggressive – so, more aggressive than watchful waiting, more aggressive than using aspirin alone, more aggressive than using phlebotomy and aspirin alone in polycythemia vera.

So, if the patient has a higher vascular risk, in general, we’re gonna need to do something more than what we consider to be the standard, and that’s where we enter into the question of cytoreductive therapy – therapies designed to lower blood counts apart from phlebotomy.

Maybe that’s gonna change. I hope it will change. Right now, the therapy for ET and PV is generally reactive. We either predict high risk and react, or if a patient is lower risk, if something changes – God forbid there’s a blood clotting event – then we may react to it.

So, ET and PV treatment are generally more reactive. In myelofibrosis, certainly, there are patients who can have lower risk and minimal systems, and there are some patients who can be observed with watchful waiting for sure, but more patients are symptomatic, more patients are gonna need therapy in myelofibrosis, and there’s sort of two big categories of therapy.

One is the risk-adapted, deciding if the patient is eligible and should consider stem cell transplant versus thinking only about medical therapy in a patient that may be transplant-ineligible.

And, the medical therapy is based on the worst symptoms for the patient. Is the symptom that’s the worst the spleen enlargement? Is it excessive fatigue? Is it weight loss, or inflammation, or fevers? If it’s that category of symptoms, we have a set of therapies. If it’s really the anemia that is the most problematic issue, then we follow a paradigm to treat anemia.

Katherine:                   

What about considerations like the patient’s health, age, genetic markers, things like that?

Dr. Stein:                     

So, of course. The comorbid illnesses can influence therapy choices, so if a patient is older and has other medical conditions, they’re not gonna be treated as aggressively.

So, in myelofibrosis, if a patient is older, with other medical illnesses, then it may be inappropriate to consider something like stem cell transplant, for sure. So, age and health comorbidities are highly influential. In terms of genetic features, if you’re asking about things like the type of mutation that a patient has, right now, we’re – in terms of vascular risk, for ET, the type of mutation matters for blood clotting risk, so if patients have different mutations, it could be treated differently. In other subtypes, like PV or myelofibrosis, in general, there’s – the mutation can be prognostic, but it may not be – it may not lead to a precise and distinct therapy just yet.

Katherine:                   

All right. What do you feel is the patient’s role in the decision for therapy?

Dr. Stein:                     

I think it’s a really important role. I think historically – and, this is decades past; this era should be well over and behind us – this era of authoritative medicine is over.

You can’t just have a doctor walk in the room and say, “This is your treatment, this is what you should do, I’ll see you later.” It’s shared decision-making, and that can be troubling for some patients. But, the idea of shared decision-making is us explaining options informing the patient and making decisions together. That’s really the paradigm for modern contemporary medicine.

Some patients have a harder time with that. A lot of patients say, “Well, doc, this is too overwhelming for me. I just want you to decide for me.” And, we try not to do that. That’s a more uncomfortable type of visit for me when a patient is very deferential and says, “Whatever you say, I’ll do.” That’s not really what we wanna hear. I wanna know that you feel really informed, that you have a good understanding because each of these treatments – any treatment, any medication has its pros and cons.

There’s no real magic bullets, and each upside has an equal downside, so you have to engage and open a dialogue, and what that means is that patients need to read and learn. That’s hard, but patients need to become proactive in their approach to their own illness, and all the patients who are listening now are doing that, trying to get more education about your relatively rare illness that’s gonna give you a much better framework to help make decisions together.

Katherine:                   

Absolutely. If a patient isn’t feeling confident with their treatment plan or their care, do you recommend that they maybe consider a second opinion or seek a specialist?

Dr. Stein:                     

Of course, yeah. These are rare diseases, and patients often – I would say that in my clinic, a lot of the patients direct their own second opinions. Oftentimes, it’s coming from the patient more so than their doctor. I think the patient community is very active, the patients are networking, and they’re finding the right specialist to get to.

I think it should be really a team approach. It’s never – it’s usually not very convenient to go to a university unless you live really close, so you wanna have someone close to home who can handle the routine, and then, someone who maybe is a little bit further away who can see you once a year, can help with the big decisions, can be part of the healthcare team. So, we generally recommend that you have someone near, and that maybe you have someone far who focuses only on MPNs as part of your team, and now, it’s a little different. Telemedicine is becoming a pretty ingrained part of medicine. It’s a little easier to have those visits with a physician who’s far away because of telemedicine.

Katherine:                    

So, now that we’ve discussed how a treatment path is determined, can you walk us through the currently available MPN treatment approaches and who they might be right for?

Let’s start with essential thrombocythemia, or ET.

Dr. Stein:                     

So, I think the first thing is taking an inventory of symptoms, seeing how symptomatic the patient might be. Again, there are some patients who are asymptomatic or have few symptoms, and they were told of a high platelet count during a routine visit, so some patients can be observed if they have few symptoms, and especially if they fall into a lower vascular risk category.

So, symptom assessment first. Second, looking at vascular risk, and there’s four categories of risk in in ET in terms of predicting the likelihood of a future blood clotting event. There’s a very low, low, intermediate and high risk group, and that’s based on a patient’s age, whether they’ve had a blood clot before, and the type of mutation they have. JAK2 mutations increase the risk of clotting.

So, if a patient falls into a higher-risk group – say they’re older than 60 with a JAK2 mutation or they’ve had a prior blood clot – those are patients who are generally treated more aggressively with cytoreduction. And then, the other thing is aspirin. We often see aspirin given to all patients with ET, but not all patients with ET necessarily need it. The role of aspirin is actually a little less clear in ET. For a very low-risk patient, there’s a potential for more harm than benefit, especially if the patient lacks a JAK2 mutation. So, the evidence base to support aspirin for all ET patients is just not there; it’s evolving.

Katherine:                    

What about polycythemia vera, or PV?

Dr. Stein:                     

So, there’s a few standards. It’s different – the aspirin question in PV is generally answered by randomized data from 16 years ago in 2004. It’s been shown that aspirin reduces the risk of clotting in PV patients, so, generally, we give low-dose aspirin to all patients.

And, hematocrit control is really important. At least, a goal of 45% is mandated in PV. And then, there are patients who might fall into a higher-risk category – older than 60 or have had a prior blood clot – they need something more. And then, I’d also emphasize that there are lower-risk patients who may not be traditional candidates for cytoreduction, but they could have symptoms that really interfere with quality of life, and symptoms alone can be the trigger to add something more to the phlebotomy and aspirin program.

Katherine:                   

What about things like interferon?

Dr. Stein:                     

So, interferons have been used in MPNs for decades and decades. So, a longstanding history with interferons. The issue has been tolerability.

These days, there’s a class called pegylated interferon that’s longer acting, and I think there’s been a lot more use, at least in the last 10 years, still much more in an academic setting than a community practice.

But, interferons have a pretty established role in MPNs, especially polycythemia vera, for sure in ET, less so in myelofibrosis.

Katherine:                    

Dr. Stein, you mentioned high-risk versus low-risk patients quite a bit. How is that risk determined?

Dr. Stein:                     

So, it’s different for each subtype. For ET and PV, when we talk about high versus low risk, we’re talking about vascular complications, risk of having a blood clot. We’re not really talking about risk of transformation. We don’t have, I think, wonderful, widely used toolkits to predict those things. We know they can happen, but our treatment is still really based on clotting for ET and PV.

And, MF – each couple of years, the tools that are available to assess prognosis become more and more. So, in MF, we’re using the most comprehensive approach – of course, taking into account things like age and demographics, but also, looking at symptoms, looking at the depth and severity of blood count changes, looking at bone marrow features like the degree of scarring, looking at the rise in blast counts, and then, looking at chromosomes and novel genetic markers. So, we’re definitely the most comprehensive in myelofibrosis at assessing prognosis.

Katherine:                   

How do clinical trials fit into treatment choices?

Dr. Stein:                     

Clinical trials are always a treatment – always an option for patients with myeloproliferative neoplasms because while we have some standards, we can definitely improve upon those standards for certain. So, clinical trials are always a therapeutic option. I think the one thing is that it may not – it’s not always the most convenient option, but it could be a really important option if available to you.

So, clinical trials basically offer something new or novel that would not otherwise be available to other patients. So, ruxolitinib was approved around 2011, but the first clinical trials were in 2007, so that’s the example I give to a patient about the benefit of a clinical trial.

The patient can get access to a drug that’s effective perhaps three to four years before it’s commercially available.

That’s really the biggest advantage, is you can get early access to something that could really help you. The downsides are that clinical trials are not usually as convenient as regular care, there’s often more visits, and there’s a lot of unknowns – unknowns about whether it will work. Some side effects are known and expected; there are others that are unknown. So, it’s a lot to think about, but I think it’s always important to consider, especially if your first-line therapy has not been effective, if it’s losing its touch, it’s a good thing to think about for a second line.

Katherine:                   

Are there emerging approaches for treating MPNs that patients should know about?

Dr. Stein:                     

Yeah, absolutely. I think the first question – I think patients are often worried that they have a really rare disease, and why would anyone do research in this area, and that’s – the research community is extremely engaged, the productivity is pretty impressive, and there’s a lot of clinical trials in the space, and I think what I try to explain is pharmaceutical companies aren’t just targeting the most common diseases.

They have interests in rare diseases, and findings in rare diseases can be extrapolated to other diseases that you might think are unrelated, but they can share features, so when you find something working in one space, it can have broad applicability. So, there’s an abundance of research in myeloproliferative neoplasms which are emerging?

In PV, I think there’s quite a possibility that there’ll be a drug approval in 2021, a novel type of interferon called ropeginterferon.

That is a drug that’s approved abroad; it’s approved in Europe, and I believe it’s approved in Taiwan, and the FDA is looking at it now. So, it’s a possibility that there’ll be a future option for patients with polycythemia vera. So, yes, it’s research now, but it could be available, and so, that’s the drug that I’m starting to talk more and more about for patients with PV.

In myelofibrosis, you have two JAK inhibitors that are approved, ruxolitinib and fedratinib, you have two others in clinical testing, momelotinib and pacritinib, and then you have a whole other class of what we call non-JAK2 type of therapies targeting the vast array of pathway abnormalities in myelofibrosis.

So, there’s a number of different clinical trial options, especially in myelofibrosis. I think that’s the disease area where there’s the most clinical trials.

Katherine:                   

Once a patient has started treatment, how do you know it’s working?

Dr. Stein:                     

That’s a good question because this is a very unique area. Yes, of course, in some respects, it’s straightforward with ET or PV. If we’re starting a medication to control a blood count in hopes of having lowered the thrombosis risk, you can look objectively at blood counts.

Okay, your hematocrit is at this goal? Yes, therapy’s working. You have not had a blood clot? Yes, therapy’s working. So, there are some objective things. In myelofibrosis, there are some objective things like measuring the spleen and seeing it reduce. You can feel that with your hands, or you can do an ultrasound. So, there are some objective parameters of success. But, in this area, patient-reported outcomes are really important, and so, a measure of success is really just asking the patient, “Do you feel like your drug is working? Do you feel better?”

It’s kind of a simple question, but it’s really important, and it’s what we ask in patients who are on certain therapies. “Do you feel like the net effect of your therapy is still positive? Do you feel like it’s helping?” Seems like a straightforward type of question, but I think the answer is extremely informative. When a patient says, “Yes, definitely, my medication is still helping me,” then I know that I don’t need to change it.

Katherine:                   

Right. Patients are often concerned about progression in MPNs. Would you explain the progression of MPN and the indicators that one might be progressing?

Dr. Stein:                     

So, I think the important but hard thing to know about MPNs is that they’re chronic progressive illnesses, but what we don’t know in an individual is how long it’ll take to progress. Is the ET or PV gonna progress in 10 years, 25 years, or 35 years? Those are difficult things to predict. And, MF is progressive as well. I think it’s a little easier to identify those patients who may progress more rapidly compared to more slowly.

So, MPNs progress, and that’s the first important thing, is that they’re chronic illnesses and patients have to be aware that they can change. ET and PV can move to myelofibrosis, and when that happens, generally, symptoms change, the spleen enlarges, and blood counts change. We start to see anemia when we didn’t before.

And, when MF progresses, the symptom burden is one thing, maybe more fatigue, unexplained fevers, drenching sweats, or weight loss. On exam, measuring the spleen and seeing it get larger, seeing a fall in the platelet count, a need for red cell transfusions or a rise in the blast count. Those are all features that we’re looking for that can indicate a progression.

Katherine:                   

Let’s talk about patient self-advocacy now, Dr. Stein. Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions.

Why is it important for patients to speak up when it comes to symptoms and side effects?

Dr. Stein:                     

I smile a little bit because patients – I get a lot of patient emails by MyChart. That’s our medical record, and it’s a secure patient email, and a lot of patients will start their message by saying, “I’m sorry to bother you.”

And, I always say, “Why do you think that? It’s my job. Please don’t apologize for reaching out to me.” So, that’s kind of the first thing. Don’t feel like you’re bothering your doctor. There are certain things that we won’t know unless you tell us, and so, I think that’s pretty clear. When we’re in a patient room and there might be a husband and wife together, and whether it’s the husband is the patient or the wife is the patient, we might ask a question, and we might get, “No, everything is fine,” but all doctors kind of sneak over to the partner, and the partner may be saying – they’re making gestures to us. There may be nonverbal forms of communication to tell us there’s something much worse than what the patient is telling you.

So, again, “advocate” meaning you have to tell us what’s going on with you. If you’re worried about something, please don’t be stoic about it. These diseases are treated a lot based on your symptoms, and so, if you don’t tell uls about your symptoms, we won’t know.

And, in terms of advocacy, I think one of the things is that these are pretty rare diseases. In an academic center, no, this is our focus, but if you’re in a community practice where the doctor’s seeing 10-15 different things during the course of a day, it’s basically impossible to keep up with myelofibrosis, especially if you have one patient in your whole practice. I can’t do that for diseases that I see that I have only one patient. The medical literature can be overwhelming.

So, patients can quickly outpace their doctor in terms of their knowledge of these diseases, but I think it’s really important to read, to learn, and to think about the illness because you may find out things through your research that your doctor wouldn’t know are available. You may find a clinical trial, a new strategy, or a new test that they simply haven’t had the time to keep up with or learn about. So, that’s what advocacy is about. Reading is really important, but you have to find a balance. I want my patients reading, but you’ve gotta find the right amount because there’s a certain amount of reading where the patients start to get overwhelmed.

All patients kind of get to this point. They take it in – like taking it in like a fire hydrant in the beginning of the disease, and it’s overwhelming, and then they start to find their balance. I think there’s a point where the reading becomes anxiety-provoking rather than ameliorating anxiety, and all patients just generally find their balance.

What I also say is if you read something that alarms you, write to me, write to us, and let us verify that because there’s a lot out there, and I think the patient communities are a phenomenal form of support, but there’s a lot of patients giving advice to each other, and sometimes that needs to be double-check – or, always, it needs to be double-checked by another doctor because sometimes, the advice is simply not – may be very individualized or not generalizable, or sometimes it’s simply inaccurate.

Katherine:                   

What would you like to leave the audience with? Are you hopeful?

Dr. Stein:                     

Oh, of course. Of course, I’m hopeful. What I leave the audience with is that things are changing; things are changing for the better, and the therapeutic choices in three years could be entirely different. I think there’s progress. Progress in medicine – some patients feel it, it’s slow, but having been in this field for a decade, there’s more and more therapeutic options emerging. Kind of what I’m looking to see most, honestly – I’m following everything really closely, but what I’m starting to think about more is paradigm shifts.

What I’d like to see in the field is to move away from a reactive type of approach and think more about early initiations of therapy, more of a proactive type of strategy, not really – because when we talk about therapeutic choices with a patient and the patient says to us – we don’t say it like this, but the way they say it back to us is, “So, wait, we’re gonna wait for something bad to happen, and then we’ll start treatment?”

I think that – I share discomfort, as – I’m uncomfortable with that approach. The reason we haven’t been proactive is because we’re kind of waiting for highly safe, highly effective therapies that could potentially change the course of the illness. That’s why we have been reserving our therapies, and that’s why our secrets sometimes include less aggressive watchful waiting with later initiation of therapy just because physicians haven’t been satisfied with their choices, but I’m hopeful that that’ll change.

Katherine:                   

Dr. Stein, thank you for joining us today.

Dr. Stein:                     

Thank you very much for having me.

Katherine:

And, thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for joining us.

What You Should Know About Progression in MPNs

What You Should Know About Progression in MPNs from Patient Empowerment Network on Vimeo

Dr. Srdan Verstovsek provides an overview of myeloproliferative neoplasm (MPN) progression and reviews indicators that essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis may progress.

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

See More From The Pro-Active MPN Patient Toolkit


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Transcript:

Dr. Srdan Verstovsek:

When we talk about ET and PV, they should be life-long conditions without much of a change. It’s uncontrolled blood cell count and thromboembolic events, which are then subject to a therapy, and the goal of therapy is to decrease the thromboembolic risk.

There is still, in some smaller proportion of the patients, a risk of a disease change on its own. We talk about the genetic testing that can reveal a change in genetic complexity of the disease, which may be responsible for a change down the road. Or abnormalities in chromosomes that can be seen at the time of diagnosis in some of the patients with ET and PV, not very often, which may predispose patients to a change down the road, a change to more aggressive condition.

So, a smaller proportion of the patients, perhaps 10 – 20 percent of the patients between ET and PV, can over time, long time, acquire fibers in the bone marrow.

That can lead to anemia actually, progressive increases in spleen, bone marrow cells in blood, that would be then a change to myelofibrosis. And a very small percent of the patients actually can change to acute myeloid leukemia, with the baby cells in the blood and the bone marrow, these are called blasts.

They should not be in the blood in the wrong person. They should be below 5 percent in bone marrow in normal person, but if they go above 20 percent, we call that acute leukemia.

So, transformation of ET or PV to myelofibrosis or acute myeloid leukemia, are fear, and obviously can lead to a shorter life expectancy. And so, one can certainly worry about that, but again, it is in a smaller proportion of the patients, and we don’t usually worry that much about it. However, the worry does exist, that’s why you are asking me about it, and the problem is we don’t have medication that would be known and proven in prevention of that biological change of the disease in some patients.

In myelofibrosis it’s similar situation, 20 – 25 percent of the patients change to acute myeloid leukemia, and we don’t have real medication that would be preventing that change.

Promising ET, PV & Myelofibrosis Therapies in Development

Monitoring MPNs: When is it Time to Switch Therapies? from Patient Empowerment Network on Vimeo

MPN specialist, Dr. Srdan Verstovsek discusses the latest research and progress for the treatment of patients with essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF).

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

See More From The Pro-Active MPN Patient Toolkit


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Transcript:

Dr. Srdan Verstovsek

When we talk about the new therapies in development, there are many in myelofibrosis in particular, and a few are in essential thrombocythemia and polycythemia vera. Let’s start with ET and PV. Here we are expecting either studies, or possibly even approval, of a long-acting interferon called Ropeginterferon that was approved a year ago in Europe for PV patients.

We gonna have, hopefully here in the United States, that drug for our patients in a year or perhaps studies in PV, or perhaps most definitely, I would say, studies in ET with this drug. That would be enhancement of what we done off-label using interferons that are approved for some other conditions. We know that interferons are biological agents active in these conditions to control the bone marrow, and perhaps even decrease the number of malignant cells in the bone marrow of patients with ET and PV, which may be beneficial down the road.

In myelofibrosis, the picture is completely different. In this setting, the life expectancy, unfortunately, is affected as we discussed, and we need therapy that would be perhaps improving that life longevity. As we know, the ruxolitinib JAK inhibitor that has been around for nine years can extend the life a few years, but not cure people.

So, helping JAK inhibitors by combinations with other active agents that would be biologically modifying that bone marrow, decrease the tumor burden, improving the quality of life or anemia, are at forefront of what is happening right now. So, combinations with Navitoclax which is Bcl-xL cell inhibitor, CPI-0610, which is BET inhibitor, Luspatercept which is anemia drug.

These are phase three studies that are planned to start soon for possible approval for combinations over JAK inhibitor alone for different problems that people face.

Or, later on in the course of the disease, JAK inhibitor may fail. What do you do then? So, we have studies announced that will be done in what we call a second line, after-JAK inhibitor. And the MDM2 inhibitor was announced. Imetelstat inhibitor in the second line. Momelotinib JAK inhibitor in the second line. Fedratinib is being studied, another JAK inhibitor. Pacritinib for patients with low platelets

These are all phase three studies. That’s means for approval of this drug, so that will be three and four, seven different phase three for myelofibrosis patients with different clinical scenarios, different clinical problems are being done, or about to be done, in very near future. So, my prospect is here. My view on that is that we will have, hopefully, at least some of these seven studies leading to approval of some new drugs for our patients with myelofibrosis.

Monitoring MPNs: When is it Time to Switch Therapies?

Monitoring MPNs: When is it Time to Switch Therapies? from Patient Empowerment Network on Vimeo

MPN expert, Dr. Srdan Verstovsek reviews factors that may indicate a treatment change for patients with essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF).

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek

The real definitions of a failure of a given therapy, it’s not easy to come by. Experts in the field, doctors that see a lot of myeloproliferative neoplasm patients, occasionally get together and try to put in place some guidelines. What would be a failure to a therapy mean for patients with ET or PV?

Would that be, for example, polycythemia vera patients too many phlebotomies when you are on hydroxyurea.

Hydroxyurea is a chemotherapy by mouth, should be eliminating need for phlebotomy, should decrease the white cells and platelets, and the spleen enlarged, and improve the quality of life.

If that’s not possible, and you have to define what that means, then you would say, you should change. So, guidelines do exist, which are always used in clinical studies to define the failure and justify a change. But they should also be applied in clinical practice to apply possible.

If you are on hydroxyurea for ET and PV, and you are not controlling blood cell count very well, you can’t take more because there are side effects from hydroxyurea, you should change. Right?

If you see a progression of the spleen, or worsening of quality of life despite the control of the blood cell count, something is wrong, maybe you should change.

In myelofibrosis is similar situation. You may be experiencing a good therapy on JAK inhibitor or anemia medication, but then after a while, spleen starts to grow, quality of worsens, or anemia develops, then you should change.

It’s not as easy to see exactly to define, but you get the point I’m sure because people are different, the benefit extent or benefit is different, pattern of a failure is different, and we have a lot of difficulties in really objectifying what this means to fail.

My approach is when I see a failure developing – nothing happens overnight. You try to modify what you do by adding another medication, adding medications for whatever is causing that failure, or modifying what you’re doing by changing the schedule or the dose. So, not to give up and say, “Oh, it’s not working,” but trying to work with the patient, and with the medications that you have in different way, for benefit to last the longest possible. 

Which MPN Treatment is Right for You? Factors to Consider

Which MPN Treatment is Right for You? Factors to Consider from Patient Empowerment Network on Vimeo.

Dr. Srdan Verstovsek, discusses how multiple factors, including diagnosis and symptom burden, determine which MPN treatment path may help improve a patient’s outcome.

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek

So, we talk about diagnosis, and then prognosis, and then go over [stem cell] transplant. Now, the transplant is done in only less than 10 percent of the patients because most of the patients are elderly. That’s why you have the disease.

They might not have a donor. They may be sick. There are multiple reasons, so transplant happens in less than 10 percent of the patients. Once we are over that, you say, “What’s wrong with the patient?” Not wrong in terms of dying, but do you have a significant anemia? Do you have an enlarged spleen? Do you have a bad quality of life when we talk about myelofibrosis? All of this that I have talked so far, applies to myelofibrosis. These are the three main reasons for initiating of therapy, usually. Significant anemia, significant bad quality of life, and significant symptom related to the big spleen. You would then introduce therapies.

For the splenic symptoms, we usually prescribe JAK inhibitors. That would be standard practice. For anemia, we have medications from injections under the skin, to some pills. No real approved therapy for anemia, but whatever we can do help patients counteract those problems because slowly over time they’ll get worse and worse, and people die with myelofibrosis between five and seven years.

So, we wanna combine medications. We’re gonna introduce medications as soon as something wrong with the patients to improve whatever is wrong so that the quality of life can continue at a decent level.

Let me go back a little bit to essential thrombocythemia and polycythemia vera, ET and PV. These two conditions are considered rather benign. They should not much effect the longevity, perhaps PV can. And if they do, the main reason for dying from ET and PV is the blood clot or thrombotic event.

This is what we say, usually. A blood clot or some bleeding usually clots in the heart, or the brain, or the lungs, can kill the person. So, we don’t usually talk about the life expectancy in terms of genetic mutations, or abnormalities in chromosomes, or something that will kill the patient outside of the blood clotting risk.

So, what we are talking about then, is after diagnosis, we are talking about the prognosis, when we talk about ET and PV, prognosis is related to what’s your thrombotic risk? So, we talk to patients with ET and PV about thrombotic risk assessment.

And typically, age over 60, or having a history of blood clot, we’ll say yes that patient is a high risk patient with ET, or high risk patient with PV, for the blood clot. And we will be treating patients for that risk in different ways.

So, it’s a little bit different angle here on what we try to achieve in ET and PV patients. More benign, more chronic, assessment of the risk of clotting and control the blood count, and occasionally when we need, control the spleen symptoms. But different ballgame and ballpark then the myelofibrosis part.  

There are, obviously, standard practice protocols in terms of what do you do? Right? So, if we are talking about ET and PV, you would say, if you are – as your remember now, we divide patients in those with the low risk of blood clot, and high risk for blood clot. For low risk, we just give people baby aspirin, and if they have PV, we phlebotomize the patients, blood-letting.

So, not much experimentation there. But there are studies that one can join if there are too many phlebotomies, for example, too many blood-letting episodes. And there are studies with medications that would be decreasing that need completely.

There are also studies in patients that are high risk for blood clotting, which typically would be treated with hydroxyurea, chemotherapy by mouth.

There are new versions of the interferons, biological agents given under the skin every two weeks, that would perhaps be taken instead of a standard practice hydroxyurea.

Not too many studies in ET and PV, really. Some. But in myelofibrosis, there are many because with ruxolitinib, for example, which has been around for about nine years, it’s a JAK inhibitor, you get in many patients good control of the splenic symptoms, but it does not last forever, and in some patients, it may benefit to some degree, but not completely.

So, there are many studies where you can add another medication to ruxolitnib a JAK inhibitor, to boost what it does more of the splenic symptoms controlled, or to add another benefit. The JAK inhibitors do not, by and large, improve the anemia, so how about adding anemia drug to ruxolitnib.

So, combination studies are many underway, so you can actually enroll – even with the newly diagnosed myelofibrosis patients, in the need of therapy, in a clinical study. Not to say, after JAK inhibitors in a second line. That’s what we call it. After JAK inhibitors you need to do something else, that second line, there are many studies because there is no other approved therapy. So, for myelofibrosis, no question in my mind, there are so many studies underway, you can be participant in study to get your result boosted by whatever else is added to what you’re doing, and discover for the large population of patients, novel therapies. 

MPN Treatment: Why Testing for Mutations Matters

MPN Treatment: Why Testing for Mutations Matters from Patient Empowerment Network on Vimeo.

Testing for mutations can influence treatment options available to patients with MPNs and provide a more in-depth understanding into their essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF) diagnosis. MPN specialist, Dr. Srdan Verstovsek reviews three key mutations that may impact treatment timing and approaches.

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek:

So, what we know is that ET, PV, and MF, these are these three, and we will use the abbreviations for simplicity, are so-called classic myeloproliferative neoplasms. Myeloproliferative means that it is disease of the bone marrow where cells grow without control.

Now, with ET we have high platelets, but ET is the disease of all the cells. In PV you have high red blood cells, and in many patients you have high white cells and platelets. In myelofibrosis, it’s paradoxically many patients present with too few cells because of reactive bone marrow fibers or fibrosis that limits the growth of the cells.

So, these are the three diseases that have underlying problem, same problem in these three conditions, which is high activity of proteins inside the bone marrow cells, proteins inside the bone marrow cells.

A cascade of protein that makes cells grow without control. We call this a JAK-STAT pathway. I had patients; they say JAK-STAT highway. It’s active all the time. This is a protein JAK too, and then the JAK2, and then other, so we call it JAK-STAT pathway.

It’s super active. Active in normal person when we need to make blood, but in the diseased person, active because of acquired mutations that affect that highway, JAK-STAT pathway or highway.

It makes it work all the time, that’s why we have so many cells. And there are three mutations, which are part of diagnostic process. You test for these. You can test in the blood or in the bone marrow sample, and these are JAK2 mutation, calreticulin mutation, and the MPL mutation.

They are almost always exclusive of each other, and about 90 – 95 percent of patients will have one or the other. They are still very few patients that have none of these three, which is interesting. And we are, in others, looking for other reasons in these few patients.

 But one of the three is present, and it’s part of the diagnostic process as well. I didn’t emphasize this before, but it is present as a part of the bone marrow evaluation. That’s where it goes. And it is therefore, helpful to test for it. But one can test for other mutations. Many patients have many other mutations that have nothing to do with the JAK-STAT pathway, and that in large part is responsible why people have different disease ET, PV, or myelofibrosis. We explain this that way because of other genetic abnormalities, other abnormalities that we cannot really describe yet.

Genetic is not the whole picture. There are other parts, I’m sure, in bone marrow environment, in other factors they control the genetic expression, and so on, that contribute why a patient with JAK-STAT hyperactivity has ET, and why another has myelofibrosis.

We don’t really fully understand that. And of course, there is a plethora of patients in between that are not all the same. So, genetics do carry a lot of weight in what happens with the patients, and we do test for that, in addition to testing for JAK2, calreticulin MPL. We test for multiple others. That’s routinely done in academic center. It’s very valuable, and it should be standard practice.

The main utility of widespread testing for additional mutations is to assess the prognosis of the patients. If we are looking at the bone marrow blood chemistry and physical exam, a splenomegaly, and presence of this driver mutation, the JAK, calreticulin or MPL.

We call them driver mutations. They drive that highway. If that is the complexity of the diagnosis, then the next step is, as you remember, the patient will say, “How long I’m gonna live?” Well, obviously, that information comes from the historical experience, and I always emphasize that. But there is valuable information from historical perspective to some intelligence to tell the patients what to expect in general terms.  Since the introduction of the genetic testing in academic centers, we have enhanced our ability to prognosticate. Initially, ten or more years ago, we would be looking at the age of the patients, how the patient fares, the symptoms, the anemia, or white blood cell count, or blasts.

These would be kind of common prognostic factors for assessment of the outcome of the patients. But now, we add information on the presence of one or the other of the driver mutations, and the presence of the number and types of these other additional, which call them somatic mutations that have nothing to do with JAK-STAT pathway.

And you can see now how the prognostication also has the flavor of complexity, and it is really not that easy, and we keep moving forward. That prognostication effort is keep moving to assess the outcome of the patients better and better for one particular reason.

If we have we a sense that a patient, based on this prognostic scoring systems, have a poor outcome, which we define as the life expectancy less than five years, then that patient should be referred to the [stem cell] transplant.

And transplant should be done because the benefit of a cure, and the risk of dying through transplant procedure – unfortunately, that’s the reality, is just justifiable if the prognostic scoring system tells you that the life expectancy is less than five years.

That’s the main role for the genetic complexity testing. Looking also at the chromosomes that might be broken. That’s done on a bone marrow sample. And dividing patients in prognosis scoring groups to guide the decision making on the transplant.  

Should MPN Patients be Retested for Genetic Mutations Over Time?

Should MPN Patients be Retested for Genetic Mutations Over Time? from Patient Empowerment Network on Vimeo.

MPN expert, Dr. Srdan Verstovsek provides insight into what factors determine whether MPN patients should undergo additional bone marrow biopsies and genetic testing over time.

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek:

We already know, everybody knows, I would think, that the living tissue does change over time. And when a patients have doubt, I tell them, “Look, my hair has not been grey all my life.” Everybody laughs. That happens with the bone marrow biopsy results, right? They change over time because the bone marrow does change over time. It’s not the same, and if you have the disease, disease cells, let’s call them malignant cells, they may acquire new mutations as they divide. If they already have some mutations that they’re present at the time of diagnosis, they are actually more prone to get more of those mutations over time.

And so, it may then be wise, and that’s what your question is about, to occasionally test patients and see whether there’s any change. Not perhaps in the number of fibers, or number of different cells, but genetically are there any new mutations which would make that disease perhaps more aggressive.

Unfortunately, the situation with repeated testing is complicated by price of the test. The testing can be done on blood, but it’s very expensive. We’re talking about thousands of dollars, and it’s not justifiable at the moment to do that every six months, or once a year for example, to see about any change because, it may not change what we do. It requires a clinical change, not just a genetic change for one to do something different, and something different would be referral to a bone marrow transplant, sooner rather than later.

So, first of all, repetitive testing would be useful in patients that are borderline for the decision of the transplant, not in everybody, because that’s the only intervention that would be affected by that testing. And then, even in these situations, you would need to have a clinically relevant abnormalities beyond just a new mutations.

That means a bigger spleen, or losing weight, or having profound anemia now. So, what basically this comes down to is, we follow the patients closely, and when there is a clinically relevant change, that would ask for a bone marrow biopsy and genetic testing, then we can justify that, and then we change what we do. So, it is cumbersome to sit tight and wait for a change, I understand that, but that’s the reality at the moment.

Which Tests Are Necessary Following an MPN Diagnosis?

Which Tests Are Necessary Following an MPN Diagnosis? from Patient Empowerment Network on Vimeo.

MPN expert, Dr. Srdan Verstovsek, explains why a bone marrow biopsy is essential following a myeloproliferative neoplasm (MPN) diagnosis and its role in confirming whether a patient has essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF).

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek:

By and large, we would like people to have a bone marrow biopsy done. In fact, I would say that myelofibrosis cannot be diagnosed without a bone marrow biopsy. Polycythemia vera, in occasional cases, can be diagnosed without the bone marrow biopsy, even by the national guidelines. And essential thrombocythemia, I would say, that should have, although that is not norm in clinical setting, a bone marrow biopsy as a part of the disease diagnostic process.

This is to say that the diagnosis of any of these three conditions, ET, PV, or myelofibrosis, does not depend solely on the bone marrow biopsy.

I’ll give you an example of the myelofibrosis. You have to have a biopsy that shows abnormalities in different cells in the bone marrow. Megakaryocytes are the key. So, cells are different in number, size,shape, and colors, and you have fibers. Sometimes you don’t even have a fibers. But then, you have to combine this.

Maybe, abnormalities in blood cell count. In the blood chemistry there is an LDH, lactate dehydrogenase chemistry test that usually is tested for. And then, you have a physical exam, enlargement of the spleen. And the type of the white cells in the blood. So, you have a combination of the bone marrow, blood chemistry test, and the physical exam that all have to come together for diagnosis to be made for myelofibrosis.

I always ask my fellow, and doctors in training, who actually makes the diagnosis? It’s the clinician. If you just look at the bone marrow, you may say there is myeloproliferative neoplasm and there are some fibers, but you have to have these other factors to make a diagnosis of myelofibrosis. And fibers, for example, do not really say that the patient has myelofibrosis. Fibers can be present in PV, polycythemia vera, in 20 percent of the patient at the time of diagnosis of PV.

So, fibers on its own doesn’t mean myelofibrosis. Of course, there are other diseases that can cause fibers, other bone marrow diseases that have nothing to do with myelofibrosis or polycythemia vera.

So, it is rather complex problem, and it is not easy to make a diagnosis. We actually looked at that, and 15 percent of the patients that come through the door here at MD Anderson, have a change in diagnosis after our own assessment. So, some experience does count, I would say does counts a lot, because of complexity.

Tips for Traveling During COVID with a Blood Disorder

Tips for Traveling During COVID with a Blood Disorder from Patient Empowerment Network on Vimeo.

PEN MPN network managers Summer and Jeff take us on the road for their annual camping trip in the Julian forest in Southern California. What are some of the challenges of traveling with a blood disorder?

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MPNs and Coronavirus: What Patients Should Know

MPNs and Coronavirus: What Patients Should Know from Patient Empowerment Network on Vimeo.

Dr. Srdan Verstovsek provides guidance for patients with myeloproliferative neoplasms (MPNs) related to coronavirus (COVID-19), stressing the necessity of continued communication with their healthcare team.

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek:

When we come to the COVID pandemic here, and the effect on our patients with myeloproliferative neoplasms, of course, that is something that is very serious. Our patients, particularly those with advanced myelofibrosis, are at a high risk of having complications from infection if they get infection. Perhaps less so for patients with ET and PV, which are more healthier, if you like, although they have myeloproliferative neoplasms.

So, our focus here is, first of all, like it is for anybody else, including myself, on prevention.

Obviously, that would be common sense and logical to try to prevent getting infected. And then, also to try to be in best possible health. Taking medications as prescribed. Being in touch with your doctor. If you cannot come to a physical exam, which is understandable because of possible increased risk through the transportation perhaps, then being in touch through telemedicine. We call, and we see our patients online. That is fine. We can do the bloodwork from the distance.

So, taking medications, being fit, and being as healthy as you can with the disease. But taking medications and controlling your risks, not allowing disease to take ahold of you, and then you get infection and you’re gonna be in trouble.

So, really communications with the doctors, taking medications, and I would say, occasional, with all precautions, visits.

Because, for example, with myelofibrosis patients, I really need to feel that spleen and the liver and see how is the patient’s weight, and not only ask over the telephone, what’s your symptoms are, or what your blood count is. I can see this on the computer. We really need, occasionally, myelofibrosis patients to come over to our clinic to be examined.

And here, in the clinic, we have extraordinary precautions. No visitors. No people to accompany patients in. So, to minimize any interactions with anybody who possible can bring any undesirable infections through the door, not just COVID, but any other. And the staff is prepared to provide excellent care with protection that is in place.

So, I think that will be my message: Prevention; staying on top of your disease; being in best possible shape with the disease; staying in touch with your doctor; and, occasionally, still visit the doctor if at all possible, particularly for myelofibrosis patients.

The Importance of Finding a Myeloproliferative Neoplasm (MPN) Specialist

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An Expert Shares Key Steps to Take Following an MPN Diagnosis

An Expert Shares Key Steps to Take Following an MPN Diagnosis from Patient Empowerment Network on Vimeo.

MPN expert, Dr. Srdan Verstovsek, provides essential advice for patients that are newly diagnosed with essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF).

Dr. Srdan Verstovsek is Chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Verstovsek, here.

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Transcript:

Dr. Srdan Verstovsek:

Obviously, it’s very stressful for any patient to be told that there was something wrong with their bone marrow. You’re talking about myeloproliferative neoplasms; these are neoplasms of the bone marrow. And one would like to first make sure that the diagnosis is correct. So, if there is any doubt, second opinion is always very good to do.

And I tell my patients come to me as a first doctor, if they have concerns, ask me questions. If they wanna go for a second opinion, please go.

The second is understand what this diagnosis means. For prognosis, for the lifestyle, educate yourself. Be educated about what this means because it’s lifelong disease. We hardly ever cure anybody. It is possible in some cases with a bone marrow transplant, but in general, this is life-long condition, any of this Myeloproliferative Neoplasms.

And then, number three, involve your family or friends. Family in particular, because caregivers needs to be informed what this means for you if there is any effect on your quality of life or on longevity. So, don’t leave the disease alone. So, these would be my three points to make people aware of what this all means when you have MPN.

Verify, educate, and share that knowledge and experience.  

An MPN Care Partner Shares Why He’s Optimistic About the Future

An MPN Care Partner Shares Why He’s Optimistic About the Future from Patient Empowerment Network on Vimeo.

Care partner Jeff Bushnell, husband of myelofibrosis (MF) patient advocate Summer Golden, explains why he’s hopeful about their future together. Jeff shares key resources that have helped him stay educated and maintain optimism.

Summer Golden and Jeff Bushnell have been married for over 20 years. When Summer was diagnosed with myelofibrosis (MF), Jeff took on the role of care partner and advocate. Summer uses her years of theatre training and comedy to cope with her condition and help others, while maintaining positivity about the future.

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Transcript:

Jeff:

It’s important to educate yourself because the more you know the less fear can overcome you. And this particular disease – the research is happening so fast, and things are changing. In my estimation, they’ll find – right now, the only cure is a stem cell transplant. It’s normally not done for older people. That in itself is innately risky. I’m convinced, probably within the next five to seven years, there will be a cure for this disease that’s not a stem cell transplant.

The research is moving that quickly on it. And if you don’t follow the disease and the people that are working on it, the specialists, you’re gonna have a much greater chance of feeling powerless and getting overwhelmed by it. As Summer believes, attitude can have a huge, huge impact on how the course of your disease runs. And a doctor would tell you the same thing.

For me, it started with Patient Power. Patientpower.info, I believe is, what it is. They have a whole section for myeloproliferative neoplasms and myelofibrosis, and they’re short videos. And you get a chance to listen to the best doctors that are the head people in this, Dr. Mesa, Dr. V [Verstovsek], and Dr. Jamieson – all the people that are really the movers and shakers. They speak. And you also get a chance to hear other patient’s stories and how they’re dealing with it. And that will give you a much better idea of what you’re facing. And you can really understand things from there. And you can get your knowledge.

Fear comes from lack of knowledge. In my job as a pilot, I flew for 50 years. I very, very rarely was afraid because my knowledge was so great and was reinforced every year by continual training that I felt prepared to handle anything that might come across to me. Knowledge is really important. It will allay your fears dramatically.

When I started online and heard about people that had been journeying with this for 10 or 15 years, initially, I had thought – well, this is a year or two, and it’ll be the end. And then I realized, plenty of people have lived with this for a long, long time. And they had a journey, and they’re doing it successfully. And that gave me confidence.

The more people you can talk to about it, the more you can put your journey in perspective. And it’s really hard to put in perspective for this particular disease because it affects everybody vastly differently. Some cancers – the progression is very, very linear. Everybody kind of goes through the same thing. This one – it depends on the mutations you have in your blood and all kinds of things like that, and some people get really bad symptoms quickly.

Others, they don’t. But the more you know about how those things affect you, the more you know and can understand about what to expect. And the more people you talk to who have it, you can find out about their journeys. It helps put yours in perspective.

I’m optimistic because I really keep up to date on what’s going on. And I see the doctors that are in the forefront of this and the research that they’re putting in and the care they have for working on this disease and the knowledge they have, and I just am quite optimistic. And as I say, I’m following the medical developments extremely closely.

I went to the ASH Conference last year. And I’ve gone to another conference that our doctor spoke at. And I’m just kinda blown away by – I’m fascinated by the science.

My advice would be find out as much as you can about it and support each other in a way that works in your own marriage.

Summer and I approach life a little bit differently. And yet, one of the reasons we do so well together is we kinda have both ends of the spectrum covered. And I sensed that when I met her 20 years ago. And we brought something to the table that each of us needed. And if you can find that in your relationship with your significant other that has the disease, what you can bring to it, what they can bring to it, you can be a tremendous support for each other.

A Care Partner’s Journey: How Life Goes on After an MPN Diagnosis

A Care Partner’s Journey: How Life Goes on After an MPN Diagnosis from Patient Empowerment Network on Vimeo.

Care partner Jeff Bushnell shares how he and his wife, patient advocate Summer Golden, have dealt with her myelofibrosis (MF) diagnosis. Jeff explains how online support and finding an MPN specialist were essential steps in helping them continue to live life to the fullest.

Summer Golden and Jeff Bushnell have been married for over 20 years. When Summer was diagnosed with myelofibrosis (MF), Jeff took on the role of care partner and advocate. Summer uses her years of theatre training and comedy to cope with her condition and help others, while maintaining positivity about the future.

 

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Transcript:

Jeff:

The worst part was initially. We didn’t get a myelofibrosis diagnosis.

It took about a month because in order to definitively diagnose it they have to take a bone marrow sample and send it to a pathologist and so on and so forth. So, all that time, I’m worrying about the possibilities. It could be leukemia or this, that, or the other thing. My way of handling and dealing with scariness – I’m a retired pilot – is to find out things, knowledge.

I spent a huge amount of time on the internet. The LLS Society has papers about it, and I read those.

And the more I got into it – once we found out it was myelofibrosis, I’ve read almost all of the papers that the doctors write for each other to find about this. That doesn’t interest Summer in the slightest. It interests me greatly. So, when we have an appointment with the doctor – when I’m talking to the doctor, it’s like two doctors talking to each other.

When Summer’s talking to her, they talk on a different plane. It’s much more about mental approach to things and that kind of thing.

And for me, when I think back to the beginning of when we had this and where we are now two years later, we’re living the life that we lived before she was diagnosed to be real honest with you.

We do everything that we did before she was diagnosed the same way we did it before, and it was a trip that probably everybody who gets diagnosed or deals with a person that has the disease takes. When it first happened, it hit us like bricks coming out of the sky hitting us on the face. Literally, when we first went to the hospital and she got the word that there was a problem – as I say, we lived in two separate houses – I literally was afraid to call her phone figuring she might be not there. I was that scared. And then, after we met our doctor, which was extremely fortuitous – when we went to the emergency room, the person that was there, she said these look like leukemia things.

So, she called the oncologist. The oncologist on call is our current doctor, Dr. Tiffany Tanaka, and she’s a specialist in this disease. It was like it was meant to be. And Dr. Tanaka asked the guy to do some other tests and then said, “Send her home, but tell her I need to see her this week.” So, we’re thinking all these horrible things. And its New Year’s weekend, so the clinic is closed for about five days, you know? We’re worrying and worrying and worrying.

We finally saw Dr. Tanaka, and it was like a breath of fresh air. This wonderful doctor has the ability to just communicate with the patients. I’m interested in the disease, so she communicated on my level. Summer is not interested in all the medical jargon, so she was able to explain to Summer what was going on and just very, very reassuring, very reassuring.

And then, I went and started getting information. That’s my way of coping with things. The first place I went was – I went to Patient Power and found a lot of information there.

And then I found the online myelofibrosis support group at Facebook. And that was very, very useful. When I started reading about the fact that some people had this for many, many years – then I said this is not – nothing’s gonna happen in the next year or two. We can go back to living. And once we learned more about it and spent more time with our doctor and Summer was able to live her life once she got taking the medicine – she takes Jakafi.

That controlled the basic symptoms, and we haven’t looked back. We just started living our life the way we had been living it before.

Is Laughter Really the Best Medicine? One Woman’s Mission to Help Others with MPNs

Is Laughter Really the Best Medicine? One Woman’s Mission to Help Others with MPNs from Patient Empowerment Network on Vimeo.

Could laugher really be the best medicine? Patient advocate Summer Golden explains how she uses comedy to cope with her myelofibrosis (MF) diagnosis and shares her mission to inspire others.

Summer Golden and Jeff Bushnell have been married for over 20 years. When Summer was diagnosed with myelofibrosis (MF), Jeff took on the role of care partner and advocate. Summer uses her years of theatre training and comedy to cope with her condition and help others, while maintaining positivity about the future.

See More From the The Path to MPN Empowerment

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Transcript:

Summer:

When I was initially diagnosed after some other false starts with an MPN, I was kind of shocked because I’ve never really been sick, and I don’t take medications, but I didn’t think about it – that sounds crazy; I can’t explain it. I just figured I’d be okay, and the main thing – I didn’t wanna give up this theater.

You know how when you’re my age, people talk about nothing but their illness sometimes? I just never been into that, so it wasn’t part of my personality.

I started doing comedy two years ago because a friend of mine was taking a comedy class, and I went to her showcase, and I thought, “I should try that, even though I’ll never be funny, I have no jokes, and I don’t know what I would say.” But, I went, and I did comedy in clubs for a while, and then I didn’t – I don’t really like drinking and dirty jokes, so I kind of got away from it off and on, and then, when I got into doing it about my myelofibrosis, then I saw a purpose in it, so I went back to it.

I was thinking about whether my life was gonna be changed, how this was gonna change me, so I emailed my comedy teacher in the middle of the night, and I said, “Do comedians ever talk about cancer, having it?” And, he said, “Only if they have it.” So, I emailed him back and I said, “I’m coming back to your class,” so I did. He assigned everyone to be in a showcase. I was gonna do mine about cancer. It was six weeks, so I had to find humor. I don’t know how I find it. I just kind of see things.

I was shocked because I thought people were gonna hate it, and I was gonna quit, and then I’d invited my doctor and two friends, so I thought I’d better not just not show up. But, people came up and said they were inspired. I was just amazed because I mainly –I don’t go out of my way to think of – I do think of things that are funny, but it’s just – it’s a real thing. I try to keep my comedy real.

It’s helped me by being in control. I don’t pay much attention to the symptoms because I’m kind of over them.

Just helped me feel like I’m doing what I can do, and so far, it seems to be working, as long as I get enough sleep.

How do I think comedy could help other people who have health problems? I can tell you one way I thought to help somebody. I wanna start a class for people, but so far, there hasn’t been a lot of interest, but I think I could really help people doing that because I know how to write comedy.

If they really wanna do that, they would be a type of person that has humor, and they could do it, but you’ve gotta realize sometimes, people get a lot out of being sick. There are a lot of rewards, and so, they might prefer to have those rewards. For my way of thinking, if they wanna do humor, it’ll make a big difference, and if somebody wants to do it, they could call me, and I’ll help them.