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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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Related Resources

 

What You Should Know About Progression in MPNs?

 

Monitoring MPNs: When is it Time to Switch Therapies?

 

An Expert Shares Key Steps to Take Following an MPN Diagnosis


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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Should MPN Patients Be Retested for Genetic Mutations Over Time?

 

MPNs and Coronavirus: What Patients Should Know

 

Monitoring MPNs: When is it Time to Switch Therapies?


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

Promising ET, PV & Myelofibrosis Therapies in Development 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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An Expert Shares Key Steps to Take Following an MPN Diagnosis

 

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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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What You Should Know About Progression in MPNs


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. 

Key Next Steps After a Lung Cancer Diagnosis: Expert Advice

Key Next Steps After a Lung Cancer Diagnosis: Expert Advice from Patient Empowerment Network on Vimeo

Following a lung cancer diagnosis, the actions that a patient takes may impact their long-term care and treatment options. Dr. Erin Schenk, a lung cancer specialist, lists key steps a patient should consider post-diagnosis.

Dr. Erin Schenk is an assistant professor in the division of medical oncology at the University of Colorado Anschutz Medical Center. Learn more about Dr. Schenk and her lung cancer research here.

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Transcript:

Dr. Erin Schenk:

As a medical oncologist who takes care of lung cancer patients, I would recommend that if you or a loved one are diagnosed with lung cancer, going to your meeting with the cancer doctor report the surgeon or the radiation doctor with a couple of main questions to ask in order to better understand your diagnosis and the treatment options.

So, the first one is what stage and stage is a descriptor that we use that talks about how far the lung cancer has spread if it’s spread at all. And sometimes, this involves additional testing to give you the best, most accurate answer. Oftentimes, patients are diagnosed with scans, but what’s also – excuse me, scans of the chest, but what’s also really important is better understanding whether or not lymph nodes in the middle of the chest are also involved.

This can require either a PET scan or occasionally procedures where tissue, the lymph nodes biopsied, and tissue samples are taken to see if the lung cancer has spread to those lymph nodes. PET scans are also able to better tell us whether or not lung cancer has spread outside of the lungs. And additionally, and MRI of the head can often be a really critical piece of information to better understand whether or not the lung cancer has spread to the brain. Unfortunately, lung cancer is one of those cancers that can spread to the brain tissue.

So, the first piece of information and more tests might be needed, is stage.

The second piece of information that’s very important is what type of lung cancer, and sometimes, this occurs hand-in-hand with better understanding stage. Usually, this involves a biopsy, so a sample of the tissue needs to be taken and then looked at underneath a microscope by a pathologist who are doctors who help us identify which type of lung cancer it is that a patient has. And then the final thing to ask your care team or your doctor is do I need additional molecular testing?

Molecular testing is a critical piece of information in order for doctors like me to help take care of lung cancer patients. Molecular testing lets us know what role immunotherapy might play in your diagnosis. It also lets us know whether or not targeted therapy which are oral pills we sometimes call TKIs are appropriate for your disease and your stage. These pieces of information, so stage, what type of lung cancer, and if molecular testing is necessary, these are, I think, the three critical pieces that you need going forward to help your cancer doctor and team better formulate a plan that is right for you.

Finally, I’d like to add in that if you are in a situation where you would like a second opinion, or you would like to get more thorough answers, I would encourage you to look for an academic center or a large medical center that has specialists who focus in on lung cancer. We are often very happy to see patients and talk with them about their treatment plan if any other tests or evaluations are needed to help you feel confident in the plan that your doctors closer to home have put together. That’s it.

Why You Should Consider a Clinical Trial for Lung Cancer Treatment

Why You Should Consider a Clinical Trial for Lung Cancer Treatment from Patient Empowerment Network on Vimeo.

Dr. Erin Schenk, a lung cancer expert and researcher, explains why patients with lung cancer should consider a clinical trial and the role trials plays in clinical care.

Dr. Erin Schenk is an assistant professor in the division of medical oncology at the University of Colorado Anschutz Medical Center. Learn more about Dr. Schenk and her lung cancer research here.

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Transcript:

Dr. Erin Schenk:

We have a very active clinical trial practice in the lung cancer world for one reason alone, and that’s that while our current therapies are good, we can still do better. Lung cancer accounts for significant cancer-related deaths in the United States and the world. And we wanna work to try and improve how well patients do and also improve how many patients we are able to cure. Clinical trials can be at any step of your workup or treatment.

So, even patients with earlier-stage disease meaning lung cancer where we can resect it with surgery, there are a number of clinical trials going on right now to try to better improve the outcomes we see with our normal standards of care. So, whether you are having a lung cancer removed by surgery whether you’re receiving chemotherapy and radiation and immunotherapy whether your lung cancer has happened to spread outside of the lungs, there are clinical trials available at every step in the game.

And I would really encourage you to ask your cancer care team or your doctor about whether or not clinical trials might be available in your area. Because often, they can help identify new targets or other ways of trying to attack the vulnerabilities of your lung cancer.

If you are considering a clinical trial, there are a number of important questions to find out from the clinical trial team as well as your cancer care team. Some of the things are really practical, logistical questions and one of those is, “How often do I need to come to clinic? How many more schedule visits do I need?”

Usually, with clinical trials, upfront so before you get on the clinical trial or once you start receiving the clinical trial medicine or therapy, often there are more frequent visits in that initial time period. But after things are – after you’ve had several treatments with the trial medicine, often it becomes more standard of care meaning visiting once every three weeks for blood work and a visit with your team and then infusion.

So, it’s often a little more work up front, and then it gets back to the usual expectations of how often you have to be in our offices. So, I think those logistical concerns are very real because especially for larger institutions, sometimes, coming to our campuses can be a bit of a challenge. So, that would be one. I would recommend discussing logistics. Discussing with your team as to why they think this would be a trial for you is important.

Occasionally, we are able to screen for certain markers or certain things that are expressed on the cancer cells and then match you with clinical trials that try to target those specific molecules or proteins or flags that are on the surface of the cancer cell. So, oftentimes, we try to match patients up to a specific clinical trial, so better understanding why that one was recommended. And then I would ask your team to also discuss what are the side effects that have been noticed.

Often with these clinical trial medicines, we don’t have a lot of experience with how well patients do on these therapies. But sometimes, we can give you an idea in terms of what we expect and what we will watch closely for. So, I think logistics are important, why your doctor or your cancer team thinks this is a good trial for you, and then finally, what sort of side effects have been noticed as best we can tell with this new trial medicine.

Lung Cancer Treatment Advances: What are Antibody Drug Conjugates?

Lung Cancer Treatment Advances: What are Antibody Drug Conjugates? from Patient Empowerment Network on Vimeo.

 Dr. Erin Schenk, a lung cancer expert, discusses emerging research around antibody drug conjugates (ADC) and how this therapy works to treat patients with lung cancer.

Dr. Erin Schenk is an assistant professor in the division of medical oncology at the University of Colorado Anschutz Medical Center. Learn more about Dr. Schenk and her lung cancer research here.

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Transcript:

Dr. Erin Schenk:

Some interesting research that’s coming to the forefront in the lung cancer field are using new medicines called antibody-drug conjugates. And so, these medicines, I think of as another type of targeted therapy. So, what happens is that cancer cells express certain proteins or certain flags on their surface that aren’t often found on other normal cells.

And what these ADC drugs are able to do is that they’re able to seek the cells that express certain flags, and then deliver a chemotherapy payload directly to those cancer cells. One trial from the recent ASCO annual meeting from this year, 2020, was looking at an ADC that targeted HER2 which can sometimes be over-expressed by lung cancer cells.

And they had good initial reports in terms of patients being able to have disease control for some time and minimal side effects.

So, I think in general the idea of ADCs or looking for surface markers on the cancer cells to try to in a more targeted fashion deliver the chemotherapy payload, I think this is a really exciting area of investigation as well as a new potential therapy for our patients with lung cancer.

Are Clinical Trials Too Risky? A Lung Cancer Expert Reviews the Facts.

Are Clinical Trials Too Risky? A Lung Cancer Expert Reviews the Facts. from Patient Empowerment Network on Vimeo.

Some patients fear that clinical trials may be too experimental and risky. Dr. Martin Edelman outlines the clinical trial process and addresses myths surrounding trials. Want to learn more? Download the Program Resource Guide here.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

Here’s the last one that I have on my list here. Clinical trials are experimental and risky.

Dr. Edelman:

Yeah. Well, so is the rest of life. So, there generally – is there risk? Yes. Essentially, every patient is always a trial because we for the most part don’t – even in the disease states where we have very active treatment – so, let’s say – for example, we were talking about the EGFR mutation. So, we have excellent drugs. We have a drug now, osimertinib – outstanding drug, easy to take, low risk of side effects.

The earlier generations – there was a lot of rash, diarrhea. That’s been pretty much done away with. But on average, patients benefit from this drug for about a year and a half.

So, that’s not great if you’re 40 or 50 years old. You want to do better. So, what are our current studies? Well, we’re looking – we’re re-addressing a question that we thought had been answered, but really it wasn’t – about, well, what’s the value of chemotherapy plus this drug? What about the value of other drugs?

So, we can’t promise anybody anything, but our current treatments are still not good enough. There are certain diseases, let’s say Hodgkin’s disease, where you know you’re gonna cure almost all the patients up front or testicular cancer, etcetera, where – again, but thanks to trials, clinical trials, we now are at that stage. We’re not there yet in lung cancer, and the reality is is every patient should really be on a study. I think it’s – and we have this problem now in that our studies have also become far more complicated to enter people in because there are many more variables one has to look at it. What’s the molecular background of the tumor? How many prior therapies?

The condition of the patient, their organ function, etcetera – and the regulatory burden has become much, much greater. But clinical patients are in clinical trials. Let’s look at the question. Are they risky? Well, everything is risky, but we do a lot to manage that risk. Patients who are in studies are observed more closely. We have to. It’s the law. There’s frequently additional personnel assigned. They’re usually getting standard of care plus a new treatment or a new treatment followed by the standard of care or some variation of that.

They’re observed, like I said, much more carefully than we would otherwise. And so, I think actually patients on trials generally will do better, and we actually have evidence. Multiple individuals have looked at this – everything from first-in-man trials or early dose escalation studies, controlled studies – that show that patients, even those on the control arm, generally do better than similar types of patients who are not treated on studies because we just are more careful.

And the physician who participates in trials is generally someone who has a greater knowledge of the disease.

The Truth About Managing Lung Cancer Treatment Side Effects

The Truth About Managing Lung Cancer Treatment Side Effects from Patient Empowerment Network on Vimeo.

Are lung cancer treatment side effects avoidable? Dr. Martin Edelman reviews effective management strategies. Want to learn more? Download the Program Resource Guide here.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

Let’s talk a little bit about some of the concerns that patients have about the side effects. Let’s see: Side effects are unavoidable.

Dr. Edelman:

Well, that’s not true. As I said, what were the side effects? If you go back a couple decades and you ask patients what were they concerned about, many of them were concerned about nausea and vomiting. And that is largely a thing of the past. Many patients will still have some queasiness with treatment, but even our most nausea-producing drugs – we really do have outstanding drugs for the prevention of that. You have to use them. You have to take them.

It’s very important to give them appropriately. There are very excellent guidelines that are out there. Sometimes, patients are still undertreated, no question about that. Not every drug has industry strong backing. There’s one drug – for example, olanzapine, (Zyprexa) was actually developed as an antipsychotic, and I always tell the patients, “No, I don’t think you’re crazy.”

But it’s at a lower dose, and we have excellent, excellent evidence that that drug given for a few evenings after chemotherapy is extraordinarily effective along with the other drugs in preventing nausea and vomiting. So, that’s one thing.

Hair loss is still somewhat inevitable with certain drugs – the taxanes. But many of our regimens don’t cause hair loss.

Or as I tell folks – only you and your hairdresser will know for sure because its hair on the pillow, but the average person won’t pick you out of a crowd. Those are big concerns still. There still are potentially life-threatening effects from chemotherapy, and we spend a lot of time educating people about that. But those are not inevitable, and it’s actually a minority of patients in lung cancer.

One should not confuse – there are different malignancies. Still, the treatments for say leukemia, though even that’s changing, can be extraordinarily toxic or the bone marrow transplant patients. Many, not just lung cancer, but in the other diseases as well – many of the things that people attribute to the drugs are more due to the disease. So, I always say, “The greatest failure and side effects to the drugs are they don’t work well enough because the side effects of the disease can be considerable.” So, that’s the bigger issue. The immunotherapeutic drugs have a rather interesting set of side effects.

They are clearly initially or frequently better tolerated than the older cytotoxics, which still have an extremely valuable place in the treatment and cure of lung cancer. The immunotherapeutics have clearly been quite beneficial, but their side effects can be subtle and far less predictable and can be very severe. Virtually, any organ in the body can be affected by this. We like to say, “If it ends in ‘itis,’ you can get it from immunotherapeutics.”

So, there are lots of side effects, no question. But they can be managed. They can be prevented. They can be treated. Sometimes, we have to abandon a drug. So, people who get severe – what we call immunotherapy-related adverse events – may not be able to continue on their drugs. But even that is not necessarily always the case.

Patricia:

This next one really gets to the heart of the doctor-patient relationship. I shouldn’t share my side effects with my healthcare team because I don’t want them to stop my treatment routine.

Dr. Edelman:

Well, you can’t prevent the side effects if you don’t know about them. And I always would tell patients, I said, “You know, if you’re having a problem, please don’t call me at 4:00 on Friday afternoon. I’m gonna end up sending you to the emergency room, which I may anyway.” But a lot of times, we can solve certain things over the phone. There are a lot of side effects that can be treated and particularly if one is aware early on. So, yeah, you should share the side effects because how’s somebody gonna know how to deal with them?

Now, the problem we run into sometimes is in a population that’s on average 60s and 70s, could be younger. There’re lots of things that can be just part of ordinary life. Everybody gets headaches, back pain, etcetera, etcetera.

We have to treat those sometimes and evaluate them much more aggressively because of the possibility of them being related to disease or drug, but it helps to sort it out. You can’t be too blasé about it because sometimes things need to be looked at very urgently, particularly with immunotherapeutic drugs. Some of the side effects that can be severe can sometimes be very subtle in their onset.

Trustworthy Resources to Help You Learn More About Lung Cancer

Trustworthy Resources to Help You Learn More About Lung Cancer from Patient Empowerment Network on Vimeo.

Expert Dr. Martin Edelman shares credible resources to help lung cancer patients become informed and empowered.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

Let’s talk a little bit about health literacy. What would you suggest patients use for online resources? What are good resources?

Dr. Edelman:

So, there are some excellent resources. The International Association for the Study of Lung Cancer has resources for patients. The National Coalition of Comprehensive Cancer Center Network (NCCN) has resources. American Society of Clinical Oncology has resources. So, those or American Cancer Society. So, there are some really reliable sources out there. And there’s a great deal that’s very unreliable – people’s Facebook pages. I’ve seen this.

Patricia:

It’s a big place.

Dr. Edelman:

Everybody always – and I think it’s important for people to understand. There will be people who will get something and have a fantastic response. I’ve used anecdotes.

The anecdotes I’ve used are to illustrate the potential hope of benefit. They’re not exceptions to the rule anymore. They’re the good case scenarios. I could have just as many anecdotes of people who didn’t benefit and stuff. And I think it is important going into this – and that’s why we are reassessing patients constantly and getting repeat scans because we don’t necessarily know always – even if something’s 90 percent effective, it means 10 percent of the time it’s not.

And each patient – we’re getting better at individualizing and personalizing therapy, but we’re not perfect yet. And we probably never will be. So, there will always be anecdotes. I think what’s – as a friend of mine puts it – the plural of anecdotes is not data. When I say, “Well, chemoimmunotherapy works.” It’s not because I have anecdotes of that, though anecdotes illustrate the magnitude of benefit.

I have data that shows that the chemoimmunotherapy regimen was compared to chemotherapy and was clearly and unequivocally superior. When I give a statistic that 60 percent of patients, 65 percent, can benefit from those types of regimens. That’s based upon prospective randomized control trials.

Is Lung Cancer Treatment Effective in Older Patients?

Is Lung Cancer Treatment Effective in Older Patients? from Patient Empowerment Network on Vimeo.

Lung cancer expert Dr. Martin Edelman tackles common misconceptions about the effectiveness of lung cancer treatment in elderly patients. Want to learn more? Download the Program Resource Guide here.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

How about this one? Treatment is not effective in older patients.

Dr. Edelman:

Treatment is highly effective in older patients. It’s interesting. So, we had long arguments about, when I started in this field, whether treatment ever worked, and there were a number of studies that showed that chemotherapy – that one platinum was better – what’s called a platinum-based agent – was better than no therapy.

And then that two drugs were better than one drug. And people would say, “Oh, well, that doesn’t work in the elderly. And they should only get one drug.” And that’s because, I guess, their burning bush on the lawn told them this. And the fact is is that then got evaluated in a controlled trial, a very nicely done study by my European colleagues. But what was crucial was that they used somewhat lower doses of chemotherapy, a little bit different schedule of chemotherapy, and it was clearly superior to a single agent. And those were even days before immunotherapeutics and these targeted agents. So, many patients will benefit. You just have to be aware of certain basic principles in geriatric medicine as well as basic principles of lung cancer care.

So, first off, if the patient is elderly but their tumor is characterized by a driver mutation, they get one of the so-called targeted agents. And these are these days very non-toxic, easy to take, and highly effective.

Patients – many are going to be eligible for immunotherapy either as a single agent or combined with chemotherapy. Chemotherapy drugs could certainly be cut in their doses and still preserve much activity and be done safely.

I had a woman with small cell lung cancer. This is now about a year and a half ago or so. And she’s in her 80s. And she came to me because she was told – oh, just sorta get your affairs in order. And her disease was what we term an extensive small cell. The staging system’s a little bit different, but she didn’t have a really vast bulk of disease. And we treated her with standard chemotherapy drugs but at somewhat lower doses and some careful TLC and some other supportive things like growth factors.

She got all of her treatment on an outpatient basis, had an excellent response. We used radiation later to consolidate her treatment, and I see her back every couple of months. I wouldn’t say that she’s necessarily cured of her disease, but she does yoga every day. She lives a full life. She sees her grandchildren. And she’s, I think – I wanna say 83-84 years old. I think she’s quite grateful for that. It’s not the numerical age.

The flipside is if somebody’s 50 years old and they’re extremely ill when they come in, then one has to be very cautious about what one does. We used to say that those patients who come in who are severely impaired should simply get supportive care and hospice services.

And actually, how would I put it? Our lives have gotten a little bit more difficult lately because as things have gotten better for patients – because I can’t necessarily say that as much because some patients may be very susceptible to the effects of – their disease may be very susceptible to the effects of immunotherapy. I had one patient who was a younger gentleman who was on a gurney. He was in his 50s, lost an enormous amount of weight , he was on oxygen. We immediately gave him fluids. My fellow – I had an excellent fellow at the time – came to me and said, “Should we admit him and send him to hospice? Or just send him to hospice?” And I looked, and he had a biomarker that indicated that he might have an excellent response to immunotherapy, so we gave him solely immunotherapy and saw him back a few days later. He was still pretty touch and go. We gave him some fluids. A week after that – still, we were kinda touch and go, but he was still with us.

And then a week after that my medical assistant, comes in, and she says, “You know, he looks a little bit better today.” And he was in a wheelchair that day. And then a few weeks after that, he had a walker, and a few weeks after that a cane and about a year after that was asking me about whether or not he could go on a cruise. Again, I still see this gentleman – a couple weeks ago. It’s now almost two years later. And the question now that we have is – should we stop his treatment? And he is restored to complete full health, has had almost no side effects of treatment.

So again, this is not every patient. Some people will be treated and get every side effect and no benefit, but I think I’ve become a lot more reluctant to say that any patient should not at least be offered the opportunity for treatment knowing what the potential side effects are. And there still are considerable and sometimes severe side effects from therapy.

Does Surgery Cause Lung Cancer to Spread? The Facts.

Does Surgery Cause Lung Cancer to Spread? The Facts. from Patient Empowerment Network on Vimeo.

Could undergoing surgery cause your lung cancer to spread? Dr. Martin Edelman debunks this misconception.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

Sure. Here’s one I hadn’t heard until just now. Surgery causes lung cancer to spread.

Dr. Edelman:

Yeah, that’s common in certain states. When I was in Maryland that was a biggie.

So, there’s a myth that the air gets to the tumor, and then it spreads. But that’s certainly not true. It certainly is possible that in a bad surgical procedure that disease can be spread, but I think historically what that was was in the days before we had as accurate of radiographic studies. So, it’s kinda interesting. I always say, “I’m not that old, and I began medical school before there were CT scans.” So, the way you would diagnose something was with a chest x-ray. That was your best chest imaging. And the brain you’d image with something called a pneumoencephalogram, which is – you don’t know what that is. Most people don’t, and they should be thankful for that. But we had no real way of knowing these things. So, what would happen is there would be a surgical exploration. They would say, “Well, it looks very localized.” But then you’d go in, and there was lots of disease all over the place.

And for the most part, that doesn’t happen anymore. Now we have CT/PET scans. We have MRIs. Patients before they go to surgery usually have had – our pulmonary physicians will usually have sampled the nodes in the middle of the chest, the mediastinum. So, it isn’t that there aren’t surprises, but there are far fewer. And certainly, a properly done operation should not spread lung cancer. I would emphasize the properly done operation. It is my strong belief that nobody should have surgery for lung cancer from other than a board certified thoracic surgeon who spends their time thinking about lung cancer, preferably in an institution with a fair volume of this.

We know – it should be no surprise to people, practice makes perfect. People who really focus in an area – people at the NCI-Designated Cancer Centers, comprehensive cancer centers – who do a lot of this have greater expertise.

Lung Cancer Treatment Decisions: Which Path is Best for You?

Lung Cancer Treatment Decisions: Which Path is Best for You? from Patient Empowerment Network on Vimeo.

Dr. Martin Edelman reviews key factors that help to determine a treatment course for lung cancer patients.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

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Transcript:

Patricia:

How are you approaching treatment decisions with your patients?

Dr. Edelman:

Well, the treatment decisions that we make – that I make are those that are in ways similar to other medical oncologists. It really depends because some of the patients may first go to a surgeon or whatever. However they come into the system, there are a few key factors in this. First is – make your decision based upon, Number 1, which kind of lung cancer. So, there are two major varieties. You have small cell and non-small cell, and they are treated – they are biologically distinct, and they are treated in distinct ways.

And then the next major consideration is the stage of the tumor, which is our way of expressing how advanced that is and deciding on both the therapy as well as conveying a prognosis and evaluating a patient for a clinical trial. And that’s based upon the size and location of the tumor; presence, absence, and location of lymph nodes; and the presence or absence and, these days, the number of metastatic areas of disease.

And then, lastly, and again depending a little bit upon the stage and interacting with all the others is what condition is the patient in? Anybody can get lung cancer, but still the median is in older individuals.

Many of these patients have compromised cardiac and pulmonary status as well as other diseases of aging, hypertension, cardiac disease, etcetera. Those people – one obviously has to tailor one’s treatments to fit those comorbidities. So, that’s sort of how the basic assessment – obviously, some patients show up with metastatic disease. We know that, but we go through a whole process for this.

The staging system that we use is complicated, and it keeps changing. We’re, gosh, up to version eight of this? I started with version three. I’m not quite sure I’ve fully mastered the current one, and the ninth edition is coming soon. And why does it keep changing? Because our knowledge of the disease keeps changing. The database keeps expanding.

We’re able to be more refined. Molecular variables have not yet fully entered into our considerations. Unquestionably, they will. But basically, one could consider lung cancer – despite the four major stages and multiple substages – that you really have three buckets that people will fit into. They have localized disease, which we will predominantly address with a localized therapy – surgery, radiation. And many of those patients, however, particularly those who might have a lymph node that’s positive, will benefit from chemotherapy to prevent recurrence.

We have patients with locally advanced disease. Primarily, those are patients who have lymph nodes located in the middle of the chest as opposed to more localized disease where if there’s a lymph node present it’s more in the lobe of the lung. Those patients with lymph nodes in the middle of the chest or larger tumors are approached with frequently a combination of chemotherapy, radiation, sometimes surgery.

And then we have patients with advanced disease who will be predominantly treated with drug therapies, which nowadays, depending upon the molecular background of the tumor, could be a targeted treatment if they have a specific mutation.

Something we see most frequently, though certainly not exclusively, in patients with scant or no smoking history, they may be approached with immunotherapy or chemotherapy combined with immunotherapy.

And there are many considerations that go into those decisions. And even in advanced stage, there are certainly roles for surgery and radiation depending upon whether there are structural abnormalities, occasionally whether there are relatively few areas or several areas of metastatic disease. And in the localized and locally advanced disease, our goal is cure in those, though we certainly are not there for every patient yet.

And in advanced disease, it’s extension of life, which is now quite considerable compared to untreated disease. And I think in certain situations, particularly those who only have a single area of metastatic disease, curative treatment is a realistic possibility. And even those with more disseminated disease, we’re now beginning to see a substantial fraction of patients who are still alive at five years or more. So, we’re beginning very cautiously to think that perhaps some of those patients may even be cured of their disease, though I’m not quite ready to say that.

How Genetic Testing Has Revolutionized Lung Cancer Treatment

How Genetic Testing Has Revolutionized Lung Cancer Treatment from Patient Empowerment Network on Vimeo.

Dr. Martin Edelman explains how genetic testing has revolutionized the lung cancer treatment landscape. Want to learn more? Download the Program Resource Guide here.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.

View more from Fact or Fiction? Lung Cancer

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Lung Cancer Treatment Decisions: Which Path is Best for You?

  

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Transcript:

Patricia:

How is genetic testing changing the landscape?

Dr. Edelman:

So, genetic testing – and in this case the testing of the tumor, not the germline, not the individual – has been very, very crucial. If you go back about 20 years ago, there was a family of drugs called epidermal growth factor receptor inhibitors or EGFR inhibitors.

And the basic science at the time made it look like these would be best combined with chemotherapy in squamous cell carcinoma. And as it turned out, combined with chemotherapy they weren’t very useful. But as single agents, there were these occasional very dramatic results.

So, that came at a time when we were able to evaluate tumor DNA, sequence it with some degree of ease at a reasonable cost. So, there was a discovery of specific mutations, which were targeted by these drugs. So, it was sort of interesting in that it was the clinical observation that led to the discoveries in biology, not really the other way around.

But then that in turn resulted in looking for other mutations, which were found, and then the development of other drugs – in some cases, the repurposing of other drugs for those. And now we have about a half a dozen very validated targets, each one of which in a small slice of the population – between say 1 percent and 5 percent – 10 percent of the lung cancer population – but these – if the patient has within their cancer that particular mutation, these are drugs that are 80 percent-plus effective and frequently can be administered with relatively little toxicity.

And usually they’ll give them benefit for one-plus years or more. So, that’s been an example of progress there.