MPN Treatments and Clinical Trials Archives

When it comes to treatment, MPN patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for Myeloproliferative Neoplasms (MPN) Treatments and Clinical Trials from Patient Empowerment Network

Are There Predictors That an MPN May Be Progressing?

Are There Predictors That an MPN May Be Progressing? from Patient Empowerment Network on Vimeo.

Many people living with MPNs are worried about progression. Dr. Raajit Rampal talks about possible indicators and discusses ongoing research to learn more about disease progression. 

Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.
 
 

Related Programs:

MPN Essential Testing | How Results Impact Care & Treatment Options

MPN Essential Testing | How Results Impact Care & Treatment Options

Understanding MPN Treatment Options _ What’s Available for MF, PV, and ET

Understanding MPN Treatment Options | What’s Available for MF, PV, and ET?

Understanding and Managing Common MPN Symptoms and Side Effects

Understanding and Managing Common MPN Symptoms and Side Effects


Transcript:

Katherine Banwell:

How do you know when it’s time to change treatments? 

Dr. Raajit Rampal:

Well, I think really two things. One is if we aren’t meeting our goals like we just talked about. But the other aspect of that is if we are incurring toxicities that are just not tolerable to the patient and that’s a reason to change therapy always. 

Katherine Banwell:

Many patients, of course, worry about disease progression. Are there key predictors or tests for progression that patients should know about? 

Dr. Raajit Rampal:

This is a key area of investigation currently. I think one of the things that patients say to us so often when we meet them is what’s going to happen to me. And right now, we don’t have great prediction tools. We can say on a population level well, there is X percent of chance of progression at 15 years. That’s useful if you’re talking about a population. That’s not really useful if you’re talking to an individual. Because if I say to somebody there’s a 20 percent chance of your disease progressing to leukemia, it doesn’t really make a difference. That’s a meaningless statement because if you’re in the 20 percent who progress, it’s not a relevant statistic anymore. 

It’s sort of a binary thing. We’ve got to do better at developing this. This is something that the MPN Research Foundation is really heavily invested in in trying to identify predictive biomarkers. 

If we can do that, then perhaps what we can do is say to a patient this is really what we think your actual risk is. And then, the next step is asking the question if we intervene early, can we prevent that progression from occurring. So, that’s where I think we need to go. We aren’t there yet. 

Katherine Banwell:

What signs or symptoms do you look for that may indicate that the disease is progressing? 

Dr. Raajit Rampal:

The blood counts are often the canary in the coal mine regardless of the disease. They can tell us if ET or PV is progressing into MF or whether MF is progressing to more of a leukemic phase. Changes in symptoms sometimes can be a harbinger of disease progression. So, Patient 2, for example, is doing really well and now, he’s having drenching sweats and losing weight. So, those types of symptoms are a sign that physical findings is the size of the spleen if it’s increasing. All of those things together give us a hint about progression.  

Katherine Banwell:

Well, is there any way to prevent progression? 

Dr. Raajit Rampal:

That is the million dollar question. Again, that’s where we ultimately need to be. We want to be able to intervene to a point where patients don’t get that sick. It would be amazing if we’d come to the point where we can intervene early and nobody progresses to late stage MF. Nobody gets leukemia. And I think that’s a worthy goal. That’s not something that we should think is too lofty of a goal. That should be our ultimate goal here. And a number of groups are investigating this exact question. It’s complicated and it’s going to take time. But I think that’s a worthwhile investment. 

Understanding MPN Treatment Options | What’s Available for MF, PV, and ET?

Understanding MPN Treatment Options | What’s Available for MF, PV, and ET? from Patient Empowerment Network on Vimeo.

How are myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) treated? Dr. Raajit Rampal reviews the available therapies for each of the MPNs. 

Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.

 

Related Programs:

MPN Essential Testing | How Results Impact Care & Treatment Options

MPN Essential Testing | How Results Impact Care & Treatment Options

Thriving With an MPN: Advice for Setting Goals and Making Treatment Decisions

Thriving With an MPN | Advice for Setting Goals and Making Treatment Decisions

Understanding and Managing Common MPN Symptoms and Side Effects

Understanding and Managing Common MPN Symptoms and Side Effects


Transcript:

Katherine Banwell:

So, what are the types of treatments available for MPNs?  And let’s start with myelofibrosis or MF. 

Dr. Raajit Rampal:

If we had had this discussion five years ago, it would be pretty simple, and it would take a minute or two. And that’s completely changing and that’s amazing, and it’s good for all of our patients.  

Right now, for patients with MF, it depends on what the issue is. If the issue is symptoms or spleen, JAK inhibitors are our first line of therapy. Three approved JAK inhibitors are currently available, two on the first side ruxolitinib (Jakafi) and fedratinib (Inrebic). And pacritinib (Vonjo) can be used for patients with really low platelet counts.  

There is a fourth JAK inhibitor that we expect to be, hopefully, approved in June of this year, momelotinib. So, the landscape is about to complete broaden in terms of just JAK inhibitors. 

But beyond the JAK inhibitors themselves, there are a number of late stage clinical trials that are combining JAK inhibitors with agents that work through a different mechanism that don’t work through inhibition of the JAK pathway. So far, these drugs have all shown promise in early phase trials. Now, the definitive Phase III trials are being done. We have to wait and see what the data tells us. But if these are positive trials, this could completely alter the landscape of MPN. 

Katherine Banwell:

There’s also transplants available, right? 

Dr. Raajit Rampal:

Correct. Transplants for more advanced patients, which comes with some major risks. And so, that has to be thought of very carefully in terms of the risks and benefit. But it is a potentially curative strategy. 

Katherine Banwell:

Let’s turn to polycythemia vera or PV. What types of treatments are available? 

Dr. Raajit Rampal:

It’s really quite a range. So, there are things like phlebotomy and aspirin, which has been the mainstay of therapy for many years. There are drugs like hydroxyurea (Hydrea), interferons, JAK inhibitors. So, ruxolitinib is approved in certain settings for treating polycythemia vera. So, the landscape is broad. There are a lot of questions going on right now with polycythemia vera with regards to how it should best be treated. Is the mainstay of phlebotomy and aspirin really what we should be doing or should we be giving patients treatment earlier on. 

And there is some data to suggest that. There is this drug called ropeginterferon (Besremi) that’s FDA-approved for polycythemia, which was compared in the study to phlebotomy and aspirin.  

And at least the data suggests that there may be better control of the disease and less progression possibly, and it’s a small number of patients, by treating patients earlier. Whereas we would have just given phlebotomy and aspirin. So, it’s something to consider. There are drugs in clinical trials as well that look promising one of which is called rusfertide, which actually works by changing the way iron is used by the body. 

Iron is a key component to hemoglobin and it is, of course, a key component to polycythemia in the sense that we phlebotomize patients to make them iron deficient and that’s how we control the disease. But this is a pharmacological way to do that. So, that drug is now in Phase III trials. So, that may also alter the landscape of treatment of PV in the near future.  

Katherine Banwell:

Finally, how is essential thrombocythemia treated? 

Dr. Raajit Rampal:

So, in some cases, with absolutely nothing as we had talked about a moment ago. There is some thought that in really, really low-risk patients. Maybe you don’t need to do anything except observe them. Whereas most patients are on an aspirin. And beyond that, we have drugs like interferon, pegylated interferon, and hydroxyurea and anagrelide, all of which can be utilized. It’s not entirely clear if there is one distinct first line treatment that is the best but these drugs are all active. JAK inhibitors have been studied in this setting. And to date, the data hasn’t led to their approval but, certainly, people have studied it.   

Katherine Banwell:

Dr. Rampal, how can you tell if a treatment is effective? Are there signs that you look for? 

Dr. Raajit Rampal:

Well, I think it’s a couple of things.  

One, are we meeting the treatment goals in terms of are we controlling blood counts with ET or PV? That’s one of the first principles in management. And with regards to MF, the same thing. Are patients’ symptoms being controlled? Is the spleen being adequately controlled? And then, there’s the symptom burden because just because the blood counts are being controlled, patients may still have symptoms, in which case, they are not being adequately treated. And then, we have to do our best to try to find a treatment strategy that does control their blood counts but also does control their symptoms. So, there is the blood count perspective but there is the symptom perspective as well. 

MPN Essential Testing | How Results Impact Care & Treatment Options

MPN Essential Testing | How Results Impact Care & Treatment Options from Patient Empowerment Network on Vimeo.

How could molecular testing affect MPN treatment decisions? Dr. Raajit Rampal explains the purpose of this essential testing and how the results may impact prognosis and care. 

Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.

 

Related Programs:

Are There Predictors That an MPN May Be Progressing

Are There Predictors That an MPN May Be Progressing?

Understanding MPN Treatment Options _ What’s Available for MF, PV, and ET

Understanding MPN Treatment Options | What’s Available for MF, PV, and ET?

Understanding and Managing Common MPN Symptoms and Side Effects

Understanding and Managing Common MPN Symptoms and Side Effects


Transcript:

Katherine Banwell:

Let’s talk about what sort of tests should be done following an MPN diagnosis. Can you tell me about those? 

Dr. Raajit Rampal:

Yeah. Fundamental to the MPN itself, the things that we really want to know is, in most cases, a bone marrow examination is needed because that will tell us really what the disease is that we’re dealing with. It will tell us about the genetics. I strongly believe we have to be comprehensive in our genetic assessments because that does prognosticate and sometimes gives us an opportunity in terms of treatment. Chromosomal analysis. These are the basic bread and butter hematology tests we want to do from the bone marrow to really understand what the patient’s disease is. 

Beyond that, I think that particularly in patients with PV and ET, it’s important that we partner with their primary care physicians to make sure that they’ve had, for example, testing for diabetes, a recent lipid profile, any cardiovascular tests, particularly measurements of blood pressure because these things are all important in terms of an ET or PV patient’s risk of having a blood clot. So, there are, again, things that are within hematology realm but then, there are other general health things that become really important in somebody who is diagnosed with PV or ET. 

Katherine Banwell:

How often should lab tests of blood work be done? 

Dr. Raajit Rampal:

It really depends on the patient. For some patients with PV, for example, they need to have their blood checked every three weeks because they’re having frequent phlebotomies. Whereas some patients with ET could probably go forward to six months between blood tests. So, it depends on the individual. 

Katherine Banwell:

How can results of biomarker testing affect treatment choices for patients with MPNs? 

Dr. Raajit Rampal:

Great question. The genetics are becoming increasingly important in our treatment decisions. So, let’s take a simple example, which is patients with ET. Calreticulin and JAK2 and MPL are the three most common mutations that we see. But they have very different invocation. So, somebody could have a calreticulin-mutated ET and based on them having that calreticulin mutation and no other factors like no history of clotting, that patient may never need to go on a medication aside from aspirin. And even early on, it’s debatable whether or not some of these patients really need aspirin at all. 

Whereas somebody who had a JAK-2 mutant ET, our guidelines and data suggests that that person, once they reach a certain age, should probably be on medication. So, that’s kind of perhaps one of our more clearcut examples of a genetic biomarker telling us how to approach treatment. 

And then, it gets more nuanced from that and more exciting and interesting in the sense that there are mutations, for example, that occur in myelofibrosis and in patients whose disease is progressing towards leukemia, such as IDH mutations. And these are things that are now targetable with FDA-approved drugs.  

And there are now clinical trials combining JAK inhibitors and IDH inhibitors for patients who have more advanced disease who have these IDH mutations. So, you go from on one end, these genomic markers being of prognostic significance and now, on the other hand, we’re getting to a point where, in some cases, they might tell us how to best treat a patient.  

Katherine Banwell:

Dr. Rampal, should all patients diagnosed with MPNs undergo molecular testing?  

Dr. Raajit Rampal:

I strongly believe that. I think that we’ve learned so much that these tests have prognostic value. 

And in some cases, it may suggest a slightly different diagnosis. I definitely think that should be the case. 

Katherine Banwell:

What should patients be asking once they have the results? 

Dr. Raajit Rampal:

What does it mean? That’s the most basic and fundamental question. It’s one thing to get a list of mutations. But the real bread and butter question is what does this mean to the disease and my prognosis and my treatment?  Those are the key questions. 

Thriving With an MPN | Advice for Setting Goals and Making Treatment Decisions

Thriving With an MPN | Advice for Setting Goals and Making Treatment Decisions from Patient Empowerment Network on Vimeo.

Dr. Raajit Rampal shares his perspective on what it means to thrive with an MPN. Dr. Rampal goes on to discuss the factors that impact therapy as well as advice for setting treatment goals for patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF).

Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.

 

Related Programs:

MPN Essential Testing | How Results Impact Care & Treatment Options

MPN Essential Testing | How Results Impact Care & Treatment Options

Understanding MPN Treatment Options _ What’s Available for MF, PV, and ET

Understanding MPN Treatment Options | What’s Available for MF, PV, and ET?

Understanding and Managing Common MPN Symptoms and Side Effects

Understanding and Managing Common MPN Symptoms and Side Effects


Transcript:

Katherine Banwell:

In your experience, what do you think it means to thrive with an MPN? 

Dr. Raajit Rampal:

It’s a great question, right. I think taking a step back, when we think about our patients with MPNs, one of the questions I always have for patients are what are your goals? And inevitably and invariably, people want two things. They want to live longer and they want to live better. And so, I think that thinking about thriving with an MPN to me is about how do we minimize the impact of an MPN in someone’s life. And that means a couple of things. One that means how do we deal with symptoms or things that are causing medical problems. 

But two, how do we deal with the anxiety of a diagnosis? In many cases in my experience, that can be just as detrimental to somebody’s well-being as the actual physical symptoms of the disease.  

Katherine Banwell:

When it comes to choosing therapy for polycythemia vera essential thrombocythemia, or myelofibrosis, it’s important to work with your healthcare team to identify what is going to work best for you. So, to begin, would you define shared decision-making and why is this critical to properly managing life with an MPN? 

Dr. Raajit Rampal:

Yeah. Shared decision-making, to me, is really about the physician or whoever is on the healthcare team providing the patient all of the information needed to make a good decision. That means what are we trying to do? What is the medication or invention going to accomplish? What are the side effects because there are always side effects.  

And what do we think that’s going to do or how is that going to impact the patient’s life? Where things get nuanced is that patients come to us because we have expertise. There are two extremes. One extreme is that the physician says this is the medication you should take. End of discussion. The other extreme though is also not helpful, which is to say to a patient here are five choices. Here are the side effects. You pick one. Our job is to lay out those side effects and the benefits but then, also help guide a decision.  

Katherine Banwell:

What are treatment goals, and how are they determined? 

Dr. Raajit Rampal:

It depends on the disease to a large extent. Now, when we’re dealing with ET and PV, the primary goal of our interventions is to reduce the risk of a clotting event or bleeding event. And that usually involves controlling the blood counts in some cases, not in all patients with ET. 

Sometimes aspirin is all we do. Myelofibrosis is a little bit more complicated because it depends on what the problem is. Not all myelofibrosis patients have the same challenges. Some have anemia that needs treatment. Some have a big spleen. Some have symptoms and some have nothing and they just need observation. So, it’s a bigger list with MF patients. But I think the first part of the discussion always is defining what the goal needs to be. 

Katherine Banwell:

What factors are considered when choosing therapy for ET, PV, and MF? 

Dr. Raajit Rampal:

I think a couple of things. One is what medication we think is going to benefit the patient best. That has to take into account the individual, their willingness to take certain medications, for example, pills versus interferon injection. Some people have an aversion to self-injection, which we have to take that into account. What are the other medical conditions that the patient is dealing with? 

And the reality is, in some cases, it’s cost because these medications, depending on a patient’s insurance, can have quite a different spread in terms of cost. Unfortunately, that is something we have to take into account.  

What Are Potential Telemedicine Risks for Myeloproliferative Neoplasm Patients?

What Are Potential Telemedicine Risks for Myeloproliferative Neoplasm Patients? from Patient Empowerment Network on Vimeo.

What risks should myeloproliferative neoplasm (MPN) patients be aware of with telemedicine? Experts Dr. AnaMaria Lopez from Sidney Kimmel Cancer Center, Dr. Krisstina Gowin from University of Arizona Cancer Center, and patient Lisa Hatfield share their perspectives and advice. Watch as they discuss potential risks in MPN patient care, how to advocate for the continuation of telemedicine, and advice for patients to work toward optimal wellness.

Download Resource Guide

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Transcript:

Lisa Hatfield: 

Are there any risks or drawbacks that you see to telehealth or telemedicine for digital health?

Dr. AnaMaria Lopez: 

The most important thing is to remember that the technology is a tool, and if the person feels that there’s a limitation, so, for example, if the patient is seen and they say their heart is racing or skipping beats or something, now, there are ways, there are electronic stethoscope, so you can really do a full exam except for palpation through telemedicine. But not everybody has that even in a clinic, but certainly in our own home, we don’t have that technology. So if a patient is expressing a concern for which the clinician really feels that needs a closer evaluation, then that’s the right next step, so we’re not…the technology is a tool to help us care for people, and if it’s not all available right there, then we need to see the patient in person. So I think that’s the risk is just sometimes people may feel limited like, “Oh, well, I’m not really sure It’s okay, I’m not really sure I need to see you, or you need to go here or go there for the care.”

And the other, which is a really big threat, is that part of the reason we did 70 years work in a couple months is because it was reimbursed, and we’re reaching the end of the pandemic, the federal…and with that, the payers may go backwards. We all know that if that happens, we will go backwards in telemedicine. [chuckle] There will just be decreased, decreased use. And it may lead to people then going back to traveling for four hours, waiting, only to be told, “Oh, you know what? There’s not this. This clinical trial doesn’t work for you.” So we don’t want to lose ground. And part of not losing ground is that we really need to continue to have advocacy around reimbursement.

Lisa Hatfield: 

Thank you, Dr. Lopez. And I feel compelled, just to follow up with one more question regarding that, because I’m very passionate about this. With some of these rules and guidelines coming to an end, I know in my particular state that I will no longer be able to access my specialist. I see a myeloma specialist. We do not have any here locally. I can access a specialist via telemedicine. I will not have that opportunity. So as all of us know, there are disparities and there are financial disparities in cancer patients. There are racial disparities in cancer patients, there are socioeconomic disparities. Telemedicine has been a tremendous…has had a tremendous impact on the care and the outcomes and the quality of life of so many patients. So as a patient and as an advocate, do you have any recommendations? Do I go to my doctor and say, “Okay, how can I move forward and still talk to my specialist, who’s out of state? Do I go to my state legislature? Do I talk to my insurance company? How can we get this to continue?” Because this has had such a significant impact on the quality of life and on the outcomes for patients, who otherwise, would not have been able to access that care.

Dr. AnaMaria Lopez: 

Yeah, I mean, I think all of the above. Partnering with other advocates, the American Telemedicine Association has a map that kind of says where are all the shifting sands regarding the different rules and legislative changes. But I think it’s led us to a place, where we are all advocates and where physicians, nurses, patients, pharmacists, everybody in the same way that we do team-based care, that we do team-based advocacy and it’s all for our patients.

Lisa Hatfield: 

Great. Thank you for that. Dr. Gowin, any last words that you may have about accessing specialists or telemedicine options?

Dr. Krisstina Gowin: 

Well, I think we covered the basics, but I just want to end with just how empowering the access to digital health interventions really is. And so I don’t think there is a one-size-fits-all approach to every patient. So what I would encourage patients to do is just to really think, “How do I compliment my care? What am I missing? How do I achieve my best wellness? And how do I get those resources in my home to make them more convenient for me?” And to start doing some research and self-advocacy to really get those resources because they are out there and in almost…in every domain, there is now a digital version that is accessible to you now.


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How Is Telemedicine Influencing Personalized Medicine for MPN Patients?

How Is Telemedicine Influencing Personalized Medicine for MPN Patients? from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) personalized medicine be influenced by telemedicine? Expert Dr. Krisstina Gowin from University of Arizona Cancer Center explains how telemedicine helps create a more personalized and precision-based approach for optimal MPN patient wellness.

Download Resource Guide

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Transcript:

Lisa Hatfield: 

Dr. Gowin, can you provide or share some examples of how telemedicine is influencing personalized medicine and MPN care, and how can MPN patients best advocate for themselves to get the latest in MPN care?

Dr. Krisstina Gowin: 

Well, I think it’s going back to some of the conversations we’ve already had, is that now with telemedicine, you can really access academic centers no matter where you are. And so rural areas now can go to academic centers, very accessible without travel, and so what that lends to is more access to precision-based clinical trials, and very often now we’re doing next-generation sequencing panels for patients with MPN. We’re looking at what are the genetic features of the disease and we may be accruing trials based on those genetic features. And so that kind of conversation really only happens at academic centers, and so I think it’s really allowing those that live far away, a few hours away, to really have those personalized and precision-based conversations. And then tying in again the aspect of integrative medicine. And then what is integrative medicine all about is personalizing your treatment plan, asking what are your goals, what is your lifestyle, what is your culture, and how do we really get you on a plan that makes sense for you, that is local for you and sustainable for you to really achieve your optimum wellness?

And so if I were counseling patients listening to this, I would say, start with the in-state academic centers and say, “What are the telemedicine services there? Is there an integrative medicine department there”? And then get a quarterback within that department and say, “Okay, this is the plan,” and then that quarterback can say, “Well, now let’s look local. What do you have? What are your resources there? Let me do some homework with you and hook you up with really evidence-based high quality providers to achieve your personalized needs in your local community.” And I think that’s how we’re really going to get all of our patients in a precision and personalized approach no matter where they live, and that’s again, the beauty of telemedicine and digital health. 


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How Will Advanced Technologies Enhance Myeloproliferative Neoplasm Care?

How Will Advanced Technologies Enhance Myeloproliferative Neoplasm Care? from Patient Empowerment Network on Vimeo.

How will myeloproliferative neoplasm (MPN) care be enhanced by advanced technologies? Experts Dr. Krisstina Gowin from University of Arizona Cancer Center and Dr. Ana Maria Lopez from Sidney Kimmel Cancer Center share information about how technology can help manage symptom burden, risk factors, and the patient experience for enhanced MPN care.

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How Is Telemedicine Influencing Personalized Medicine for MPN Patients

Transcript:

Lisa Hatfield: 

So, Dr. Gowin, do you have anything to add on, how MPN care or just cancer care, in general, could change with different technologies? We didn’t touch a lot on things like artificial intelligence and that type of thing, and we can speak to that or any other type of technology that you’re familiar with.

Dr. Krisstina Gowin: 

Well, I think the artificial intelligence aspect is really going to change the paradigm again on how we’re designing, studies. And I think one of the biggest challenges that we have in myeloma and as well as myeloproliferative neoplasms, is to think about how do we optimally sequence our therapies to achieve best survival, right? And I think this is a wonderful problem to have. We have now not only one JAK inhibitor on the market, but several and more in the pipeline and several other therapeutic targets. And so now the question is which therapy and when do we employ it? So things like artificial intelligence will help us to answer that question with machine learning decision tree analysis, all of that is going to be answered through those kind of platforms. And so I think that is going to be a shift we will see in the next five years is many different machine-based learning algorithms to better understand those problems we cannot have tackled traditionally otherwise.

Sensors though is another one, right? And so a big thing in MPNs is not only addressing the blood counts and reducing risk of thrombosis, and to address symptom burden, but it’s really addressing lifestyle because it’s things like cardiovascular disease, stroke that really we’re worried about as some of the sequelae of having the disease and what we’re trying to prevent with therapeutics. And so even going back to the NCCN guidelines, it’s addressing cardiovascular risk factors as part of our core treatment goals. And so how do we really do that? And it’s really through lifestyle medicine and that’s where the sensors come in. And so now we have, these Fitbits and smartphones that connect to our Apple watches and we have Garmins and all these wonderful devices that are prompting us to move more, prompting us to be cognizant of our heart rate and stress response prompting us to meditate. And so I can envision those evolving over time and connecting to the EMR and being very seamlessly interwoven into our clinical trials. And we’re already doing that. In fact, we’re talking about doing one very soon in MPN patients. And so I think the sensors are gonna be another big way that we’re going to be integrating, into our clinical trials and symptom management tools.

Lisa Hatfield: 

That’s fascinating. Thank you for that. And Dr. Lopez, do you have anything to add about other technologies and how they may affect cancer care in the future?

Dr. Ana Maria Lopez: 

Sure. When Dr. Gowin mentioned the sensors, it just reminded me, we’re building this new building, patient care building and oncology will be there. And I did a tour recently, and we’re used to go to the doctor, you stop in, they get your blood pressure, they get your weight, et cetera. Here, you’ll walk in directly to your exam room and you check in at a kiosk, so you just kinda check in [chuckle] with a little robot kiosk, and then it’ll tell you where you’re going. You’ll go to Room 3, let’s say, and Room 3 will say, “Welcome, Lisa.” [chuckle] And so you know that you’re in the right place. And you’ll walk in, there’s your gown, you’ll sit in the exam chair, and the exam chair automatically is gonna take your vital signs. So it just seems, really these built-in aspects to the technology. And one of the things, again, what I just love about this work is that it’s a very interdisciplinary, multidisciplinary. And one of the projects that we were working on, which it ties into this, when I was in Arizona with the telemedicine program is we worked with the College of Architecture and with this concept of smart buildings.

Dr. Ana Maria Lopez: 

So it’s kind of like that. Why should you do these different sensors that detect, but that it could also detect. You might walk into the room and you might be really nervous as you might be really cold, and it would detect that and it would warm the room for you. Or you might be coming in and be having hot flashes and it would just cool the room for you. So the technology has so much potential to really improve the patient experience.

Lisa Hatfield: 

And that’s amazing to me. I think that would be incredible to walk into a building to have that experience, as long as it doesn’t take away the compassion and care I get from my providers. I am so fortunate to have extraordinary providers, so I don’t think it will ever take over that aspect of it, I think that is a fear people have, especially with artificial intelligence and that type of thing, I think it can only go so far. Can’t provide the humanness that’s required for patient care, so yeah.

Dr. Ana Maria Lopez: 

Yeah. These are tools. 


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Will Advancing Technologies Allow MPN Clinical Trials in Patient Homes?

Will Advancing Technologies Allow MPN Clinical Trials in Patient Homes? from Patient Empowerment Network on Vimeo.

Will myeloproliferative neoplasm (MPN) clinical trials at patient homes be enabled by advancing technologies? Experts Dr. Krisstina Gowin from University of Arizona Cancer Center and Dr. Ana Maria Lopez from Sidney Kimmel Cancer Center share their perspectives on clinical trials models that work well with remote models and an update about the hospital at home concept.

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Transcript:

Lisa Hatfield: 

Do you think that technology has progressed enough that clinical trials might allow a patient to be at home, maybe in a more remote area and monitored remotely? Whereas in the past, that same clinical trial required them to be at the facility? Do you think that we’ve progressed to that point in some clinical trials?

Dr. Krisstina Gowin: 

You bet. Yeah. I think COVID out of necessity has forced us to do that. And I have in my own clinical trials, even with pharmacologic clinical trials conducted telemedicine visits that were approved by the sponsor. So the paradigm is shifting, and particularly when it is oral therapeutics, I think that’s really accessible when they’re IV subcutaneous, I think that has different challenges. Obviously you can’t do that as remote, but when their oral therapeutics are non-pharmacologic intervention, such as our integrative interventions, I think it really lands well to a more remote experience.

Lisa Hatfield: 

Okay. And then would that require communication between the local oncologist and maybe someone like yourself, the investigator on that clinical trial to know what is going on with that particular patient? I assume that that communication would be ongoing?

Dr. Krisstina Gowin: 

Absolutely. Always.

Lisa Hatfield: 

Okay. Yeah. All right, great. Well, thank you for that information. So, Dr. Lopez, kind of a similar question for you. What are some examples of how technology is influencing cancer care right now?

Dr. Ana Maria Lopez: 

Yeah, let me just add on the clinical trial question.

Lisa Hatfield: 

Oh, Yes.

Dr. Ana Maria Lopez: 

That there’s also the opportunity. Again, there were so many things that we thought, “Oh no, we just can’t be done.” But because of the necessity, necessity is the mother of invention, we do remote consent, so that was a big deal in the past. We can also do a televisit ahead of the appointment, and screen for the cancer clinical trials, people travel large distances for studies and instead of traveling four or five hours, and then to be told, “Oh, actually you don’t meet the criteria.” To be able to do all of that at a distance, to get the records, to get the images, to review all that needs to be reviewed. And then to say yes, and not only yes, but we can also do your consent at a distance in some situations.

And then when you come, there’s actually the more substantive, perhaps even the treatment. There’s also a large movement around hospital at home and that these patients that are eligible for that would be able…with digital support, be able to get hospital level care in some cases at home. So some of that may involve infusion, some of that…again, but that visual connectivity and in the past you really had to kind of conceptualize it, and it was kind of space aging to talk about it. But we now, we’ve all done FaceTime, so I think we all really can understand what it entails, so tremendous shifts and, we want to try to keep that momentum going for our patients. So, I do think that, there’s so many ways that technology has impacted cancer care, even when we talk about the electronic record and patients accessing the electronic record and patients having the opportunity to go into a portal and to see their labs, to see their reports…

To be able to track their changes. All of that is really, really powerful. You know, patients with…the most common I think is patients with diabetes who track their blood sugar sometimes to the minute and they can say, “Oh, I ate that and now I see the impact.” So the opportunity for monitoring, the opportunity for also bringing in experts. So let’s say there’s a patient with a rare disease and the expert is elsewhere, there might be the opportunity to bring people together. We do tumor boards. That’s just part of what we do in cancer care. And also as many…there are health systems now so that it’s not one hospital, it’s multiple hospitals together where we can bring all of those folks together, bring in local expertise, regional expertise, national expertise, all for the patient’s benefit.

So there are so many ways that technology even something as simple as the note. Now this is something we experimented with and it’s still in experimentation phase, but there were these Google classes where you could interact with the patient and as I’m talking, the Google glass would record kind of the conversation and would come up with some sort of a structure for the note. So for what that encounter had been like. So there are lots of ways of how do you capture natural language in real time to really help the workflow, the documentation process. So I think there’s aspects to help the patient, to help the families, to help the clinical teams and to help everybody work together. 

Lisa Hatfield: 

Great. Thank you. And you talked about the patient portal, and I’m one of those patients at fault of seeing a lab result before my doctor saw it and calling him or sending a message via MyChart saying, “Hey, this is going up. What’s wrong with this?” So I’m sure you don’t have to mention any names. I’m sure you’ve seen the challenges of, digital health too, are having that patient portable or portal accessible to patients. So anyway, just wanted to throw that out there that I’m sure that brings challenges to you. Also few little challenges here and there.

But at the same time, that’s so good, right? It’s so good that patients are engaged. It’s so good that you’re engaged. And I think as long as we’re communicating that yes, you may see this before me, so you may have questions and then, we just get together and answer the questions. 


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Is Technology Accelerating Progress in Myeloproliferative Neoplasm Care?

Is Technology Accelerating Progress in Myeloproliferative Neoplasm Care? from Patient Empowerment Network on Vimeo.

Is myeloproliferative neoplasm (MPN) care being advanced by technology progress? Expert Dr. Krisstina Gowin from University of Arizona Cancer Center discusses how MPN care has benefited from technology advances and explains two MPN technology tools and how they assist in MPN patient care.

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What Are Potential Telemedicine Risks for Myeloproliferative Neoplasm Patients

Transcript:

Lisa Hatfield: 

So, Dr. Gowin, a couple of questions for you. Is technology playing a role in accelerating progress in MPN care?

Dr. Krisstina Gowin: 

Oh, absolutely. And I think some of the ways that it really accelerates progress is pulling us together. So what we need to recognize is that myeloproliferative neoplasms truly is a rare disease, and we just celebrated Rare Disease Day. But there’s a lot of challenge in treating patients and progressing the field forward in rare diseases because you can’t do the big clinical trials. It’s hard to come together because everything’s siloed, and there’s just a couple patients here, a couple patients there in each practice. But with digital health and clinical trials that are offered on a digital platform, it pulls the nation together and even the world together. And we’ve seen that. I’ve done an international survey-based analysis and I had 858 MPN patients from 52 countries participate in that survey. And so that just shows how it pulls the world together. And for the web app that we just discussed, we had 93 patients say they were interested within three weeks, and within actually a week, we identified them all and then took three weeks to actually accrue them to the trial. So it really speaks to A, how MPN patients are digitally engaged and excited about these kinds of platforms. And then B, how effective it really can be to pull the groups together.

So yes, I think it’s…and that’s really how we’re going to get progress is through these kind of interventions with a rare disease. And I hope it’s okay if we jump back to something you said, Dr. Lopez, which is, I think telemedicine is so so important to bring everyone together. And in particular, I see that on the transplant ward. And so in myelofibrosis, that’s the only curative therapy. And so many myelofibrosis patients actually go through allogeneic stem cell transplantation. And my goodness, that is a socially isolating experience. Patients are in the hospital, not uncommonly for at least 30 days and then have to be near their transplant center for three months, which often is away from home. So to pull in their support system, both through the acuity of the transplant themselves and then the couple of months after is so crucial to a successful transplantation. And I think through FaceTime and also the MPN support groups, which is very robust, the patient advocacy and the way the MPN network sticks together on a digital platform, I think is really unique and offers unique support.

Lisa Hatfield: 

Thank you. And then what role does technology play in the disease symptom management, and in particular, in clinical trials too. What role does technology play with clinical trials?

Dr. Krisstina Gowin: 

Well, I think it helps us through different, clinical trial accrual patterns, we can see who’s eligible where, so it helps us identify patients. It helps us to, understand the different kind of precision-based medicine approaches so we can start to pool the data, say for, particular mutations… ASXL1 mutations. And so it helps us in the precision medicine aspect of clinical trials and now we’re looking at symptom management and how do we really integrate that. So large survivorship platforms like Carevive, if you’ve ever heard of Carevive, is now integrating our validated symptom assessment form into the Carevive platform. So now we can really collect that data and use that to mine it for potential kind of retrospective analysis. So it’s helpful for clinical trials as well as for our clinicians and clinics to really identify changes in symptom burden.

And just as Dr. Lopez was mentioning, that we can track these over time and it can flag and say, “Oh, your symptoms are changing, they’re increasing over time,” and maybe we need to be thinking about that. And so Carevive is really kind of an electronic medical record driven it’s really a healthcare driven platform, but now there’s patient ones too. And I just learned about this two weeks ago, I was at an MPN conference in Phoenix and learned about MPN Genie. And so MPN Genie apparently, is tracking…patients are putting their symptoms in and that’s shooting that information to the electronic medical record to their doctors. And so I think that’s fantastic, ’cause, we now get that information real time and we can change our clinical management, maybe bring that patient in sooner, maybe do a bone marrow earlier. We never would’ve identified that if it weren’t for those kind of digital engagements, so I think it’s a really exciting time. And I think we’re going to see more and more of these new platforms and ways for, different EMRs and smartphones to be communicating back and forth between patients and providers. 


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How Can MPN Patients and Care Partners Stay Aware of Telemedicine Options?

How Can MPN Patients and Care Partners Stay Aware of Telemedicine Options? from Patient Empowerment Network on Vimeo.

Telemedicine is often an option for myeloproliferative neoplasm (MPN) care, but how can patients and care partners stay aware of options? Expert Dr. AnaMaria Lopez from Sidney Kimmel Cancer Center shares her experience introducing healthcare providers to caring for patients via telemedicine and some of the benefits telemedicine provides versus an in-person visit.

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Transcript:

Lisa Hatfield: 

And some patients might be a little more reluctant to use telehealth or telemedicine. How can patients and their care partners feel more confident in voicing their concerns or communicating with their healthcare teams regarding any telemedicine options that are out there?

Dr. AnaMaria Lopez: 

So you mentioned that I had been the founding medical director of the Arizona Telemedicine Program, and it was such a wonderful experience because skepticism, and I really respected that. It was brand new, and we had our system in the library. And the library, it was down in the basement, so it was very metaphorical. I would meet the new clinician at the entrance of the library. We would walk down the stairs together and often, the conversation was, “Okay, I’m doing this for you. I’m doing it one time. We’ll see how it goes.” And I was always so reassuring that if for some reason, because ultimately the clinician needs to feel comfortable, “Yes, this works, or no, it doesn’t. And if you have any doubt and you feel that you need to see the patient in person, you just need to say that,” ‘I need to see the patient in person.’” And inevitably as we’re walking up the stairs, “Oh, I know you called me because I was on call. Just call me anytime. Don’t call the on-call person. This was great. I loved it.”

So inevitably, people really like it, and it’s good. You see the patient in their own environment, you can interact. You often get insights that you may not have gotten otherwise just because of where you are and how comfortable they feel in their own space. So I think, for me it’s the proof is in the pudding. Give people the opportunity, have the right supports and technology in place, and often it’s a very positive experience. 


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What Telehealth Tools Impact Myeloproliferative Neoplasm Care?

What Telehealth Tools Impact Myeloproliferative Neoplasm Care? from Patient Empowerment Network on Vimeo.

How are myeloproliferative neoplasm (MPN) patients impacted by telehealth tools? Experts Dr. AnaMaria Lopez from Sidney Kimmel Cancer Center and patient Lisa Hatfield discuss specific situations and telehealth tools that benefit patients for improved MPN care. 

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Transcript:

Lisa Hatfield: 

Dr. Lopez, can you also speak more broadly to innovative telehealth tools that are making an impact on symptom management and overall cancer care?

Dr. AnaMaria Lopez: 

Sure. So one of the things that we know, is that, for example, people have appointments every three weeks, or they have appointments once a month with the oncologist, and a lot can happen in that time. So setting up systems that are assisted by technology, so that patients can report their symptoms in real time can be very helpful. And some of this may require…it may not be a common way where the person may be familiar going to a computer or going to their phone to kind of say, “This is how I’m feeling.” So that may require some engagement education, but often regardless of age, regardless of background, people find that really easy and find that so helpful to be able to say, “Oh, was it two weeks ago that I had that?” As opposed to just saying, “Hey, I just had this,” and then it can happen anytime day or night that the patient can report. And that way there’s…it’s just so helpful to have an intervention in real time.

The other part that’s good is that often some of these systems can kind of track. So we can look at it together and say, “You know what? Your fatigue tends to be a couple of weeks after therapy, so how can we either prepare for that?” Or just to have the reassurance that, “Yes you have that depth, but it gets better and you get through it.” So being able to look retrospectively and identify that can be helpful and I think also just the ease for people to be able to connect with multiple specialists, sometimes to have multidisciplinary visits where not only does the patient meet with everyone, but the patient can see that we are all meeting and interacting together. So all of those are incredible tools, one of my favorites though, one of my favorites is patients who are in the hospital and patients who are in the hospital a long time, on some occasions. So and even if a person’s not there a long time, it can feel like a long time, so to use the technology, not just to connect the patient, the healthcare team, but to use the technology to connect the patient with his or her family. And I think especially…I mean, a lot of people have smartphones, but it’s using your minutes, sometimes the Internet may not be so strong. So to use the technology that would be used for the clinical piece to have that available in the inpatient setting so that patients can feel connected.  

Lisa Hatfield: 

Yeah, that’s a really great thought that you brought up, too. I know when the pandemic was in full swing, but patients were starting to go back into the office to see their provider. For me, I was not allowed to take my husband in with me, so I went in alone. I was far enough along in my journey. I didn’t necessarily need a care partner with me, but some patients do, maybe a newly diagnosed patient. So that is a really great point. Say, a patient has to come in by himself or herself, is that a technology they can use? Are you willing to let them use their phone to maybe FaceTime during that call or we had to use the actual physical landline because my phone did not connect, the signal wasn’t strong enough. But do you allow that during your appointments to have patients contact somebody?

Dr. AnaMaria Lopez: 

Absolutely.

Lisa Hatfield: 

Okay. That’s great. Yeah.

Dr. AnaMaria Lopez: 

And also there’s pandemic, but there’s also…people live everywhere. So you could say their son could be in California and I’m in Philadelphia and this way it’s okay, we’ll just beam them in. 


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How Can Myeloproliferative Neoplasm Patients Use Integrative Health?

How Can Myeloproliferative Neoplasm Patients Use Integrative Health? from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) patients benefit from integrative health? Experts Dr. Krisstina Gowin, Dr. AnaMaria Lopez, and patient Lisa Hatfield discuss common symptoms of MPN patients, integrative health techniques, and benefits of including integrative medicine.

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Transcript:

Lisa Hatfield: 

So I have multiple myeloma, and, of course, that comes with side effects from the different therapies and symptoms of their own. We have a great integrative health center at our cancer center here locally where I live, and I’ve used it for acupuncture for some of my symptom management. I’ve also watched you on different platforms, through webinars and patient support groups where you describe different integrative health techniques and that type of thing. So I’m wondering…two questions. The first part is, what symptoms do MPN patients face the most? And then how can they use integrative health to do that, particularly as it relates to telemedicine? Are there telemedicine options for integrative health? I suppose things like acupuncture, maybe not, but other types of, of integrative health, and can they get a consult for integrative health? Can they even go as far as getting a consult? So if you can answer those questions, the symptoms they face, how to use integrative health, and if they can get a consult for integrative health, that would be great. We’d appreciate that.

Dr. Krisstina Gowin: 

Yeah. Well, Lisa, I want to take a moment just to validate your journey that you’re going through and to congratulate you for your self-advocacy to go look for those integrative therapies to support yourself. And for MPN patients, I will say that it’s a really unique group, and so all cancer patients experience symptoms. But in myeloproliferative neoplasms, it’s really kind of this heterogeneous what we call a symptom burden. And so most patients will experience fatigue about 80 percent of MPN patients. But then beyond that, there’s really a whole slew of different sequelae that can be associated with the disease, which you may or may not think about when you’re thinking about MPNs, such as psychosocial issues, sleep issues, sexual issues.

And then we have kind of the classical issues that happen with MPNs, such as dizziness, but we talked about the fatigue, bone pain, itching, abdominal discomfort from an enlarged spleen and early satiety, or feeling full quickly. It’s really a huge symptom complex, if you will. And we now have validated measurement tools to better understand those. It’s the MPN symptom assessment form, which has really, I think, revolutionized how we look at MPN. It’s no longer just treating the blood counts. We’re treating the patient as a whole, and even within our NCCN guidelines, kind of how we as oncologists go through the algorithms of how to change therapy and how we look at patients. We now have symptoms in there. So even if blood counts are controlled, we may change therapies or even do a bone marrow based on symptoms alone. So symptoms are a huge thing in MPN. So getting to your second question for integrative health.

So I think that MPN…the patients in the community are really early adopters for digital engagement, which is fantastic. Everyone’s very engaged, and I’ve had the opportunity to work on meditation apps, yoga apps, a wellness-based app here from the University of Arizona, and patients just really accrue fast. Everyone’s so excited. And most of these, though, were very small kind of pilot trials, looking at feasibility, can’t we really do these things? But most of them as well are showing some impacts on depression, anxiety, sleep, and total symptom burden. So I do think that these modalities through digital platforms certainly can make a difference on the symptoms. And we’ve seen that with meditation. We’ve seen it with yoga, and we’ve seen it with a seven domain wellness app. And is it the digital engagement? I don’t think so.

I think it’s likely the integrative therapies that they’re receiving through that platform, right? We know meditation works, we know yoga works, perhaps not so well in MPNs. We need to build that evidence base, but other solid cancers, we know those interventions really work. But it’s wonderful to get that kind of early data, say it not only works, but it also works when you’re doing it at home, when you’re doing it on a digital platform. And so I would encourage all patients listening to this to, yes, look at what’s around you, what are the resources, what are the clinical trials? Looking at these different digital modalities for integrative medicine, but also to go get an integrative consultation.

And as Dr. Lopez already had mentioned, she does all of her integrative medicine via telemedicine now, which is fantastic. And so you, it’s really, it’s that, you know, your fingertips. You now have access to wonderful oncologists like Dr. Lopez to guide you in this journey. And the journey is not only allopathic Western medicine, but it’s treating you as a person, you as a whole symptom complex. And that’s really what integrative medicine aims to support you through. 

Lisa Hatfield: 

And you mentioned that Dr. Lopez also does her integrative health via telemedicine. So I’m going to ask, Dr. Lopez, can you speak to that a little bit more? How do you do that with patients? Do they just contact you and set up an appointment for an integrative health consult or appointment? And do you conduct some of that yourself, or do you send them to particular resources in the community?

Dr. AnaMaria Lopez: 

Sure. So, yes, patients can make an integrative oncology appointment directly. I really like to do the consults through tele simply because I can…as I was mentioning, it’s like a virtual house call to really get a sense of the patient. Often a partner, significant other, caregiver might be present as well, and as we know there’s the survivor and there’s the co-survivor. So including both can be very helpful to some people, and I think the initial intake…again, as Dr. Gowin was saying, it depends so much on what the person wants to do. So the first opportunity for coming together is simply, “Where are you? What are your goals? What’s important to you? And of the panoply of options, which might be the easiest or the one that you are most interested in?”

And so depending on what it is, we might work together, we might also bring in others if the person is really interested in making lifestyle changes, let’s say related to nutrition. The person might work closely with a nutritionist for some period of time and then come back, and we’d come together and reassess. You mentioned the acupuncture, and you can’t do acupuncture at a distance, but you can certainly teach people about the points and consider acupressure for certain points. So there are so many ways to engage and interact, but yes, I think like a lot of medicine, it’s a team-based approach.


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How Has COVID-19 Impacted Myeloproliferative Neoplasm Care?

How Has COVID-19 Impacted Myeloproliferative Neoplasm Care? from Patient Empowerment Network on Vimeo.

How has myeloproliferative neoplasm (MPN) care been impacted by COVID-19? Expert Dr. Krisstina Gowin from University of Arizona Cancer Center shares information from an MPN care study by the Mayo Clinic on some of the impacts experienced by patients. 

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What Advances in Cancer Care Relate to Digital Health

What Advances in Cancer Care Relate to Digital Health?

How Can MPN Patients and Care Partners Stay Aware of Telemedicine Options

How Will Advanced Technologies Enhance Myeloproliferative Neoplasm Care

Transcript:

Lisa Hatfield: 

So the pandemic has resulted in significant changes to many aspects of daily living for many of us, but for patients living with cancer like myself, there are different realities that we’ve had to deal with. Do we go in for our monthly blood draws, or do we wait a couple of months? So question for, Dr Gowin, can you give us an overview of the impact that COVID-19 has had on MPNs or MPN care?

Dr. Krisstina Gowin: 

Absolutely. Well, there was a really wonderful study that was done, really led out of Mayo, by Jeanne Palmer and Ruben Mesa, and it was an international study, and it looked at 1,500 MPN patients. And they asked questions like, “How many of you are actually having telemedicine?” And this was in 2020, kind of at the beginning. And over half of them had already been engaging in telemedicine. And about a quarter of them felt that their care actually was delayed a little bit and that there were actually consequences to that delay, so that really speaks to an international kind of change in the paradigm of how we’re delivering care for MPN patients. The other thing is the lockdowns, the lockdowns that were occurring for us here in the US and really internationally. And what they did is, they asked patients their MPN symptom burden, and those that were on lockdown, not surprisingly I think to all of us, had a significantly higher symptom burden.

So I think that really speaks to that A, yes, there was a very large impact of COVID on the development of telemedicine and the need for telemedicine. But it also underscores the need for symptom management that we now have a group of patients that are having a higher symptom burden, probably likely secondary to more sedentary behavior, more anxiety, more depression, but a higher symptom burden because of COVID. And so we really need not only more therapeutics and perhaps non-pharmacologic interventions to support their symptom burden, but it needs to be delivered on a digital platform.


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What Advances in Cancer Care Relate to Digital Health?

What Advances in Cancer Care Relate to Digital Health? from Patient Empowerment Network on Vimeo.

How has digital health brought advances in cancer care? Expert Dr. AnaMaria Lopez from Sidney Kimmel  Cancer Center discusses cancer care advances and benefits from telemedicine, remote monitoring, and the re-emergence of a traditional patient care option.

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How Has COVID-19 Impacted Myeloproliferative Neoplasm Care

How Has COVID-19 Impacted Myeloproliferative Neoplasm Care

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Will Advancing Technologies Allow MPN Clinical Trials in Patient Homes

Transcript:

Lisa Hatfield: 

So, Dr Lopez, I’d like to start with you to talk about cancer care and technology and how far we’ve come with technology and cancer care. We’ve made a fair number of strides in cancer care as they relate to digital health. Can you speak to that a bit?

Dr. AnaMaria Lopez:

Sure. I always say that the COVID pandemic allowed us to advance telehealth in a couple of months what probably would have taken 70 years. So we went from maybe 10 percent of the visits being done through tele to 90 percent plus. So a huge, huge change, and a lot of lessons learned, both in the, how do we do it clinically as well as how do we integrate this? How do we integrate this into clinical trials and to move beyond? We really thought telemedicine and really thinking telehealth because there are so many technologies, so there could be monitors, there could be…a lot of us were doing work in patient-recorded outcomes, how do you integrate that? How do you make it easy for patients to use this? And maybe it’s not simply the patient, but it’s also the patient and the caregiver who can help with this reporting. What are really the implementation efforts that need to be done? I could go on and on because there are so many lessons learned, and it really shook things up. So people are thinking of this as a new technological revolution. So technology plays a big role in care and certainly a very big role in cancer care.

Lisa Hatfield: 

And just out of curiosity, Dr Lopez, do you have a lot of your patients who still continue to see you via digital health or telehealth, who prefer that?

Dr. AnaMaria Lopez: 

Yes. Yeah. For example, in some of the psychiatry literature, which I think is a little bit unexpected because you think psychiatry is such an intimate interaction. Well, a lot of patients actually feel safer when it’s digital, when it’s through tele. So yeah, I do. And we were talking earlier about doing integrative medicine, and almost all of my integrative medicine patients, we do at a distance. I really think of it as it’s a way to bring back the house call.


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Managing Life With an MPN | What You Need to Know

MPN expert Dr. Raajit Rampal shares advice for making treatment decisions for patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). Dr. Rampal also reviews tips and tools for managing symptoms and side effects and provides an update on new and emerging MPN therapies.
 
Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.
 
 

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Transcript:

Katherine Banwell:

Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today’s webinar is a continuation of our Thrive series. And we’re going to discuss how to manage life with an MPN.  Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you.  

Let’s meet our guest today. Joining me is Dr. Raajit Rampal. Dr. Rampal, welcome. Would you please introduce yourself.    

Dr. Raajit Rampal:

Hi. Thank you so much for having me. I’m Raajit Rampal from Memorial Sloan Kettering Cancer Center where I focus on myeloproliferative neoplasms. 

Katherine Banwell:

Thank you so much for being with us today. 

Dr. Raajit Rampal:

My pleasure.  

Katherine Banwell:

As we do with each of the webinars in our Thrive series, let’s start with this question. In your experience, what do you think it means to thrive with an MPN? 

Dr. Raajit Rampal:

It’s a great question, right. I think taking a step back, when we think about our patients with MPNs, one of the questions I always have for patients are what are your goals. And inevitably and invariably, people want two things. They want to live longer and they want to live better. And so, I think that thinking about thriving with an MPN to me is about how do we minimize the impact of an MPN in someone’s life. And that means a couple of things. One that means how do we deal with symptoms or things that are causing medical problems. 

But two, how do we deal with the anxiety of a diagnosis? In many cases in my experience, that can be just as detrimental to somebody’s well-being as the actual physical symptoms of the disease.  

Katherine Banwell:

When it comes to choosing therapy for polycythemia vera essential thrombocythemia, or myelofibrosis, it’s important to work with your healthcare team to identify what is going to work best for you. So, to begin, would you define shared decision making and why is this critical to properly managing life with an MPN? 

Dr. Raajit Rampal:

Yeah. Shared decision-making, to me, is really about the physician or whoever is on the healthcare team providing the patient all of the information needed to make a good decision. That means what are we trying to do? What is the medication or invention going to accomplish? What are the side effects because there are always side effects.  

And what do we think that’s going to do or how is that going to impact the patient’s life? Where things get nuanced is that patients come to us because we have expertise. There are two extremes. One extreme is that the physician says this is the medication you should take. End of discussion. The other extreme though is also not helpful, which is to say to a patient here are five choices. Here are the side effects. You pick one. Our job is to lay out those side effects and the benefits but then, also help guide a decision. 

Katherine Banwell:

What are treatment goals and how are they determined?  

Dr. Raajit Rampal:

It depends on the disease to a large extent. Now, when we’re dealing with ET and PV, the primary goal of our interventions is to reduce the risk of a clotting event or bleeding event. And that usually involves controlling the blood counts in some cases, not in all patients with ET. 

Sometimes aspirin is all we do. Myelofibrosis is a little bit more complicated because it depends on what the problem is. Not all myelofibrosis patients have the same challenges. Some have anemia that needs treatment. Some have a big spleen. Some have symptoms and some have nothing and they just need observation. So, it’s a bigger list with MF patients. But I think the first part of the discussion always is defining what the goal needs to be. 

Katherine Banwell:

What factors are considered when choosing therapy for ET, PV, and MF? 

Dr. Raajit Rampal:

I think a couple of things. One is what medication we think is going to benefit the patient best. That has to take into account the individual, their willingness to take certain medications, for example, pills versus interferon injection. Some people have an aversion to self-injection, which we have to take that into account. What are the other medical conditions that the patient is dealing with? 

And the reality is, in some cases, it’s cost because these medications, depending on a patient’s insurance, can have quite a different spread in terms of cost. Unfortunately, that is something we have to take into account. 

Katherine Banwell:

Let’s talk about what sort of tests should be done following an MPN diagnosis. Can you tell me about those? 

Dr. Raajit Rampal:

Yeah. Fundamental to the MPN itself, the things that we really want to know is, in most cases, a bone marrow examination is needed because that will tell us really what the disease is that we’re dealing with. It will tell us about the genetics. I strongly believe we have to be comprehensive in our genetic assessments because that does prognosticate and sometimes gives us an opportunity in terms of treatment. Chromosomal analysis. These are the basic bread and butter hematology tests we want to do from the bone marrow to really understand what the patient’s disease is. 

Beyond that, I think that particularly in patients with PV and ET, it’s important that we partner with their primary care physicians to make sure that they’ve had, for example, testing for diabetes, a recent lipid profile, any cardiovascular tests, particularly measurements of blood pressure because these things are all important in terms of an ET or PV patient’s risk of having a blood clot. So, there are, again, things that are within hematology realm but then, there are other general health things that become really important in somebody who is diagnosed with PV or ET. 

Katherine Banwell:

How often should lab tests of blood work be done? 

Dr. Raajit Rampal:

It really depends on the patient. For some patients with PV, for example, they need to have their blood checked every three weeks because they’re having frequent phlebotomies. Whereas some patients with ET could probably go forward to six months between blood tests.  

So, it depends on the individual. 

Katherine Banwell:

How can results of biomarker testing affect treatment choices for patients with MPNs? 

Dr. Raajit Rampal:

 question. The genetics are becoming increasingly important in our treatment decisions. So, let’s take a simple example, which is patients with ET. Calreticulin and JAK2 and MPL are the three most common mutations that we see. But they have very different invocation. So, somebody could have a calreticulin-mutated ET and based on them having that calreticulin mutation and no other factors like no history of clotting, that patient may never need to go on a medication aside from aspirin. And even early on, it’s debatable whether or not some of these patients really need aspirin at all. 

Whereas somebody who had a JAK-2 mutant ET, our guidelines and data suggests that that person, once they reach a certain age, should probably be on medication. So, that’s kind of perhaps one of our more clearcut examples of a genetic biomarker telling us how to approach treatment. 

And then, it gets more nuanced from that and more exciting and interesting in the sense that there are mutations, for example, that occur in myelofibrosis and in patients whose disease is progressing towards leukemia, such as IDH mutations. And these are things that are now targetable with FDA-approved drugs.  

And there are now clinical trials combining JAK inhibitors and IDH inhibitors for patients who have more advanced disease who have these IDH mutations. So, you go from on one end, these genomic markers being of prognostic significance and now, on the other hand, we’re getting to a point where, in some cases, they might tell us how to best treat a patient. 

Katherine Banwell:

Dr. Rampal, should all patients diagnosed with MPN’s undergo molecular testing? 

Dr. Raajit Rampal:

I strongly believe that. I think that we’ve learned so much that these tests have prognostic value. 

And in some cases, it may suggest a slightly different diagnosis. I definitely think that should be the case. 

Katherine Banwell:

What should patients be asking once they have the results? 

Dr. Raajit Rampal:

What does it mean? That’s the most basic and fundamental question. It’s one thing to get a list of mutations. But the real bread and butter question is what does this mean to the disease and my prognosis and my treatment?  Those are the key questions.  

Katherine Banwell:

So, what are the types of treatments available for MPNs?  And let’s start with myelofibrosis or MF. 

Dr. Raajit Rampal:

If we had had this discussion five years ago, it would be pretty simple, and it would take a minute or two. And that’s completely changing and that’s amazing, and it’s good for all of our patients.  

Right now, for patients with MF, it depends on what the issue is. If the issue is symptoms or spleen, JAK inhibitors are our first line of therapy. Three approved JAK inhibitors are currently available, two on the first side ruxolitinib (Jakafi) and fedratinib (Inrebic). And pacritinib (Vonjo) can be used for patients with really low platelet counts.   

There is a fourth JAK inhibitor that we expect to be, hopefully, approved in June of this year, momelotinib. So, the landscape is about to complete broaden in terms of just JAK inhibitors.  

But beyond the JAK inhibitors themselves, there are a number of late stage clinical trials that are combining JAK inhibitors with agents that work through a different mechanism that don’t work through inhibition of the JAK pathway. So far, these drugs have all shown promise in early phase trials. Now, the definitive Phase III trials are being done. We have to wait and see what the data tells us. But if these are positive trials, this could completely alter the landscape of MPN. 

Katherine Banwell:

There’s also transplants available, right? 

Dr. Raajit Rampal:

Correct. Transplants for more advanced patients, which comes with some major risks. And so, that has to be thought of very carefully in terms of the risks and benefit. But it is a potentially curative strategy.  

Katherine Banwell:

Let’s turn to polycythemia vera or PV. What types of treatments are available? 

Dr. Raajit Rampal:

It’s really quite a range. So, there are things like phlebotomy and aspirin, which has been the mainstay of therapy for many years. There are drugs like hydroxyurea (Hydrea), interferons, JAK inhibitors. So, ruxolitinib is approved in certain settings for treating polycythemia vera. So, the landscape is broad. There are a lot of questions going on right now with polycythemia vera with regards to how it should best be treated. Is the mainstay of phlebotomy and aspirin really what we should be doing or should we be giving patients treatment earlier on. 

And there is some data to suggest that. There is this drug called ropeginterferon (Besremi) that’s FDA-approved for polycythemia, which was compared in the study to phlebotomy and aspirin.  

And at least the data suggests that there may be better control of the disease and less progression possibly, and it’s a small number of patients, by treating patients earlier. Whereas we would have just given phlebotomy and aspirin. So, it’s something to consider. There are drugs in clinical trials as well that look promising one of which is called rusfertide, which actually works by changing the way iron is used by the body. 

Iron is a key component to hemoglobin and it is, of course, a key component to polycythemia in the sense that we phlebotomize patients to make them iron deficient and that’s how we control the disease. But this is a pharmacological way to do that. So, that drug is now in Phase III trials. So, that may also alter the landscape of treatment of PV in the near future. 

Katherine Banwell:

Finally, how is essential thrombocythemia treated? 

Dr. Raajit Rampal:

So, in some cases, with absolutely nothing as we had talked about a moment ago. There is some thought that in really, really low-risk patients. Maybe you don’t need to do anything except observe them. Whereas most patients are on an aspirin. And beyond that, we have drugs like interferon, pegylated interferon, and hydroxyurea and anagrelide, all of which can be utilized. It’s not entirely clear if there is one distinct first line treatment that is the best but these drugs are all active. JAK inhibitors have been studied in this setting. And to date, the data hasn’t led to their approval but, certainly, people have studied it.  

Katherine Banwell:

Dr. Rampal, how can you tell if a treatment is effective? Are there signs that you look for? 

Dr. Raajit Rampal:

Well, I think it’s a couple of things.  

One, are we meeting the treatment goals in terms of are we controlling blood counts with ET or PV? That’s one of the first principles in management. And with regards to MF, the same thing. Are patients’ symptoms being controlled? Is the spleen being adequately controlled? And then, there’s the symptom burden because just because the blood counts are being controlled, patients may still have symptoms, in which case, they are not being adequately treated. And then, we have to do our best to try to find a treatment strategy that does control their blood counts but also does control their symptoms. 

So, there is the blood count perspective but there is the symptom perspective as well. 

Katherine Banwell:

How do you know when it’s time to change treatments? 

Dr. Raajit Rampal:

Well, I think really two things. One is if we aren’t meeting our goals like we just talked about. But the other aspect of that is if we are incurring toxicities that are just not tolerable to the patient and that’s a reason to change therapy always. 

Katherine Banwell:

Many patients, of course, worry about disease progression. Are there key predictors or tests for progression that patients should know about? 

Dr. Raajit Rampal:

This is a key area of investigation currently. I think one of the things that patients say to us so often when we meet them is what’s going to happen to me. And right now, we don’t have great prediction tools. We can say on a population level well, there is X percent of chance of progression at 15 years. That’s useful if you’re talking about a population. That’s not really useful if you’re talking to an individual. Because if I say to somebody there’s a 20 percent chance of your disease progressing to leukemia, it doesn’t really make a difference. That’s a meaningless statement because if you’re in the 20 percent who progress, it’s not a relevant statistic anymore.  

It’s sort of a binary thing. We’ve got to do better at developing this. This is something that the MPN Research Foundation is really heavily invested in in trying to identify predictive biomarkers. 

If we can do that, then perhaps what we can do is say to a patient this is really what we think your actual risk is. And then, the next step is asking the question if we intervene early, can we prevent that progression from occurring. So, that’s where I think we need to go. We aren’t there yet. 

Katherine Banwell:

What signs or symptoms do you look for that may indicate that the disease is progressing? 

Dr. Raajit Rampal:

The blood counts are often the canary in the coal mine regardless of the disease. They can tell us if ET or PV is progressing into MF or whether MF is progressing to more of a leukemic phase. Changes in symptoms sometimes can be a harbinger of disease progression. So, Patient 2, for example, is doing really well and now, he’s having drenching sweats and losing weight. So, those types of symptoms are a sign that physical findings is the size of the spleen if it’s increasing. 

All of those things together give us a hint about progression.  

Katherine Banwell:

Well, is there any way to prevent progression?  

Dr. Raajit Rampal:

That is the million dollar question. Again, that’s where we ultimately need to be. We want to be able to intervene to a point where patients don’t get that sick. It would be amazing if we’d come to the point where we can intervene early and nobody progresses to late stage MF. Nobody gets leukemia. And I think that’s a worthy goal. That’s not something that we should think is too lofty of a goal. That should be our ultimate goal here. And a number of groups are investigating this exact question. It’s complicated and it’s going to take time. But I think that’s a worthwhile investment. 

Katherine Banwell:

Let’s talk about MPN symptoms and treatment side effects. Here’s a question we received from a viewer before the program. How common is peripheral neuropathy in primary myelofibrosis? 

And what is the best treatment for it? 

Dr. Raajit Rampal:

Well, by itself, it’s not a very common symptom of MF by itself. Can it be a symptom? Sure. But there are also a number of things that can cause peripheral neuropathy. So, I’m not sure there’s a best treatment.   

But what needs to be done is a thorough investigation. There can be a number of causes. It could be nerve injury. It could be a deficiency in vitamins like B12. There are a lot of things that could cause it. So, that type of a symptom needs to be thought of in a broad way in terms of diagnosis.  

Katherine Banwell:

Jeff sent in this question. How could I manage the itching? Are there new treatments or strategies to live with itching? 

Dr. Raajit Rampal:

Very common thing. And it’s an interesting thing explaining to when we teach our trainees about this symptom, we have to impress on them the fact that itching is not the itching that everybody else experiences. 

This is a very profoundly different symptom. It’s debilitating for so many people. I have patients who go to the Emergency Room for that. That’s how terrible it could be. There are a lot of things that could be tried. JAK inhibitors, in my experience, work very well for itching but not in everybody. We use sometimes antihistamines that can work well. Sometimes, antidepressants can work well, not because they’re treating depression but because of other properties that they have. And sometimes, UV light therapy can be useful tool here, too. A lot of patients swear by it. 

 Katherine Banwell:

Another common side effect is fatigue. Do you have any advice for managing this symptom? 

Dr. Raajit Rampal:

Fatigue is the most common symptom across MPNs. And it is also one of the most difficult things to treat. Part of the issue is trying to figure out what does fatigue mean to the patient.  

When someone says they’re tired, does that mean they’re sleeping all of the time? Does that mean they don’t have get up and go? The first step is always understanding what does fatigue mean to the patient? And then, the second is trying to dissect that. In some cases, it’s related to anemia, in some cases, it’s not related to anemia and it’s just the disease itself.  

And in some cases, you have to think outside of the box about general medical issues like thyroid dysfunction that could be at play here. So, there isn’t one best fit. 

But the first test is always to dig deep. When someone says they have fatigue to dig deeper and try to figure out what is that really. 

Katherine Banwell: 

What other common symptoms do you hear about from patients? And what can be done about those?  

Dr. Raajit Rampal:

There are a lot of different things. It’s a spectrum. So, I think that itching and fatigue are very common. Feeling full early is, that’s a big thing, particularly in myelofibrosis patients.  

Bone pain, that’s another big one, particularly in myelofibrosis. There is not one therapy that is best for all. I think the JAK inhibitors, certainly, benefit many of these symptoms. But they don’t benefit everybody and not to the extent that makes it tolerable for everybody. So, often times, we struggle with this and try a lot of different things. But, again, I think one of the things to always remember is we don’t always want to say that this must be because of the MPN. Sometimes, symptom is arising because of another medical condition that’s going on concurrently. 

Katherine Banwell:

That’s good advice. Thank you. Let’s answer a few more audience questions we received. This one is from Calvin, “If your hematologist says you’re stable and responding well to Hydrea, should you still seek out a second opinion?” 

Dr. Raajit Rampal:

It’s never wrong to seek out a second opinion. I strongly believe that, especially when you’re dealing with a disease that’s rare like this. 

And even seeking out a second opinion, even if you’re under the care of an expert in the field is never a wrong thing. I think that no one person knows everything. And sometimes, people’s experience and perspective is different. So, I don’t think that’s a bad thing ever.  

Katherine Banwell:

As a follow-up to Calvin’s question, is it sufficient to just look at what the blood tests reveal? Or does having  bone marrow biopsy dictate what treatment you should follow? 

Dr. Raajit Rampal:

I think the bone marrow is important, particularly at initial diagnosis or when there is a change. The blood counts are the canary in the coal mine. So, they tell us is there something else going on that we’re not thinking about. And that’s when the bone marrow becomes important. So, I definitely think bone marrow is important at certain points in the disease.  

Katherine Banwell:

Sandra has this question, “Are there new treatments for polycythemia vera being researched beyond interferon?” 

Dr. Raajit Rampal:

Yeah. So, we talked about rusfertide as an example of this. And there are, certainly, other drugs that have been evaluated in this space. So, there is a lot of work going on for this disease, which is really encouraging. 

Katherine Banwell:

Carolyn sent in this question, “Is there a possibility of bone marrow fibrosis reversal in myelofibrosis without a stem cell transplant?” 

Dr. Raajit Rampal:

The answer is yes. So, even with JAK inhibitors, we see that about a third of patients will have a reduction in bone marrow fibrosis. And this is a key question being investigated with some of the newer therapies that are being introduced into the treatment of myelofibrosis. And, certainly, we’ve seen data to date that suggests that the fibrosis can be reduced if not potentially eliminated in some cases.  

Katherine Banwell:

Dr. Rampal, should all patients diagnosed with MPNs undergo molecular testing? 

Dr. Raajit Rampal:

I strongly believe that. I think that we’ve learned so much that these tests are prognostic value. 

And in some cases, it may suggest a slightly different diagnosis. I definitely think that should be the case. 

Katherine Banwell:

What should patients be asking once they have the results? 

Dr. Raajit Rampal:

What does it mean? That’s the most basic and fundamental question. It’s one thing to get a list of mutations. But the real bread and butter question is what does this mean to the disease and my prognosis and my treatment?  Those are the key questions. 

Katherine Banwell:

Andrew wants to know does Jakafi cause other mutations to develop? 

Dr. Raajit Rampal:

That’s a really good question. Right now, we don’t think the answer is necessarily yes. We have seen that in some patients where the disease has progressed on Jakafi, mutations have emerged. 

But the problem is that genetic testing has limits of detection. In other words, the mutation appears, it may not have just appeared or been caused by the drug but that it may have been below our limits of detection and actually grew while the patient was on therapy, which does not mean that the drug caused the mutation but that it was allowed to emerge during treatment with the specific drug. So, that is an area of investigation.  

Katherine Banwell:

Well, thank you, Dr. Rampal. And please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them answered on future webinars.  

You mentioned earlier clinical trials. And I’d like to dig a little bit deeper. Where do these fit into the treatment plan? 

Dr. Raajit Rampal:

I think they should always be considered. None of the therapies that we have do we consider curative. And in many cases, standard therapy is fine given a patient’s clinical situation. In a case where standard therapy is not working or where we think that a patient’s prognosis is particularly challenging, or if they have mutations that may confer resistance to current therapies. 

I think in those scenarios, a trial should always be considered. 

Katherine Banwell:

So, if a patient is interested in possibly participating in a clinical trial, what kinds of questions should they be asking their healthcare team? 

Dr. Raajit Rampal:

All of these trials are different. I think the first thing is to discuss what’s the risk, what’s the benefit of any given trial or drug. What stage and development is it? What’s the evidence to support it? And what can I expect from it?   

Katherine Banwell:

What about cost? 

Dr. Raajit Rampal:

So, trials, in general, have two components. One is what we call standard of care meaning that things we would do normally for in the course of a patient’s treatment would be billed to a patient’s insurance as if they weren’t on a trial. 

Almost all trials, the study drug or any tests that are being done specifically with regards to the study drug are all covered by whoever is sponsoring the trial.  

Katherine Banwell:

How do patients find out about where the clinical trials are taking place? 

Dr. Raajit Rampal:

Usually, their physician should either, if they’re in a specialized center, they’ll have access there. But if they’re interested in trials and they’re being seen, for example, by a physician in the community who doesn’t necessarily specialize, asking for a referral to a major center where that MPN expertise is not an unreasonable approach to that. There is also clinicaltrials.gov where patients can go look for ongoing trials for their particular diagnosis.  

Katherine Banwell:

So, if patients want to learn more about MPNs, what sort of resources would you recommend? 

Dr. Raajit Rampal:

The thing I always say to patients is the internet is a very dangerous place for a variety of reasons. We have to, I think, do a good job of communicating to patients what are the resources. And the ones that I always point patients to are, for example, the MPN Advocacy International, the MPN Research Foundation, The Leukemia & Lymphoma Society, and the American Cancer Society. Those are sources of information that are vetted by physicians. 

Some of that information is specifically for patients. Those, to me, are good sources for patients to read.  

Katherine Banwell:

Dr. Rampal, as we close out our conversation, I wanted to get your thoughts on where we stand with progress and MPN care. Are there advances in research and treatment that make you hopeful? 

Dr. Raajit Rampal:

Without a doubt. I think I’ve seen more progress in the last three years than I’ve seen in the last 10 years. And we have so many new drugs coming forward, new questions that we’re trying to answer, tough questions as you alluded to. The question about prognosis but also intervening early to prevent progression of disease. These are things that are difficult questions that we are trying to dig into now. So, I think we should be optimistic. We are seeing so many excellent developments. We’ll have to see how far they’re going to take us. I don’t think we know the answer to that. But this is an exciting time.  

Katherine Banwell:

Dr. Rampal, thank you so much for joining us. 

Dr. Raajit Rampal:

My pleasure.  

Katherine Banwell:

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 being with us today.