How Can You Thrive With an MPN? Advice for Navigating Care.

How Can You Thrive With an MPN? Advice for Navigating Care. from Patient Empowerment Network on Vimeo.

How can you thrive with an MPN? In this animated explainer video, an MPN specialist and myelofibrosis patient discuss how to make informed decisions about your care and live a full life with an MPN.

 

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Finding an MPN Treatment Approach That Is Right For You

How Can Patients Navigate Care and Thrive With an MPN?

How Can Patients Navigate Care and Thrive with an MPN?


Transcript:

Brian: 

Hi, I’m Brian. Nice to meet you! Many years ago, I was diagnosed with a condition called myelofibrosis. At first, it was a scary to learn that I had cancer, but once I found the right treatment option for me, I’ve been living a full life.  

Meet, Dr. Liu – my doctor. 

Dr. Liu: 

Hi! I’m Dr. Liu, and I’m a hematologist specializing in the care of people with myeloproliferative neoplasms or MPNs.   

MPNs are a group of blood cancers that are characterized by the bone marrow overproducing a certain type of cell. The three types of MPNs are essential thrombocythemia, or ET,  polycythemia vera or PV, and myelofibrosis, or MF. 

As Brian mentioned, with the right treatment, it is possible to live a full life and to thrive with an MPN. 

Brian: 

It’s so true. Navigating my care has been much easier because I partner with my healthcare team – participating in decisions makes me feel like an important member of the team. 

Dr. Liu: 

That’s right, Brian. When considering treatment, it’s important to weigh all of your options.  

While your healthcare team is the expert when it comes to the clinical side of your disease, you as the patient, are the expert on how treatment will impact YOU and your lifestyle.  

Brian: 

And as someone who knows my needs well, my wife is another key member of my team.  She comes with me to appointments and takes notes during visits, and when it is time to make decisions about my care, we both feel well-informed about the options. 

So, Dr. Liu – what factors should be considered when choosing an MPN treatment?  

Dr. Liu: 

Well, it’s important to note that everyone’s MPN is different so what may work for one person, may not work for another. In general, we consider certain factors,1 such as: 

  • The type of MPN, whether it is ET, PV, or MF. 
  • The patient’s age and overall health. 
  • Test results, including blood work or any genetic testing that has taken place. 
  • The symptom burden, which basically means how much the disease symptoms are interfering with a patient’s quality of life. 
  • Any pre-existing health issues. 
  • Finally, and most importantly, the patient’s preference.  

Brian: 

And I like to make informed decisions. So, when considering therapy, I also did some research on my own, and then discussed the information with my healthcare team. It helped my wife and me understand what we’d learned, and confirmed our decision. 

Dr. Liu, what sort of questions should patients ask their doctor when considering a treatment plan? 

Dr. Liu: 

Great question. When choosing therapy, patients should ask: 

  • How is the treatment administered, and how often will I need treatment? 
  • What are the potential side effects of the treatment? 
  • How will the effectiveness of the treatment be monitored? 
  • And, what are options if this treatment doesn’t work for me? 

Brian: 

That’s great advice. Once you’ve begun treatment, it’s important to continue to share how you are feeling with your healthcare team – be sure to mention any side effects or symptoms you may be having with your team. 

Dr. Liu: 

That’s right, Brian. If you speak up about what’s bothering you, we can usually find a way to manage the issue. 

It’s also important point to tell your doctor if you’ve missed a dose of your medication. Many of the newer MPN therapies are self-administered, and it’s important to let us know so we can adjust the plan if necessary. 

So, what steps should you take to thrive in your life with an MPN? 

Brian: 

  • First, understand and participate in treatment decisions. Be sure to share your personal preferences. 
  • Then, communicate regularly with your healthcare team – don’t wait to share information only when you have an appointment.  
  • And, utilize your whole team – nurses, nurse practitioners, and others, are all there to help you. 
  • Use your patient portal. You can view lab work and test results, or even use the messaging feature to communicate with your team. 
  • Bring a friend or loved one to appointments and always write down any questions or concerns in advance.  

Dr. Liu: 

And, most importantly, remember you are at the center of your care. Advocate for yourself! 

To learn more, visit powerfulpatients.org/MPN to access a library of tools. Thanks for joining us! 

How Are ET, PV and Myelofibrosis Monitored?

How Are ET, PV and Myelofibrosis Monitored? from Patient Empowerment Network on Vimeo.

MPN specialist and researcher Dr. Joseph Scandura reviews tools that are used to monitor patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF), including routine blood work and symptom management

Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.

 

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How to Treat PV-Related Itching


Transcript:

Katherine Banwell:

I would imagine monitoring patients is different for each of the MPNs. So, how are patients typically monitored over time, and let’s start with essential thrombocythemia?  

Dr. Scandura:

Yeah. I think – again, it’s similar. You know, what’s near-term, what’s long-term? And so, in all of these diseases, thrombosis risk is a near-term risk. That’s something that I am monitoring in certain ways to help mitigate that risk. In ET and PV, I approach them similarly. Blood counts are certainly – these are diseases of the blood forming system. Certainly, monitoring blood counts I find helpful. But the reality of it is, in ET, there is not a clear linkage between blood counts and risks.  

And so, I like to keep the platelet count near normal if I can. But I also recognize that it may not be worth suppressing all of the blood counts to achieve that landmark, because it’s not clear that that’s really reducing the risk any more than just having somebody on a medication that helps control the blood counts. In polycythemia vera, different blood counts are very important. The red blood cells are kind of like part of the clotting risk. We know from clinical trials that keeping the red blood cell parameters within certain ranges reduces the risk of clotting. And so, what I monitor in polycythemia vera is the hematocrit. In women, I like to keep it below 42. In men, I like to keep it below 45.  

But I don’t just – I’m not a slave to the hematocrit. I am keeping an eye on the other blood counts and the other red blood cell parameters. So, for instance, what’s the size of the red blood cells? That tells me a little bit about what’s going on in the blood formation for that patient. And what’s the number of red blood cells? So, sometimes people can have very small red cells, because they’re a little iron-deficient and have a huge surplus of the number of red blood cells. And that tells me a little bit about how their blood forming system is responding to therapy.  

Iron deficiency in polycythemia vera is very prominent. I personally believe it’s a very major driver of symptoms in patients who are receiving phlebotomy as part of their care. And it’s something that I monitor and really counsel patients on. My goal is to make phlebotomy independent, but it can take a while.  

Everybody starts out iron-deficient, and then we take iron out of their body through blood with the phlebotomy. And that makes them more iron-deficient.  

Katherine:

Right. 

Dr. Scandura:

I monitor symptoms from patients, and sometimes that can tell me that their disease needs to be – their treatment needs to be tweaked a little bit, even something as simple as aspirin. People can sometimes have burning in the skin or itching that is sometimes responsive to changing the aspirin dose or how it’s given, once a day versus twice a day.  

And that simple thing can be a big change for a patient who’s kind of, literally, climbing out of their skin or wishing they could and to try and find something that is helping.   

I had a patient the other day. He had COVID. I said, “Oh, you should probably get this medication.” Do you have your primary care physician? Who’s taking care of you?” And he goes, “Well, to be honest with you, you’re my guy.” And so, it’s true. I see this patient a lot. And so, sometimes they forget. If I’m not paying attention to their blood pressure, the risks or treatment of diabetes, cholesterol, lipids, their screening programs for mammogram or colonoscopy, health maintenance issues, I do keep an eye on that in patients, because I do think it’s a part of the MPNs.  

I think that there are excess risks for patients for some of these factors. Certainly, if you think of it as three strikes, they get a strike for having an MPN. I don’t want them to have any other strikes. So, diabetes, hypertension, those are strikes that I can potentially, at least, treat or refer them to somebody to help comanage with me. And so, that’s kind of my general approach. 

Katherine:

What about patients who have myelofibrosis? Are they monitored more closely? 

Dr. Scandura:

Yeah, I think it depends a little bit on the patient. Patients with early myelofibrosis often don’t have any symptoms or near-term risks much different than those from ET or PV. As the disease can progress, then some of these patients have more profound problems with symptoms, which I may be trying to find a solution to make them feel better. And also, blood counts can become more of an issue.  

Transfusions in some patients who are very high white blood cell count, the spleen is often quite enlarged. Although, in my experience, most patients aren’t really bothered by the size of their spleen as the physicians are. But it is something where I think, on average, they’re monitored a little bit more closely to quite a bit more closely depending on the patient. 

Katherine:

What happens if someone suddenly has a change in blood counts? What do you do? 

Dr. Scandura:

Yeah. I mean, repeat it. That’s the first thing. Also, check what’s going on. It’s not uncommon in patients with MPNs that I’ll see them and the counts are a little bit out of whack, the white count is much higher than it’s been, and questioning them. “Oh, yeah. I had X, Y, or Z last week or the week before.” It used to be a upper respiratory tract infection, or they had a minor surgical procedure.  

And sometimes the responses to these things can be accentuated in patients with MPNs. And so, if that’s what of this story, I certainly would repeat it and let things calm down a little. And that’s often all it is. I’m much more of a monitor of the trends. So, one-time measure doesn’t generally excite me. It might make me want to have a follow-up a little more – in a shorter period of time. Of course, it depends on what the change is. But, for most of the changes that we observe, they’re relatively minor. And I will monitor them over time.  

If I see a trend where something is progressively increasing or decreasing over time, then I start thinking about what else is going on. And that’s always in the context of what’s going on with the patient. How are they feeling? What’s their physical exam like? What are the other laboratory values like?  

Katherine:

When is a bone marrow biopsy necessary? 

Dr. Scandura:

I would say a bone marrow biopsy is absolutely necessary at the time of diagnosis. I personally do not routinely monitor by bone marrow biopsy unless it’s part of a clinical trial.  

But I do perform a bone marrow or want to look at the bone marrow morphology if there is one of these changes or at least a trend that I want a little bit more information about. And so, if – or if it’s been a very long time since somebody has had a bone marrow. If it’s been five or ten years, then sometimes I may recommend we look just so we can collect a little bit more up-to-date information.  

But I don’t routinely do a bone marrow, but I will do it if there are laboratories that are kind of trending in the wrong direction, there’s symptoms, there’s physical findings that I’m just not sure about. And I think it would help me be more sure as to what’s going on and be able to discuss that with the patient. Sometimes, just to say, “Hey. Look, we were worried about this, but the bone marrow looks really good.”  

Thriving With an MPN | Tips for Managing Worry and Anxiety

Thriving With an MPN | Tips for Managing Worry and Anxiety  from Patient Empowerment Network on Vimeo.

Dr. Joseph Scandura explains the role of shared decision-making when deciding on an MPN treatment, and why it’s so important for patients to take an active role in their care.

Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.

 

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Advice for Choosing MPN Therapy: What’s Right for You?


Transcript:

Katherine Banwell:

Can you talk about shared decision-making? Why is it so important for patients to work closely with their healthcare team on choosing a therapy? 

Dr. Scandura:

Because these are therapies that last for a long time. And, hopefully, the patients and the relationship last for a long time. And so, I think that everybody has to be comfortable with the decision about a therapy. And my personal goal is to try to make sure that everybody understands the rationale for a therapy, the potential ups and downs with the therapy, which every drug has, every approach has, and what I’m kind of watching and monitoring. I’m a very – I think that communication relieves a lot of anxiety. I think that the unknown is far scarier than the known, even if it’s not perfect. And so, I think shared decision-making has a role in relieving some of the scariness of unknown.  

If we’re discussing to come to a decision, that means that my job is to give you the knowledge that I have so that you can tell me the knowledge about you and what you’re feeling and what you want back. And that back and forth is what helps me do a better job of taking care of the patient and helps the patient understand what’s going on and relieve some of the stress of the unknown. So, I think it’s a very synergistic approach. I don’t think I could practice medicine in another way.  

Katherine:

Managing the worry associated with a diagnosis or concerns even about progression can lead to a lot of anxiety and fear amongst patients. Why is it important for them to share what they’re feeling with their healthcare team? 

Dr. Scandura:

I would say this. If our goals are to have people – I mean, this is what I say to patients – I want you to think about this disease when you’re here. And, then, when you’re not here, my goal is to have you not thinking about this disease because you’re feeling okay and you’re comfortable and confident in what’s going on.  

So, I want to make it a clinic visit disease. That’s not always possible. But, for many patients, it is. I don’t want somebody to become – to start thinking like a sick person when they’re not. I don’t want the diagnosis to be the disease, right? I want the person if they’re feeling well, to recognize that. Live your life; move on with things. But, at the same time, these kinds of diagnoses are scary.  

Katherine:

Yeah. 

Dr. Scandura:

And so, it is normal with a new diagnosis or a change in the diagnosis to go through a period of time where you have to adjust. And so, that’s normal, and you have to work your way through it. Some people want to work that all out internally, and that’s good to a certain extent as long as they have good supports at home. But I often want to know how they’re doing, how they’re working through that so I can get a gauge of how it’s affecting their life and the duration where this adjustment is going on.  

So, somebody who’s still adjusting to a new diagnosis two years after the diagnosis, and they’re otherwise clinically well, that’s getting into the range where it’s not normal. You might need additional help. You might need counseling. And, in some patients, that might include some medications for a short period of time. The goal is to have the disease affecting you only in so far as it’s affecting you, not the idea of the disease. 

So, that’s a – again, it’s a conversation. There are lots of resources. People, being individuals, deal with things in their own way, and I just try to help understand with them how it’s affecting their life. And, if it seems to be more than I would expect, I’ll tell them that.  

And then we can discuss that. It doesn’t mean we have to do something today, but I will tell them, “I think this is maybe a little bit more. Why are you so worried? I think you’re doing great.” 

Katherine:

Yeah. Yeah. Can a social worker or somebody else on the healthcare team help with these emotional needs that patients have? 

Dr. Scandura:

Absolutely. We have great social workers. I tap into them all the time. We also have a group of psychiatrists who are really interested in kind of psychiatry that’s related to oncology and the diagnoses and how it impacts care. I mean, this is New York City, so everybody has a therapist. But a lot of patients have preexisting connections to healthcare providers or support systems. I think, for some patients, groups are helpful.  

MPNs and Pregnancy: Why Close Monitoring Is Important

MPNs and Pregnancy: Why Close Monitoring Is Important  from Patient Empowerment Network on Vimeo.

What complications can arise from an MPN during pregnancy? Dr. Joseph Scandura, from Weill Cornell Medicine, explains how pregnant women are monitored during pregnancy and in the postpartum period. 

Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.

 

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How Should You Participate in MPN Care and Treatment Decisions?


Transcript:

Katherine Banwell:

“What complications can arise from an MPN during pregnancy?” 

Dr. Scandura:

Well, look, pregnancy – here you have two things, one of them common and complicated and the other one uncommon and complicated. So, common is pregnancy, but every pregnancy is different. And there’s a lot of changes going on in the body, and there’s certain risks that can go along with that as well. So, clotting risks sometimes can be increased in pregnancy. And then you have an MPN, where you have a clotting risk on top of that. The pregnancy really changes what kinds of medications we can think about using. And so, there are certain medications that we use comfortably in patients that would be an absolutely forbidden medication in a pregnant woman.  

And so, it depends a little bit on what’s going on with the patient. But, if they have a history of clotting, then certainly, we would think about wanting to control the blood counts. It depends a little bit on what the disease is how we would do that. Interferons are commonly used in pregnancy, and they are safe in pregnancy and can improve the outcomes in some patients with pregnancy.  

But short of that, in patients, for instance, who are very thrombotic risk, sometimes we have to sort of balance the risk of having a clot and something that can interfere with the pregnancy and the risk of bleeding. So, it’s not uncommon that people are on blood thinners during pregnancy at some point, but it really depends on the individual patient. What we do here is we keep very close contact with the patients.  

And all of our patients are seen by the high-risk OB/GYN. So, it’s not the general obstetrics people who are monitoring the patient, so they’re much more closely monitored for complications of pregnancy. And we are seeing them more frequently during pregnancy to help, from the MPN side, to try to optimize and minimize the risks of clot. And that doesn’t end as soon as the baby’s out. If breastfeeding, their clotting risk is not normalized after pregnancy, as soon as the baby comes out. And so, you know, there’s an adjustment for several months afterwards where we’re still kind  of thinking about this person a little bit differently than we would if they were not or had not been recently pregnant. 

What Are Indicators of MPN Progression?

What Are Indicators of MPN Progression?  from Patient Empowerment Network on Vimeo.

What are signs that essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF) may be progressing? Dr. Jeanne Palmer, of the Mayo Clinic in Arizona, reviews factors that may indicate progression and discusses how blood counts are used in disease monitoring.

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

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Understanding Treatment Options for ET, PV, and Myelofibrosis

Understanding Treatment Options for ET, PV, and Myelofibrosis


Transcript:

Katherine Banwell:

Okay. Katie had this question. “What are the signs of progression from PV to MF or AML, both clinically and in blood tests, and when do you need a new bone marrow biopsy to check for this happening?” 

Dr. Jeanne Palmer:

So, in terms of progression, there are several things that we see happen. 

I think most importantly is, let’s say you have PV, and you’ve always been on medication, and it’s been hard to control. And all of a sudden, you don’t need medication to control it anymore, or the same thing for essential thrombocythemia. You have been taking medication, and all of a sudden your platelets go down, and you don’t need to take drugs anymore. A lot of times people are like, “Oh, that means I’m fixed and I’m well,” not necessarily, you really need to make sure to talk to your healthcare provider and potentially get a bone marrow biopsy. 

Now, the other thing – sometimes the blood counts will actually drop too low, so you’ll have somebody who has PV, who has always been too high and then all of the sudden they come in, and their hemoglobin is very low, and they’re anemic, and that’s another situation where you do that. So, anytime the blood counts start to drop is concerning. 

Now, it’s a continuum, so the blood counts may drop as you’re at the point of transitioning but it doesn’t – it’s not like if your blood count is dropping you say, “Oh my God, I have myelofibrosis, I need a bone marrow transplant tomorrow.” That’s not necessarily the case. This is generally a transition type process. 

Also when the spleen starts to get enlarged. Now, the spleen can be enlarged even in the setting of just ET or just PV, so spleen enlargement does not necessarily mean you’re transforming, but it can be one of the things that we would see that would indicate that. 

Katherine Banwell:

Okay. 

Dr. Jeanne Palmer:

And then finally white blood cell count increasing can often be a sign of that. Now, in terms of progression to AML, that is generally something we’ll see in the blood. AML or acute myeloid leukemia, is indicated by the presence of blasts at greater than 20 percent. Now, many patients with myelofibrosis, in particular, but even PV and ET, may have blasts in their peripheral blood. Blasts are normal. If I did a marrow on every healthy person out there, they are going to have some blasts, because these are the first part of the development of white blood cells. So, they’re like baby white blood cells. But what the problem is, is when they start to grow too much. 

And so in the setting of myelofibrosis and even sometimes with these other diseases, the blasts will be in the peripheral blood primarily because the bone marrow is damaged and doesn’t hold them in very well. It becomes AML when it gets greater than 20 percent, so that blasts of greater than 20 percent in the peripheral blood or in the bone marrow but a lot of times we find it in the peripheral blood is where we indicate this has progressed to AML. 

Katherine Banwell:

Yeah.  

Dr. Jeanne Palmer:

Blasts of greater than 10 percent are also something that we really want to pay attention to, because that would suggest that the disease is starting to become more aggressive. Now, blasts vary, so for example, I’ve had patients go up to 11 and then drop back down to 3 or 4, and then they say around 3 or 4 or 5. So, you always want to make sure to double-check because one blast count at 11 percent, whereas it’s very important to address, may not necessarily reflect that you need to change in treatment at that time. Again, these blood tests, I always tell people, do not freak out over one blood test. 

Make sure you get at least a couple of them to really confirm what you are looking at. 

How to Treat PV-Related Itching

How to Treat PV-Related Itching  from Patient Empowerment Network on Vimeo.

Dr. Jeanne Palmer, from the Mayo Clinic in Arizona, explains pruritis, which is the itching related to polycythemia vera (PV), and discusses strategies for managing this MPN symptom.

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

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Understanding Treatment Options for ET, PV, and Myelofibrosis


Transcript:

Katherine Banwell:

We received some audience questions prior to the program today. This one is from Jacqueline, “What can I do to minimize pruritus or itching due to PV? A typical histamine blocker like Claritin or Zyrtec has done nothing whatsoever.” 

Dr. Jeanne Palmer:

Yeah. Unfortunately, the itching of this is not as much mediated by an allergic type reaction or histamine. It’s a lot related to that microvasculature, those tiny little blood vessels. Things like avoiding hot showers, as we talked about, taking cooler showers or not even taking showers, just like cleaning yourself with a washcloth can be helpful. There are certain medications that we can use sometimes that help. 

Now, first of all, Jakafi is extremely effective for itching. Of course, it does have side effects. It’s not always approved for your disease, so for example, it’s not approved for essential thrombocythemia. But JAK inhibitors can be helpful in that setting. There are also medications like gabapentin, which is a medicine that we use to treat peripheral neuropathy and that can actually be helpful because actually the itching, a lot of it is related to nerves not functioning right, so gabapentin can be helpful. 

And a really old-school medicine that I sometimes use, especially if the itching is most prevalent at night, is a drug called doxepin, and that’s been around for a very long time, but it can be extremely sedating and has to be used with caution, especially in patients who are older. 

How Can Patients Navigate Care and Thrive With an MPN?

How Can Patients Navigate Care and Thrive With an MPN?  from Patient Empowerment Network on Vimeo.

What does it mean to thrive with an MPN? Dr. Jeanne Palmer, an MPN specialist from the Mayo Clinic, shares advice on navigating MPN care and stresses the importance of communicating openly with your healthcare team.

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

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Understanding Treatment Options for ET, PV, and Myelofibrosis

Understanding Treatment Options for ET, PV, and Myelofibrosis

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How Should You Participate in MPN Care and Treatment Decisions?

How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions


Transcript:

Katherine Banwell:

What does it mean to you to thrive with an MPN?

Dr. Jeanne Palmer:

I think living with an MPN can be very difficult. I think there is a number of things. First of all, there’s always the worry of what’s going to happen in the future. Many of these MPNs can start as fairly, for lack of a better term, as benign issues and can convert to something much more serious. So, I think living with that sort of timebomb in the back it can be extremely stressful. So, figuring out how to live with the fact that there is some degree of uncertainty.

I think the other thing is making sure to understand your disease. These are very rare disorders and even if you go to a hematologist-oncologist specialist, a lot of times they don’t have all the information because they don’t see a lot of them every year. So, it’s really important to make sure that above and beyond that you understand what’s going on in your body so that when new things happen, new symptoms happen, you’re able to really address them as opposed to sort of living with something that may make you feel poorly that’s not being addressed.

So, again, I think the biggest piece of this is seeing how do you live with uncertainty and how do you make sure you understand your disease well enough that you know what’s going on in your own body. 

Katherine Banwell:

Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions. Why do you think it’s important for patients to speak up when it comes to symptoms and side effects?

Dr. Jeanne Palmer:

Well, there is a lot of things. This is a disease, again, that we can direct our therapy many times towards symptoms, and so when we think about how do I direct my therapy, so how do I treat somebody, symptoms are an incredibly important part of it. And there is nothing worse than having a patient come and see me who I see every six months, because they’ve been pretty stable and they’re like, “Oh, for three months I’ve been feeling awful.” And you’re like, well, “Why didn’t you let me know, we could do something about this?”

So, if there is something that doesn’t feel right, it’s very, very important to talk to your healthcare provider. I would much rather be bothered and handle something earlier on than miss something and really have a lot more catch-up to do afterwards.

The other thing is symptoms may indicate a blood clotting event. We know that patients will have a higher risk of blood clotting. These are extremely important to identify early on because if they go unchecked, they can cause more damage. 

Katherine Banwell:

Dr. Palmer, was we close out this conversation I wanted to get your thoughts on where we stand with progress in helping people live longer and truly thrive with MPN. What would you like to leave the audience with?

Dr. Jeanne Palmer:

So, I think that the first thing is make sure you understand your disease. Don’t hesitate to ask for a second opinion. It’s always good to make sure you talk to someone who can really explain so you feel like when you go home you understand what’s going on in your body. Make sure you understand what symptoms to look for, what things to be aware of, because a lot of times people come in and they have no idea that, oh, these symptoms are actually related to their disease.

The other thing to make sure is that you’re very honest with your provider on how you’re feeling. A lot of times people come in and they say, “Oh, how are you feeling?” “I feel fine,” but then they start to ask very specific questions and they’re like, “Oh yeah, I’m really tired, my fatigue is an 8 out of 10,” or something.

So, make sure you’re really honest with your provider. When they ask you how they’re doing, this is not a social visit, this is a visit where they need to know your symptoms, so you don’t need to say I’m fine like you normally would if you were walking down the street.

The next thing is to always make sure to know where there’s clinical trials because we are making enormous great leaps and bounds in this field. It’s a really exciting time for myeloproliferative diseases, and there’s a number of new drugs that are being tested and coming out. So, it’s always important, if the opportunity is available and you can do it, clinical trials are a great way to get treatment.

Plus, you are giving back, because these are things that help us learn whether something works or not. So, you’re not as much a guinea pig, you never get a sugar pill. It’s one of those things will you will always get the treatment you need and then they may add something to it or you may be in the situation where there is no treatment, so they try something.

But clinical trials, I have to emphasize, are a great way to get therapy and really are how we know everything that we know about treatment for these diseases.

Katherine Banwell:

Yeah. It sounds like there’s a lot of progress and hope in the field.

Dr. Jeanne Palmer:

Oh, absolutely

What Are Common MPN Symptoms?

What Are Common MPN Symptoms? from Patient Empowerment Network on Vimeo.

Dr. Jeanne Palmer, an MPN specialist, reviews the most common symptoms associated with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF).

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

Related Programs:

 
How to Treat PV-Related Itching

How to Treat PV-Related Itching

How Can Patients Navigate Care and Thrive With an MPN?

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Thriving with an MPN What You Should Know About Care and Treatment

Thriving with an MPN | What You Should Know About Care and Treatment


Transcript:

Katherine Banwell:

Would you walk us through the common symptoms of each of the MPNs? Let’s start with essential thrombocythemia. 

Dr. Jeanne Palmer:

Right. So, there are a number of shared symptoms throughout all the diseases and when we start to figure out how to categorize them, they call into several different categories. The first one is inflammation-related symptoms. We know that the inherent pathway that’s dysregulated or that causes these diseases to happen can also result in significant inflammation in a person, that can result in things like fevers, night sweats, weight loss, and overall feeling really fatigued and poorly, which is something that it seems to be much more prevalent in patients with MPNs, all sorts of them, actually. 

The next set of symptoms is related to microvasculature, so all the little blood vessels. And sometimes we think, oh, maybe that’s because there’s too many red blood cells or platelets and the blood become viscous. It’s probably more related to the actual dysregulation of that JAK2 pathway, which is inherent to all the myeloproliferative diseases and as a result, the little blood vessels can clamp down and that can give people headaches, visual changes, numbness and tingling in the hands and feet, and even can cause sort of a painful rash called erythromelalgia in the body. 

So, these are things that can happen that are probably less appreciated side effects of the disease. And finally, there’s spleen-related symptoms. The spleen is in the left upper quadrant of the abdomen and it’s an organ that generally is about 12 centimeters in length, 10 to 12, but in patients with myeloproliferative diseases it can be enlarged. And as a result of an enlarged spleen people can have feeling like they get fuller early. So, if you’re eating a meal, all of the sudden you can only eat half of that meal versus the whole meal. 

Discomfort or pain in the left upper quadrant. Sometimes it’s much more noticeable when you like bend over to tie your shoes. And then sometimes people can actually, when the spleen gets really big, the blood flow can be impaired towards the end of it which can cause some of the spleen tissue to die, and that can be painful. So, these are things that if somebody does start to notice that they’re having fullness in the left upper quadrant, pain, stuff like that, that that may be related to spleen symptoms.  

Katherine Banwell:

What about PV or polycythemia vera, what are the symptoms? 

Dr. Jeanne Palmer:

So, all of these sorts of relate to all of the myeloproliferative diseases. So, one other one that I didn’t mention, and this is actually more in PV than others, is itching. Itching can be absolutely unbearable when somebody has PV. It’s particularly noticeable after taking a shower. So, a lot of times I’ve met patients who are like I haven’t been able to take a shower in years, because it causes such a high degree of itching. 

Katherine Banwell:

Why a shower? Is it different from having a bath?  

Dr. Jeanne Palmer:

Water on the body that can cause the problem. So, if people take hot showers, it’s even worse. Although I think that people sort of react to it differently. Usually what patients end up doing is more like sponge bath type of things, rather than actually being exposed to the water. 

 Taking colder showers or cooler showers can sometimes help mitigate that. But the itching, and even in the absence of a shower, people can have pretty severe itching, and that can also be one of the major side effects. 

Thriving With MPNs: Your Role in Managing Your Treatment and Care

Thriving With MPNs: Your Role in Managing Your Treatment and Care from Patient Empowerment Network on Vimeo.

 How can patients thrive with a myeloproliferative neoplasm (MPN)? Dr. Jeanne Palmer discusses treatment approaches, strategies for managing disease symptoms and treatment side effects, and advice on how patients can be proactive in their care.

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

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Expert Perspective: Hopeful MPN Research and Development


Transcript:

Katherine Banwell:

Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to talk about how to live and thrive with an MPN. We’re going to discuss MPN treatment goals and how you can play an active role in your care. 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. 

Well, joining us today is Dr. Jeanne Palmer. Dr. Palmer, welcome. Would you please introduce yourself? 

Dr. Jeanne Palmer:

Thank you so much. I am so happy to be here and to help participate in this. My name is Jeanne Palmer. I am a hematologist at Mayo Clinic in Arizona. I specialize in MPNs as well as bone marrow transplant, and I am thrilled to be here. 

Katherine Banwell:

Thank you for taking the time out of your busy schedule to join us today, Dr. Palmer. We start all of the webinars in our Thrive series with the same question and that is, what does it mean to you to thrive with an MPN? 

Dr. Jeanne Palmer:

I think living with an MPN can be very difficult. I think there is a number of things. First of all, there’s always the worry of what’s going to happen in the future. Many of these MPNs can start as fairly, for lack of a better term, as benign issues and can convert to something much more serious. So, I think living with that sort of timebomb in the back it can be extremely stressful. So, figuring out how to live with the fact that there is some degree of uncertainty. 

I think the other thing is making sure to understand your disease. These are very rare disorders and even if you go to a hematologist-oncologist specialist, a lot of times they don’t have all the information because they don’t see a lot of them every year. So, it’s really important to make sure that above and beyond that you understand what’s going on in your body so that when new things happen, new symptoms happen, you’re able to really address them as opposed to sort of living with something that may make you feel poorly that’s not being addressed.  

So, again, I think the biggest piece of this is seeing how do you live with uncertainty, and how do you make sure you understand your disease well enough that you know what’s going on in your own body. 

Katherine Banwell:

Yeah. That’s helpful to understand, especially as we move through today’s program, and we’re going to cover the three classic MPNs, polycythemia vera, essential thrombocythemia and myelofibrosis. One part of thriving with an MPN is managing the symptoms of the disease. Would you walk us through the common symptoms of each of the MPNs? Let’s start with essential thrombocythemia. 

Dr. Jeanne Palmer:

Right. So, there are a number of shared symptoms throughout all the diseases and when we start to figure out how to categorize them, they call into several different categories. The first one is inflammation-related symptoms. We know that the inherent pathway that’s dysregulated or that causes these diseases to happen can also result in significant inflammation in a person, that can result in things like fevers, night sweats, weight loss, and overall feeling really fatigued and poorly, which is something that it seems to be much more prevalent in patients with MPNs, all sorts of them, actually. 

The next set of symptoms is related to microvasculature, so all the little blood vessels. And sometimes we think, oh, maybe that’s because there’s too many red blood cells or platelets and the blood become viscous. It’s probably more related to the actual dysregulation of that JAK2 pathway, which is inherent to all the myeloproliferative diseases and as a result, the little blood vessels can clamp down and that can give people headaches, visual changes, numbness and tingling in the hands and feet, and even can cause sort of a painful rash called erythromelalgia in the body. 

So, these are things that can happen that are probably less appreciated side effects of the disease. And finally, there’s spleen-related symptoms. The spleen is in the left upper quadrant of the abdomen and it’s an organ that generally is about 12 centimeters in length, 10 to 12, but in patients with myeloproliferative diseases it can be enlarged. And as a result of an enlarged spleen people can have feeling like they get fuller early. So, if you’re eating a meal, all of the sudden you can only eat half of that meal versus the whole meal.  

Discomfort or pain in the left upper quadrant. Sometimes it’s much more noticeable when you like bend over to tie your shoes. And then sometimes people can actually, when the spleen gets really big, the blood flow can be impaired towards the end of it which can cause some of the spleen tissue to die, and that can be painful. So, these are things that if somebody does start to notice that they’re having fullness in the left upper quadrant, pain, stuff like that, that that may be related to spleen symptoms. 

Katherine Banwell:

What about PV or polycythemia vera, what are the symptoms? 

Dr. Jeanne Palmer:

So, all of these sorts of relate to all of the myeloproliferative diseases. So, one other one that I didn’t mention, and this is actually more in PV than others, is itching. Itching can be absolutely unbearable when somebody has PV. It’s particularly noticeable after taking a shower. So, a lot of times I’ve met patients who are like I haven’t been able to take a shower in years, because it causes such a high degree of itching. 

Katherine Banwell:

Why a shower? Is it different from having a bath?  

Dr. Jeanne Palmer:

Water on the body that can cause the problem. So, if people take hot showers, it’s even worse. Although I think that people sort of react to it differently. Usually what patients end up doing is more like sponge bath type of things, rather than actually being exposed to the water. 

Taking colder showers or cooler showers can sometimes help mitigate that. But the itching, and even in the absence of a shower, people can have pretty severe itching, and that can also be one of the major side effects. 

Katherine Banwell:

Much of the time the chosen treatment for MPNs manages the symptoms of the condition. I’d like to review the different types and classes of treatment for the three MPNs. So, let’s start with essential thrombocythemia again. When is it time to treat, and what are the options available?  

Dr. Jeanne Palmer:

Right. So, with essential thrombocythemia, that’s the disease that sometimes we don’t need to treat. 

So, we basically have a risk stratification system and this risk is based on age, history of a blood clot, the presence or absence of a JAK2 mutation. So, for example, if somebody is 28, does not have a JAK2 mutation, which is again one of those driver mutations, and never had a blood clot, they actually don’t necessarily need to do anything and just be monitored.  

Somebody who is less than 60 and has a JAK2 mutation or who is greater than 60 and does not have a JAK2 mutation, in that setting, a lot of times you can use aspirin. Now, it gets a little bit gray in terms of that over 60 without the JAK2 mutation with regards to whether at that point you really should start taking some medicine to lower the platelets. 

Now, if somebody has a JAK2 mutation, is greater than 60 or has had a blood clot, hands down they need to take medicine to lower the platelets, in addition to aspirin or whatever blood thinner they may need. So, for example, if you have a blood clot in a vein, a lot of times you need to take a blood thinner and that will be a lifelong thing. And again, we do these risk stratifications because we know there is a certain risk of clotting associated with the risk of essential thrombocythemia. 

So, for example, somebody who is less than 60 and does not have a JAK2 mutation, never had a clot, their risk of clotting is probably very close to that of the normal population. Whereas if you’re higher risk and have a JAK2 mutation and greater than 60 or have had a history of a clot, the risk of clot is probably about 4 percent per year. So, this is something that can vary quite widely, and even though that 4 percent per year on the short-term doesn’t sound like a lot, if you take it additive over years, that’s why we generally try to be aggressive about lowering the platelets.  

In lowering the platelets, the goal is to get less than 400 and doing that can be done through several different medications. The most commonly used medications is a drug called hydroxyurea, which has been around for a number of years, and a drug called anagrelide which is probably a little less commonly used, because it has some more GI side effects and headaches associated with it. 

In some cases, especially in younger patients with this disease, we can consider using interferon, which is an injection of a cytokine, which are one of the chemicals that regulates the immune system within the body. But this interferon can actually help lower the platelets and there is a question of whether it may affect the biology of the disease as well. 

Katherine Banwell:

Let’s turn to polycythemia vera or PV, what are the different options available for treating it? 

Dr. Jeanne Palmer:

So, for polycythemia vera, everyone needs to be on aspirin. 

And additionally, everyone needs to make sure to keep their blood count low, to manage their hematocrit, which is one of the measures of red blood cells. So, in men it’s generally recommended to keep below 45 and in women it’s recommended to keep below 42 percent.  Now, the studied number was 45 percent and that was a study that was done, I don’t know, it was probably about 10 plus years ago, that actually showed that by keeping the blood hematocrit less than 45 percent you reduce the risk of having negative events like cardiovascular events and heart attacks. Because women tend to run with a lower blood count than men, it’s been extrapolated that 42 percent should be the number used for women. 

Now, this can be done by phlebotomy, which essentially is bloodletting. It’s kind of like donating blood except for that the blood unfortunately can’t be donated to anybody, it has to be discarded. But the phlebotomy is one way to do that, and the reason that works is because it makes somebody iron deficient. So, whereas if this is normal, if you’re iron deficient you become anemic. If your baseline hematocrit is here, making you iron deficient brings you back to normal. So, even though we always associate iron deficiency with anemia, iron deficiency in the setting of polycythemia vera is actually kind of a treatment of sorts. 

Now, once somebody gets above 60 and 60 seems to be sort of the magic age in these diseases, once somebody gets above 60, it is recommended that cytoreductive therapy is used, which means therapy or treatment that will bring down the red count. And again, for this one, hydroxyurea is an option as well as interferon. And there is recently an approval, actually FDA approval for a newer interferon called ropeginterferon or Besremi, which can help just bring down the red blood cells but it is the first interferon that’s actually been FDA approved for this indication. 

Katherine Banwell:

Are JAK inhibitors used as well? 

Dr. Jeanne Palmer:

They are. So, if somebody doesn’t respond well to hydroxyurea, the approval for ruxolitinib is actually for patients who have failed hydroxyurea. Although it’s something that we often consider especially in people who have a lot of symptoms. So, the itching, one of the things that can really help itching actually is Jakafi. If people have night sweats, they have weight loss, spleen related symptoms, those are the patients that will benefit from Jakafi. Additionally, if they are on hydroxyurea and can’t seem to get control of their blood count, Jakafi is a good option to help control the blood counts as well. 

Interferon is a very nice option because there’s great data that shows that you may actually be able to lower the percentage of JAK2 burden. 

So, we’d look at something called an allele burden, which is the percentage of cells that are involved – have the JAK2 mutation. Now, we don’t know whether lowering this percentage necessarily translates to long-term better survival, but I think there is enough data out there, and there is a good biologic underpinning for saying that this actually can help. But yes, Jakafi is another thing. 

And the really exciting thing is that there is a newer agent called rusfertide, which is a hepcidin mimetic, which is basically taking a protein in your body that helps metabolize iron and by making it externally and giving it to somebody that it can actually help bring down the hematocrit without having some of the other side effects we know with some of the other medications. That is currently in Phase III studies, so hopefully in the next couple of years we’ll see approval for that. 

Katherine Banwell:

Oh, that’s great news. And finally, how is myelofibrosis treated? 

Dr. Jeanne Palmer:

So, myelofibrosis is a little bit of a different animal. When you have something like essential thrombocythemia or PV, a lot of this is managing symptoms, preventing blood clots, but if you do appropriate treatment and management of these diseases you could probably live close to a normal life expectancy.  

So, I never typically pin a survival on it. With myelofibrosis, it’s a little bit different because there is a survival. Instead of saying you can live close to normal life expectancy, it backs up to saying how many years do I think you can live with this disease. Now, of course, we are horrible at predicting how many years anyone can live, so we have to take that all with a grain of salt. But we can at least sort of risk stratify people. 

And the first thing that’s really important is to figure out whether somebody is a transplant candidate or not and if, based on age, disease risk features, stuff like that, or whether we think they ever will be a transplant candidate. So, that kind of helps us sort of think about what your path moving forward is.  

Now, the current FDA-approved treatment for myelofibrosis, there are three JAK inhibitors approved, which is like Jakafi, which was the first approved one but there is also Inrebic or fedratinib and Vonjo or pacritinib and these have all been approved over the years. 

The role of JAK inhibitors and treatment of myelofibrosis is symptoms-based. So, for example, a lot of patients with myelofibrosis will have weight loss, night sweats, big spleens, really feeling fatigued and poorly and in this setting, the JAK inhibitor can be very helpful. And you don’t have to have a JAK2 mutation, a lot of times people say, well, I don’t have the JAK2 mutation so how can a JAK inhibitor help. So, the JAK inhibitor works on this pathway, which is called the JAK/STAT pathway, irrespective of mutation. 

So, if you are having symptoms and you have myelofibrosis, JAK mutation, excuse me, the JAK2 mutation does not predict who is going to have a response. And people who, regardless of which mutation you have, may actually benefit from it. 

So, the JAK inhibitors, though, are extremely effective at reducing symptom burden as well as reducing the spleen size. And we know that if a spleen is big and we can make it shrink that, that probably is a surrogate marker for living longer, and I think it’s because inflammation does a lot of wear and tear on the body. So if you can reduce the inflammation and the spleen shrinks, which generally go hand in hand, then you might help somebody live longer. It is not changing the biology of the disease, though, however, it doesn’t change the pathway and that this disease is kind of projecting ahead in terms of creating – it changes, as it goes along, may acquire new mutations or something like that which makes the disease become more serious. 

Right now, the approved therapies for it are JAK inhibitors and the Jakafi, ruxolitinib was the first one approved. Inrebic was approved several years back, or fedratinib. 

And then the most recent one that was approved is Vonjo or pacritinib and that’s a drug that is a JAK inhibitor that is actually very good for people with low platelets. The reason I bring that up is because if we think of what’s the biggest limiter of JAK inhibitors, JAK inhibitors bring down red blood cells, and they bring down platelets. So, when somebody has low platelets it’s very hard to use a JAK inhibitor, because we’re not really able to increase the dose well enough to get that inflammatory reduction because of the fact that the blood counts will drop too low. 

So, now drugs like Vonjo exist which, due to several other mechanisms associated with the drug are actually much more tolerated in somebody with low platelets. So, if you have low platelets, you can actually take the Vonjo, hopefully get the same degree of JAK inhibition to help the spleen shrink, help the symptoms get better without necessarily making the platelets substantially worse. A lot of times they do drop, it doesn’t help bring up the platelets, but it does help people tolerate more JAK inhibition, which ultimately will help with symptoms.  

Dr. Jeanne Palmer:

So, one thing I also wanted to add about myelofibrosis treatment is sometimes people present, they don’t have a lot of symptoms, they don’t have a lot of spleen related problems but they have anemia or low blood counts and these can be incredibly hard to treat. 

Even with symptoms and low red blood cell count or anemia or low platelets, it can be challenging to treat because many of these medications lower that. To treat the anemia there are several things that we can do. One of the first ones is using erythropoietin, and so there are many agents, they go by the names of like Procrit or darbepoetin alfa, that actually stimulate red blood cell growth by – like we give a recombinant hormone that helps red blood cells grow. This is normally something produced by the kidney. 

So, one thing that’s important before going on one of these injections is to make sure that the kidney is not already producing enough. So, for example, if the kidney said, oh geez, I really need more red cells and is making lots of this hormone, erythropoietin, giving more of it is not going to help the system. But in people who don’t have a really high level it can be very beneficial. 

The other thing that can help with anemia, specifically, is a drug called danazol (Danocrine).  

It’s been around for a very long time. There are multiple presumed mechanisms of action, but one of them is that it is kind of a testosterone derivative. So, this is a medicine that can often help increase red blood cells in probably about 40 percent of people, and it’s a pill that you take twice a day. 

Another option, sometimes we use thalidomide or lenalidomide (Revlimid). These are medications that have been used quite frequently in the setting of multiple myeloma and even a little bit in myelodysplastic syndrome, so some other blood disorders.  

But in the setting of myelofibrosis, they can be helpful with anemia and sometimes are combined with prednisone or a corticosteroid. And then finally, in terms of drugs that are being tested and hopefully will be approved at some point in the future. There is a drug called momelotinib, which is another JAK inhibitor that actually has some mechanisms that may also help improve hemoglobin. 

So, this is something I’m really looking forward to and we anticipate may be approved by the end of the year. And finally, there is another drug called luspatercept (Reblozyl). Luspatercept may work in the setting where your kidneys are already producing enough erythropoietin. So, the luspatercept is an injection that you receive once every three weeks.  

It is currently FDA-approved for the treatment of myelodysplastic syndrome but this is something that has been shown to have some efficacy in myelofibrosis as well. So, this could be another therapeutic option for patients with myelofibrosis. 

It is also important, especially for people who have polycythemia vera myelofibrosis to make sure that your iron has been checked and B-12 has been checked, because just because you have a bone marrow disorder doesn’t necessarily mean you don’t have a nutrition deficit that may be able to help improve your hemoglobin somewhat. But these are important things to talk to your doctor. I do not recommend just starting to take iron or B-12, however, if you’re anemic because in many cases you are not deficient and taking too much iron can actually be damaged. 

Katherine Banwell:

Yeah, that’s great advice.  

When would you consider a stem cell transplant? 

Dr. Jeanne Palmer:

So, the stem cell transplant is based on disease risk. There is a number of ways we assess disease risk. The first two ones that were published a number of years back were the DIPSS score, which is Dynamic International Prognostic System Score, or the DIPSS Plus, which basically is the DIPSS and then you add to it a few other clinical features. This symptom score is based largely on things that we can see without even a bone marrow biopsy, so things like symptoms, age, number of white blood cells, whether somebody has anemia. And then the number of something called blasts, which is very immature white blood cells. The DIPSS Plus takes into account low platelets, need for transfusions, and chromosome abnormalities, which is the only test among that that needs to be from a bone marrow biopsy. 

Now, these were created prior to Jakafi being commercially available. So, we have to take a little bit of a grain of salt with those because of the fact that Jakafi probably has changed how long people can live with this disease. 

Now, more recently they’ve tried to account for these other molecular changes. So, when we take the genetic landscape of these diseases, we have the known driver mutations, so the JAK2 mutation which I have talked about, also calreticulin and MPL.  

These three mutations all affect that one pathway, the JAK/STAT pathway, so they all affect the pathway that drives the disease and they are known to be kind of mutually exclusive and definitely contribute to the formation of the disease.  

Some of these other mutations are called somatic mutations. They could be checked by things next generation sequencing or genetic analysis. There’s a number of different names that people use for this testing, but we look for mutations that are present and these mutations, number one, can sometimes tell us risk. So, there’s certain mutations that are high risk. Other times it can actually give us other opportunities for therapy, especially of the disease progresses. But these mutations are important to know for risk stratification. For example, if somebody has DIPSS score that is maybe not super high risk, but then they have one of these mutations, we know that that probably makes their disease a little bit more aggressive. 

And that’s when we think about transplant, is when we know that the disease probably has an average life – when somebody gets to the point in their disease where we estimate their life expectancy is around five years, recognizing that we’re not very good at this. That is the type of point when we start to think about transplant. But the timing of transplant is something that’s extremely difficult and a very personalized decision. It’s something that it’s really important to understand the disease risks, how we assess them and the caveats of these disease risk assessments as we move forward planning and timing of transplant and that’s something that is, again, a very, very important discussion to have at length with your physician. 

And I always recommend, there is quite a few of us out there who actually specialize in transplant for myelofibrosis and having discussions with somebody who really understands the biology of the myelofibrosis and important because it’s very different than a lot of the other diseases that are transplanted.   

Katherine Banwell:

Yeah. Well, speaking of that, patients can sometimes feel like they’re bothering their healthcare team with their comments and questions. Why do you think it’s important for patients to speak up when it comes to symptoms and side effects?  

Dr. Jeanne Palmer:

Well, there is a lot of things. This is a disease, again, that we can direct our therapy many times towards symptoms, and so when we think about how do I direct my therapy, so how do I treat somebody, symptoms are an incredibly important part of it. And there is nothing worse than having a patient come and see me who I see every six months, because they’ve been pretty stable and they’re like, “Oh, for three months I’ve been feeling awful.” And you’re like, well, “Why didn’t you let me know, we could do something about this?” 

So, if there is something that doesn’t feel right, it’s very, very important to talk to your healthcare provider. I would much rather be bothered and handle something earlier on than miss something and really have a lot more catch-up to do afterwards. 

The other thing is symptoms may indicate a blood clotting event. We know that patients will have a higher risk of blood clotting. These are extremely important to identify early on because if they go unchecked, they can cause more damage. 

Katherine Banwell:

With many of the treatments available as pills now, patients have a role in self-administering their treatment regimen. What happens if a patient forgets to take a medication? Does it impact its effectiveness? 

Dr. Jeanne Palmer:

Generally no. I think the ones that would are certain blood thinners you really don’t want to miss and you don’t want to miss the doses on it. With drugs like Jakafi, if you miss one dose you probably won’t notice it, but if you miss multiple doses you can actually get very sick from that. So, some of these medications are really important to be consistent on. 

Now, I know this could be a challenge. I mean I don’t take very many medications and I sometimes have a hard time keeping track of what I take, so I know that this can be a difficult thing to do. So, one thing is if you really find you’re struggling with it, setting an alarm on your phone or your Apple Watch or whatever… 

Katherine Banwell:

…device. 

Dr. Jeanne Palmer:

Device you have can be a really helpful way of doing it. Also having a pill box. They make pretty amazing pill boxes these days that can account for taking drugs once a day, twice a day, three times a day. I’ve even seen them up to four times a day, although generally the most you’ll probably have to take a medicine for a myeloproliferative disease is twice a day. But those are different ways that can really help make sure you’re consistent about taking your medication. 

Katherine Banwell:

And if a patient misses a dose, do they need to call their healthcare team and let them know? 

Dr. Jeanne Palmer:

Not just for one missed dose. If like, for example, they’re run out and they say, “Oh, geez, I don’t have any and many of these drugs are specialty pharmacy,” so they need to be mailed, and you know that you’re going to be missing it for a while. Or let’s say you look at your pill bottle and go, “Oh shoot, I only have so many pills left,” it is helpful to call because a lot of times, for example, if somebody is on Jakafi and they know they’re going to run out of their pills four days before they’re going to get their next shipment in, then what I sometimes do is I lower the dose a little bit to make sure they maintain a dose throughout that time. 

But this is something you definitely want to do under the advice of a healthcare provider. You don’t want to just all of the sudden go, “Oh, well I’m going to run out so I’m just going to change my dose,” and kind of do that. 

Katherine Banwell:

Yeah, yeah. We received some audience questions prior to the program today. This one is from Jacqueline, “What can I do to minimize pruritus or itching due to PV? A typical histamine blocker like Claritin or Zyrtec has done nothing whatsoever.” 

Dr. Jeanne Palmer:

Yeah. Unfortunately, the itching of this is not as much mediated by an allergic type reaction or histamine. It’s a lot related to that microvasculature, those tiny little blood vessels. Things like avoiding hot showers, as we talked about, taking cooler showers or not even taking showers, just like cleaning yourself with a washcloth can be helpful. There are certain medications that we can use sometimes that help. 

Now, first of all, Jakafi is extremely effective for itching. Of course, it does have side effects. It’s not always approved for your disease, so for example, it’s not approved for essential thrombocythemia. But JAK inhibitors can be helpful in that setting. There are also medications like Gabapentin, which is a medicine that we use to treat peripheral neuropathy and that can actually be helpful because actually the itching, a lot of it is related to nerves not functioning right, so gabapentin can be helpful. 

And a really old-school medicine that I sometimes use, especially if the itching is most prevalent at night, is a drug called Doxepin and that’s been around for a very long time, but it can be extremely sedating and has to be used with caution, especially in patients who are older. 

Katherine Banwell:

Here is a question from Daniel. “How often should a person with PV have hematology appointments, and how often should you have blood tests?” 

Dr. Jeanne Palmer:

Well, that is something that you need to discuss with a provider, because everyone’s a little bit different. If I have somebody who I’m managing on a medication, they’ve been rock solid stable, it may be every few months that I check blood and maybe every six months that I see them.  

If I have somebody who have been particularly difficult to control and I’m sort of adjusting medications or they’re having symptoms, then I try to check blood more regularly, like on a monthly basis. But again, this is something that – I have checked blood as frequently as every two weeks, especially in somebody who has an extremely high red blood cell count that I’m trying to lower. I have checked blood as infrequently as every three months. Again, in somebody who is not undergoing treatment, say, for example, who has essential thrombocythemia, sometimes I check blood even less. So, it really is something that can vary from every two weeks to every six months. 

Katherine Banwell:

Okay. Katie had this question. “What are the signs of progression from PV to MF or AML, both clinically and in blood tests, and when do you need a new bone marrow biopsy to check for this happening?” 

Dr. Jeanne Palmer:

So, in terms of progression, there are several things that we see happen. 

I think most importantly is, let’s say you have PV, and you’ve always been on medication, and it’s been hard to control. And all of a sudden, you don’t need medication to control it anymore, or the same thing for essential thrombocythemia. You have been taking medication, and all of a sudden your platelets go down, and you don’t need to take drugs anymore. A lot of times people are like, “Oh, that means I’m fixed and I’m well,” not necessarily, you really need to make sure to talk to your healthcare provider and potentially get a bone marrow biopsy. 

Now, the other thing – sometimes the blood counts will actually drop too low, so you’ll have somebody who has PV, who has always been too high and then all of the sudden they come in, and their hemoglobin is very low, and they’re anemic, and that’s another situation where you do that. So, anytime the blood counts start to drop is concerning.  

Now, it’s a continuum, so the blood counts may drop as you’re at the point of transitioning but it doesn’t – it’s not like if your blood count is dropping you say, “Oh my God, I have myelofibrosis, I need a bone marrow transplant tomorrow.” That’s not necessarily the case. This is generally a transition type process. 

Also when the spleen starts to get enlarged. Now, the spleen can be enlarged even in the setting of just ET or just PV, so spleen enlargement does not necessarily mean you’re transforming, but it can be one of the things that we would see that would indicate that. 

Katherine Banwell:

Okay. 

Dr. Jeanne Palmer:

And then finally white blood cell count increasing can often be a sign of that. Now, in terms of progression to AML, that is generally something we’ll see in the blood. AML or acute myeloid leukemia, is indicated by the presence of blasts at greater than 20 percent. Now, many patients with myelofibrosis, in particular, but even PV and ET, may have blasts in their peripheral blood. Blasts are normal. If I did a marrow on every healthy person out there, they are going to have some blasts, because these are the first part of the development of white blood cells. So, they’re like baby white blood cells. But what the problem is, is when they start to grow too much. 

And so in the setting of myelofibrosis and even sometimes with these other diseases, the blasts will be in the peripheral blood primarily because the bone marrow is damaged and doesn’t hold them in very well. It becomes AML when it gets greater than 20 percent, so that blasts of greater than 20 percent in the peripheral blood or in the bone marrow but a lot of times we find it in the peripheral blood is where we indicate this has progressed to AML. 

Katherine Banwell:

Yeah. 

Dr. Jeanne Palmer:

Blasts of greater than 10 percent are also something that we really want to pay attention to, because that would suggest that the disease is starting to become more aggressive. Now, blasts vary, so for example, I’ve had patients go up to 11 and then drop back down to 3 or 4, and then they say around 3 or 4 or 5. So, you always want to make sure to double-check because one blast count at 11 percent, whereas it’s very important to address, may not necessarily reflect that you need to change in treatment at that time. Again, these blood tests, I always tell people, do not freak out over one blood test.  

Make sure you get at least a couple of them to really confirm what you are looking at. 

Katherine Banwell:

Thank you, Dr. Palmer. And to our viewers, please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them answered on future webinars.  

Dr. Palmer, was we close out this conversation I wanted to get your thoughts on where we stand with progress in helping people live longer and truly thrive with MPN. What would you like to leave the audience with? 

Dr. Jeanne Palmer:

So, I think that the first thing is make sure you understand your disease. Don’t hesitate to ask for a second opinion. It’s always good to make sure you talk to someone who can really explain so you feel like when you go home you understand what’s going on in your body. Make sure you understand what symptoms to look for, what things to be aware of, because a lot of times people come in and they have no idea that, oh, these symptoms are actually related to their disease. 

The other thing to make sure is that you’re very honest with your provider on how you’re feeling. A lot of times people come in and they say, “Oh, how are you feeling?” “I feel fine,” but then they start to ask very specific questions and they’re like, “Oh yeah, I’m really tired, my fatigue is an 8 out of 10,” or something. 

So, make sure you’re really honest with your provider. When they ask you how they’re doing, this is not a social visit, this is a visit where they need to know your symptoms, so you don’t need to say I’m fine like you normally would if you were walking down the street. 

The next thing is to always make sure to know where there’s clinical trials because we are making enormous great leaps and bounds in this field. It’s a really exciting time for myeloproliferative diseases, and there’s a number of new drugs that are being tested and coming out. So, it’s always important, if the opportunity is available and you can do it, clinical trials are a great way to get treatment. 

Plus, you are giving back, because these are things that help us learn whether something works or not. So, you’re not as much a guinea pig, you never get a sugar pill. It’s one of those things you will always get the treatment you need and then they may add something to it or you may be in the situation where there is no treatment, so they try something. 

But clinical trials, I have to emphasize, are a great way to get therapy and really are how we know everything that we know about treatment for these diseases. 

Katherine Banwell:

Yeah. It sounds like there’s a lot of progress and hope in the field.  

Dr. Jeanne Palmer:

Oh, absolutely. 

Katherine Banwell:

Thank you so much, Dr. Palmer, for joining us today.  

Dr. Jeanne Palmer:

You are very welcome, my pleasure, and it’s always fun to do these things, so thank you for having me.   

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 joining us today. z

What Are the Signs of MPN Progression?

What Are the Signs of MPN Progression? from Patient Empowerment Network on Vimeo.

Dr. Abdulraheem Yacoub, an MPN specialist, explains how essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF) may progress from one disease to the next, including potential signs and symptoms of MPN progression. 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 

Related Programs:

 
What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Myelofibrosis?

What Are Treatment Options for Myelofibrosis?


Transcript:

Katherine:

We have a couple of questions from the audience. This one is from Sarah. She writes, “I’ve been living with essential thrombocythemia for three years, and have been relatively stable. Of course, I’m worried about progression to PV or MF. What is my chance of progression, and what are the signs of progression?” 

Dr. Yacoub:

That’s a very good question. And unfortunately, we’re very good at describing those numbers. Unfortunately, our tools at interfering are not as good. So, in general, patients with ET, statistically speaking, have a life expectancy that is not different from their age match peers. And Sarah’s story will be not too indifferent from her sisters and her mother, in terms of what’s going to happen to her long care and her health, provided she gets good medical care. The exception to that is that there is a transformation risk. For ET we caught around a 4 percent every 10 years in which ET will actually change into a different cancer, a higher risk cancer.   

Could be MF, could be MDS, could be acute leukemia. And that will be a much more serious diagnosis. So, it’s about 4 percent in 10 years. We do have a – or we extrapolate some of the data from other cancers. So, certain mutations are more favorable, certain mutations are more risky. And we try to forecast that but worried it’s really hard to predict that since it’s such a long journey with disease. The first symptoms or the findings, when patients start suspecting that their disease has changed, is that the pattern of symptoms that they have are different.   

They often become worse. So, they have more constitutional symptoms, more tiredness, more fevers, more night sweats, losing weight, not being able to eat a full meal, abdominal distension, the spleen gets bigger.  

So, these are some of the feelings that patients can experience that lead to this. Other objective things is when the blood tests change in a less favorable way. So, for patients with ET who always run at 800,000 platelet count, if they’re suddenly 200, and that’s in the normal range, but that’s actually not good news, because the cancer changed. And this change is not favorable. So, as the doctors run routine labs, if they see the sudden change in labs, that’s also abnormal.  

If the doctor can feel that the spleen gets bigger every time, that’s also concerning. If the patients suddenly have anemia or very high white cell count or immature white cells in the blood, that’s also a concern.  

So, that’s why it’s great or important to establish a baseline symptom burden.  A baseline spleen, a baseline bone marrow biopsy with mutation analysis, so that patients have a clear reference point to where they started, and if things change, they can always go back to that point and compare.