Session 1 Replay - Understanding MPNs: Accessing the Best Care & Treatment Options

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Topics include: Treatments and Understanding

The recent myeloprolifative neoplasm (MPN) town meeting in partnership with the University of Colorado Anschutz Medical Campus featured renowned experts and clinicians and a well-informed patient panel sharing the latest news in treating and living well with MPNs. Tune in now to hear leading medical experts discussing treatment options and research for essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF). You’ll also learn about the phases of clinical trials, when to consider this treatment option, and tools for communicating with your healthcare team, to ensure you are receiving the best care for your condition. 

Produced in Partnership with the University of Colorado Anschutz Medical Campus. Sponsored by Incyte Corporation.

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Transcript

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

Hello. Greetings from Aurora, Colorado just outside Denver. The campus of CU Anschutz, a huge medical campus here. And we’re so grateful they’re hosting us here. I’m Andrew Schorr, and over the next two, three hours, we’re gonna cover a lot for people living with MPNs, myeloproliferative neoplasms, long-term.

And we’re gonna talk about how we can access the best care or the best treatment and really live well. So, this is an interactive town meeting wherever you are. And we have a bunch of people in the room. And hopefully we’ve gotten a look at that. We’re gonna be a community now of people from around the world and sitting here in Colorado to get answers from leading experts who are behind me and others who will join us and some very experienced patients as well.. So, I wanna get Nelson to stand up for a second. Maybe we can get a shot. Maybe Joe, if we could get a shot of Nelson—this is Nelson Patz. And you’re gonna meet him in a little while. Nelson is a very strong guy. He goes to the gym every day, has myelofibrosis. He moved to Colorado to be with his daughters because he thought he was dying. And he wasn’t on the right medicine.

And he connected with one of these providers here, Dr. McMahon, and he’s gonna tell you the story by making the connection, also getting some financial assistance. He’s here today and doing well, right?

Nelson Patz:        

Doing excellent.

Andrew Schorr:  

Okay. Have a seat, Nelson. This is Becky VanNoy, right?

Becky VanNoy:    

Mm-hmm.

Andrew Schorr:  

Maybe you could stand up, so everybody could see you. And you’re gonna meet Becky for a minute—living with ET for many years.

Becky VanNoy:    

Ten years.

Andrew Schorr:  

Ten years. Okay. So, you’re gonna hear Becky’s story of living with this chronically, hoping it doesn’t develop into something more serious and having a wonderful relationship with her provider. So, thank you. And you’ve been married how many—40 years?

Becky VanNoy:    

41 years on Monday.

Andrew Schorr:  

Oh, congratulations. Nelson, you have a birthday in May.

Nelson Patz:        

Next week.

Andrew Schorr:  

Next week. And you’ll be how old?

Nelson Patz:        

71.

Andrew Schorr:  

71. Happy birthday, Nelson. Okay. I think we’re ready to get going, Jamie. Jamie’s our producer, so I’m gonna make sure I do it right. Okay. I’m gonna sit down, and I’m gonna introduce—and I’m gonna get them to tell you who they are too. This is also one of my longtime best friends, Dr. Naval Daver from MD Anderson. But what’s your position at MD Anderson down in Houston?

Dr. Daver:            

Sure. First of all, thank you for having me. It’s always good to work with Patient Power. I think you’re doing a fantastic job in MPN. And hopefully, we’ll move into other diseases as well. I’m an Associate Professor in the Department of Leukemia at MD Anderson Cancer Center, and I also direct the Leukemia Research Alliance. We have a number of trials that are under the alliance. And I focus a lot on MPN and AML research. So, we have a number of clinical trials in MPN and more so now in AML which is of course a think people are worried about progressing to. So, we will touch on both those aspects hopefully later today.

Andrew Schorr:  

Perfect. Okay. And over there, now we’re talking to the—do you say Coloradans? Did I get it right? The Coloradans. Dr. Brandon McMahon. Why don’t you say it right and give us your title?

Dr. McMahon:     

Yeah. So, I’m Brandon McMahon. And I’m actually a new Coloradan if that is what you say. I came here about a year and a half ago. I was at Northwestern University for just over nine years. Then I came here, specifically to help develop an MPN program along with Lindsey. Pardon me. Pardon me. The allergies here are not so good this time of year. But anyway, yeah. So, I came here about a year-and-a-half ago. And my interests are in developing clinical trials, looking at new drugs, new interventions, but also, I’m very interested in a better understanding of why some people develop bleeding and clotting problems with these diseases.

So, I’m collaborating with a couple of labs on campus. And for those of you who have seen me in the office, I often will have asked you for some samples to see if we can better clarify some of the features of the blood that may be different that may predispose bleeding and clotting. So, that’s my story.

Andrew Schorr:  

Okay. And next to you is Lindsey Lyle. And Lindsey, why don’t you introduce yourself? She’s been a regular on Patient Power both in Houston where she used to be with the team down there, and now she’s here. Lindsey, introduce yourself and what you do here.

Lindsey Lyle:        

Okay. Thank you, Andrew. So, I’m Lindsey Lyle. I’m a Physician Assistant. And I, like Andrew said, started my career at MD Anderson in Houston and used to work with Dr. Daver and then made the move back to Colorado where I’m from about three years ago, where now I get to work with Dr. McMahon. So, I work in the blood disorder center where we take care of patients with all he malignancies, but I have a special interest in MPNs. So, I work closely with Brandon monitoring patients on clinical trials and just collaborating with him to provide the best care we can for our MPN patients.

Andrew Schorr:  

Okay. And Lindsey was telling me earlier that she was on a program recently where they’re really working hard to educate community doctors and nurses about these rare conditions, because some of you maybe have gone to some place where maybe they don’t hardly have another patient with one of these conditions which is one of the things we’ll talk about today. Is it important to at least, if you can, connect with a academic medical center like MD Anderson or the University of Texas or Anschutz here to really connect with the latest or make a bridge between your community doctor, which is what I did at Seattle, and a doctor at one of these major centers so everybody’s on the same page?

So, we’ll talk about that. But Lindsey’s been very devoted to that education.

Lindsey, let’s just start with you for a second. We have on our slide there ET, MF, PV. How are these related? What are they? Is it something in your bone marrow? Where does it come from? What is it?

Lindsey Lyle:        

Great question. So, MPN, or myeloproliferative neoplasm is sort of an umbrella term for basically a process in the bone marrow is going out of control, essentially. So, it can come in different flavors is the way I like to talk about it. And so, essential thrombocythemia is a problem where the bone marrow, there’s a clonal process in the bone marrow meaning that it’s not reactive, but actually, there’s a mutation causing platelets to grow without control. And so, there are too many platelets being made in the bone marrow. And then those go out into the peripheral blood. And that’s why we can see elevated platelets on a blood draw. So, that’s the main feature of the disease of essential thrombocythemia.

Myelofibrosis is the most progressive type of myeloproliferative neoplasm. And just to be said, these are not necessarily a continuum. ET does not always progress to myelofibrosis. But it can. It can over time. But myelofibrosis is a bone marrow disorder in which too many white blood cells are usually being produced. And there’s fibrosis or scarring in the bone marrow which makes it more difficult for the bone marrow to make healthy cells such as red blood cells or platelets. So, those are oftentimes low. And then because of this hyper proliferation or these cells that are growing without control, sometimes patients have very large spleens in myelofibrosis.

This is a disease that, like Andrew said, can be categorized as lower risk or higher risk depending on different features of the disease, which we’ll get into in a little bit. But lastly, within this group of MPNs, polycythemia vera can be manifested in elevation of all three cell lines. So, you have too many white blood cells, too many red blood cells, and too many platelets. But the difference is that polycythemia Vera is characterized by elevated red blood cell counts. So, the other two, essential thrombocythemia and myelofibrosis don’t have an elevated red blood cell count above normal. But polycythemia vera does. So, that’s the distinguishing feature of that process.

Andrew Schorr:  

Okay. Thank you. So, Brandon, I’m gonna try to be a student. And you tell me if I get a good grade, okay? Because I wanna make sure we understand this. So, platelets, that’s what helps—if you cut yourself, that’s what helps you heal, right? It’s stickiness in the blood.

Dr. McMahon:     

Yup.

Andrew Schorr:  

So, these platelets that—what would be the normal—if you had a blood test, what would be the normal range of platelets?

Dr. McMahon:     

There’s a little bit of variability, but it’s a very broad range. About 150 to about 400 thousand roughly.

Andrew Schorr:  

Okay. But if somebody had ET, how high could their platelet number go?

Dr. McMahon:     

Sometimes, depending on the person, it could be in the million range. So, the number is reported as 150 to 400, but it’s 150,000 to 400,000. So, it could be up as high as a million, a million-and-a-half, and sometimes even higher than that.

Andrew Schorr:  

Okay. And polycythemia vera, what would be indications of that in the blood count?

Dr. McMahon:     

So, the big key that people will notice is their hemoglobin and hematocrit are going to be elevated. And as Lindsey mentioned, oftentimes the way blood count is high, and the platelet count is high with polycythemia vera, but not always. But oftentimes the key feature of p. vera is that the hemoglobin and the hematocrit are elevated.

Andrew Schorr:  

Okay. And in myelofibrosis, we talked about scarring in the bone marrow. And I thinktell —me if I’m right—the bone marrow in your long bones and in your hips, that’s where we make our blood. That’s the blood factory, right?

Dr. McMahon:     

Correct.

Andrew Schorr:  

Okay. So, if we get scarring, that can gum everything up.

Dr. McMahon:     

It can. And it can also, especially in the case of myelofibrosis, even though the blood cells are predominantly made in the bone marrow, if the bone marrow’s less effective, then organs that used to make blood cells when you were a fetus, the spleen being one of them, can get bigger and bigger and bigger, because that tries to take over and tries to make red blood cells and other cells in place of the bone marrow.

Andrew Schorr:  

In some people who develop a large spleen, it can go down to your hip.

Dr. McMahon:     

Very large. Yes. Can be very painful or cause a lot of symptoms with that too.

Andrew Schorr:  

But treatment has made a big difference.

Dr. McMahon:     

Big difference. Yes.

Andrew Schorr:  

Okay. And the idea of having to remove the spleen is very uncommon now.

Dr. McMahon:     

Very rarely do we recommend a splenectomy. And it would typically only be in very specific situations for patients because there can be complications with the surgery. And now as you mentioned, since we do have a lot more availability with treatments that are not surgical, people can have improvement with their spleen.

Andrew Schorr:  

Okay. Dr. Daver, let’s talk about diagnosis and monitoring for a little bit. So, I mentioned earlier, when I was diagnosed, they looked at my blood. And they could see certain mutations or changes that shouldn’t be there. And that led to a diagnosis of myelofibrosis. But you also have bone marrow biopsies. So, could you help us understand how do you know when somebody comes to you whether they have one of these conditions and which condition and how advanced it may be?

Dr. Daver:            

Sure. Yeah. So, I think each one of those present little bit differently. So, in patients who have ET or p. vera, they will usually be referred to us because they have elevation in either hemoglobin and/or platelets. And it’s important to note that it’s not always a uni model meaning that ET patients may not always have just high platelets. They may also have high platelets and high hemoglobin. And the same is true for p. vera where they may present with a high hemoglobin hematocrit but also may have elevated platelets. So, the most common referral we will get is a person who is seen by his community doctor.

Maybe he started having fatigue, weakness, abdominal distension, or maybe it was just a routine bloodwork for work related, insurance related things. And they say, “Well, the hemoglobin came out at 17. We’re concerned about this. Let’s go to a tertiary center.” And they will come to Colorado or MD Anderson. Same thing with ET. They will say, “Platelets are at seven, eight hundred. We don’t find any clear reason why that should happen. So, let’s refer you.

So, the first thing we do in these patients is rule out what we call secondary erythrocytosis which means a secondary elevation of your hemoglobin. So, there are conditions that we know for example, people who have COPD, lung diseases, heavy smokers, marathon runners who have a physiological increase in hemoglobin to keep up with the decreased oxygen diffusion from the underlaying lung condition or because they’re doing marathons, etcetera. And we will rule that out by a good history. So, you talk to them, you get that idea. If you don’t find that clear association, then we will say the next thing to do is look at two things—one is molecular mutations.

And the three most common mutations we look for either on blood or bone marrow are JAK2, which is the most common mutation seen in 97 percent of PV and about 50 percent of ET. And the second one is CALR called calreticulin which is seen in about 20 percent of ET and very uncommon in PV. So, with these mutations, in the PV cases, we almost 100 percent can diagnose in 97 percent plus 2 percent MPL or other mutations. And in ET, about 70 percent will have that.

Andrew Schorr:  

All right. Let me just explain. Okay. You be the professor. So, a mutation. So, okay. So, we have genes that we’re born with. Do we have blue eyes or brown eyes or dark skin or light skin or eventually no hair? And then we have other kinds of cancer genes that develop where the cells go a little haywire. And that’s what you’re talking about is is there the JAK2 gene that starts showing up where it shouldn’t or the—you even mentioned a new one that many people have here, MPL or calreticulin. And does that then correlate with one of these diagnoses?

Dr. Daver:            

Right. So, normally, we should not have mutations in those genes. So, we have the genes, but there’s actually a point in the gene that is looking abnormal from how it should look in a normal person. So, that’s the mutation where your gene is now abnormal. And we can pick those up…

Andrew Schorr:  

From the blood test?

Dr. Daver:            

…quite easily now from blood tests, right. So, if we find those mutations in the right clinical scenario—and that’s important because rarely, these mutations can be seen in people without disease. This is something that’s new emerging. But if we have a clinical patient with high hemoglobin, with high platelets, with the mutation, that helps you confirm the diagnosis.

Andrew Schorr:  

Okay. And we had on the screen a second ago just a bone marrow biopsy. So, could we get a shot of the audience for just a second, Alan? One of you guys just—there we go. So, raise your hand among the patients if you’ve had a bone marrow biopsy. Okay. Few people. All right. And hopefully, it was not too bad an experience. It’s not something you’d be overjoyed to have. But I’ve had like, ten along the years.

Why, Dr. Daver, do you ask sometimes for a bone marrow biopsy? What’s the point?

Dr. Daver:            

So, usually, the reason we do a bone marrow biopsy is because in spite of having clinical symptoms and being able to check the mutations on blood, one is really to confirm the diagnosis and then to know which of these conditions it is, PV, ET, myelofibrosis. Only a pathologist looking at a bone marrow can tell you that. So, you could have a patient with myelofibrosis, PV, ET who could have high platelets, high hemoglobin. But the treatment is quiet different for these conditions. So, to confirm and differentiate between these three very accurately, you need a bone marrow.

The second thing is in some cases, we see that even at presentation, patients are already progressing to what we would consider high-risk myelofibrosis or progressive myelofibrosis. And those are people who need more early and more aggressive treatment either with ruxolitinib (Jakafi) or even with combinations of Jakafi with other agents. And so, we don’t wanna miss that and delay treatment. And so, those are two main reasons we do a bone marrow biopsy.

Andrew Schorr:  

Okay. And also, we talked about scarring in the bone marrow. So, the bone marrow biopsy can also tell you the percentage of scarring.

Dr. Daver:            

Correct. The percentage of scarring. Yes. 

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Page last updated on May 29, 2019