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Health Educator Turned MPN Patient Speaks to Importance of Specialized Care

Health Educator Turned MPN Patient Speaks to Importance of Specialized Care from Patient Empowerment Network on Vimeo.

Myeloproliferative neoplasm (MPN) patient and health educator Julia Olff helped others navigate the healthcare system before her diagnosis with myelofibrosis and later with ET. Watch as she shares her unique experience and how finding MPN specialists can help patients in receiving optimal care.  

See More From the MPN TelemEDucation Resource Center

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Transcript:

Julia Olff:

Well, as a health educator and as a former hospital administrator when I was diagnosed with myelofibrosis, I feel like I had a particular reaction to the diagnosis that might be different from others who didn’t work in healthcare. I was both fascinated, which may sound really bizarre, that I was being diagnosed with an illness that I did not feel. Also, an illness I had not heard of, although I knew about some of the treatments that might be proposed because of my work as a health educator. So, I feel like I kind of went into health educator mode and health navigator mode, and that gave me a leg up in terms of knowing very early on, that once my ET transformed to myelofibrosis, I knew that I needed to see an MPN expert. And because I worked on health education because I worked in hospitals, I understood that I want, I needed to see a physician who had a depth of expertise, who had a volume of patients, who had a lot of experience with the drugs that existed. Although, in 2008, there was no approved, drug for myelofibrosis, but I knew I needed to go to the place where there was…

Where I had a better chance of getting the latest treatment, and I was diagnosed by a community oncologist who was lovely and one of the nicest people, and one of the nicest physicians I’ve seen, but it was clear he was not steeped in MPNs, because he treated patients across a spectrum of cancers. So, in that way, I think I started out in a different place, I also know that hospitals and healthcare can be very overwhelming, and I had a bit of the language and the world and some of the sort of…I understood a little bit more, I think about what my physicians might have been sharing with me, and if I didn’t, I felt empowered and not that this is easy by any stretch and it continues to be a challenge, but I knew that I needed to ask questions. I knew that I needed to read more about my illness, I knew I needed to vet my doctor as well, and I also figured out over time that as I was going to have this illness, hopefully in the sense that I hope I continue to live well with myelofibrosis and stay alive, that I was going to be seeing an MPN expert for a long time.

So, I think that influenced my point of view, I kind of take it as a job, so as to my personality, so I have a health notebook, I need to have one from the very beginning, I knew I couldn’t remember everything, I had to write it down. I knew I had to track what I was feeling so I could share it with my doctor, and I knew that from being a health educator, I think no one told me to do those things, and certainly, physicians don’t really know to tell you that, so I think in a lot of ways, I was approaching my illness in a very serious manner because I had experience in healthcare, and the last comment I’ll make is, I think from navigating the system, navigating health insurance, I knew a little bit from my experience as a caregiver already, and also from a health educator, I understood this is another area where I needed to empower myself or ask questions, or not take some of the information that may have been shared with me initially as on face value, that it was okay to ask more. As I said, I’ve had this illness for 13 years, I’ve also been caregiving for an adult child with illness, and every time I call the insurance company to ask a question about an explanation of benefits or why something isn’t covered, and learn a tiny bit more, and I add that to sort of my toolkit. 

MPN Patient Q&A: How Did You Avoid Obstacles to Receiving the Best Myeloproliferative Neoplasm Care

MPN Patient Q&A: How Did You Avoid Obstacles to Receiving the Best Myeloproliferative Neoplasm Care from Patient Empowerment Network on Vimeo.

 In 1991, there were few myeloproliferative neoplasm (MPN) experts. Many MF, ET, and PV patients were misdiagnosed and often received dismissive care. MPN patient Nona Baker shares how her diagnosis with two MPNs – essential thrombocythemia (ET) and polycythemia vera (PV) changed her life.

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:

How Do I Best Communicate My Concerns Without Feeling Dismissed


Transcript:

Dr. Nicole Rochester:

Hello and welcome. I’m Dr. Nicole Rochester, I’m a physician, a health advocate, the CEO of your GPS Doc, and the host for today’s Patient Empowerment Network program. I’d like to start by thanking our partners, MPN Alliance Australia and MPN Voice for their support. Today we’ll be doing an MPN patient question and answer session, talking directly to a patient living with an MPN for over 30 years. The goal is to help learn how to avoid obstacles to the best MPN care. Following this program, you will receive a survey and we’d be delighted to get your feedback, this helps inform future programs that we produce, please remember that this program is not a substitute for seeking medical care, so please be sure to connect with your healthcare team on what the best options may be for your medical care. I am proud and honored to introduce Nona Baker. Nona was diagnosed in 1991 with essential thrombocythemia, also known as ET, and then in 2004 with polycythemia vera also known as PV. Nona is a staunch patient advocate and the co-chair of MPN Voice where she counsels MPN patients around the world on how to connect to the best care. We are so happy that you have tuned in to learn about Nona’s journey and tips that she has for you and your family as you face an MPN diagnosis as well as how to navigate your care and gain clarity on your path to empowerment. Thanks for joining us, Nona.

Nona Baker:

Thank you and thank you to Patient Empowerment Network for giving me this opportunity to share my experience and hope for other patients as they navigate their way through the MPN diagnosis and treatments.

Dr. Nicole Rochester:

Wonderful, so Nona in 1991, when you were first diagnosed, there were very few experts in MPN. Many MF, ET, and PV patients were misdiagnosed, and they often received dismissive care, because there were just so many unknowns at the time, and sadly, this was part of your journey and we’re going to learn a little bit more about that shortly. We received a number of questions about how you navigate treatment early in the course of your diagnosis, your initial diagnosis was actually more of an assumption, and I’d love for you to briefly speak more about that.

Nona Baker:

Thank you. It was a fairly scary time, I have to admit because so little was known about MPNs or MPDs in those days, myeloproliferative disorders, blood disorders, and my journey was very much a checkered journey, starting with being sent to an orthopedic surgeon, who I then had to go into physiotherapy for painful feet and insoles in my shoes. I was sent to a rheumatologist who took one look at my blood work and that’s when he said, I think you’ve got an alcohol problem. My husband actually was sitting beside me and he said to him, he said, I think you’ve got that wrong, she doesn’t really drink. And the doctor then turned around, they said, Well, maybe the machines have got it wrong, so that was quite a scary thing, it was…I knew there was something wrong, but I’d been what we call here around the hoses, and it wasn’t until I…the rheumatologist asked for a new set of blood work that he called me two days later that I’ve made an appointment for you to see a hematologist and his call was on a Saturday morning on Monday, two days later, and then I can tell you I was really scared.

Nona Baker:

Really, really scared. The other thing it did is I kind of didn’t trust what I was being told, the one thing I was told was there were only 12,000 known patients in the country, I’m not sure that gave me a great deal of confidence, but yeah, it wasn’t an easy start it was very scary.

Dr. Nicole Rochester:

Wow, I appreciate you sharing that, and I’m sure that many people with MPNs and other rare diagnoses can relate to that journey. Well, let’s take a look at your brief vignette that sheds a little more light on your unconventional path to care…let’s watch.

Wow, well, the good news is Nona, we have come a long way, but of course, we still have a ways to go. Would you agree with that?

Nona Baker:

I couldn’t agree with that more. I hear so many patients through my work with caring forums that we do from London, that go out around the world who go and don’t get the right information and get quite scared still by what’s going on, and I think things like we’re doing now today help empower people to know that they can actually claim ownership of their MPN and ask for and have a right… Well, certainly in this country to ask for a second opinion and get to the right care to meet their needs.

Dr. Nicole Rochester:

Absolutely, and you are speaking my language as a health advocate, I am always talking with people about the importance of using their voice, standing up for themselves and seeking information, and asking questions, so I love that you have opened our program with that. So with that, let’s go ahead and get our questions, the first question comes from Susan, and Susan asks, “After the initial shock of your diagnosis, were you worried about limited treatment options and specialists, and then what was your next step?”

Nona Baker:

Was I worried? Well, I was just generally anxious because it’s this thing of not being in control of one’s body and having to surrender that control to another person, so that’s the scary bit for me, and then I did something a little bit stupid in hindsight because it was the early days of the internet, man, I did Dr. Google, not a good plan, because particularly in the very early days, there was some really, really sort of dreadful prognosis is almost sort of go from right, you will…which, of course, here I am, 30 years on. And so, I think that I would be very cautious even now in using Dr. Google, I would go to safe sites where they are medically monitored because I think a little knowledge can be very dangerous.

Dr. Nicole Rochester:

That is so true. And I just want to highlight that because in medicine, we often kind of jokingly talk about Dr. Google, but it really is a phenomenon, and while there’s this balance of patients with rare diseases being able to find information and empower themselves, but then as you mentioned, known a lot of the information on the internet has not been vetted, some of it is not scientifically accurate, and it can literally have you pulling your hair out as you read these accounts and start to really create more worry as opposed to creating action stuff. So, I appreciate you sharing that.

Our next question is from Alice and Alice says, “I’ve noticed among women, minority groups and underserved communities, that there’s often a dismissive tone or atmosphere when you speak up and share your concerns,” and she wants to know, “Nona, do you feel that being a woman played a role in your initial diagnosis?” And she also like to understand how to communicate concerns with the care team when you feel that you’re being dismissed.

Nona Baker:

That’s an interesting question, I have to be honest and say I didn’t experience that, but I’m well aware of that. And it goes on, and it’s really disempowering to feel that, so I have huge empathy to hear that. I think if I had experienced it, which I obviously didn’t experience it, my key tip here would be when going for an appointment with a clinician, take a notebook and a pen and write down what you want to ask them, and write down their answers, and preferably if you can take somebody with you, because then you have that opportunity afterwards to digest what you’ve been told, and that in itself is empowering because you can then make further choices.

Dr. Nicole Rochester:

I love that, Nona. Also, advice that I always give to clients, and you’re right, having someone with you and writing things down is so important, especially in these situations where you’re getting a diagnosis, there’s a lot of uncertainty. We know that a lot of the information that’s shared in medical appointments goes in one ear and out of the other, particularly if we’re anxious or concerned or worried, so having that second person in the room is so incredibly important. I appreciate that advice. All right, our next question comes from Charles. He says, some patients living with two MPNs have said that they’re living with two cancers,” and he goes on to say that he’s been confused as to whether MPNs are cancers or blood disorders. Do you feel comfortable speaking to that and setting the record straight based on how you counsel other advocates in this space, he also mentions that his wife is living with ET and PV as well, and that sometimes the language can be very confusing.

Nona Baker:

I absolutely agree. And interestingly, we did a virtual forum for…at the weekend and one of the research projects, there has been only impacting on families, and it’s very interesting that the language can be very…again, disempowering the word cancer, I think the conventional word cancer is almost…it’s a deaf nail, but actually, when I challenged on the medication, I had the word cancer was used, I went to my primary GP physician, and I asked him,” nobody’s told me I’ve got cancer. What’s this?” Because at the time, it was a blood disorder and it said cancer, and he said,” Do you know what cancer means, Nona?” He said, “It means a proliferation of cells, but these are confined to the bone marrow.” But what happened for us as patients, as we started off, or certainly I did with a blood disorder, and then the World Health Organization, because of this perforation of cells re-classified that as a neoplasm, a neoplasm is just another word for cancer. So, it hasn’t changed since I was diagnosed, but the words have changed. And the scariest is in the word neoplasm suddenly here in the UK, it’s been an advantage, because we have access to much better drugs than we would have had if we’ve just been a disorder. I can’t speak for other health authorities or other countries, because each country is different, but I think it’s just simplifying it.  Simplifying the language. That’s empowering in itself.

Dr. Nicole Rochester:

I agree, and language is everything, and I think the key is what you said, that while there is a proliferation and while some may use the word cancer that it is confined, and I think that that provides a lot of clarity. Alright, we also have a question from Julie. Julie says, “I was given the run-around early on in my journey and wasted valuable time,” and she wants to know, what are some questions or actions to take at the outset when ruling out MPNs?”

Nona Baker:

That’s a difficult question because I think everybody is different and every health service is different. I think if you’re in an area where the clinicians don’t necessarily know too much about MPNs, that can be problematic. We’re a small country here and we have access to some really good hospitals that specialize in MPNs. I think, again, it’s going back with your piece of paper saying, can we rule out that I’ve got an MPN and I’ve read about MPNs, I have the symptoms, whether it’s fatigue or whether it’s itch for PV or whatever the symptoms are, and I’ve seen that that can be a symptom of an MPN. And again, take a piece of paper, and say can we rule that out? You know, I think that’s empowering.

Dr. Nicole Rochester:

I agree. Nona and I think when counseling patients who have had misdiagnosis or long road to accurate diagnoses, what you just said is key, and a lot of times it’s a matter of opening up the minds of your physicians and your healthcare team, and like you said, if they’re not familiar with MPNs, then they may go down a path of giving you a different diagnosis, but if you’ve done a little research or if you have some concerns, just saying, could it be this…I know that you think I have this condition, but based on what I’ve read, based on what I’ve learned, could it be an MPN? And a lot of times just that suggestion is enough to kind of shift the conversation, so I think that’s wonderful advice. Alright, our next question comes from Edna. And Edna says that in your in yet you stated that you were diagnosed at 41 and that you are a busy mom and that you were working, and she wants to know, “How did you share this diagnosis with your children and how did it impact your work in your career?”

Nona Baker:

It’s a very interesting question, and I think my children, because I had sort of my mom’s painful feet and I have packets of mushy peas that used to be put on my feet because they were painful because of the obviously thick blood, and my younger son has done a lot of fundraising for MPN Voice, and he talked about how as an 8-year-old, he’d grown up with me having these symptoms that I haven’t done much about, and I know I’ve always taken the view for me, and this is only for me, that I don’t let my MPN define who I am. You know, I think it’s part of my life. It isn’t my life, because my fear would be after that initial anxiety and fear that if I allowed it to take over my life, it would actually really impact my younger…my young children…in terms of my work, I only work part-time. You know, the other thing is, yes, I got a lot of fatigue, but I think what I’ve learned over the years is to put your hands up and say, you know, I’ve hit a wall whereas I just take five minutes.

Nona Baker:

Just take that time. Whereas sometimes it’s difficult when you’re a mom with young children, and I think now, people tend to explain it a bit to their young children, when mom’s tired, it’s not because it’s anything you’ve done it, because I remember patients describing it to have children is…it’s like a car, when the oil in the car gets too thick, the car slows down and sometimes the car needs to stop, and she equated her blood as the oil in the car that sometimes it just slows down and then has to stop gets a bit of refueling, I thought that was a good definition for young children.

Dr. Nicole Rochester:

I love that, I love that, and I’m a pediatrician by training, so I love putting things in clear terms for kids, and I think that’s really important to just make it simple for them. I also really like what you said, Nona about the self-care part, I think that can be really difficult sometimes for even women who may not have chronic diseases, but certainly for women and moms who have chronic diseases and feeling that, feeling guilty when they take time for themselves, even if it’s in the context of their illness, and so needing to rest and explaining that and normalizing that mom needs to take a nap, I think is incredible, and I love that your son is involved in the advocacy work that you do for MPN. All right, we have a question from James. James says, “Are there specific lifestyle changes that you may, following your diagnosis that brought relief to any symptoms that you were having?”

Nona Baker:

Well, the first change I had to make was I used to smoke, and then my hematologist said to me that affects the red cell count, and that was the incentive to absolutely give up smoking there, and then that was my first lifestyle change, and I haven’t regretted it for a single day. Other lifestyle changes, not really, other than just becoming aware that you know to fight fatigue doesn’t help, sometimes you have to surrender to it, but definitely give up smoking and I… you know, I think that…well, nowadays people don’t smoke, but we’re talking 30 years ago, so…yeah, 30 years is pretty well since I’ve had a cigarette…

Dr. Nicole Rochester:

Well, kudos to you for giving up smoking that…that is a challenge. So that’s wonderful. All right, we have a question from Janet. Janet says, I have noticed that many MPN patients develop a second MPN over time, and she wants to know. She wants to know, “Were you surprised about your PV diagnosis over a decade after your first diagnosis, or is this something that you were perhaps prepared for by your medical team?”

Nona Baker:

Well, my second diagnosis came by chance because I had a problem with fibroids, which necessitated having a hysterectomy, which so, the natural venesection was taken away, and then it evolved to a… I don’t know whether that’s the reason, but then I was diagnosed with PV, which means that I have PV with high platelets now is I think the way in my hematologist describes it, but it’s certainly under control with the medication and with venesection from time to time. So, was I surprised? I don’t think after my journey, I don’t think anything surprised me really, I sort of…I think, again, I took ownership of it and just got on with it, really.

Dr. Nicole Rochester:

Excellent, thank you, Nona. As we prepare to close, is there one tip or one piece of advice that you would like to give to individuals with MPN?

Nona Baker:

Don’t be afraid to ask a question, because I think living with a fear of something is really not good for one’s general health because fear and anxiety can, I think, impact a physical illness if you’re living with a lot of fear in a lot of anxiety, and I know this is easy for me to say because I’ve had a relatively easy journey, and I’ve met patients who’ve had a really, really tough time and I know through Pan-voice, people that were diagnosed either shortly after me or some before who had a bone marrow transplant, you know, their life is obviously better, but my goodness…what they went through to get where they are now. But I think the whole thing that we’ve been talking about really is just find that voice, and even if it’s not with the clinician, share it with a friend, you don’t sit on fear, share it with a friend, have a body, have an ally, and one of the things we do at MPN Voice, which actually I think has helped enormously, is we have a buddy program there where you would be…you will be matched with somebody who has been diagnosed for at least two years that can buddy you along emotionally, because I don’t underestimate the emotional impact that that affects a lot of us.

Nona Baker:

And I think we need to have that voice to say, yes, it is a bit of a shock, but I’m not going to let it define me and wreck my life. If you can do that, I think life will be easier.

Dr. Nicole Rochester:

That is awesome. Don’t sit on fear. I’m going to carry that with me. Nona, I appreciate that. Well, that’s all the time that we have for questions. Nona, I want to thank you for taking this time to share your story with me and for everyone watching, and just to recap, we’ve learned that avoiding obstacles to the best MPN care means remembering that everyone’s journey is going to be different. We learned the importance of not allowing your disease to consume your life, and we’ve also learned the importance of using your voice because we are truly our own best advocates, it’s these actions that are key to staying on your path to empowerment. Thank you so much again for joining us, Nona, this has been amazing.

Nona Baker:

Thank you for giving me the time to speak to the patient community.

Dr. Nicole Rochester:

I’m Dr. Nicole Rochester, thank you again for joining this Patient Empowerment Network program. 

MPN Patient Q&A: How Did You Cope with a Second MPN Diagnosis?

MPN Patient Q&A: How Did You Cope with a Second MPN Diagnosis? from Patient Empowerment Network on Vimeo.

For myeloproliferative neoplasm (MPN) patients diagnosed with a second MPN, how can they cope or react to the diagnosis? Watch as MPN patient Nona shares her experience with her second MPN diagnosis as part of her patient journey.

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:

How Do I Best Communicate My Concerns Without Feeling Dismissed

 


Transcript:

Dr. Nicole Rochester:

All right, we have a question from Janet. Janet says, “I have noticed that many MPN patients develop a second MPN over time,” and she wants to know, “were you surprised about your PV diagnosis over a decade after your first diagnosis, or is this something that you were perhaps prepared for by your medical team?”

Nona Baker:

Well, my second diagnosis came by chance because I had a problem with fibroids, which necessitated having a hysterectomy, which saw the natural venesection was taken away, and then it evolved to a…. I don’t know whether that’s the reason, but then I was diagnosed with PV, which means that I have PV with high platelets now is I think the way in my human toll describes it. But it’s certainly under control with the medication and with venesection from time to time. So, was I surprised? I don’t think after my journey, I don’t think anything surprised me really, I sort of…I think, again, I took ownership of it and just got on with it, really.  

MPN Patient Q&A: What Lifestyle Changes Did You Make?

MPN Patient Q&A: What Lifestyle Changes Did You Make? from Patient Empowerment Network on Vimeo.

 Should myeloproliferative neoplasm (MPN) patients make lifestyle changes after diagnosis? Watch as MPN patient Nona explains lifestyle changes she made following diagnosis to improve her quality of life.  

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:

How Do I Best Communicate My Concerns Without Feeling Dismissed

Key Considerations When Making Prostate Cancer Treatment Decisions


Transcript:

Dr. Nicole Rochester:

All right, we have a question from James. James says, “Are there specific lifestyle changes that you may, following your diagnosis that brought relief to any symptoms that you were having?”

Nona Baker:

Well, the first change I had to me was I used to smoke, and then my hematologist said to me that affects the red cell count, and that was the incentive to absolutely give up smoking there, and then that was my first lifestyle change, and I haven’t regretted it for a single day. Other lifestyle changes, not really, other than just becoming aware that you know to fight fatigue doesn’t help, sometimes you have to surrender to it, but definitely give up smoking and I… you know, I think that…well, nowadays people don’t smoke, but we’re talking 30 years ago, so…yeah, study is pretty well since I have a cigarette.  

MPN Patient Q&A: How Did Your MPN Diagnosis Impact Your Life?

MPN Patient Q&A: How Did Your MPN Diagnosis Impact Your Life? from Patient Empowerment Network on Vimeo.

For a myeloproliferative neoplasm (MPN) patient, how can diagnosis impact your life? Watch as MPN patient Nona shares her experience as a working mom, and Dr. Nicole Rochester shares her perspective about self-care. 

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:


Transcript:

Dr. Nicole Rochester:

All right, our next question comes from Edna. And Edna says that in your…you stated that you were diagnosed at 41 and that you are a busy mom and that you were working, and she wants to know, “How did you share this diagnosis with your children, and how did it impact your work in your career?”

Nona Baker:

It’s a very interesting question, and I think my children, because I had sort of my mom’s painful feet, and I have packets of mushy peas that used to be put on my feet because they were painful because of the obviously thick blood, and my younger son has done a lot of fundraising for MPN Voice. And he talked about how as an 8-year-old, he’d grown up with me having these symptoms that I haven’t done much about, and I know I’ve always taken the view for me, and this is only for me, that I don’t let my MPN define who I am. You know, I think it’s part of my life. It isn’t my life, because my fear would be after that initial anxiety and fear that if I allowed it to take over my life, it would actually really impact my younger…my young children…in terms of my work, I only work part-time. You know, the other thing is, yes, I got a lot of fatigue, but I think what I’ve learned over the years is to put your hands up and say, “You know, I’ve hit a wall,” whereas I just take five minutes.

Nona Baker:

Just take that time. Whereas sometimes it’s difficult when you’re a mom with young children, and I think now, people tend to explain it a bit to their young children, when mom’s tired, it’s not because it’s anything you’ve done it, because I remember patients describing it to have children is…it’s like a car when the oil in the car gets too thick, the car slows down and sometimes the car needs to stop, and she equated her blood as the oil in the car that sometimes it just slows down and then has to stop gets a bit of refueling, I thought that was a good definition for young children.

Dr. Nicole Rochester:

I love that, I love that, and I’m a pediatrician by training, so I love putting things in clear terms for kids, and I think that’s really important to just make it simple for them. I also really like what you said, Nona about the self-care part, I think that can be really difficult sometimes for even women who may not have chronic diseases, but certainly for women and moms who have chronic diseases and feeling that feeling guilty when they take time for themselves, even if it’s in the context of their illness. And so, needing to rest and explaining that and normalizing that mom needs to take a nap, I think is incredible, and I love that your son is involved in the advocacy work that you do for MPNs.  

MPN Patient Q&A: What Questions Should I Ask If I Suspect I Have an MPN?

MPN Patient Q&A: What Questions Should I Ask If I Suspect I Have an MPN? from Patient Empowerment Network on Vimeo.

For patients who suspect they have a myeloproliferative neoplasm (MPN), what questions should they ask? Watch as MPN patient Nona shares her advice for approaching questions with your doctor, and Dr. Nicole Rochester explains how to empower yourself to shift doctor-patient communication.

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:

How Do I Best Communicate My Concerns Without Feeling Dismissed

 

Key Considerations When Making Prostate Cancer Treatment Decisions


Transcript:

 Dr. Nicole Rochester:

All right, we also have a question from Julie. Julie says, “I was given the run-around early on in my journey and wasted valuable time.” And she wants to know, “What are some questions or actions to take at the outset when ruling out MPNs?”

Nona Baker:

That’s a difficult question, because I think everybody is different and every health service is different. I think if you’re in an area where the clinicians don’t necessarily know too much about MPNs, that can be problematic. We’re a small country here, and we have access to some really good hospitals that specialize in MPNs. I think, again, it’s going back with your piece of paper saying, “Can we rule out that I’ve got an MPN and I’ve read about MPNs, I have the symptoms,” whether it’s fatigue or whether it’s itch for PV or whatever the symptoms are, “and I’ve seen that that can be a symptom of an MPN.” And again, take a piece of paper, and say, “Can we rule that out?” You know, I think that’s empowering.

Dr. Nicole Rochester:

I agree. Nona and I think when counseling patients who have had misdiagnosis or a long road to accurate diagnoses, what you just said is key. And a lot of times it’s a matter of opening up the minds of your physicians and your healthcare team, and like you said, if they’re not familiar with MPNs, then they may go down a path of giving you a different diagnosis. But if you’ve done a little research or if you have some concerns, just saying, “Could it be this…I know that you think I have this condition, but based on what I’ve read, based on what I’ve learned, could it be an MPN?” And a lot of times just that suggestion is enough to kind of shift the conversation, so I think that’s wonderful advice.

MPN Patient Q&A: How Do I Best Communicate My Concerns Without Feeling Dismissed?

MPN Patient Q&A: How Do I Best Communicate My Concerns Without Feeling Dismissed? from Patient Empowerment Network on Vimeo.

What can myeloproliferative neoplasm (MPN) patients do to improve communication when the feel like their concerns aren’t being heard? Watch as MPN patient Nona shares her advice for preparing for appointments, and health advocate Dr. Nicole Rochester offers advice on how to help calm anxiety at appointments.

This program provides one patient’s perspective. Please talk to your own doctor to make healthcare decisions that are right for you. 

See More from Best MPN Care No Matter Where You Live

Related Resources:

 


Transcript:

Dr. Nicole Rochester:

Our next question is from Alice and Alice says, “I’ve noticed among women, minority groups and underserved communities, that there’s often a dismissive tone or atmosphere when you speak up and share your concerns,” and she wants to know, “Nona, do you feel that being a woman played a role in your initial diagnosis?” And she also likes to understand how to communicate concerns with the care team when you feel that you’re being dismissed.

Nona Baker:

That’s an interesting question, I have to be honest and say I didn’t experience that, but I’m well aware of that. And it goes on, and it’s really disempowering to feel that, so I have huge empathy to hear that. I think if I had experienced it, which I obviously didn’t experience it, my key tip here would be when going for an appointment with a clinician, take a notebook and a pen and write down what you want to ask them and write down their answers. And preferably if you can take somebody with you, because then you have that opportunity afterwards to digest what you’ve been told, and that in itself is empowering because you can then make further choices.

Dr. Nicole Rochester:

I love that, Nona. Also, advice that I always give to clients, and you’re right, having someone with you and writing things down is so important, especially in these situations where you’re getting a diagnosis, there’s a lot of uncertainty. We know that a lot of the information that’s shared in medical appointments goes in one ear and out of the other, particularly if we’re anxious or concerned or worried, so having that second person in the room is so incredibly important. I appreciate that advice.  

An MPN Expert’s Top Three Tips for a Telemedicine Visit

An MPN Expert’s Top Three Tips for a Telemedicine Visit from Patient Empowerment Network on Vimeo.

As a myeloproliferative neoplasm (MPN) patient, what steps can be taken to prepare for telemedicine visits? ExpertDr. Jamile Shammofrom Rush University Medical Center provides her key tips to help ensure an optimal telehealth visit. 

See More From the MPN TelemEDucation Resource Center

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Transcript:

Dr. Jamile Shammo: 

When preparing for a televisit, I think it’s so important to know whether or not you would have a connectivity issue. A lot of times I’m trying to connect with the patient and then we realize that their phone isn’t equipped to handle the televisit and that is kind of disappointing to find that out a minute before you try to connect then that visit becomes a telephone encounter, which is again, less satisfying for some patients. I mean it does the job, but again, it doesn’t provide me with the exam…part of the exam that I’d like to do, at least in that way. So, I think prepare yourself and make sure that your device is able to connect and actually most clinics will have a person that may be able to help you navigate through the televisit pieces that would help you get through and connect with the provider, and then obviously with an MPN or any other visit, patient with a heme malignancy, it would be helpful to make sure that you have a blood draw or if your physician would like to have a blood draw in my case, I always like to have a CBC beforehand or perhaps a chemistry or maybe ion studies or what have you, to have that so that there will be something to discuss and make sure that your physician has had those results before you have the visit. 

Sometimes it is also disappointing that the patient thinks I’ve received those results when I actually haven’t and I have no control over that, so that would be the other piece. All of us do our best so that we can make sure that all those pieces are in place so that we can conduct the visit. And I know it’s a lot of work on everybody’s part. But in the end, what matters is that we are providing the best care possible in those very challenging times. 

What Opportunities and Challenges Does Telemedicine Present for MPN Patients?

What Opportunities and Challenges Does Telemedicine Present for MPN Patients? from Patient Empowerment Network on Vimeo.

For myeloproliferative neoplasm (MPN) patients, what does telemedicine offer in terms of opportunities and challenges? Expert Dr. Jamile Shammo from Rush University Medical Center shares situations when telemedicine versus in-person visits can help provide optimal MPN patient care.

See More From the MPN TelemEDucation Resource Center

Related Resource:


Transcript:

Dr. Jamile Shammo: 

I think the medicine has provided a tremendous opportunity for us to take care of patients in general, MPN patients in particular during the pandemic. We obviously wanted to minimize the exposure of patients to COVID during the pandemic, but patients who have MPN as well as other hematological malignancies needed to have CBCs frequently to make sure that the treatments that they were on were safe, that they were doing what they were supposed to do in terms of controlling their counts. So, then there was no escaping that. And they also needed to get ahold of their doctor, so being able to do both, perhaps away from the hospital in some type of clinic and being able to connect with the physician online to discuss the results of the CBC that they had obtained in perhaps a less populated lab was tremendous. And granted, this had made it feasible to care for patients during the pandemic. But now that we are sort of emerging from the pandemic, people are realizing that perhaps those technologies are there to stay, and perhaps there’s a subset of patients that may still be able to benefit and take advantage from those resources, so we are learning as we go who may be able to continue to do this. 

I have to say though, that that may not be for every patient, and I still feel like there’s a particular type of MPN patient that will benefit from seeing the physician and having a full exam once every so often. And we can talk about the particular application that that may be, but granted telemedicine has certainly provided a tremendous advantage during COVID.  

So, when I think of the patient that might benefit most from seeing the physician via televisit, for example, it would be someone who perhaps has a stable disease. Someone who I may want to monitor perhaps every three to six months, someone who may have stable counts, and we’re just talking to about their symptoms and monitoring those types of things every so often. And perhaps I look at the labs and you can discuss their symptoms and whether or not they have splenomegaly and issues like that. Someone who may already be on a stable dose of medication and we don’t have to do any dose adjustments and even if we have to do those adjustments, perhaps we could do labs a little more frequently, so that would be all right too, but someone in whom I would like to initiate in treatment, someone in whom the disease may be progressing a little too quickly, someone who I may want to do an exam and assess their spleen, I suppose you could send them to an ultrasound facility and obtain an MRI or a CT, or an ultrasound of the imaging study that is. But there’s nothing like an actual exam of the patient. You are thinking about the disease progression, so those sorts of patients in which the disease is actually changing its pace, you may want to take a look at it, the full smear look and examine the skin for certain TKI and signs and symptoms of low platelets and that sort of thing. Look in the mouth for ulcers and things of that nature. Those are the patients that I feel like would benefit the most from seeing their physician of course, the patient who has questions and that that could be probably beyond what a televisit could do. I think those would be the types of situations where you would like to have a physical presence and discuss things that would be of extreme importance to the patient’s physical health, psychological health, and of course, labs that you may want to obtain beyond the regular CBC that a standard lab could obtain outside of your institution. There are specialized labs that not every leg outside of your own tertiary care center may be able to provide, and that is something that we need to all the time. Let’s say a patient may require a bone biopsy, well then you have to have them physically be in your place, and then you might as well, then see them, examine them and do all of the labs, and that’s the other thing that we would like to do is perhaps to bundle all of the tests that you would be minimizing the exposure of patients to frequent visits so that you would be again, lessening the exposure, potentially infections.

What Impact Does Telemedicine Have on Clinical Trials for MPN Patients?

What Impact Does Telemedicine Have on Clinical Trials for MPN Patients? from Patient Empowerment Network on Vimeo.

For myeloproliferative neoplasm (MPN) patients, what impact does telemedicine have on clinical trial access? ExpertDr. Jamile Shammofrom Rush University Medical Center explains the current environment for clinical trial access and her perspective on how trial access should be approached in the future for improved MPN care. 

See More From the MPN TelemEDucation Resource Center

Related Resource:


Transcript:

Dr. Jamile Shammo: 

So, there’s no doubt that COVID has certainly impacted our ability to enroll patients on clinical trials. There have been a lot of governing bodies that have created various rules and regulations around that to facilitate enrolling patients on clinical trials, and I think right now we are seeing that this has become feasible, such that we are able to enroll patients yet again on the clinical trial. So, now I think that we have the vaccine that is available, it has become a little bit more feasible and possible to do so. So, this should not stop us. I think we should continue to seek better treatments for MPN patients actually the only way to do so is by you know, only patients on trials, because we certainly don’t have a perfect way to provide care at the moment, we always need to come up with better ways and that would be one way to do so. 

The MPN community truly should partner with their physician and learn as much as possible about their disease and about available treatment options, and perhaps show some support for available clinical trials because this is the only way that we can perhaps understand how we can do a better job in treating patients who have MPNs. 

Ask the MPN Expert – Dr. Joseph Scandura

Ask the MPN Expert – Dr. Joseph Scandura from Patient Empowerment Network on Vimeo.

MPN specialist Dr. Joseph Scandura from Weill Cornell Medicine answers patients’ burning questions.


Transcript:

Andrew:
Greetings from southern California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program where you can ask an MPN expert your question. I’ve been living with an MPN, a myeloproliferative neoplasm myelofibrosis, since 2011. So, believe me, I have questions and I want answers just like you. I want to thank the Incyte Corporation for its financial support, but tell you, of course, that all the editorial control is our expert and our producers and me. Nobody tells us what to ask or what to say.

Okay, let’s meet today’s MPN expert. Joining us from New York City is Dr. Joseph Scandura. He is with Weill Cornell Medicine in New York City and he is also the scientific director of the Richard T. Silver Myeloproliferative Neoplasm Center at Weill Cornell Medicine. Dr. Scandura, welcome and welcome back to Patient Power. We’ve had you before. Thanks for being with us.

Dr. Scandura:
Thanks for having me, Andrew.

Andrew:
Okay, and I should mention that Dr. Scandura is a physician-scientist, so you can see that whiteboard behind him. He spends time in the lab, as well as seeing patients, in-patients, and in clinic. So, he is meeting us, but also working on a cure and we’re gonna talk more about that and hopefully, we can get there. Okay, are you ready for our first question Dr. Scandura?

Dr. Scandura:
All set.

Andrew:
Okay. So, this one comes from Philip who writes and he says, “I’m a 63-year-old male with PV, polycythemia vera. What does it mean that my blood is too thick?”

Dr. Scandura:
What it’s probably referring to, the term too thick is a little bit – can be generalized in a lot of different directions. It’s a colloquial term, not really a medical term, but what people often are referring to there in the context of polycythemia vera is too many red blood cells. If you think of the blood vessels in your body as being highways, they can only accommodate a certain amount of traffic. And you being in southern California are probably aware of this, that sometimes there’s too many people trying to get on the highway at the same time and that slows everything down. You could consider the highways too thick in that situation and that’s what’s really happening in polycythemia vera.

There’s too many red blood cells. There’s about 1,000 red blood cells for every of the white blood cells there, so the most common blood cell type and they occupy about half of the whole blood volume. And when you have too many being produced, they end up causing traffic jams in the blood vessels and that is what people are trying to describe when they’re saying the blood is too thick.

Andrew:
And you’re at risk for stroke and blood clots?

Dr. Scandura:
Yeah. So, it has a lot of both short-term and long-term consequences. Short-term certainly it provides a risk of having abnormal blood clots. That can be in an artery, so that could be a stroke, an artery in your brain, or an artery in your heart, a myocardial infarction or heart attack. It can also be a clot in a vein and so these, I’m sure you’ve seen them on TV, the advertisements for DVT or deep venous thrombosis or pulmonary embolism which is usually a clot in a vein that then has broken off and traveled through the circulation and landed in the lung where it can cause symptoms there. And so, the short-term risks of a clot are certainly elevated in people with polycythemia vera when the blood counts aren’t controlled.

Andrew:
Okay. Just one follow up question. Philip was wondering about this too. So, we see ads on TV whether it’s the DVT medicine ads or the blood thinner ads. Does that apply to people with PV?

Dr. Scandura:
It can. We treat people with PV to reduce the risk of a clot, but some people are diagnosed with a clot at the same time they’re diagnosed with PV and some people, even with the best of treatment, end up developing a clot. If it’s a clot in the vein, then one of the things that is a standard of care is to administer drugs that colloquially again are referred to as blood thinners. In this context, it has a different meaning and this is a group of drugs that interfere with the blood clotting system. So, these are proteins, not cells, and it’s what – if you ever have cut yourself and you feel just with your fingers, it gets a little sticky between the fingers. That’s actually clotting.

It’s a little bit like Jell-O. It starts out liquid and then it solidifies and that’s what your body does to help prevent bleeding. It forms this sort of polymer fiber that ends up being part of the plug. And what the blood thinning medications, the so-called blood thinning medications, do is they interfere with that process. Either given by injection or given by pill, the ultimate goal is to reduce the formation of that sort of sticky acellular clot. And that’s more of a treatment and can be a preventative for future clots as well, but it’s a little different than what we were talking about before in terms of too thick blood from too many red blood cells.

Andrew:
Too many cells versus the quality of the cells.

Dr. Scandura:
Yeah, but not even the cells. A lot of the blood clotting factors are produced by your liver. They’re not from the cells themselves that are floating around in the blood.

Andrew: I’ve never understood that before. So, thanks for explaining. I should also just say one thing about Philip. He shared with us that he has AFib. So, when somebody, and that’s not uncommon atrial fibrillation, does that complicate all the treatment for somebody with PV?

Dr. Scandura:
Well, one of the risks with AFib, some of them can be just related to the heart, it can disturb a little bit in how the heart functions and if people have some mild symptoms, AFib can make symptoms worse just from a heart function standpoint. But one of the things that’s related to, again some of the commercials you see on TV and the rationale for blood thinners, is the heart – the atrium, the left atrium which is really what fibrillates, which is just – normally the heart is pumping like this, all together coordinated. And what fibrillation means is it’s sort of not doing that. It’s going like this and what happens is the blood and the surface of the heart ends up not being pushed out normally.

And sometimes actually clots can form on the surface of that fibrillating heart and then when they get pushed out, they can travel. And because it’s usually the left atrium where this happens, when they travel they go into the arteries and then they can form clots and that can be stroke is the big thing people worry about. So, you can have atrial fibrillation that puts you at risk for stroke and that’s why people think about anti-coagulation medications to prevent that risk. And so, again, that’s another rationale for blood thinner, although it has nothing to do with the blood being too thick. It has to do with atrial fibrillation itself.

Andrew:
Okay. So, two things going on. Here’s a question we got in from Julie. Julie says, “What is the significance of a very low allele burden in a JAK2 positive patient?” And may you could define allele for us too.

Dr. Scandura:
Sure. So, as you know, we have some of our genes from mom and some of our genes from dad and the genes that we get are always in these two copies. And so, one copy from mom, one copy from dad, and they’re mixed and matched while we’re being sort of grown up from the embryo. But what happens in MPN is sometimes one of those copies, always starts with one of them, becomes mutated and that can be for instance, in the most common mutation, in the JAK2 gene, JAK2 V617F, a particular mutation that’s associated with abnormal function of the JAK2 gene product. And so, if we have just one copy in a cell, then one copy’s normal and one copy is mutant.

So, if we are talking about that one cell, that variant allele frequency, so that’s the abnormal gene. The proportion of all the genes that are abnormal would be 50%. Right? One abnormal, one normal. But now we think about all of the blood cells, trillions upon trillions of blood cells and then we have to take sort of an average of all of those cells. Some of them will be normal, some of them will be MPN cells, some of them will have one copy normal, one abnormal, some two abnormal, and some both normal. And so, when we look in a composite from a blood draw which is generally what people are sending, it’s a representation of how many abnormal alleles are present among all of the alleles of all of the DNA from the blood cells that’s been selected.

So, what a low variant allele frequency means that the proportion of mutant alleles in that sample of your blood is low. So, low would be maybe 10% or 5% or something like that and what is the significance of that? It’s an area a little bit of some debate, but there’s certainly a number of studies that have shown a correlation between the variant allele frequency in blood and the disease type itself. So, for instance, essential thrombocythemia, or ET, generally has a lower bearing allele frequency than myelofibrosis for the same mutation. And polycythemia vera is often in between.

Andrew:
While we’re talking about genes, I just wanted to bring in this question from Jocelyn because we’ve been learning are we JAK2 positive, are we CALR positive, these others that you’ve been discovering. So, Jocelyn said, “In 2006 I tested positive for JAK2 V617F. In 2018 I was told that I’m not JAK2V617F positive, but that I’m CALR positive. So, is it common for mutations to change and what does it mean?”

Dr. Scandura:
So, it’s not common for the mutations to change in terms of going away if they’re present, although there are certainly examples of this happening. It’s not common. What is probably a little bit more common is sometimes people have one mutation or a couple mutations and then sometimes more mutations are found later. And that often, not always, is linked to the disease changing its character itself. So, somebody with polycythemia vera having more of a fibrotic phase of the disease. In this situation, it’s a little hard to know exactly what happened, but there is a fair amount of variability from one laboratory or one test type to another in terms of sensitivity and the specificity of what is being detected.

So, JAK2 may have been at a very, very low level, could have been an erroneous measure, or it could have been at a relatively low level and the calreticulin mutation wasn’t tested for. And then later somebody retested with a different test that wasn’t sensitive enough to pick up the JAK2 mutation and they looked for a CALR and now that’s coming up positive.

So, the testing modality, the type of test that’s being done, and its individual sensitivity is an important part of this story and it’s a little hard, I think even for many physicians, to sort of get their heads around because it’s not like a blood count where you have international standards and basically a half-dozen equipment makers everybody uses across the world. There’s a lot of different technologies, each of which have little wrinkles to them that can limit somewhat exactly what’s being reported.

Andrew:
Okay. Here’s another one we got. This was actually asked by several people. Nick, Maggie, and Philip all want to know related to phlebotomy. What are the goals of phlebotomy as a treatment and how does it work and when do you know when it’s time to switch from phlebotomy to medication?

Dr. Scandura:
Right. So, I just came from a conference the end of the week and this is a topic of debate among physicians. When, whether to do phlebotomy? Whether phlebotomy therapy by itself is sufficient? What are the alternatives and when to make those decisions? I would say, I can tell you what my own feeling is. I feel that there is good support to justify that, but to be totally honest, there are physicians who feel differently than I do and I don’t know if any of us can claim to be absolutely correct. But I think we can all agree that the goal of phlebotomy in the short term is basically to take cars off the highway.

If you go back to the analogy of having too many cars on the highway causing thickened blood or this sludging from the red blood cells, this is a therapy specific to polycythemia vera, is that phlebotomy is just a very simple way of taking blood out of the system, taking cars off the highway. So, if you were to imagine and I frequently imagine this in New York City, is all of the sudden a third of the cars disappeared, it’d be a lot easier to get around. And so, that’s really what the goal of phlebotomy is, is to make it a little easier on your body to pump the blood around because there’s less resistance to having all that traffic in the vessels. How much? Go ahead, you had a question.

Andrew:
I was just gonna say, but debate about when to leave phlebotomy behind and have medication try to do the job when you prove one or others that may be coming.

Dr. Scandura:
So, I think the first goal is to get people under what would be considered control. So, an adequate level of traffic. And the numbers that are generally accepted by people in the field is having a hematocrit, that’s the portion of blood occupied by red blood cells, in males it’s below 45% and in females below 42%. Although we can all argue about that a little bit. I think people settle down around those numbers.

When is too much? My personal feeling and this is where there isn’t great data, so you’re left with opinion, but my personal feeling is it depends a little bit on the patient, the convenience, and I find that people who are getting phlebotomy more than four or five times a year, it ends up being a real burden on them in terms of the amount of time that they’re having, poor control of their polycythemia vera, and the amount of time required for phlebotomy, and the amount of risk of things like iron deficiency which can cause symptoms.

And then there’s some suggestion, I wouldn’t say great data, that maybe iron deficiency or repeated phlebotomy may be a risk in the long term, although I think that data is not very clear. My biggest determinant is patients, in my experience, just get a little fed up with getting phlebotomy when it gets above four, five, six times a year.

Andrew:
Okay. Thank you for that. I should mention to our audience again if you want to send in a question, whether we can use it on this program or a whole bunch we’ll be doing coming up, send it to mpn@patientpower.info. Okay, so here’s a question we got from Nick and all of us wonder about it. How often or do we need bone marrow biopsies so that you, as our doctor, and we are well informed about what’s going on?

Dr. Scandura:
So, another area where there isn’t – you know, in medicine we look for the perfect data. We’ve controlled – we treated one group of very similar patients one way, we’ve treated another group of people another way, and we compare and see who does better. What’s the better approach? This hasn’t been done for how often to check bone marrow. I think bone marrow evaluation is very important. Personally, I generally follow how the patient is doing as the primary determinant and if there are any signs that something is changing. And those signs can be how the patient is feeling, new symptoms that are arising, but oftentimes it can be just in how the blood counts are responding.

You’re on a stable dose of a medication for several years and all of a sudden it stops working or all of sudden it starts working too well. You have very low blood counts whereas before you were okay. That suggests to me something’s changing. The bone marrow is the factory for all the blood cells. So, if you wanna know what’s happening with the production in the factory you really have to look into the factory and see what’s going on. And so, that’s my personal threshold for doing a bone marrow, when I’m seeing something that’s suggesting that the factory is not functioning the way it was the last time I looked.

Andrew:
Okay. And for those of us who’ve had many bone marrow biopsies, and I have, hopefully where it’s done is someone who does it frequently. Usually, the anxiety we have is worse than the exam itself. It takes 15, 20 minutes, whatever and someday I’ll tell you the story of the lady down at MD Anderson who believed in voodoo and talked to the bone marrow as she was pulling it out. And it was so weird, that I was so distracted, I didn’t feel a thing, but anyway I understand it’s important.

Here’s a question that will help our friends with ET. This is from Michelle. She says – well actually now she has post-ET myelofibrosis. She says she has ASXL1 and TP53 gene mutations. Does the mere existence of these predict aggression and poor outcome? That’s what she worries about that those have been found.

Dr. Scandura:
Well, obviously every individual has their own history that they’re developing and so exactly what this means for you, for an individual, is different than what it would mean for a population of people with similar mutations. That’s really what we know in medicine. We look at people in a cross section and we say people who we can put into this bin tend to behave in that way, but within that bin, there are individuals who don’t act that way, the way that the others do. So, I would in myelofibrosis, in MDS, in polycythemia vera, P53 mutations are an area of some concern, as is ASXL1 mutations are also an area of some concern.

In ET it’s less well established and so I think because, if this was just ET and you had those mutations, I think many people, myself included, would say well, maybe we don’t know perfectly, but it is an area of some concern. I’m gonna keep a closer eye on you. Now that it has already evolved into myelofibrosis, I would say this is probably more like myelofibrosis where we know that P53 mutations, TP53 mutations, and ASXL1 mutations, can sometimes be some of the harder ones for us to treat. It’s something that, if an allogenic transplant is something that is possible, should at least be considered and discussed.

It doesn’t – speaking with a transplanter, getting typed doesn’t mean you have to get a transplant, but it gives you information and so I think that that would be a reasonable thing to do. Again, the decision at the end, it may not be the right decision for you, but it is something that is information for you to use in making informed decisions.

Andrew:
Right. I did have a consultation with Dr. Castro, who was at the time here in San Diego, exactly about that. Not to take action, but just to have the relationship and be typed, et cetera. Here’s a question we got from Paul. He says, “I was diagnosed in 2009. I take a weekly dose of 90 mg of interferon. How long can a patient continue to take interferon and what indicates a move to change treatment?”

Dr. Scandura:
So, we have people who have been treated for 20 plus years with interferon. So, I don’t know if there is a known duration which is too much. For many patients it’s a very well tolerated therapy, can be quite effective, and I think that it is one of the few medications that seems to have some disease-modifying activity. However, when to change? If it looks like it’s not working, it’s time to think about changing and that can be adjusting the dose, but I think if somebody has been on it for a long while, that’s when I think thinking about additional therapy, either adding another medication to the interferon or changing completely to a different medication.

Clinical trials, there’s a lot of activity in MPNs in clinical trials. Thankfully, over the past five years or so, it’s really been increasing. There’s a lot of options. There’s some drugs that we’re really pretty excited about right now in terms of thinking they might have some nice activity and talking to somebody about what might be a suitable treatment for you if the interferon was not working anymore.

Andrew:
Okay. Here’s a – again we’re getting similar questions from a number of people. So, Ragita, Nankin, Raven if I’m saying the name right, and Jacquelin sent in basically this question. How common is it in patients with MPNs to have bone pain? What causes it? Is there anything that can help with the pain?

Dr. Scandura:
So, bone pain is always on the list of symptoms reported by patients with myeloproliferative neoplasms. I wouldn’t say, in my experience, it’s one of the more common ones. It might be a little bit more common early in disease. Sometimes things like phlebotomy that you can actually have a rebound where the bone marrow is a little bit revved up to try to replace all those cells that were taken out, that can cause some bone pain. It can be seen in myelofibrosis occasionally and sometimes when the disease is becoming more aggressive or is having a – changing its pace. But the cause of bone pain, we think of as being related to sort of expansile pain.

So, the bone marrow, the factory for all the blood cells, sometimes is just working so hard that it causes, it irritates the bone fibers that are around the surface of the bone. There’s very little in the way of pain fibers inside the bone, but on the surface of the bone you have a lot and that expansile pain, that gives that sort of vague, achiness people often describe as bone pain. The treatment for bone pain in some ways is determined by what the cause is. If it’s just, for instance, a rebound after phlebotomy, it can last a day or two and then go away. And so, short-term symptomatic treatment with non-steroidal anti-inflammatories, NSAIDs like Motrin, can be helpful or Tylenol even.

But occasionally patients report a real benefit from things like histamine blockers which the mechanism for that is entirely unknown, but there’s certainly a population of patients who feel like the bone pain has gone away with medications like Claritin you can get over the counter. It’s worth a try. They are very well tolerated medications and not all patients have any symptomatic benefit, but a subset of people do. If the bone pain is related to the cells being too active, a very proliferative feature of the disease, sometimes it dictates treatment.

So, if you were on phlebotomy alone, well maybe it’s time to change to a more cytoreductive therapy and see if that can help with the pain. Sometimes it prompts additional evaluation. If you’ve never had bone pain, all of a sudden the blood counts are a little different, you have bone pain, it might be something somebody would think about doing a bone marrow evaluation for. Again, looking in the factory which is probably where the cause is coming from.

Andrew:
I have a couple more topics I want to cover just before we close. We’ll go just a little bit longer if that’s okay. So, Robert wrote in and said, “How does a stem cell transplant cure myelofibrosis?”

Dr. Scandura:
So, I’ll go back to that factory analogy. If you think of the bone marrow as being sort of corrupted by these MPN cells. You have, normally this is a very orderly factory. It’s producing a number of different lines if you think of it as a car factory. You can be producing red blood cells maybe your sports cars, and your white blood cells, your infection-fighting cells, as sedans, and platelets as SUVs, but it’s all very orderly and it should be proceeding in a regular way. And you get MPNs and somebody has just turned up the volume and are just cranking out a lot of cells. And sometimes that production starts becoming abnormal too and that’s more like in a myelofibrotic setting.

And so, what is the point of a stem cell transplant is really to clear out that factory, get rid of all the workers in there, and replace them with completely different workers to come in, set up shop, clean up the factory, and start normal blood cell production. There’s another part of it is, it’s not just the blood cells, it’s actually the immune system. And so, you’re giving the recipient an entirely new immune system. You have to wipe out the old immune system to allow the new donor cells to get a hold in the bone marrow and then they have to be educated to sort of relearn how to fight off infections and to figure out who is who.
So, graft versus host is one of the complications where those cells from another person come into the recipient and say, “Ah, I don’t know you. I’m going to attack.” And so, that can be a problem. It can be a short-term problem. It can be a long-term problem. It can be mild and it can be severe, but there’s another edge of that sword which is what we think of as graft versus leukemia effect, or in this case it would be graft versus MPN effect where some of those donor cells recognize the little differences between them and the MPN cells and wipe them out. And so, that’s really what you’re trying to do is allow that new immune system to find the bad actors and wipe them out.

Andrew:
Okay. You touched on something I think we’ve got to ask about and that is people are hearing in the blood cancers now the experimental and in some cases an approved approach called CAR-T, chimeric antigen receptor T-cell therapy, but again immunotherapy to train the T-cells to fight your ailment. What do you think about that in MPNs? Does it have promise?

Dr. Scandura:
I mean, it definitely has promise. It’s been a challenge in myeloid disease as a whole, so AML, MDS, MPNs have not been the first diseases where this has been shown to be successful, more lymphomas where it has had a lot of traction and some nice responses. What it really is it’s a living drug and this can be done in a couple different ways. They can be cells from yourself that then are treated in the laboratory so that they start recognizing these immune cells. You start tricking them into saying, “I’m going to attack this particular thing.” Even though they weren’t really trained to do that, they are now being tricked into doing that.

And so, in a disease like a B-cell lymphoma, most of them express a particular protein that’s on B-cells, CD19. So, if you take these CAR-T cells and you say, “Well, go out and kill everything you see that has CD19 on it”, it will wipe out a lot of those lymphoma cells. In myeloid diseases like MPNs, it’s a little harder. The targets are not so clear-cut and they’re shared with normal cells. There’s one area where I think it has the most promise is calreticulin because the mutation in calreticulin isn’t a tiny little mutation. It’s a mutation that causes a whole new end of the protein that doesn’t exist in the body otherwise.

And some of the calreticulin actually gets onto the surface of the cells so it’s displayed to the immune system, and so this is an area where I think there’s some promise for CAR-T cells to target those calreticulin mutant cells. There may be other targets as well and I think we’ll learn as time goes along. People are trying to target molecule CD123 which is expressed on certain abnormal stem cells. The problem is it’s expressed at relatively low levels on those cells. It’s also expressed on normal cells and it’s expressed at higher levels on much more common cells. So, it makes it a somewhat imperfect target, and also difficult from a drug standpoint because there’s a lot of people wearing the same mask, only some of them you want to kill. So, it can be a problem.

Andrew:
Okay. You have quite the analogies. But, I’m just gonna ask you about two more questions and then we’re gonna have to go. This came in from Linda who says, “I am CALR positive and I have many symptoms. What causes vision symptoms for me and migraines? Can that be tracked to the CALR somehow?”

Dr. Scandura:
It’s common in a subset of people with MPNs. Sometimes it’s linked to the platelets themselves, to the white blood cell count, so I would certainly unless there’s a reason not to try aspirin, that’s something that can help with patients. It may also be an indication for cytoreductive therapy, so actually trying to lower the blood counts. I don’t know exactly what disease that Linda has, but it’s one that I would think is a symptom that would warrant therapy because it can be quite bothersome.

The vision changes is something that may be related to the migraines, but it’s also something that might prompt a visit to an ophthalmologist so they can actually look at the blood vessels in the back of the eye and sometimes what happens is you can have a little irritation of the blood vessels or even clots in those blood vessels and that’s something that would definitely trigger a change or new therapy.

Andrew:
Dr. Scandura, our audience is saying, “Please, one more question, one more question.” So, if I can a couple more. Philip said, “Is iron deficiency a new normal if you have PV and you’ll, therefore, have weakness, fatigue, maybe even some cognitive issues because of anemia as well?”

Dr. Scandura:
Yeah, so I sort of fall in the camp, as I mentioned before, there’s some debate in the field and I sort of fall in the camp that if you’re getting symptoms from iron deficiency, it might suggest that something other that phlebotomy could be beneficial or could relieve that symptom. Everybody, if you take enough blood out of them, is going to become iron deficient and, in fact, most people diagnosed with polycythemia vera, if tested, actually meet the criteria for iron deficiency, not because they actually don’t have enough iron in their body, but because all of the available iron is soaked up in making red blood cells. Red blood cells are red because of iron.

So, if you think about all of the iron in your body and all of the places it’s used for metabolism and everything else, there’s a lot of enzymes that actually use iron as part of their catalytic site. A large proportion of all of the iron in our body goes to making red blood cells. In polycythemia vera, that regulation is completely abnormal and you end up just making a lot of red blood cells that aren’t needed and it soaks up all the iron. Then when you start doing phlebotomy, you’re taking all of that extra iron and you’re taking it out, but the bone marrow still wants to try to make red blood cells. So, it continues to scavenge as much of the iron as it can.

So, iron deficiency is pretty common and if you need a lot of phlebotomies it’s universal. Some patients, in my experience, meet all the criteria for severe iron deficiency have very little in the way of symptoms. Others meet criteria for mild iron deficiency, but they’re quite symptomatic. And so, in those instances, you need to individualize a little bit. At least give a try to a different therapy and allow the iron stores to normalize and see if that improves the symptoms.

Andrew:
Okay. You used the word individualize and that’s where I wanted to wrap up. So, you alluded to earlier, that you were encouraged by new medicines coming for MPNs and you have your whiteboard behind you where you’re charting things and I hope, Joe, coming up with a cure of tomorrow for all of us. How encouraged are you in the near term and the longer term for beating back or even curing these diseases?

Dr. Scandura:
I think we’re gonna cure these diseases, I do. I don’t know if it’s gonna be this year, but I think that the number of tools we have to understand how these diseases work and the number of new drugs that are being developed that have real promise, like real mechanistic reasons why they should work, I think is going to yield, reap rewards over time. People have heard this for a long time. The war on cancer has been going on for a long time, but I think we didn’t have the tools that we have now for that entire duration. Right now, we can sequence a genome in a week of a person. Now, do you need to do that? No, but it allows you to get a level of information that was in the past, really just fantasy world, science fiction, and now it can be done on a routine basis.
There are, virtually all of our patients, have sequencing for 40 plus genes. It allows us to know a little bit more about what their risks are, and also gives us a spectrum of targets to start hitting. There’s models that are better than what we’ve had in the past for many of the cancers that have been targeted. Breast cancer models, you know, there’s some decent breast cancer models, but they’re very complex tumors. MPNs, for better or for worse, if you look at the spectrum of genetic complexity, they’re really pretty simple meaning that they have one to half a dozen mutations.

Now mutations aren’t the whole story, but it’s a good starting point and if you only have maybe 10, 15 genes that are currently mutated in a disease, it’s trackable. You can figure this out. You can figure out what they’re doing to allow them to win and once you know that, you start figuring out how to beat them back. And so, I think that their time is gonna come. I don’t know if it’s this year as I said, but I think it’s definitely doable.

You know, CML, when I was a kid, when I was in medical school my parents had a good friend with CML who died with CML. Now, it just wouldn’t have happened. He would have been fine, but he was on, for a long time, ineffective therapy, transformed to an acute leukemia as they all did, and then it becomes really untreatable. And now we have these magical drugs, semi-magical drugs, that for the vast majority of people just – it’s a pill a day. It’s amazing.

Andrew:
Well, you’ve got that work on your whiteboard and in the lab and your colleagues around the world and you had told me before the program started that you all are collaborating better now than ever before. So, Dr. Joseph Scandura from Weill Cornell in New York City, thanks for what you do as a physician-scientist and thanks for spending time with us today.

Dr. Scandura:
It was my pleasure and thanks for helping patients through what is a difficult ordeal I think in terms of adjusting to a diagnosis and getting information.

Andrew:
Well, thank you for joining us and Weill Cornell folks have been great and send our best to Dr. Silver too. He’s in his 90s and still going strong.

Dr. Scandura:
Yeah, he’s traveling today.

Andrew:
Thank you so much for being with us for this Patient Empower Network program. Thanks to Incyte for helping fund our series. We appreciate their commitment to the MPN community and as always, I just sign off by saying I’m Andrew Schorr and remember, knowledge can be the best medicine of all.

Ask the MPN Expert – Dr. Naveen Pemmaraju

Ask the MPN Expert – Dr. Pemmaraju

“Ask the Expert” session with MPN specialist Dr. Naveen Pemmaraju from The University of Texas MD Anderson Cancer Center.


Transcript:

Andrew: And greetings from near San Diego, Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this patient empowerment network program, and thanks to Incyte Corporation for helping make it possible. They have no editorial control. I’m a myelofibrosis patient, have been for several years, so I’m vitally interested in this. Welcome to our MPN community, and welcome to one of our favorite experts joining us from MD Anderson Cancer Center in Houston. There’s Dr. Naveen Pemmaraju. You can see behind his desk there all the working on the cures of tomorrow, but Naveen, thank you so much for being with us.

Dr. Pemmaraju: Thanks for having me, Andrew. My pleasure.

Andrew: Okay. Let’s get right started. We’ve gotten all sorts of questions in. If you have a question, send it to MPN@patientpower.info, but we’ve gotten a lot already and I’ll start to buzz through them over the next 30 minutes. This question is from Cynthia and she says, “I was diagnosed with ET (Essential Thrombocythemia). I’m JAK2 positive,” so she has that JAK gene. “When I was 66 years old,” now she’s 68, I’m about to be 68 as well, “What markers on my blood work, asides platelets, are important for my doctor to watch? What indicates a need for another bone marrow biopsy?”

Dr. Pemmaraju: Well, thanks, Andrew. And thanks to the question from Cynthia out there. This is very important. This is what we talk about day-to-day, week-to-week in the clinic. There are a couple of perimeters outside of the platelets. One, I would say the most important for us to watch are the other of the big two. That’s your hemoglobin number, also known as anemia; if it’s too low, or polycythemia, if it’s too high, and then the white blood cell count is also very important. Again, if too high, or too low, it can tell us what’s going on.

With ET, the key thing is it can transform, or change into any of the other MPNs. For example, PV (polycythemia Vera), myelofibrosis, or – and I hate to mention it, but it does happen five, maybe seven percent of our patients, where the disease can go to Acute Myeloid Leukemia, AML. So, distinct blood count changes, either too high, or too low, can give us clues if the MPN is changing, or in fact, going to AML.

And so, the answer for a repeat bone marrow is based on that, which is, let’s look together, patient and provider to see if there are subtle or avert changes in the blood counts that are markedly different from the previous visit, rather than having a pre-prescribed, every three months, or every six months type of a deal.
Andrew: But, Naveen, with all you’re doing now with sophisticated testing, do you still have to poke us in the hip, or couldn’t they just do it from our arm?

Dr. Pemmaraju: I wish, Andrew. I think this is very important. I think with the juxtaposition, you have this sophisticated gene panel testing, JAK2 CALR, MPL, and yet we’re still sticking a needle in people’s backs in a very painful procedure. Nothing still has overmatched as the gold standard, the bone marrow aspiration biopsy. So, for now, we’re – pun intended, I guess – stuck with this procedure. But your point is a good one. For example, with bone marrow transplant, can you believe it nowadays, they’ve moved from not having to exclusively do it from bone marrow source to peripheral blood, so I think you’re on the right track and we need to work on different ways of accessing this important information.

Andrew: Okay. One thing about bone marrow biopsy, it doesn’t have to be painful. It’s uncomfortable, but it doesn’t have to be painful if you have somebody experienced doing it.

Dr. Pemmaraju: I wanna emphasize how right that is because at least here, at our center at MD Anderson, as you know, we have a team that is dedicated to doing it many, many people, many repetitions doing it, so there might be local discomfort, but a lot of our patients do not experience pain. I’m glad you brought that up.

Andrew: Right. And that’s been my experience both there, and at other major centers. Okay. Here’s a question from Denise. Denise says, “I have PV and I’m trying to improve my health by making smoothies containing large amounts of dark green vegetables, such as spinach, kale, and watercress. I’ve been warned by some members of our community that these foods will increase iron and raise the hematocrit, putting me at risk. Is that true? And should people with PV avoid these foods that are high in vitamin K?”

Dr. Pemmaraju: Well, this is an important question and I remember five to 10 years ago we would say things like, “Well, we don’t really know the answer,” or you know, “Diet doesn’t really have anything to do.” But now with more and more understanding of the total therapy for patients and approach to the whole body, I think this is an important question. So, yes, iron levels do matter. Too low, then you’re iron deficient. (That can definitely happen in our patients.) Too high, potentially may fuel the fire, if you will, for polycythemia Vera.

So, I think iron levels are important to watch and certainly can be increased by what our question is being asked about. But there’s another aspect, too, that some of the medications that we prescribe and take. One example is Coumadin, or Warfarin that a lot of our patients know, which is a high-level blood thinner. It’s an anticoagulant. And man, oh, man, that is exquisitely dependent on the vitamin K pathway. So sensitive, that in some patients in some cases even salad consumption, or spinach, so healthy foods because of the vitamin K level in them can alter this level. It’s called the INR. And so, it’s something we have to watch out for.

So, not only in terms of iron metabolites, but also drug-to-drug interactions. So, it is always best to mention these things when we’re going on new medications.

Andrew: Right. Talk to your doctor.

Dr. Pemmaraju: Talk to your doctor.

Andrew: What you’re doing –

Dr. Pemmaraju: Everything.

Andrew: – what you’re eating. Yeah. Okay. Here’s a question from Sally. Sally says, “I have ET with the MPL mutation. So, I have JAK, but there’s also MPL. I believe, not much is know about my mutation. Can you shed light on it, or me and our community here today?”

Dr. Pemmaraju: Yeah, great question. So, when I look at these mutations as the big three, I go back to the time of William Dameshek, who hypothesized in the ‘50s and ‘60s that MPNs would be a unified group of diseases; ET, PV, and MF. And now, 67 years later, we’ve proven that. So, JAK2, we’ve known about since 2005. The most common, most major recurring mutation, fifty to 60 percent of patients of myelofibrosis. Then in 2013, 2014 the CALR mutation was elucidated. Can you believe, that’s only been four, five years. That’s the second most common. But there’s a third of the big three. That’s the least common, the MPL; MPL mutation.

That’s a mutation in something called the thrombopoietin receptor (TPO), which is in charge of helping to stimulate and make platelets. So, in terms of MPN patients, it does make sense and it has something to do with platelets, and that axis. It is the least common; by far the less common of these three, so I would say maybe something to the point of three to seven percent of our patients will have it.
Up until recently, we didn’t know if it had any prognostic significance, but our Italian colleagues published a very nice paper in Blood a few years ago, independent of the IPSS risk, that I’m sure we’ll talk about later. That if you just take patients with myelofibrosis, not ET and PV, you can stratify our patients based on the mutation risk. And not everyone knows about this.

For example, in this scoring, CALR mutation alone is the best prognosis for our patients. JAK2, or MPL is what’s called an intermediate prognosis, and the so-called triple negative, if you don’t have any of these big three, the implication being that you likely have something else, like ASXL1, then those patients tend to have the worst prognosis. So, MPL helps us to diagnose and confirm an MF diagnosis, and it also may have prognostic significance in our modern era.

Andrew: Okay. I don’t want people to freak out because this is a moving target as they learn and say, “Oh, my god. I have triple negative…

Dr. Pemmaraju: That’s right.

Andrew: Right. Okay? Because there’s progress going on all the time.

Dr. Pemmaraju: Well said.

Andrew: This is what they’re learning now. Okay. Now. Here’s the big one and you mentioned it. You said, a small percentage of us with myelofibrosis have the risk of progressing to AML. I know there been a lot of new drugs now either approved, or in development for AML. And some that help people who have secondary AML.

Dr. Pemmaraju: Oh, yes.

Andrew: Right? But tell us about the risk of progression, and then what do you do about it?

Dr. Pemmaraju: Right. So, that’s a great topic, and as you know, you’ve been doing this for so long now. This has often been a very not great part of the conversation, but it’s an exciting time for our patients based on the research I’m about to share. So, the first part is progression to AML, which we touched on earlier. We do have an objective prognostics scoring system. Actually we have so many scoring systems now that many of our patients are starting to see them.

But the IPSS, or International Prognostics Scoring System, was first developed by our colleague, Cervantes et al, this is right before 2010, so ’07 to ’09. That one is supposed to be applied at diagnosis and based on five of these risk factors, we can prognosticate, or tell which of our patients are at a higher risk for AML. And so, a lot of our viewers ask that. So, it is true. Age – over a certain age, white count of 25,000, circulating blasts, constitutional symptoms, and anemia made up that original five.

Since that time, there are dynamic scoring systems, DIPSS, DIPSS+ and others that include, or modify as some of those risk factors. So, we can tell, at least based on a textbook impression, who has a higher likelihood of going to AML. Once our patients go to AML, there’s a lot of hope now. There have been four – count them – four new FDA approvals for AML in just the last 12-24 months. They apply to different segments of AMLs, two of them are targeted therapy, so one drug called Midostaurin hits the FLT3 inhibitor, one drug hits the IDH1 and 2. Actually those are two separate drugs.

The drug you were referring to had a code name CPX-351, or VYXEOS, and it does have an approved label for so-called secondary, or therapy-related AML. Although I will caution our viewers that – MPN patients, specifically, we’re not included in those early data sets. They were more geared towards patients with MDS leading to AML, but the principle is there for us.

And then finally there’s another drug called Gemtuzumab or Mylotarg. So, you have four FDA approvals, ongoing clinical trials with combination therapy, excitement and ongoing investigation for CAR-T cells, optimizing stem cell transplant, and then combining possibly MPN drugs with AML drugs in a clinical trial setting. So, I think this is actually a very, very important time to talk about AML in all of our MF and MPN sessions.

Andrew: Okay. Just one brief question, and – if someone like me, where I’m on Jakafi myself, but if that sort of poops out, or that, or another medicine is not working for me, and I’m developing AML, do you feel now it’s a more hopeful time than it’d had been previously?

Dr. Pemmaraju: I do, Andrew, and you know me. I used the word ‘hope’ very seriously and very carefully. Before with AML – and I mean, just five to seven years ago, it was not as hopeful of a time for us, as researchers, and for our patients. Not just because of the FDA approvals. That obviously is very encouraging and applies to a lot of our patients, but also because of the funding, the research, and the ideas for combination chemotherapies, and the emergence of these immune therapies. I think it’s a hopeful time for all of us involved with AML.

And specifically as you were mentioning this secondary, or post-MPN, or post-MDS AML, which is largely been an urgent unmet medical need.

Andrew: Okay. And just to everybody understands, AML, Acute Myeloid Leukemia, so it’s acute and as has been in the past a five alarm fire and now they’re developing medicines for that. All right. Let’s go on. Bonnie has a question. “I have myelofibrosis and my only symptom is that my spleen is enlarged. I’m on Hydroxyurea, but hesitant to switch to Jakafi or Ruxolitinib. Assuming no real discomfort and just moderate weight loss and stable blood counts, does an enlarging spleen itself cause problems?” And also, I guess the bigger question, Naveen is, is there a penalty for waiting if there is a treatment that might line up with what you got?

Dr. Pemmaraju: You’re right on. Yeah. This is very, very serious, important topic. So, for the first part of the question, the spleen itself being enlarged can in fact cause some really, really big problems for a lot of our patients. It is true, as the questioner’s asking, that one can have mild splenomegaly. So, a spleen that’s slightly enlarged, not yet causing physical symptoms of early satiety, which means getting full fast, or physical discomfort, but a lot of patients do have that. So, yes, a big spleen alone can cause not only local problems, but also systemic. Because again, it’s a disease of cytokines; messengers and proteins that are being scattered all throughout the body, causing the body to feel flu-like symptoms, or fatigue.

Now, the studies for Ruxolitinib are very specific. These are two Phase 3 studies, they are called Comfort 1 and 2, published in the New England Journal five six years ago now. And they did include patients with intermediate to, or high-risk disease. Or intermediate to high-risk disease, shall we say. And although the spleen itself doesn’t come out in the scoring system, some position is that it should be patients with more advanced, or higher scoring diseases than say someone with lower risk disease.

So, with the trial data that we have we know a couple of things. 1) The drug got approved in those more advanced patients. 2) There was early crossover that was allowed. So, one of the comfort studies, Andrew, was as you know, randomize to placebo. So, no active therapy, and one was best available therapy. Even with the early crossover allowed to the Ruxolitinib, both are not showing overall survival benefit. Translated into more layman’s terms, what that means is, it does appear, maybe, possibly, that if you got the drug at the beginning early on, there looks to have been some long-term benefit.

We won’t know that until further studies are done, and those studies are being planned. Those are called ‘early intervention’ studies, so people at a lower, earlier stage, low risk, Intermediate 1, and I think we’ll all be eager to see how that benefits our patients.

Andrew: Okay. Great. So, the penalty for waiting – right now it appears there could be a penalty. In other words, it could be better to get on it. Don’t wait. If you and your doctor agree, there’s a therapy for you.

Dr. Pemmaraju: I think that’s exactly the resource position to take, which is I think that – I believe that there are a subset of our patients who exactly fit what you said. They are symptomatic, out of proportion to the risk scores that are available. The spleen is highly enlarged, although they have lower intermediate risk by IPSS. And that’s exactly the are of investigation for myself, and our colleagues. And even in the clinic there might be some role to assessing patients as you said like that. So, it shows you the limitation of these text book scoring systems and how much research we have left to do.

Andrew: Okay. Well, you know more than you did before, so, I –

Dr. Pemmaraju: Well said.

Andrew: – I’m happy about that. Okay. So, here’s a question from Jane. She says, “I have myelofibrosis, but it’s not progressing, and I’m CALR negative.” So, that’s one. “I’m JAK negative.” That’s two. “And I’m waiting to hear if I’m actually triple negative, as you said, which would be JAK, CALR, and MPL. Are there medicines to slow progression for me?”

Dr. Pemmaraju: Well, that’s the ultimate question. Isn’t it? So, the first concept is this triple negative. And if our viewers have heard that before you have, that was borrowed from the breast cancer literature, which was a similar sentiment, which is having the top three markers negative. And just in that case, as in RMF, the supposition is the same, that that means that you have a higher risk disease.

But going from negative to positive, what it does mean now with the new sequencing and molecular studies that are coming out, is that it really looks like 90 percent, maybe even close to a 100 percent of patients, have some form of a molecular driver. And those other mutations you’re going to start to hear about are becoming common; ASXL1, TP53, EZH2, IDH, etc. etc. So, triple negative may mean that we don’t have those big three, but there might be something else that’s driving the MF, and it means that it’s a higher risk to progress to AML and for some patients to not do as well.

But this questioner brings up a very good point. What the textbook risk score says does not have to imply to each individual patients. So, just because the finding is that, okay. Triple negative patients as a population may do worse, it may not apply to that individual patient. So, in this person’s case, maybe they’ve been diagnosed very, very early. That’s a good thing. Maybe the driver mutations and the triple negative matter, which is what I think. So, ASXL1 mutation vs. some other ones.

And then finally, each patient is different. Everyone’s case is different. You have other co-morbidities, other underlying drivers of disease. So, I think that’s the good point. But, we do have to say, at least for right now, I like your phrase ‘of a moving target’. The understanding that if you are this triple negative disease in this classical sense, should mean that you are a higher risk at some point to progress, as compared to others in your group, and so, possibly closer monitoring and observation is necessary.

Andrew: Right. And see an MPN specialist. Because what if there’s a drug in development that’s an AS – What is it? AS –

Dr. Pemmaraju: ASXL1.

Andrew: Inhibitor. And that’s driving your bus. Right? Maybe you wanna be in that trial. [

Dr. Pemmaraju: Absolutely right. Clinical trials are important for all of our patients with any rare cancers, or any cancers in general.

Andrew: Right. Okay. Let’s go on. I just wanna take this question from Susan. It really rang true for me. Susan writes, “Is it common for an ET patient to experience numbness in the scalp, ears, and face? I’m currently on 1,500 milligrams of Hydrea daily.” And I wonder if you can broad this out because I was telling you before the program, I’m getting every once in a while – I wake up with a little prickliness. Not itchy, and I go back to sleep, but is that related to my MPN? So, she has scalp questions, is it the MPN, ET whatever? Is it the medicine?

Andrew: This is coming up in my clinic on a weekly basis. The short answer is, yes. It’s always due to the MPN. And I’m here to tell you why. This is an underappreciated part of what we do as healthcare providers in patients. For anyone who’s ever filled out the Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score:, developed by Ruben Mesa, his colleagues, now shortened down to a nice, nifty 10 ques – sheet. You know, actually, peripheral neuropathy is one of those 10 questions. Do you have numbness and tingling? So, even though we don’t talk about it, that’s our fault, peripheral neuropathy is a thing. It’s a common aspect of the MPN.

No. 2 is – and you brought this up to me nicely as well, is some of the mediations that we prescribe at the chemotherapeutic level can also cause nerve damage and neuropathy. JAK inhibitors either as a class, or some of these individual ones, both FDA approved in clinical trials have been associated with either a central, or peripheral neuropathy. So, I think that’s another big aspect.

And then finally, I hate to say it, but these drug-to-drug interactions of all of these medicines can cause that. When you factor that, plus vitamin deficiencies, thyroid deficiencies, iron abnormalities, restless leg syndrome, our patients have a host of reasons to have neuropathy. Usually this is an intermittent phenomenon, a come and go phenomenon. When it starts to become more of a permanent phenomenon and progressive, that’s a big concern, and that would really require a separate neurologic work up.

True, there are some chemotherapy drugs that can cause that, but I would say that would necessitate a multi-disciplinary approach; neurologists and all of this kind of thing.

Andrew: Okay. You used the name of a drug that people take. Is a blood thinner, Coumadin. Somebody may take diabetes medicines, I also have Chronic Lymphocytic Leukemia and take medicines for that. Okay. So, if we’re developing some of this and we have an MPN, which of the many doctors we have doo we go to first – do we go to you as our MPN specialist? Do we start there?

Dr. Pemmaraju: Yes. Absolutely. I think the phrase and the motto of every MPN expert that you’ll meet (and you and I know all of them now) is, ‘Tell us everything.’ Because I will tell you what. Now that we have more understanding – not full understanding yet, but more understanding of the biology of these diseases, it turns out that a lot of things that are happening are due to MPN.

One example I’ll give you, Andrew, our colleague and friend, Claire Harrison has pioneered this phrase called, ‘presenteeism’. Presenteeism. Not absenteeism, as we learned when we were younger. The concept that our patients with MPN are there, they’re here at work, with their loved ones, they’re at dinner, but they’re not really there. That’s also a question on the questionnaire; inability to concentrate. Subtle, subtle, subtle, but this is part of the MPN process. We’re not talking about it enough, but programs like this will get the message out there. So, tell your MPN doctor everything because more than likely they know it’s part of the MPN.

Andrew: I gotta tell my wife. I’ve been married 33 years.

Dr. Pemmaraju: This is all recorded, so you can tell her.

Andrew: All right. Esther, where are you? Okay. No. Let’s go on. So, Heather sent in this question. “My local hematologist, oncologist will only give me a phlebotomy after my hematocrit is over 51. What is the standard marker? I have PV and I’m really struggling with symptoms.”

Dr. Pemmaraju: I actually have data to share with you and your viewers. So, before four years ago, we did use to do it either based on convention, symptom burden, or a pre-designed abstract number. But now we have data. So, our Italian colleagues, Barbui and colleagues published in the New England Journal about four years ago a very nice paper that starts to answer this question. They randomize patients with P. Vera to two groups. They called it a liberal group, where you could get phlebotomies at any number essentially just like what’s being asked here, and then a more stringent group, which they came up with the hematocrit goal of 45 and below. Or below 45.

And the trial was actually stopped early because it showed a four-fold decrease in cardiovascular morbidity and mortality. That means, four times less chance of people having cardiac events or cardiac deaths in the stringent phlebotomy group. That is when you put the goal below 45. Yes, it’s only one study, but it’s with several hundred patients with P. Vera in a nice controlled situation. So, that has become a lot of us – for us, the de facto of standard of care.
So, I would advise, if you’re a higher risk patient with P. Vera, the so-called triple therapy approach, where you’re doing, you know, baby aspirin if you qualify. The phlebotomy goal of 45 and below, and then of course, cytoreductive therapy if you need it in the higher-risk situation. So, 45 and below, it should be validated, we should do more studies here in the States, but that’s something that I think we can use with high-level data.

Andrew: Okay, thanks. Here’s a question we got in from Kimberley. She says, “My daughter is 22, she was diagnosed in 2013 with ET, and she’s been on Hydroxyurea, but is decided she no longer wants to take the med. What should she be aware of, or cautious about, given that she’s no longer taking it?”

Dr. Pemmaraju: Ugh. Well, this is an area that’s very dear to me and very important to my research. With our group here, with Dr. Serge Verstovsek and my colleagues, we just published a paper on our experience with adolescents and young adults with MPN, or AYA. As its own separate field, AYA cancer has become a very important understanding that really didn’t exist, in my opinion, 20 years ago. But our patients are not always older patients. So, young patients can get MPN, too. Yes, patients in their teens and twenties can get them just like this questioner.

So, this is a type of patient that I’m seeing quite commonly in the clinic. Couple of points to say. One is, who can blame her? Who wants to take a life-long, indefinite oral chemotherapy that may or may not have short-term and long-term side effects? In our study what we found is, approximately 10 percent of our patients met this definition. The NCCN gives it, I think, age 16 to 39. So, younger than 40. And out of those patients, I was surprised to see that a good seven percent had a thrombotic event. That means a blood clot, either at the time of diagnosis just prior to, or just after. Well, that’s a pretty good clip, and that would be more than the general population than what you would expect.

The problem with the young patient with MPN has several issues. One is, what about at the time of fertility and pregnancy? Two, what about at the time of surgical procedures? I’m talking about routine things, such as dental and other care. And then three, as they start to transition into their older adult years. So, in this patient’s case, this is a very difficult thing. We don’t have many drugs. We have Hydroxyurea, we have Interferon, which possibly might be better for a younger patient. If someone has myelofibrosis, there’s no age requirements. So, if you qualify, then the JAK inhibitor, as a class.

But this is just one of those in-between, vulnerable populations, and we really don’t have great treatments for in general, an AYA cancer, and specifically here. And so, the main thing that we would say to this person is, really, really close follow-up early on with an MPN expert, as you always advocate. Two, is at the time of fertility planning, pregnancy in our family planning is to have high-risk maternal-fetal experts involved early on. (I think, this is something important.) And three, really cautious planning in and around surgical procedures, looking for bleeding and blood clots. I think those are some basic guidelines for anyone to follow.

Andrew: Well, great advice for mom and daughter. I wanted to post this, just a quick question from Caroline who lives in the United Kingdom is diagnosed with primary myelofibrosis four years ago at age 49. And she said, “I’ve tried to find others with myelofibrosis of a similar age, but so far no luck.” So, is being diagnosed at her age, age 49 with myelofibrosis, unusual?

Dr. Pemmaraju: There you go. That’s perfect. So, that also goes along with our “Young people get MNPs as well.” This was a disease – first of all a disease, now we recognize it as a cancer that was thought to be 60, 70, 80, 90 and older. And now we realize that there’s a significant subset of our populations diagnosed in their teens, twenties, thirties, and forties. So, we definitely want our question – our viewer to know, no, you’re not alone at all. Please, see our paper that we just put out there and several other of my colleagues, including Brady Stein and others.

Two is, my goodness. Not only you’re not alone, but I actually believe – and I know you know this too – that a lot of rare cancers are sometimes are under diagnosed and underappreciated. It does require expert bone marrow, expertise, someone to identify it, someone to do a bone marrow. And lastly, for this patient looking for other patients, I would refer them to sources, such as this one. Patient Power, support groups on Facebook, we have a Twitter feed, as you know, a grassroots Twitter, that’s investigators initiative called #MPNSM (myeloproliferative neoplasm on social media).

So, there are lots of different ways for this person to connect with not only younger patients with the disease, but also as a support group, virtually. And I think platforms, such as Patient Power, have frankly revolutionized the way people have obtained information, have communicated with each other, and specifically for a patient like this in the UK, who is not able to connect with me. And when there are people all over the world waiting to talk to her.

Andrew: Right. I wanna call at our friends in the United Kingdom, MPN Voice.

Dr. Pemmaraju: Oh, yes.

Andrew: It’s Claire Harrison, who you mentioned, wonderful, devoted.

Dr. Pemmaraju: Outstanding.

Andrew: She’s an expert, out of London, helps run it. So, please, connect with them. Okay, here is a question from Erin, as we’re getting near the end of our program. “Can ET ever cause systemic inflammation? And is that what causes symptoms? The inflammation.”

Dr. Pemmaraju: Yes, yes, and yes. So, inflammation, I think, used to be a word that may have been potentially, if I may say, a wastebasket term, but now is a very specific term. So, now we know that a lot of our hematologic disorders and malignancies lead to a high level of inflammation. That means tissue damage. Tissue injury. That’s what inflammation means. There are some conditions that the patient does not even have a blood cancer diagnosis, but has a molecular mutation, that’s called CHIP (clonal hematopoiesis of indeterminate potential), and those patients appear to have a higher likelihood of cardiovascular disease and death. That’s New England Journal of Medicine. The likely pathway is inflammation.

In our patients with MPN, even the quote on quote, earlier stages, such as ET and PV. This is a disease of cytokines and inflammation. So, high levels of abnormal messengers and signals. So, yes, inflammation is part of the disease, patients have a higher rate of cardiovascular events and death. That’s inflammation. And then of course, the bone marrow milieu itself, as it progresses to myelofibrosis has an up ramp, if you will, of cytokines and inflammation. Last part of it is the therapies that we’re working on are trying to either target inflammation itself, or to bring down that level.

Andrew: Okay. I wanna see if – Here’s a – one that just popped in as we get near the end of our program. Roger says, “Are there any drugs being studied that improve anemia in patients with a low hemoglobin?” What’s the easiest way to find out about clinical trials if you live out of the state, or out of the country where this trial may be –?

Dr. Pemmaraju: Yes. Your best resource to look that up is run by the Federal Government, the NH, it’s called clinicaltrials.gov, that’s dot G-O-V. This is an outstanding website, well curated, updated as quickly as they can, and it has a nice search function. You can search by investigator, disease type, condition, and there’s even a box for ‘other’ where you can type in something like ‘myelofibrosis’.

There are several drugs in development. These drugs are known as Luspatercept and Sotatercept, for example. And they’re a class of drugs that are anemia targeting in myelofibrosis and myelodisplastic syndrome. So, the answer is, yes. And you can find out these types of clinical trials either online at this website, or at other websites. But this is an important, urgent, unmet medical need that we are working on, and there are active clinical trials for patients to enroll on.

Andrew: Well, okay. And the last thing I would ask you about – and this always comes up, Naveen, but I wanna hear what you have to say is somebody we have people with ET, we have people with PD, MF, and we talked at one end about acute myeloid leukemia. What do we know about progression now? So, if I’m sitting there with ET, am I necessarily going to go onto PV, or MF? Or anywhere along the line, and how do we know?

Dr. Pemmaraju: We do know a little bit more. So, the answer is no. So, a lot of our patients do stay in the chronic phase, as you’re asking. So, if you’re ET, or PV – and our European colleagues have really done these nice population studies, where the majority – the vast majority of patients with ET and PV are expected in the modern era to have normal life expectancies as long as you’re mitigating in some bleeds, clots, and these type of events.

But for the minority, who don’t have a normal life expectancy, you’re talking about progression to AML, which is a minority of all these. Right? Maybe 5-7 percent of cases at the most. There are some things we have identified. One is that there are some dynamic acquisition of molecular mutations that are happening at the time of progression. And what I mean by that is, there are new injuries to the DNA that people appear to be picking up. So, two important studies our colleague, Raajit Rampal showed that the acquisition of TP53 mutation, which is the guardian of the genome present in 50 (five, zero) percent of human cancers. That looks like it’s more common when ET and PV are trying to take off to AML.

Another study by our Mayo colleagues just published in Blood Advances showed that other mutations, such as PTPN11, or RUNX1, just to name some particular ones, and then we’ve known about ASXL-1 now for a while. So, rapidly change in blood counts in concert with new molecular mutations, and then a baseline if you have high-risk mutations. That seems to be a way for us to predict who might transform faster than others.

Now, that’s an addition to the traditional risk factors that you and I have already discussed, the IPSS risk, or etc. So, there are some ways that we can monitor. A lot of these may be in the research setting. Some are ready for the clinic, but there are some ways now.

Andrew: Okay. So, ladies and gentlemen, I hope this program is been worthwhile for you. Remember that the big meeting of Dr. Pemmaraju and his colleagues from around the world with thousands of hematologists is this the American Society of Hematology meeting, which once again, will be, yay, near me, in San Diego.

Dr. Pemmaraju: Very good.

Andrew: Esther and I’ll just drive over. And the Patient Power team will be there, the Patient Empowerment Network team will be there. So, we’re there for you. So, look for more programs as we go through the fall, and certainly in December, when this meeting happens. And that’s where a lot of the research that Dr. Pemmaraju talks about is presented.

Dr. Pemmaraju: Right.

Andrew: And then we’ll have more news. So, we’re living with these long-term conditions, thank god for most all of us, and it’s a moving target, as I’ve described. I wanna thank you for joining the Patient Empowerment Network program, for sponsoring this program. We thank Incyte Corporation for its support, and Dr. Naveen Pemmaraju from M.D. Anderson, and the Leukemia Department there, thank you for being a partner in this, and just explaining things, and your passion. And Naveen, again, back to your whiteboard back there. Figure it out.

Dr. Pemmaraju: It’s all there. Yes, sir, Andrew.

Andrew: It’s all there. Figure it out. Okay? All right. Thank you so much for being with us from around the world. We love it. We’ve got a community. This is what it’s all about and we’ll have future ask the expert programs. I’m Andrew Schorr near San Diego. Thanks to the Patient Empowerment Network for making all this happen. Remember, knowledge can be the best medicine of all.

Living Well with MPNs – Tips and Strategies for Managing Symptoms and Side Effects of MPNs

Tips and Strategies for Managing Symptoms and Side Effects of MPNs

As part of our Living Well with MPNs webinar series a panel of MPN experts and patients discussed managing life with an MPN. The panel shared advice on managing fatigue, itching, night sweats, enlarged spleen and other symptoms. The experts explained why symptoms occur and stressed the importance of communication with your healthcare team. Tune in to learn more.

Events

Ask the Expert “Live” | MPNs

Do you have a burning question for an expert in myeloproliferative neoplasms (MPNs)? On Tuesday, October 30th at 3:00pm Eastern time (2:00pm Central, 12:00pm Pacific), Patient Power will host a LIVE, 30-minute “Ask the Expert” session with MPN specialist Dr. Joseph Michael Scandura at Weill Cornell Medicine.

Don’t miss this opportunity to voice questions about your condition and hear directly from an expert. Send in your questions to mpn@patientpower.info, and we’ll do our best to get it answered during the live program.

You must register in advance to receive a link to the program and instructions for attending. 

Register Here

Please note that we try to respond to all appropriate questions, but cannot provide specific medical advice over the internet. We always recommend that you seek care from your own doctor, or an MPN specialist, that’s how you’ll get the best treatment for you.


Guests

 

Register Here

Ask the MPN Expert – LIVE

Do you have a burning question for an expert in myeloproliferative neoplasms (MPNs)? On Tuesday, August 28 at 5:00pm Eastern time (4:00pm Central, 2:00pm Pacific), we will host a LIVE, 30-minute “Ask the Expert” session with MPN specialist Dr. Naveen Pemmaraju from The University of Texas MD Anderson Cancer Center.

Don’t miss this opportunity to voice questions about your condition and hear directly from an expert. Send in your questions to mpn@patientpower.info, and we’ll do our best to get it answered during the live program.

You must register in advance to receive a link to the program and instructions for attending. 

Register Here

Please note that we try to respond to all appropriate questions, but cannot provide specific medical advice over the Internet. We always recommend that you seek care from your own doctor, or an MPN specialist, that’s how you’ll get the best treatment for you.

Guest

Register Here