MPN Newly Diagnosed Archives

Your MPN diagnosis is just a starting point. Even though the path ahead may seem unclear or even insurmountable, armed with knowledge you can take control.

Let us help you become empowered to understand your diagnosis, to confidently ask questions, and to identify providers that are the best fit for you.

More resources for Myeloproliferative Neoplasms (MPN) Newly Diagnosed from Patient Empowerment Network.

What’s YOUR Role in Making Myelofibrosis Treatment Decisions?

What’s YOUR Role in Making Myelofibrosis Treatment Decisions? from Patient Empowerment Network on Vimeo.

How can you play a role in your myelofibrosis care? Dr. Joseph Scandura shares his personal philosophy on patient care and the important role of shared decision-making.

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

Related Programs:

Have You Had These Essential Myelofibrosis Tests?

What Are the Considerations When Choosing Myelofibrosis Therapy?

Expert Perspective: Promising Myelofibrosis Treatment Research


Transcript

Katherine Banwell:

Dr. Scandura, what is the role of the patient in making treatment decisions? 

Dr. Scandura:

My personal philosophy is I view myself and my interactions with patients as a partnership. And I have and I bring to this partnership medical knowledge, some scientific knowledge, experience treating patients, understanding the diseases and the biology of the diseases. 

What patients bring is their personal histories, what they want and need from therapy, what their expectations are, where their fears and concerns might be. And as we share our information, I think that provides the opportunity to come to an understanding where the patient can make an informed decision and I can support that decision, that we know what the groundwork has been between us. And so, I spend, often, a lot of time in the beginning with patients kind of trying to understand who they are as people and what they need and expect. And everybody, as you might imagine, is an individual.  

And I present to them the information, and I try to encourage questions so that I know that they understand the information that I’m giving so that they can make a decision in their best interest. And so, I think shared decision-making is the only model I practice.  

Now, patients have different needs, particularly some of my older patients. And, culturally, there are some differences where they don’t want to take that role of being the decision-maker. And so, then my role changes a little bit, and it becomes more to make sure they’re comfortable and understand the direction that we’re going in and, again, always trying to encourage people to take ownership. 

I think, in New York City, that’s not so common. People are pretty well-informed and interested and more than willing to express their opinions.  

And so, I would say it can be very rewarding to come to a decision where patients feel their needs are being met.  

Expert Perspective: Promising Myelofibrosis Treatment Research

Expert Perspective: Promising Myelofibrosis Treatment Research from Patient Empowerment Network on Vimeo.

Dr. Joseph Scandura shares optimism about myelofibrosis therapy in clinical trials, including excitement about anti-fibrotic agents and how they work.

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

Related Programs

What Are the Considerations When Choosing Myelofibrosis Therapy?

How Does Inhibitor Therapy Work to Treat Myelofibrosis?

What’s YOUR Role in Making Myelofibrosis Treatment Decisions?


Transcript

Katherine Banwell:

Dr. Scandura, you mentioned promising research in myelofibrosis treatment. What are you most excited about right now? 

Dr. Scandura:

I think there are a couple drugs that have been in clinical trials that have had activity in a significant subset. So, anywhere from 20 to 50 percent of patients where the bone marrow fibrosis is actually reversed. 

And this is really something that we haven’t seen with other agents. And the approved agents, when that does happen, it’s really in a vast, vast minority of patients. And so, these newer drugs and, often, they’re used in combination with other approved drugs, can reverse the fibrosis in the marrow. And that is what I find most intriguing and exciting. They seem to be well-tolerated medications with predictable and reversible side effects when they do exist. And I think that time will tell if the promise is long-lived or if it’s short-lived. I mean, obviously, new drugs we don’t have the experience with that we really need. 

The clinical trials that are available now with some of these agents are in the last stages before the companies go to the FDA seeking approval for use. 

And so, we don’t have their results from those studies yet. They’re just opening, so sometimes the excitement doesn’t bear out when we do the rigorous clinical trials. But I’m actually quite optimistic about some of these agents, and I think that there is going to really be a sea change in how we treat patients and some of the outcomes we can expect from our therapies.  

COVID-19 Vaccination: What Do Myelofibrosis Patients Need to Know?

COVID-19 Vaccination: What Do Myelofibrosis Patients Need to Know? from Patient Empowerment Network on Vimeo.

 Should myelofibrosis patients get the COVID-19 vaccine? Dr. Joseph Scandura discusses the risks and benefits of vaccination.

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

Related Programs

What Are the Considerations When Choosing Myelofibrosis Therapy?

Expert Perspective: Promising Myelofibrosis Treatment Research

What’s YOUR Role in Making Myelofibrosis Treatment Decisions?


Transcript

Katherine Banwell:

Is the COVID-19 vaccine safe for patients with myelofibrosis, and how does the vaccine affect treatment? 

Dr. Scandura:

So, I will flip that question around a little bit. I live in New York City.  

If I cross the street, the decision to cross the street is potentially a life-or-death decision. And whatever minor decision you’re making, there are always risks and there are always potential benefits. So, I might get home, I might get run over by a cab. And so, I try to mitigate those risks as I can by crossing in certain streets, looking both ways. So, when we talk about vaccine, we also have to talk about the other part of it. What is the risk of not being vaccinated? And so, we know COVID-19 is a severe illness in a subset of patients, we know that if you take all people, about 1 percent of people die from COVID. 

 If we take all people from the vaccine who have been vaccinated, the number of serious side effects is very, very, very, very small, so, like .000, you know, something percent. 

So, very low. It doesn’t mean it’s zero, but it’s very, very low. So, just looking at those numbers, I say for virtually everybody, the risk/benefit is in favor of vaccination. In patients with myelofibrosis, we’ve had the opportunity collectively across the world to gather experience and look at patients with myeloproliferative neoplasms and how they responded to COVID when they were infected with COVID. And worldwide, the toxicity of COVID in patients seems to be quite high. And so, patients with myelofibrosis may be at higher risk from COVID. 

I can’t say that they absolutely are because this is imperfect data, but that’s the experience that has been published so far.  

We really don’t know anything about the experience of patients to the vaccine. Actually, at my center, we have a myeloproliferative diseases center, and we are trying to collect that information because patients often ask, and I don’t have any results from that. But I think that, all told, there is no reason to expect higher symptoms in patients with myelofibrosis from vaccination. And what we do know is that the risk of not being vaccinated is probably higher than the risk of being vaccinated.   

What Are the Considerations When Choosing Myelofibrosis Therapy?

What Are the Considerations When Choosing Myelofibrosis Therapy? from Patient Empowerment Network on Vimeo.

 When choosing a myelofibrosis treatment, how do you determine what might be best for you? Dr. Joseph Scandura shares expert advice, including a review of inhibitor therapy and stem cell transplant.

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

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How Does Inhibitor Therapy Work to Treat Myelofibrosis?


Transcript

Katherine Banwell:

What are the considerations when choosing treatment for myelofibrosis?  

Dr. Scandura:

I would say in broad strokes, the primary considerations are the patient, what they want, the disease, what our options are, and the overall condition in terms of what are our possibilities for therapy and what is the risk/benefit of some of these different therapies. So, in myelofibrosis, although there’s been a huge amount of research over the past 10 years, really blossoming and are very impressive in, I think, an exciting way, there really are only two therapies that are approved by the FDA in the treatment of myelofibrosis, and those both affect one class of agents. These are JAK2 inhibitors, and those can be ruxolitinib (Jakafi) and fedratinib (Inrebicare the two drugs that are approved. 

Now, we have a number of therapies that have been used off-label, meaning without FDA approval, so often and for so long that they’re considered alternative standards of therapy. These can be growth factors; these can be biological agents in certain situations. And then, clinical trials is really increasingly a common therapeutic option for patients.  

And then, on the most aggressive side, is hematopoietic stem cell transplant and allogeneic transplant getting blood-forming cells from another person and replacing the entire blood system through transplant. 

Katherine Banwell:

So, who is right for a stem cell transplant? 

Dr. Scandura:

I would say, first and foremost, an informed patient about the risks of transplant and a patient for whom a donor exists, and a good quality donor. Transplant is not an option for some people or if a donor can’t be identified, obviously. 

And it’s a patient for whom the risk balance, the risk/benefit balance is tipped so that the potential toxicity, frankly, of transplant is warranted. Transplant is our most aggressive therapy. Virtually every patient will have significant side effects from transplant. Some of them are short-lived, some of them can be chronic. People die from the consequences of transplant. And so, it’s not something that is considered in patients who are necessarily doing well or are frail. The risk of transplant versus the benefit may not be in favor of transplant at that time.  

My approach for transplant is to get advice from transplant physicians. I’m not a transplant physician, but I have colleagues who I refer to. 

And I refer in myelofibrosis fairly universally fairly early, with the rationale being that this is information. It is not a plan; it is to speak to a transplant, what kind of donor exists. If no donor exists, then transplant is not on the table. If we have a very good, high-quality donor, then this is something that wouldn’t make the decision in itself, but it’s kind of something we can keep in our hip pocket in case we need it. And I think it’s important for patients to understand and have a full and complete discussion with a transplant physician so they understand what that means. You know, it is a significant commitment of time and morbidity, and it comes with risks. 

It is also our only curative therapy. And so, it’s a double-edged sword, and I think informed patients and understanding what the options are are the gateway to any consideration of transplant.   

Primary vs. Secondary Myelofibrosis: What’s the Difference?

Primary vs. Secondary Myelofibrosis: What’s the Difference? from Patient Empowerment Network on Vimeo.

Are primary and secondary myelofibrosis different? Dr. Joseph Scandura, a specialist in myeloproliferative neoplasms (MPNs), explains the diagnoses and shares insight into each type.

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

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What’s YOUR Role in Making Myelofibrosis Treatment Decisions?


Transcript

Katherine Banwell:

Dr. Scandura, would you start by introducing yourself?  

Dr. Scandura:

Sure. My name’s Joe ScanduraI’m an assistant professor at Weill Cornell Medicine in New York City. I’m a physician scientist. My laboratory studies blood formation, normal and malignant, and clinically I treat people with  myeloid neoplasms, particularly, and myeloproliferative neoplasms.  

Katherine Banwell:

Would you define myelofibrosis for us, and also provide an explanation of primary versus secondary myelofibrosis? 

Dr. Scandura:

Sure. Myelofibrosis is in the class of diseases called myeloproliferative neoplasms. And, really, its sort of marker feature is scarring in the bone marrow.  

Clinically, this comes along most commonly and fairly universally with anemia, and there can be abnormalities of both the white blood cell count and the platelet count, sometimes, often in the beginning, being too high. And then they can also become too low. 

It tends to be a progressive disease, or on the face on which it progresses is different in different people and there are a variety of different features that can go along with risk. But every individual, of course, is individual.  

A primary myelofibrosis is what we refer to when the diagnosis is made and there’s no antecedent, there’s no precursor malignancy. And so, you come in and the diagnosis is myelofibrosis, and we can’t find anything that came before it.  

Secondary myelofibrosis is what we refer to when somebody has another blood disorder, usually essential thrombocythemia or polycythemia vera, and in a small subset of these patients, the disease can change, what we call evolve or progress into a fibrotic phenotype or associated with the marrow scarring, and a lot of the features of myelofibrosis. Although there are some subtle differences between primary and secondary, they’re more similar than different in terms of their clinical features and how we treat them. 

How Does Inhibitor Therapy Work to Treat Myelofibrosis?

How Does Inhibitor Therapy Work to Treat Myelofibrosis? from Patient Empowerment Network on Vimeo.

What is inhibitor therapy? Dr. Joseph Scandura reviews approved JAK inhibitor therapies and explains how they work to treat myelofibrosis.

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

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Which Gene Mutations Impact Myelofibrosis Treatment Options?

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What’s YOUR Role in Making Myelofibrosis Treatment Decisions?


Transcript

Katherine Banwell:

How does inhibitor therapy work to treat myelofibrosis? 

Dr. Scandura:

So, the therapies that we have now that are approved therapies that are in this class are  ruxolitinib (Jakafi) and fedratinib (Inrebic) 

Both of these agents act to block signaling through a protein called JAK2. You can think of JAK2 as being part of the antennae system that a cell uses to communicate with the rest of the body. And so, our blood-forming cells have a lot of input from the body saying, “Okay, we need some of these kinds of cells, we need some of those kinds of cells,” and it’s a very adaptive system. And JAK2 is involved in a lot of the signaling in this as part of the antennae system.  

And what happens in the myeloproliferative neoplasms is that signaling is a bit excessive. 

And so, it’s like the volume is turned up too loud and the signaling is causing the cells to do things, make too many cells, make the wrong kinds of cells, and JAK2 is part of that signaling system. So, these inhibitors kind of help turn down the volume of the signaling in these blood-forming cells. They are drugs that have good activity in improving symptoms, they have great success in reducing the size of the spleen, they can be useful for a few years to many years. They are not curative therapies. We don’t think of them as therapies that change the course of disease, but they certainly have an important role in helping people feel better. There are other inhibitor therapies that are in clinical development. 

So, clinical trials of some of these drugs have really impressive activity, but none is approved yet by the FDA.  

I hope and expect we’ll have a couple more drugs available in the coming years. And there’s a lot of excitement in clinical trials in terms of some of the activities that are being seen, and really quite tolerable therapies, so not a lot of side effects for patients. And so, I think it’s kind of an exciting time for physicians and for patients and a lot more options now and, I think, a lot more options coming down the line.

Which Gene Mutations Impact Myelofibrosis Treatment Options?

Which Gene Mutations Impact Myelofibrosis Treatment Options? from Patient Empowerment Network on Vimeo.

Are there specific mutations that may affect myelofibrosis treatment choices? Dr. Joseph Scandura explains the factors that are considered when deciding a myelofibrosis therapy, including a discussion of high-risk and low-risk disease.

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

Related Programs

Have You Had These Essential Myelofibrosis Tests?

What Are the Considerations When Choosing Myelofibrosis Therapy?

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Transcript

Katherine Banwell:

Are there gene mutations that affect myelofibrosis treatment choices? 

Dr. Scandura:

Yeah. So, you know, the primary mutations in JAK2 or CALR or MPL in myelofibrosis aren’t that helpful in guiding therapy.  

And we look at the other genes for co-ocurrent mutations and those, as I was mentioning before, can come into one of two categories. So, there are a number of genes that we know tend to confer a higher risk, and so we call those high molecular risk mutations. And people who have higher molecular risk tend to have a more aggressive disease. 

Now, I want to add a word of caution because when we talk about patients and risk, we’re talking about groups of patients. For any individual, everything kind of boils down to it happens, or it doesn’t happen. And so, there’s nobody is 50 percent dead in five years, right. You either are or you’re not. And so, when we talk about risk, then we’re talking about risk of bad things happening like death or other complications of the disease, we’re trying to guide treatment decision-making and guided discussion based on a chance.  

But all of those things, for any individual, there are people who have high risk who do quite well for a long period of time, and people who don’t have high risk who don’t do as well as you think they should. And so, it’s a part of a conversation, it helps guide discussion, but it is not something carved into stone, and nobody has a perfect ability to predict anybody’s future. 

And all of these things are our best tools to estimate, but they are not a future; they are a possibility. And so, people who have higher molecular risk, we might think about more aggressive treatments than people who have lower molecular risk.  

Have You Had These Essential Myelofibrosis Tests?

Have You Had These Essential Myelofibrosis Tests? from Patient Empowerment Network on Vimeo.

What are the essential tests that should follow a myelofibrosis diagnosis? Dr. Joseph Scandura reviews the necessary laboratory testing, along with a discussion of next generation sequencing, and explains how often bone marrow biopsies should take place.

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

Related Programs

Which Gene Mutations Impact Myelofibrosis Treatment Options?

What Are the Considerations When Choosing Myelofibrosis Therapy?

How Does Inhibitor Therapy Work to Treat Myelofibrosis?


Transcript

Katherine Banwell: 

What testing should take place following a myelofibrosis diagnosis? 

Dr. Scandura:  

So, a diagnosis of myelofibrosis always comes after a bone marrow exam and a physical examination. Often, patients have an enlarged spleen and blood count testing and a variety of other laboratory tests. So, after that and a diagnosis is made of myelofibrosis and, sort of, coincident with the diagnosis, we often look for molecular markers of myelofibrosis. So, these are malignancies of the bone marrow, cancers, if you will, on the bone marrow, although the term is scarier than or is different than what we think of for many malignancies in how it acts. But the myelofibrosis, this is a disease that’s characterized by, really, mutations in the malignant cells, the abnormal cells. 

They’re really just one of three genes. And so, JAK2 being one of the genes, calreticulin or CALR being another one, and MPL one.  

And more than 90 percent are people having mutation in just one of those three genes. And so, often at the time of diagnosis, tests for those mutations are done, and they help eliminate the possibilities of other causes of myelofibrosis – infections, rheumatological diseases. Sometimes, you can have marrow fibrosis but they don’t go along with mutations and the same clinical situation. And so, at the time of diagnosis, we usually know something about a mutation in JAK2, CALR, or MPL.    

More commonly now, and it’s increasingly common over the past 10 years in, I would say, in New York City and many places across the country, we also look more broadly for other common mutations in the MPN cells. And these are what we refer in the batch as next generation sequencing or NGS panels, and we use the term panels because we’re looking at from a few tens to even 100 or a couple hundred genes for mutations that occur far less frequently than in JAK2 or MPL or CALR.  

But they occur often enough that some of them we use to help guide treatment decision-making or approach to therapy. The reality of it is that that the technology to sequence and identify mutations has really outstripped our knowledge of what to do with all of that information. 

And, for the vast majority of people, it comes down to do you have a marker, a genetic marker that tends to go along with higher risk, meaning a higher likelihood of something that we don’t want to have happen. And in that instance, although it may be looking at a hundred or so genes, it comes down to a binary thing – either you have or you don’t have. 

Katherine Banwell:

Is there any other testing that you usually want to do? 

Dr. Scandura:

Laboratory testing, for sure and, as I mentioned before, a bone marrow exam. But physical examination, some people might do imaging of the spleen size. Honestly, I don’t routinely do that outside of the setting of the clinical trial. I don’t really think it dictates therapy very often. 

And if the spleen is so small that you can’t feel it on physical exam, it probably isn’t clinically meaningful anyway in terms of something to treat. It might be there, but it doesn’t really change things too much.   

Katherine Banwell:

How often should patients have a bone marrow biopsy? 

Dr. Scandura:

So, I’ll answer there is no standard in terms of monitoring for myelofibrosis with the marrow or otherwise. My personal approach is I do a marrow when I think it’s going to help medical decision-making. And so, for a patient who’s got early myelofibrosis, who’s been very stable, responding well to therapy, that could be three, five years between marrow exams. 

For somebody who’s being considered for a clinical trial, oftentimes, a marrow exam is required before they start on the clinical trial and at various intervals afterwards. If there’s somebody who had been stable and something is changing, like the blood counts are changing or his symptoms are changing, or any of a number of clinical features, then I might look in the marrow to see what’s happening there, to see if explains and can help guide a treatment approach to help people feel better. So, there is no single standard, but my personal approach is to do a bone marrow exam when I think it’s going to help make a decision.  

What You Need to Know Before Choosing a Cancer Treatment

What You Need to Know Before Choosing a Cancer Treatment from Patient Empowerment Network on Vimeo.

Download Guide

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What steps could help you and your doctor decide on the best treatment path for your specific cancer? This animated video explains how identification of unique features of a specific cancer through biomarker testing could impact prognosis, treatment decisions and enable patients to get the best, most personalized cancer care.


If you are viewing this from outside of the US, please be aware that availability of personalized care and therapy may differ in each country. Please consult with your local healthcare provider for more information.


Related Programs:

 

PEN-Powered Activity Guides

Digitally Empowered™


TRANSCRIPT:

Dr. Jones:

Hi! I’m Dr. Jones and I’m an oncologist and researcher. I specialize in the care and treatment of patients with cancer. 

Today we’re going to talk about the steps to accessing personalized care and the best therapy for YOUR specific cancer. And that begins with something called biomarker testing.

Before we start, I want to remind you that this video is intended to help educate cancer patients and their loved ones and shouldn’t be a replacement for advice from your doctor.

Let’s start with the basics–just like no two fingerprints are exactly alike, no two patients’ cancers are exactly the same. For instance, let’s meet Louis and another patient of mine, Ben. They both have the same type of cancer and were diagnosed around the same time–but when looked at up close, their cancers look very different.  And, therefore, should be treated differently.

We can look more closely at the cancer type using biomarker testing, which checks for specific gene mutations, proteins, chromosomal abnormalities and/or other molecular changes that are unique to an individual’s disease.

Sometimes called molecular testing or genomic testing, biomarker testing can be administered in a number of ways, such as via a blood test or biopsy. The way testing is administered will depend on YOUR specific situation.

The results could help your healthcare team understand how your cancer may behave and to help plan treatment. And, it may indicate whether targeted therapy might be right for you. When deciding whether biomarker testing is necessary, your doctor will also take into consideration the stage of your cancer at diagnosis.

Louis:

Right! My biomarker testing results showed that I had a specific gene mutation and that my cancer may respond well to targeted therapy.

Dr. Jones, Can you explain how targeted therapy is different than chemo?

Dr. Jones:

Great question! Over the past several years, research has advanced quickly in developing targeted therapies, which has led to more effective options and better outcomes for patients.

Chemotherapy is still an important tool for cancer treatment, and it works by affecting a cancer cell’s ability to divide and grow. And, since cancer cells typically grow faster than normal cells, chemotherapy is more likely to kill cancer cells.

Targeted therapy, on the other hand, works by blocking specific mutations and preventing cancer cells from growing and dividing.

These newer therapies are currently being used to treat many blood cancers as well as solid tumor cancers.  As you consider treatments, it’s important to have all of the information about your diagnosis, including biomarker testing results, so that you can discuss your treatment options and goals WITH your healthcare team.

Louis:

Exactly–Dr. Jones made me feel that I had a voice in my treatment decision. We discussed things like potential side effects, what the course of treatment looks like and how it may affect my lifestyle.

When meeting with your healthcare team, insist that all of your questions are answered. Remember, this is YOUR life and it’s important that you feel comfortable and included when making care decisions. 

Dr. Jones:

And, if you don’t feel your voice is being heard, it may be time to consider a second—or third—opinion from a doctor who specializes in the type of cancer you have. 

So how can you use this information to access personalized treatment?

First, remember, no two cancers are the same. What might be right for someone else’s cancer may not work for you.

Next! Be sure to ask if biomarker testing is appropriate for your diagnosis. Then, discuss all test results with your provider before making a treatment decision. And ask whether testing will need to be repeated over time to identify additional biomarkers.

Your treatment choice should be a shared decision with your healthcare team. Discuss what your options and treatment goals are with your doctor.

And, last, but not least, it’s important to inquire about whether a targeted therapy, or a clinical trial, might be appropriate for you. Clinical trials may provide access to promising new treatments.

Louis:

All great points, Dr. Jones! We hope you can put this information to work for you. Visit powerfulpatients.org to learn more tips for advocating for yourself.

Dr. Jones:

Thanks for joining us today. 


This program is supported by Blueprint Medicines, and through generous donations from people like you.

Advocacy Through Various Mediums with an MPN Patient and Caregiver

Advocacy Through Various Mediums with an MPN Patient and Caregiver from Patient Empowerment Network on Vimeo.

What is patient advocacy and how can you advocate? MPN Network Managers Jeff and Summer discuss the various ways in which they advocate. In addition to volunteering with PEN, Jeff actively participates in a support group. Summer who is living with MPN has decided to advocate through her humor. Make sure to watch to see a snippet of her stand-up routine! 

“Our challenge to you is, as a patient find a way to give your knowledge of how you’re handling your disease to others and you too can become a strong patient advocate.” 

Want to connect with Jeff and Summer? Email them at question@powerfulpatient.org or text EMPOWER to (833)213-6657.

Patient Profile: Alexis Chase, PhD

Patient Profile

Alexis Chase, PhD

“To be empowered you have to be open, to want to do it, and to accept where you are.” – Dr. Alexis Chase, An MPN Empowered Patient

Dr. Alexis Chase has had a pretty interesting life, but she doesn’t think that makes her unique. She says she thinks all women have interesting lives. Born congenitally blind in her right eye she was given the name Alexis Elizabeth Lucia Chase. “I’m very proud of my name,” she says explaining the origin. Alexis was the name of a doll her mother had as a girl, and it means protector of mankind. Elizabeth is a family name, and Lucia represents Saint Lucia, the patron saint of the blind. Her mother was a nurse and her father, who was the first to recognize she had a vision issue, had a degree in biology. She was very close with her parents who instilled in her a strong foundation in her Roman Catholic faith. While she was born in Connecticut, she spent most of her adult life in Georgia as a divorced mother who built a successful 27-year career in the prison corrections system. She worked her way up to warden and earned two PhDs, one in religious counseling and one in criminal justice and corrections. After her retirement she became an international advocate and consultant of gender and women’s rights issues that include vocational training, post-incarceration reintegration, and female prisoners with children. She has travelled as far as Afghanistan in her advocacy work, and she is also the proud nana to a cat named Nathan Edgar Chase. She’s done a lot, and much of what she’s accomplished, she’s done while living with cancer.

The first time she was diagnosed with cancer was in 1976. She was in the first trimester of a high-risk pregnancy when she was diagnosed with ovarian cancer. Her doctors thought it would be best to terminate the pregnancy, but she refused. She was determined to have the baby, her daughter, and as soon as she was born, Dr. Chase began treatment for her cancer, opting for an experimental drug that she says saved her life.

At the time, her parents, her desire to live for her daughter, and her strong faith gave her the support she needed. “They were right there with me,” she says of her parents who she is grateful to for her faith. “It’s my great equalizer. My rope of hope,” she says and adds that she can pull on her faith anytime and in any place. “You’ve got to believe in something greater than yourself because definitely we’re not it,” she says.

She’s had no recurrence of the ovarian cancer, but in 1996, during a regular wellness checkup, she was diagnosed with myeloproliferative neoplasms (MPNs), a group of blood cancers that affect the function of bone marrow and can cause a number of complications. In Dr. Chase’s case her MPNs includes iron deficiency, anemia, diseases of the blood and blood forming organs, and hypothyroidism. MPNs are chronic conditions that can transform into another blood cancer and can affect people at any age but are more common in older adults. MPNs are also progressive. Dr. Chase had no symptoms for the first four years after her diagnosis, and wondered if she’d been misdiagnosed, but in 2000 she says she just started to feel like something wasn’t right and that’s when her blood counts started to change. She began taking medication, but in 2020 it stopped working and her cancer team worked to find other medications and therapies to treat her.

MPNs are rare and she doesn’t know anyone else with the same diagnosis, but she says she has an incredible support network through her daughter, her friends that are like family, her church, and her cancer team. “They take great care of me,” she says, but she also takes great care of herself. In fact, she’s very meticulous about taking care of herself. She carefully takes her medications, and she makes herself a priority. She focuses a lot on her mental health and she stresses the importance of mental health for all cancer patients. She says she finds three ways to laugh at herself every day and she chooses six words every day that represent how she’s doing and to help her feel empowered. A recent example, “I feel surrounded by grace today”. Also, part of her self-care is taking the time to listen to calming and soothing sounds and inspirational messages and quotes.

She says it’s a blessing to have the cancer she has because she is able to handle it and it makes her take time to smell the roses. She’s handled it so well that during her career as a prison warden she never let on that she was sick. She managed to schedule her appointments around her work so no one would know. She didn’t want her illness to affect her career.

Always an empowered patient, she’s been known to walk out of a doctor’s office when a situation doesn’t feel right. “It’s important for people to feel like they are being heard and more importantly that they are being listened to.” She says “It’s also important to know what’s going on with your care. You know your body better than anybody.” Dr. Chase likes the Patient Empowerment Network (PEN) because of the resources it provides to help others feel empowered in their own care. “I found PEN and love that I can access it anytime,” she says. She feels it’s important for patients to take charge of their own care plans. “To be empowered you have to be open, to want to do it, and to accept where you are,” she says. Her recommendation to other patients is to read, and comprehend, everything they can about their illness. “If you don’t understand it, you need to have someone explain it to you,” she says and also recommends keeping a journal. “We have to have something tangible. We can’t remember everything.”

While she continues to accomplish a great deal while living with cancer, it’s not always easy. “The chronic cancer fatigue, it will get me. I fight it because I feel like once I give in it would overtake me,” she says. She does experience shortness of breath and plans her days around her energy level. “It slows me down, but I don’t let it stop me. I push myself because I know the next day or the next day I won’t be able to.” Along with continuing her consulting and advocacy work, Dr. Chase loves to travel and hopes to go to Turkey to see the Virgin Mary’s house. “You never know what God has laid out for you,” she says. “I’m still here. I’ve been symptomatic for 20 years and I’m still here.”


Read more patient stories here.

The Pro-Active MPN Patient Toolkit: Find Your Voice Resource Guide

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Shared Decision-Making: The Patient’s Role in Treatment Choices

Shared-Decision Making: The Patient’s Role in Treatment Choices from Patient Empowerment Network on Vimeo.

What is the role of the patient when it comes to treatment choices? Dr. Brady Stein details how he partners with patients in decision-making for their MPN care. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


Related Resources

MPN Symptom or Treatment Side Effect? Know the Difference

An Expert Shares Key Steps to Take Following an MPN Diagnosis

How Often Should You See Your MPN Doctor?


Transcript:

Katherine:                  

What do you feel is the patient’s role in the decision for therapy?

Dr. Stein:                   

I think it’s a really important role. I think historically – and, this is decades past; this era should be well over and behind us – this era of authoritative medicine is over.

You can’t just have a doctor walk in the room and say, “This is your treatment, this is what you should do, I’ll see you later.” It’s shared decision-making, and that can be troubling for some patients. But, the idea of shared decision-making is us explaining options informing the patient and making decisions together. That’s really the paradigm for modern contemporary medicine.

Some patients have a harder time with that. A lot of patients say, “Well, doc, this is too overwhelming for me. I just want you to decide for me.” And, we try not to do that. That’s a more uncomfortable type of visit for me when a patient is very deferential and says, “Whatever you say, I’ll do.” That’s not really what we want to hear. I want to know that you feel really informed, that you have a good understanding because each of these treatments – any treatment, any medication has its pros and cons.

There are no real magic bullets, and each upside has an equal downside, so you have to engage and open a dialogue, and what that means is that patients need to read and learn. That’s hard, but patients need to become proactive in their approach to their own illness, and all the patients who are listening now are doing that, trying to get more education about your relatively rare illness that’s going to give you a much better framework to help make decisions together.

Katherine:                  

Absolutely. If a patient isn’t feeling confident with their treatment plan or their care, do you recommend that they maybe consider a second opinion or seek a specialist?

Dr. Stein:                   

Of course, yeah. These are rare diseases, and patients often – I would say that in my clinic, a lot of the patients direct their own second opinions. Oftentimes, it’s coming from the patient more so than their doctor. I think the patient community is very active, the patients are networking, and they’re finding the right specialist to get to.

I think it should be really a team approach. It’s never – it’s usually not very convenient to go to a university unless you live really close, so you want to have someone close to home who can handle the routine, and then, someone who maybe is a little bit further away who can see you once a year, can help with the big decisions, can be part of the healthcare team. So, we generally recommend that you have someone near, and that maybe you have someone far who focuses only on MPNs as part of your team, and now, it’s a little different. Telemedicine is becoming a pretty ingrained part of medicine. It’s a little easier to have those visits with a physician who’s far away because of telemedicine.

Self-Advocacy: Advice for Being a Pro-Active MPN Patient

Self-Advocacy: Advice for Being a Pro-Active MPN Patient from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) patients be more pro-active in their care? Dr. Brady Stein shares advice to help patients educate themselves about the disease, while finding the right balance of knowledge to prevent them from feeling overwhelmed. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


Related Resources

Tools to Help You Learn More About MPN Clinical Trials

How Often Should You See Your MPN Doctor?

Which MPN Treatment is Right for You? Factors to Consider


Transcript:

Katherine:

Let’s talk about patient self-advocacy now, Dr. Stein. Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions.

Why is it important for patients to speak up when it comes to symptoms and side effects?

Dr. Stein:                   

I smile a little bit because patients – I get a lot of patient emails by MyChart. That’s our medical record, and it’s a secure patient email, and a lot of patients will start their message by saying, “I’m sorry to bother you.”

And, I always say, “Why do you think that? It’s my job. Please don’t apologize for reaching out to me.” So, that’s kind of the first thing. Don’t feel like you’re bothering your doctor. There are certain things that we won’t know unless you tell us, and so, I think that’s pretty clear. When we’re in a patient room and there might be a husband and wife together, and whether it’s the husband is the patient or the wife is the patient, we might ask a question, and we might get, “No, everything is fine,” but all doctors kind of sneak over to the partner, and the partner may be saying – they’re making gestures to us. There may be nonverbal forms of communication to tell us there’s something much worse than what the patient is telling you.

So, again, “advocate” meaning you have to tell us what’s going on with you. If you’re worried about something, please don’t be stoic about it. These diseases are treated a lot based on your symptoms, and so, if you don’t tell uls about your symptoms, we won’t know.

And, in terms of advocacy, I think one of the things is that these are pretty rare diseases. In an academic center, no, this is our focus, but if you’re in a community practice where the doctor’s seeing 10-15 different things during the course of a day, it’s basically impossible to keep up with myelofibrosis, especially if you have one patient in your whole practice. I can’t do that for diseases that I see that I have only one patient. The medical literature can be overwhelming.

So, patients can quickly outpace their doctor in terms of their knowledge of these diseases, but I think it’s really important to read, to learn, and to think about the illness because you may find out things through your research that your doctor wouldn’t know are available. You may find a clinical trial, a new strategy, or a new test that they simply haven’t had the time to keep up with or learn about. So, that’s what advocacy is about. Reading is really important, but you have to find a balance. I want my patients reading, but you’ve got to find the right amount because there’s a certain amount of reading where the patients start to get overwhelmed.

All patients kind of get to this point. They take it in – like taking it in like a fire hydrant in the beginning of the disease, and it’s overwhelming, and then they start to find their balance. I think there’s a point where the reading becomes anxiety-provoking rather than ameliorating anxiety, and all patients just generally find their balance.

Is My MPN Treatment Working?

Is My MPN Treatment Working? from Patient Empowerment Network on Vimeo.

During myeloproliferative neoplasm (MPN) treatment, specific blood tests and diagnostic measurements help to gauge a patient’s treatment response. Dr. Brady Stein details the criteria he assesses in monitoring the efficacy of a therapy, including patient-reported outcomes.  

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


Related Resources

What Are the Treatment Options for Myelofibrosis?

Monitoring MPNs: When is it Time to Switch Therapies?

MPN Symptom or Treatment Side Effect? Know the Difference


Transcript:

Katherine:                  

Once a patient has started treatment, how do you know it’s working?

Dr. Stein:                   

That’s a good question because this is a very unique area. Yes, of course, in some respects, it’s straightforward with ET or PV. If we’re starting a medication to control a blood count in hopes of having lowered the thrombosis risk, you can look objectively at blood counts.

Okay, your hematocrit is at this goal? Yes, therapy’s working. You have not had a blood clot?

Yes, therapy’s working. So, there are some objective things. In myelofibrosis, there are some objective things like measuring the spleen and seeing it reduce. You can feel that with your hands, or you can do an ultrasound. So, there are some objective parameters of success. But, in this area, patient-reported outcomes are really important, and so, a measure of success is really just asking the patient, “Do you feel like your drug is working? Do you feel better?

It’s kind of a simple question, but it’s really important, and it’s what we ask in patients who are on certain therapies. “Do you feel like the net effect of your therapy is still positive? Do you feel like it’s helping?” Seems like a straightforward type of question, but I think the answer is extremely informative. When a patient says, “Yes, definitely, my medication is still helping me,” then I know that I don’t need to change it.