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.

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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.

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What Are the Considerations When Choosing Myelofibrosis Therapy?

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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.

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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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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.

Related Programs

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

Expert Perspective: Promising Myelofibrosis Treatment Research

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.