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Is the COVID-19 Vaccine Safe for Myeloma Patients?

Is the COVID-19 Vaccine Safe for Myeloma Patients? from Patient Empowerment Network on Vimeo.

 Should myeloma patients get the COVID-19 Vaccine? Dr. Joshua Richter encourages all patients to get the vaccine but notes important considerations around treatment.

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

See More From Engage Myeloma


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Myeloma Treatment Decisions: What Should Be Considered?

What Standard Testing Follows a Myeloma Diagnosis?

An Expert’s Take on Promising Myeloma Treatment and Research


Transcript:

Katherine:

Is the COVID-19 vaccine safe for patients with myeloma?

Dr. Richter:

Absolutely, 100 percent yes. Everybody with myeloma should absolutely get the vaccine. What’s a little more complicated is the timing of it. So, one is in relation to stem cell transplant or CAR T-cell therapy. If you’ve had one of these, obviously, consult with your provider. But the general recommendation is to wait about 60 to 90 days after a high-dose therapy like that. And it’s not a question of safety, it’s a question of efficacy. Vaccines are like vegetables, seeds, you have to put them in the ground to grow. If you give yourself a vaccine right after a stem cell transplant, well, your bone marrow is not ready to work with it. It’s like planting a seed in the desert.

You want to make sure your immune system can take in that vaccine and give you immunity. So, you have to wait at least 60 to 90 days. The other question is, what happens if you’re getting continual therapy? And we don’t know the answer for most of these drugs, but one of the things is dexamethasone (Decadron), which is a steroid. Almost all myeloma therapy comes with some steroids. And we like to separate the vaccine from the steroid dose by a little bit if we can. Again, always important to talk with your care team as to risk/benefit about holding certain treatments.

An Expert’s Take on Promising Myeloma Treatment and Research

An Expert’s Take on Promising Myeloma Treatment and Research from Patient Empowerment Network on Vimeo.

 Myeloma research is advancing quickly. Dr. Joshua Richter, a myeloma expert, shares his excitement about emerging treatments in development.

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

See More From Engage Myeloma


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Myeloma Treatment Decisions: What Should Be Considered?

Myeloma Treatment: When Should a Clinical Trial Be Considered?

What Standard Testing Follows a Myeloma Diagnosis?


Transcript:

Katherine:

When it comes to myeloma research and emerging treatment options, what are you most excited about, specifically?

Dr. Richter:

So, I think the big thing that I’m excited about from myeloma that we’re on the cusp of is T-cell engagers and T-cell based therapies. And, essentially, we all have T cells in our body, and T cells are a part of our immune system. They attack bacteria, viruses, and cancer.

And one of the best cancer fighters that exists is our own immune system. And the old way of treating cancer and blood cancers like myeloma was just to give medicines that suppressed all of the immune system, the good and the bad. Now, we’re trying to be more precise, and there’s certain parts of the immune system that we don’t want down, we want up. So, they help attack the cancer.

And the two biggest technologies are something called CAR T and something called bispecific antibodies. CAR T stands for chimeric antigen receptor T cells.

And, basically, what that is is we collect your T cells, we engineer them in the lab to rev them up and target the cancer. And we can put them back into you and they attack the cancer, very exciting. And then we have something called a bispecific antibody that has two arms. And as we infuse this medicine into you, one arm grabs onto the cancer cell, the other arm grabs onto your T cell and makes that T cell activate and attack the cancer cell.

And a lot of these drugs are in clinical trials as well. So, we’re very excited about moving from, you know, just lowering everything, the good and the bad, to being more precise and saying, no, no, no. There are some cells that we want way, way up.

Katherine:

Right. Right. So, you’re – you’re being much more specific now.

Dr. Richter:

Mm-hmm.

Myeloma Treatment: When Should a Clinical Trial Be Considered?

Myeloma Treatment: When Should a Clinical Trial Be Considered? from Patient Empowerment Network on Vimeo.

 At what point should a clinical trial be an option for myeloma treatment? Dr. Joshua Richter shares his perspective on the appropriate time to weigh clinical trial participation and the potential benefits.

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

See More From Engage Myeloma


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Is the COVID-19 Vaccine Safe for Myeloma Patients?


Transcript:

Katherine:

When should a clinical trial be considered for myeloma treatment?

Dr. Richter:

So, clinical trials are an extremely important component of how we manage myeloma. And I think there are a lot of myths and misconceptions about trials. Trials are not always things to do after everything else failed. From my standpoint, at every point along the way, we should always consider clinical trials, because they offer something really amazing. They offer us access to drugs way before they’re approved.

And the benefit of not waiting until the end, after you’ve been through everything else, is two-fold. One, in order to get on a trial, you need to fit certain criteria, inclusion, and exclusion criteria. You need to have myeloma, but you can’t be so sick from other medical problems that you’re not going to tolerate that treatment well. As such, unfortunately, some patients after they’ve been through all the other therapies may not qualify for a clinical trial, and that can be really upsetting.

The other benefit of doing a clinical trial early on is if you go on a new drug and it doesn’t work, you have all of the other standard of care options available at a moment’s notice. But if it does work and you gain access to a drug way before it’s approved, and it happens to work extremely well in you, you can have an unbelievably long remission and still have all of the drugs that are available. And, potentially, in that time on the drug, new standard of care drugs are approved. It even deepens the well that you can reach into to grab more options. So, at all times along the way, it’s always important to weigh the risks and benefits of what we call standard of care treatment versus clinical trial options.

Myeloma Treatment Decisions: What Should Be Considered?

Myeloma Treatment Decisions: What Should Be Considered? from Patient Empowerment Network on Vimeo.

When deciding on a myeloma treatment, what factors affect your choice? Dr. Joshua Richter shares key considerations, the patient role in making decisions, as well as key questions to ask about treatment

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

See More From Engage Myeloma


Related Programs:

Which Myeloma Patients Should Consider Stem Cell Transplant?

Myeloma Treatment: When Should a Clinical Trial Be Considered?

Is the COVID-19 Vaccine Safe for Myeloma Patients?


Transcript:

Katherine:

Dr. Richter, would you please start by introducing yourself?

Dr. Richter:

Sure, my name is Dr. Joshua Richter. I’m an Assistant Professor of Medicine at the Tisch Cancer Institute Icon School of Medicine at Mount Sinai and the Director of Myeloma at The Blavatnik Family Medical Center at Chelsea at Mount Sinai.

Katherine:

Great. Thank you. When making a treatment choice, what are three key considerations for myeloma patients?

Dr. Richter:

Absolutely. So, whenever we decide on treatment options, we consider three main topics: patient-related factors, disease-related factors, and treatment-related factors. So, patient-related factors are easy. How old or young are you? How fit or frail? Do you have any comorbidities, meaning other medical problems like heart disease or diabetes?

Disease-related factors are another important one. How aggressive is your disease? Is it rising up very quickly? Is it very slowly? Do you have something that we call extramedullary disease which means myeloma outside the bone marrow in the mass that we call a plasmacytoma? And that influences how we treat things.

And the last is treatment-related factors. What treatments have you, previously, had, how did you respond to them, and what side effects did you have?

If you developed a lot of neuropathy with one drug, we may not want to choose a drug that continues to have that type of side effect profile.

Katherine:

What’s the role as a patient in making treatment decisions?

Dr. Richter:

The role, from my standpoint of the patient, is honesty. You don’t get extra points for being in pain. I want to hear from you. I want you to tell me what your concerns are, short-term, long-term. I want you to tell me about little problems that you don’t – it’s not that you don’t want to bother your care team, we want to know.

Because something little may mean something big to us. So, all we want is for your well-being. And the better we keep those lines of communication open, the better.

Katherine:

Are there questions that patients should consider asking about their treatment plans?

Dr. Richter:

Absolutely. I think in a day and age where there’s so many different options, I think it’s always important to ask the care provider, what are the alternatives to this? Or why did you select this treatment for me? Because many times, there are alternative answers. So, in myeloma, there are a lot of options that may be good for someone. And the physician team may say we recommend this drug, and the patient may have trouble getting back and forth to clinic for logistical reasons. And there may be an all-oral alternative that if you don’t ask, we may not know that that’s going to be your preference. So, really that dialog is crucial.

What Standard Testing Follows a Myeloma Diagnosis?

What Standard Testing Follows a Myeloma Diagnosis? from Patient Empowerment Network on Vimeo

What tests will you have following a myeloma diagnosis? Are there additional tests you should request? Dr. Joshua Richter provides an overview of key testing for myeloma and why each test is necessary.

Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.

See More From INSIST! Myeloma


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Myeloma Treatment Decisions: What Should Be Considered?

Myeloma Treatment: When Should a Clinical Trial Be Considered?

Is the COVID-19 Vaccine Safe for Myeloma Patients?


Transcript:

Katherine:

What standard testing follows a myeloma diagnosis?

Dr. Richter:

So, the standard testing that follows a myeloma diagnosis is multifaceted. So, the first one is blood work. And we draw a lot of blood tests to look at the bad protein that the cancer cells make. So, we send tests like a protein electrophoresis which tells us how high that bad protein is. We send immunofixation. That test tells us what type of bad protein it is. You’ll hear names like IgG kappa and IgA lambda.

These are the different types of bad proteins made by myeloma cells. Oftentimes, we’ll send urine tests to find out how much of that bad protein that was in the blood is coming out in the urine. We will, typically, do a bone marrow biopsy. It’s a test where we put a needle into the back of the hip bone to look at the marrow itself. And we’ll use that marrow to figure out how much myeloma there is, any other characteristics like the genetic changes in those cells.

The other big thing is imaging. So, the classic imaging that we do with myeloma is something called a skeletal survey. It’s, basically, a listing of X-rays from head to toe. But nowadays, we have newer techniques, things like whole body low-dose CAT scans, something called a PET-CT scan, and MRI scans. And your care team may have to figure out which one is right for you at what given time.

Katherine:

Mm-hmm. Are there additional tests that patients should ask for?

Dr. Richter:

Absolutely. One of the most important things from myeloma has to do with the genetic risk stratification.

So, for almost all cancers, the staging has a very big impact. And people will often think of cancer in stages I, II, III, and IV, and they’re managed very differently depending upon what stage it is. Myeloma has three stages, stage I, II, and III. But the most important thing is, actually, beyond the staging is what’s called the cytogenetics risk stratification. So, it’s really important when the bone marrow is sent to be sure that it is sent for, kind of, advanced techniques. Because you really want that snapshot of exactly what the genetic profile is, because that gives us information of A) how to treat, and B) prognostic, you know, who will tend to do better or worse based on this information. And even though that may not tell us which drugs to use, specifically, it may say, should we do something like a transplant or not? Should we consider a clinical trial early or not?

Katherine:

I see. How do test results affect treatment choices?

Dr. Richter:

So, test results can affect treatment choices in a number of ways. Probably, the most common one is thinking about the routine blood tests like your CBC or complete blood count and your chemistry, which looks at things like your kidney function. Some drugs tend to have more toxicity to the blood counts. So, if your blood counts are very low, we may choose drugs that don’t lower the blood counts very much.

Kidney function which we, usually, measure by something called the creatinine. Creatinine is made by the muscles and cleared out by the kidneys. So, if your kidneys aren’t working very well, you don’t pee out creatinine, and that creatinine level will rise in the blood. If your creatinine level is high, we may choose certain drugs that don’t affect the kidneys or not metabolized or broken down by the kidneys.

The genetic studies that we use – we’re not quite at this base yet where we can say, if you have this genetic abnormality in your myeloma, we should use this drug except there’s some really great data on the cutting edge about a drug called venetoclax.

Venetoclax is a pill that’s used to treat other diseases like lymphoma and leukemia. And it turns out that people who have what’s called a translocation (11:14) which means part of the 11th chromosome and part of the 14th chromosome in the cancer cells swap material.

Those people respond amazingly well to venetoclax. So, we’re starting to have what we would call precision medicine where we find your genetic abnormalities, not that you got from your parents or passed to your kids, but the genetics inside the tumor cells to tell us which treatments will work best for you.

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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Have You Had These Essential Myelofibrosis Tests?

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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.

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What Are the Considerations When Choosing Myelofibrosis Therapy?

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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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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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Have You Had These Essential Myelofibrosis Tests?

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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.

Related Programs

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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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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

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Expert Perspective: Promising Myelofibrosis Treatment Research


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.

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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.  

How Can Myeloma Patients Advocate for the Best Care?

 

How Can Myeloma Patients Advocate for the Best Care? from Patient Empowerment Network on Vimeo.

Dr. Peter Forsberg shares advice for myeloma patients on why it’s important to speak up about symptoms and side effects, how to become a better partner in their care, and the role of a second opinion.

Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.

See More From The Pro-Active Myeloma Patient Toolkit

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What Should You Know About Myeloma Treatment Options?

Transcript:

Katherine:                  

What is some key advice that you give patients when they’re considering their treatment options?

Dr. Forsberg:             

Well, I think one important one is to always feel comfortable communicating with your provider. I think that there no by the book questions, list of questions, that’re the right questions to ask. I think the more important thing is trying to establish a good working relationship with your treatment team. Myeloma is much more of a marathon than it is a sprint. So, getting comfortable with your team, getting comfortable with a relationship and a partnership that can be often many years in duration, are really critical steps.

So, I think laying that foundation, feeling comfortable asking questions, trying to understand why. Understand how and what are tools to monitor what the myeloma will be and what indicates success or a need for something else. Those would all be critical pieces that I would encourage patients to feel empowered to be part of.

Katherine:                  

Patients can sometimes feel like they’re bothering their healthcare team with the comments and the questions. So, why is it important for patients to speak up when it comes to their symptoms and side effects?

Dr. Forsberg:             

Well, I think feeling comfortable being vocal about what’s going on is one of the key issues to navigating myeloma successfully. Being aware of issues, even if they may seem minor or insignificant, they may be an indicator for something that is emerging in terms of a treatment related side effect that we wanna be aware of. There are treatment side effects that we are willing to work through. But it can be very broad in terms of the spectrum of how we maneuver through different side effects.

And additionally, we always want to be aware of any issues that may be going on that could be a sign for what’s happening with the myeloma. So, trying to be vocal. Not only to understand what’s going on, what our treatments are, how successful are we at any given point in time, where things stand. But also, to make sure that you are putting things on your provider’s radar are key. So, lots of folks want to be good and compliant patients and we certainly appreciate that hope. But being assertive in terms of issues that may be coming up or questions that you may have, can really make for a much more successful long-term relationship in terms of how we manage the myeloma.

Katherine:                  

Well, do you have suggestions on how a patient could feel more confident in speaking up and becoming a partner in their care?

Dr. Forsberg:             

Well, certainly using tools like, if you found your way to this material, I think is a great first step.

Becoming a little bit more versed in the myeloma, in the language of the myeloma, what these tests that we use are. What their results might be. Using a number of great patient specific organizations are great first steps. So, being proactive about learning, to some degree about the myeloma. And then feeling comfortable asking that first questions. Once you begin the process of unlocking the myeloma and demystifying what it is and what these tests mean and where we stand, then that can really build on itself and allow folks to feel more in control of their myeloma and their myeloma journey.

Katherine:                  

And if a patient isn’t feeling confident with their treatment plan or their care, do you recommend that they seek a second opinion or consult a specialist?

Dr. Forsberg:             

Well, I never think it’s a bad idea to think about a second opinion or seeing a myeloma specialist. Even if you feel very comfortable with your treatment plan. Myeloma’s a unique disease and our approaches for it may be somewhat different, person to person.

And your needs as a myeloma patient my change and they may change somewhat abruptly. So, having seen someone who specializes in myeloma as part of your care team, and usually it is a care team. And there’s different models we sometimes work with in terms of both local or primary oncologists, as well as more specialized academic oncologists. We’re used to working through all sorts of models to provide the best possible care for patients. So, I never think it’s a bad idea to ask about that. Because having that more robust team is usually mostly benefit without adding a lot of headache. 

Is My Myeloma Treatment Working?

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

How can a myeloma patient know if their treatment is working? Dr. Peter Forsberg explains tests involved in determining if myeloma treatment is effective and factors that may indicate that it’s time to switch therapies.

Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.

Download Program Resource Guide

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources:

What Key Tests Should Follow a Myeloma Diagnosis?

What Key Tests Should Follow a Myeloma Diagnosis?

Myeloma Treatment Decisions: What’s Right for You Resource Guide

Transcript:

Katherine:                        

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

Dr. Forsberg:              

So, we’re lucky in myeloma in that we have some pretty easily accessible tools to evaluate how our response is going. How the myeloma is responding to treatment. How we’re sustaining that response and if we may be losing it at some point in time. And a lot of those come down to those blood tests I mentioned before.

The tools that measure protein levels or antibody levels in the blood, whether that’s intact antibodies or fragments of antibodies. So, that is that serum protein electrophoresis or serum free light chain levels.

Sometimes in conjunction with urine collections, which can measure abnormal antibodies in the urine. Those are ways that we can monitor on a month-to-month basis, how well the myeloma is responding to treatment. How well we are sustaining in a response or remission status. Or if it might be starting to come back.

We do at times use those in conjunction with other tests that look at things like bones using X-rays, MRIs or higher resolution scans like a PET scan. Or things like bone marrow biopsies which we may do at specific time points to evaluate the myeloma in different ways.

Whether that’s to evaluate a remission and see how deep that response might be, correlating it with blood work. Or if the myeloma come back, making sure we understand the characteristics of it. So, we’re lucky to be able to draw on tools that are not very invasive using bloodwork and sometimes urine. But we may couple that at certain other points in time with more substantial evaluations as well.

Katherine:                  

What could indicate that it’s time to switch therapies?

Dr. Forsberg:              

So, the most common indicator may be a change in one of those tests that I just mentioned. If we notice that there’s an increasing level of an abnormal antibody in the blood, one that’s usually produced by the myeloma, that may be our first indicator that the myeloma has become more active and that we need to change our treatment approaches. Other times people may develop symptoms from the myeloma that shows that it is becoming active and those would be our indicators. So, those are different ways that we help to monitor the myeloma. One is assessing the bloodwork and other things that we monitor pretty closely.

The other is being vigilant for new problems that may come out. So, we end up spending a lot of time with folks over the years with the myeloma and some of that may feel a bit routine, but we’re always trying to make sure that we’re attentive to new issues as they come up.