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Which MPN Treatment Is Right for You? What You Need to Know

Which MPN Treatment Is Right for You? What You Need to Know from Patient Empowerment Network on Vimeo.

What should you know before deciding which treatment is best for YOUR MPN? MPN expert Dr. John Mascarenhas reviews key factors–including essential testing–that guide treatment decisions for essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). Dr. Mascarenhas also provides an overview of available treatment types and why he’s hopeful about the future of MPN research.

Dr. John Mascarenhas is Associate Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai. Learn more about Dr. Mascarenhas, here.

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Transcript

Katherine:

Hello, and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to discuss how to access the most personalized therapy for your MPN and how you might benefit from key testing. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you.

All right. Let’s meet our guest today. Joining me is Dr. Mascarenhas. Welcome. Would you please introduce yourself?

Dr. Mascarenhas:

Thanks for having me. My name is John Mascarenhas. I am an associate professor at the Icahn School of Medicine here at Mount Sinai in New York City, and I am a clinical investigator in myeloproliferative neoplasms, and I direct the Adult Leukemia Program here.

Katherine:

Excellent. Thank you so much for taking the time out of your busy schedule to join us today. Before we delve into our discussion, let’s start with a term we’ve been hearing a lot about recently. How would you define “personalized medicine?”

Dr. Mascarenhas:

So, it’s a good question because I think it’s poorly defined in many ways because it can mean different things I think to different people. And, it’s a definition that’s in evolution. So, I think at its core, personalized medicine tries to embody the concept of creating an evaluation and management plan that is specific of tailored to that patient on multiple levels.

On a personal level, on an objective level of what the patient’s objectives are with their therapy or their disease, and then on a biologic level in terms of the type of disease, and now increasingly, on a molecular level. So, in some cases, it may be personalized therapeutics that are specific or targeted to certain mutations that the patient may have. And, that’s kind of where things are evolving from a treatment perspective. And to me, personalized medicine should be the goal of any interaction with a patient that you have to personalize the approach. Because, every patient that we meet is quite different and distinct from the next patient, and their own sensitivities, understandings and desires can be quite different. So, you want to personalize that approach to that patient at the most basic level.

Katherine:

Yeah. Well, that’s really helpful as we move through today’s program, which is going to cover the three classic MPNs, polycythemia vera, essential thrombocythemia and myelofibrosis. So, for someone who has one of these conditions, can you help us understand how one may progress to the next?

Dr. Mascarenhas:

So, these are a very heterogeneous or variable group of diseases that are under an umbrella called the myeloproliferative neoplasm. So, MPNs can really present and behave and have very different clinical courses. So, I think it’s very important for patients to realize that these are rare diseases, that that has a complexity to it, because they don’t always have the ability or the privilege to know other patients or people in their lives that may have these diseases. So, it could be very frightening from a level of feeling isolated or alone with a diagnosis like this and not having familiarity, but also, that these are vague diagnoses in the sense that when you have breast cancer, one can kind of conceptualize that there is a mass in the breast, for example, and that that can be staged. It can go to the lymph nodes in the armpit, it could spread below. And people can kind of understand that concept. I think it’s a little bit more challenging when you talk about MPNs, because it’s a little bit more abstract.

These diseases are within the bone marrows at diagnosis. So, they’re not staged in a physical way, and they are complex because they can lead to high blood counts, low blood counts, different types of symptoms, and the approaches really have to be personalized. They are all three interrelated because there are commonalities. So, there are certain clinical commonalities and also biologic commonalities. So, for example, the JAK2 mutation, the JAK2V617F mutation is seen in all three diseases. So, it’s not specific to one or the other.

It’s more common in polycythemia vera, but in about 50 percent of patients with ET, and 50 percent of patients with MF, you can see this mutation. So, the mutation alone doesn’t really tell us what the disease is. It just tells us you have one of these diseases. And, there are other mutations. So, a bone marrow biopsy then becomes integral in helping subtype the patient and then create that treatment plan and that outlook that’s specific for that disease.

And as you mentioned, to make it even more complicated, these diseases can overlap not just biologically, but in a continuum. So, patients with ET or polycythemia vera can progress in some cases to myelofibrosis. And, all three diseases in a minority of patients can progress or evolve into acute myeloid leukemia, which is a more aggressive form of bone marrow cancer.

Katherine:

Well, let’s turn to testing. And, you did just mention this. You touched on it a moment ago. What tests are necessary to help understand a patient’s specific disease at diagnosis?

Dr. Mascarenhas:

Usually, the blood counts are the first opening door test that allows some understanding of, again, either an abnormal production of red blood cells, platelets or under production of these cells. And, that’s really where often the evaluation begins. And then, there are further blood tests that often are done.

And I would say almost indefinitely or almost definitely one should have a bone marrow biopsy that helps categorize the type of myeloproliferative neoplasm because there can be overlap in how the blood counts can look from one disease to the next and overlap in the mutations like the JAK2 mutation. So, sometimes, the blood counts and the molecular testing are not enough, and a bone marrow biopsy looking under the microscope at the different types of cells, the proportion of cells, whether there’s fibrosis where there’s others other types of cells that shouldn’t be there, and they’re looking at the chromosomes and the flow cytometry, these are associated tests. As well as almost probably anywhere anyone goes at this point, they’re going to get next-generation sequencing, which is looking at multiple genes and mutations, and that gives a more broader, deeper sense of the disease.

So, those really become the integral parts. In some cases, patients will end up getting imaging of their abdomen to see if they have an enlarged spleen or enlarged liver.

Although that’s not always necessary, that is often part of the workup. So, it’s bloodwork, it’s bone marrow biopsy, sometimes imaging is usually the cornerstone.

Katherine:

And, what is molecular or biomarker testing?

Dr. Mascarenhas:

So, molecular testing today really means – at one point, it really meant looking at PCR for specific gene mutations.

So, for example, we would look at the JAK2 and we would say, “In a given person, is this gene mutated?” We all have JAK2 gene, but in patients with these diseases, they’re more commonly mutated which means altered in the blood cells. And, it’s very important for a patient to understand not in every cell in their bodies, but in their blood cell compartment. And, that helps us understand and start characterizing their disease, and sometimes that mutation can be measured. It can be at a low level. It could be a high level. And, that’s all put together in trying to understand the molecular basis of these diseases.

Today, next-generation sequencing has really taken over and that’s looking at more than just one gene.

Its sequencing could be 40 genes, it could be 200 genes, to get a sense of the complexity of the disease and looking for certain mutations which are considered biomarkers that can portend prognosis or I think increasingly, we’ll see may inform treatment decisions and may even be targets themselves of therapies.

Katherine:

Right. Should all patients diagnosed with ET, PV, or MF undergo biomarker testing? Is that necessary?

Dr. Mascarenhas:

I would say it’s part of the modern evaluation and management of patients today. I don’t think that that was true 10 years ago. But I think the field has matured. I will say I’m the first person to acknowledge to patients that we get a lot of information back, and the truth is we don’t often know what to do with all of that information. So, sometimes we get information back that can cause anxiety because you can see mutations in genes. But they don’t always inform us on how to educate the patient about their disease or tell us what to do with the treatment.

So, there is still a lag as there normally would be between the testing of the results that we get, and then the actual knowledge of what to do with that. And, that’s still a process that’s in evolution.

Katherine:

Right. Some patients may not know if they’ve received these important tests. So, what key questions should they ask their physician about testing?

Dr. Mascarenhas:

Well, I think it’s important that the patients feel empowered to understand sort of where the field is and what key questions you would ask a physician, hematologist who’s taking care of you. So, I think all patients should be aware of their diagnosis, the name of the diagnosis, the subtype, but also do they have any of the key driving mutations, the JAK2 mutation, the calreticulin mutation, the MPL mutation, and that’s usually done off of a bone marrow biopsy sample, but it can be done off peripheral blood. And, they may not always know that it’s done. So, I think having a discussion with the position to understand there are criteria that exist called the World Health Organization criteria that are updated frequently and should set a standard throughout the world of how you diagnose and establish these diagnoses.

So, I think it’s important for physicians to be able to convey to the patients with confidence, “We follow these criteria and you have these criteria and we’ve done this testing that shows that you have these mutations.” And not just regurgitate what they found, but help them understand and navigate with that means, which again, I will point out that sometimes we don’t know. But I think it’s important for physicians to convey sometimes that some of the findings that they may see, for example, patients look on portals these days and they can look at their labs and stuff like that. And, we don’t always have a terrific answer or an informed answer for everything that we get back. And, we will potentially in 10 years from now, but sometimes at the moment, we don’t. But I think a discussion about the meaning of the labs that are obtained is probably good for the patient to understand what’s being done.

Katherine:

Absolutely. It sounds like each person’s situation is unique and should be considered before making any treatment choices. Can you talk about how the results of these tests may affect prognosis and treatment?

Dr. Mascarenhas:

So, we do have risk stratification systems that we use for essential thrombocythemia, polycythemia vera, and myelofibrosis. I’ll talk about myelofibrosis because that’s probably a little bit more of a complex and sophisticated model. It’s also changing, and we update it frequently. And, these models are imperfect, so I always warn patients to not put all of their money in one basket when we talk about risk stratification. They broadly help us understand where a patient is in their disease course. So, for example, in myelofibrosis, historically, the DIPSS, the Dynamic International Prognostic Scoring System is used, which considered five clinical variables that have been shown to be independently prognostic. So, at age over 65, the presence of blasts or circulating immature cells in the peripheral blood, anemia, hemoglobin less than 10, symptoms, fevers, night sweats, weight loss or a high white count over 25,000, you those points up.

And patients can do this online. There are calculators that you can calculate your DIPSS score. And, you’ll see that there are four different risk groups that range from low risk to high risk, and they are associated with median survivals. We now know that mutations influence those, have influence on prognosis. So, there are a group of high molecular risk mutations like ASXL1, SRSF2, IDH1/2. So, there are mutations that also have prognostic significance, and we incorporate them into the decision-making.

And, essentially, and this is where I think patients have to be very careful, physicians have to be very careful with conveying this. With these risk models whether they are clinical variable risk models or these integrated molecular risk models, each category is associated with a median survival, that’s based on retrospective studies. But that doesn’t tell the patient specifically what they should expect in terms of survival. And, I always fear that patients, when they look at these things, or even physicians when they convey them that they may inadvertently misrepresent or convey what those really mean.

And, I think the purpose of those risk stratifications is really to help guide a risk adapted treatment approach that’s reasonable and is weighted for benefit to risk of the disease. So, for example, if you have advanced disease with a high-risk score of intermediate to or higher, bone marrow transplant in certain patients may be a warranted therapy to consider. So, they really help inform treatment.

Katherine:

Right. You mentioned a couple of these already. But, outside of testing, what other factors should be considered when choosing treatment?

Dr. Mascarenhas:

I think patient expectation. So, sometimes physicians and family will impose what they want for a patient, and that may not be what the patient really wants. So, I have learned over the years that it’s crucial to make sure that you understand the patient and what the patient’s expectations, desires, and that’s influenced by the life they’ve lead or the remaining life that they want to live and their own personal religious and spiritual beliefs.

] So, I think knowing your patient and understanding what their expectations are, it’s fundamental, and sometimes, it’s overlooked. So, understanding that, I think, is very crucial. And then, dividing what are the objectives of the treatment in a given patient? Is it really to improve anemia in some patient versus perhaps a different patient, it may be to improve their quality of life and reduce their symptom burden. And then in other patients, it may be purely trying to cure the disease with therapies that may be aggressive, which may not be appropriate for an older patient where toxicity could outweigh any potential benefit of survival or longevity. So, you really have to have a discussion with the patient or caregivers, and then define what are the goals in that individual to personalize that approach for that patient.

Katherine:

Right. Right. And, there’s the patient’s overall health, comorbidities, other things like that?

Dr. Mascarenhas:

Yeah, because we are not treating a disease in isolation usually. So, patients come with baggage posed of past diseases, current diseases.

And sometimes patients are not “fit” for certain types of therapies because they may be sick or they may have organ dysfunction that would make certain types of treatment approaches ill-advised because the toxicity could be higher. So, absolutely, you need to know their comorbid index, how much comorbidities they have and also their performance status, how active and how well they are in general.

Katherine:

Right. Are there specific biomarkers that may affect prognosis or treatment?

Dr. Mascarenhas:

So, yes and no. I mean, I think that’s an area of intense interest and research. So, we have identified certain biomarkers that have, as I mentioned, prognostic significance, and that may influence treatment decisions. So, patients who have, for example, as we discussed next-generation sequencing and we see their mutations that are present, if they have an accumulation of high molecular risk mutations, that may give us a sense that perhaps that patient may not enjoy the full benefit and duration of benefit of, for example, a JAK inhibitor as another patient that has a less complex disease.

And, that doesn’t necessarily mean that the therapy is not appropriate for the patient. But it may help us plan and be prepared to move on to the next therapy sooner or to be more vigilant for changes that would tell us it’s time to move on. So, I think they help us maybe get a general sense of things and put things into perspective. They don’t always necessarily inform us on a change in therapy immediately or the next or the most immediate therapy. But I do think that that will change because I would predict in the next five to 10 years, I think that the number of available drugs for myelofibrosis, for example, will likely double from what it is now. I think we will have an armamentarium to choose from, and what we will learn from trials that are ongoing is there may be certain profiles, mutations, chromosomal profiles, other clinical variable profiles that we will learn from these trials that will help us to find upfront, “Well, this profile really should go with his medication. That profile should go with that medication.”

An early of example that would be we’re learning that not all patients with the JAK2 mutation are created equal, that you can have different burdens of JAK2 mutation. And, patients with low burden JAK2 mutation, for example, may fare better with up a specific JAK to inhibitor like pacritinib than patients who get treated with other JAK inhibitors like ruxolitinib.

So, there are differences even within patient defined by mutation that may help us predict which of the JAK inhibitors, as an example, may be more appropriate as a first-line therapy. So, I think that will evolve more so over the next five to 10 years.

Katherine:

Dr. Mascarenhas, what is inhibitor therapy and how does that work?

Dr. Mascarenhas:

So, inhibitor therapy in general are usually oral drugs for the most part, small molecule inhibitors that are geared and usually specific but not totally specific because then they can have off-target effects, but geared to inhibiting usually an enzyme that is overactive or is contributing to the pathophysiology of the disease.

I think in MF, probably one of the best examples is a JAK2 inhibitor. So, there are a number of JAK2 inhibitors that have been in clinical testing. There are two that are approved, ruxolitinib (Jakafi) and fedratinib (Inrebic), which are excellent drugs in inhibiting JAK2 protein itself in the cells that could be either upregulated or hyperactive in the signaling pathway, and it quiets down a signaling pathway in the hematopoietic cells that leads to a lot of the manifestations of the disease, namely symptoms and spleen.

So, one of the clear benefits of JAK inhibitors that was established many years ago and reinforced by multiple drugs that are either approved or in late-stage testing is these drugs are excellent in improving the symptom burden in the patients and reducing their spleen. Unfortunately, as a class, we’ve not seen these drugs induce remissions or cure patients. So, there’s still interest in developing, obviously, non-JAK inhibitor therapies. But inhibitors in general are inhibiting proteins that are either inappropriately activated or part of a cascade of signaling molecules that are contributing to the disease.

And they are not chemotherapeutic, which might be an important point to make. In past days, we’ve relied heavily in hematologic malignancies in using chemotherapies which are nonspecific and just kill dividing cells whereas inhibitors typically are targeted, and in some sense, it’s personalized to the disease with toxicity profiles that are usually quite distinct from the traditional chemotherapies that we use.

Katherine:

Well, outside of inhibitor therapy, let’s review other treatments for patients. Depending on the patient, it seems like ET or PV may be easily managed. So, how are they treated? Let’s start with essential thrombocythemia or ET.

Dr. Mascarenhas:

So, ET is a disease in which first and foremost, we’re trying to reduce the risk of thrombosis, clotting, and/or hemorrhage bleeding. So, typically, ET patients are risk stratified by low risk or high risk.

It’s almost simply based on their age, whether they’ve had a clot in the past, and some systems now even incorporate other factors like mutation status. And, you tailor the treatment based on their risk score. So, low risk ET patients don’t necessarily need to be treated. They can be followed expectantly and watched. The height of the platelet count does not predict thrombotic risk. So, we don’t treat the platelet count per se. A high-risk patient is at high risk for clotting. So, these patients almost invariably are getting aspirin at a baseline, and they are often on cytoreductive therapy. And sometimes, that is chemotherapy like hydroxyurea (Hydrea). Sometimes it’s a non-chemotherapeutic option and like anagrelide, and sometimes it’s a biologic therapy like interferon alfa either 2a, PEGASYS, or 2b ropeginterferon. And, these are therapies that have rationale, that have clinical data, that have demonstrated reduction in risk of clotting, which again is the reason why we treat high-risk ET patients.

Katherine:

And, what about PV, polycythemia vera?

Dr. Mascarenhas:

So, in polycythemia vera, it’s similar to ET. We risk stratify patients low and high risk based on age and clotting histories. And whether you’re low or high risk, we give PV patients aspirin or at least once daily, and we look to keep their hematocrit below a threshold of 45 percent. And sometimes in women, we even go lower, to 42 percent. But the idea is that controlling the hematocrit, which is one of the red blood cells indices, you reduce the risk of having clots, and that’s been shown actually many years ago and reinforced in a very well-known study called the CYTO-PV study in Italy documented that if you keep the hematocrit less than 45 percent, so, stringent control versus allowing for less stringent control between 45 to 50, that you reduce by fourfold the number of cardiovascular events that can occur.

So, we know that controlling the hematocrit is important, and that can be done, again, with hydroxyurea, interferon, and ruxolitinib. The JAK2 inhibitor has also proved specifically for patients who had an intolerance or refractory hydroxyurea, but also importantly as a drug that can address, probably better than most drugs in this field, the symptom burden that could be problematic for some of those patients. But it’s really about controlling the hematocrit.

Katherine:

Yeah. Since myelofibrosis is a progressive condition, I imagine that makes it more difficult to manage. So, what else is available for patients with myelofibrosis?

Dr. Mascarenhas:

The first line of treatment is typically a JAK inhibitor, although I would say that there are a subset of patients – well, there are patients we sometimes meet that have very low risk disease. They don’t have those clinical variables we discussed before that could uptick their risk score, and some of those patients can be watched.

And interestingly, there are a subset of patients that can have an indolent or slow form of the disease where they don’t have aggressive changes in their disease, their blood counts, their symptoms, their spleen, and don’t need immediate treatment. Most patients would benefit from a JAK inhibitor, although there are a subset of patients where their issue is less simple in spleen burden and it’s more anemia.

So, we take those patients where anemia’s the focus, we look at their erythropoietin level, which is their endogenous hormone level that regulates red blood cell production. If it’s low, we give them a lab-based form of erythropoietin, something called PROCRIT or Aranesp. If it’s high, we will move on. We can use a drug called danazol, which is a synthetic male androgen which can improve hemoglobin levels in 20 to 40 percent of patients. Or, we can use a drug called lenalidomide, which is an immunomodulatory drug. And, more recently, there’s a drug in testing called to luspatercept, which is an active activin receptor ligand trap. So, there is a growing armamentarium of drugs that can be used to try to alleviate the anemia which is present and can be a significant issue in about a quarter of patients with myelofibrosis upfront at time of diagnosis or about 75 percent through the course of their disease. So, that’s an unmet need that still requires attention and may alter the treatment plan for a given patient.

Katherine:

What about stem cell transplants?

Dr. Mascarenhas:

So, we relegate stem cell plant transplants for those patients as mentioned before that are higher risk because we think that the potential benefit-to-risk ratio is in favor of transplant.

Transplant is really a modality that is the only modality that offers the potential for cure, but it’s also a modality that poses a significant risk of morbidity and mortality associated with it. So, it has to really be taken very seriously. It can’t be the kind of treatment you would think of as a last resort at the last minute. Once you see a transplanter, if they’re interested in that therapy and see it early on in the disease course, in my opinion, to start that dialog and then figure out when is the optimal time to employ a bone marrow transplant, which is not a surgical procedure. It’s often thought to be surgical. It’s not a transplant of an organ. It’s a transplant of hematopoietic cells. So, it’s really an infusion of stem cells that then end up in the person’s bone marrow, and they create a whole new hematopoietic system and immune system. And with that, you can have an immune system that then goes after the myelofibrosis stem cells.

That’s called graft-versus-leukemia effect. But with that included graft-versus-host disease, which is when the new graft, the new immune system doesn’t always recognize well the person’s own tissues, whether it’s the liver, or the lung, or the skin, and you can have immunologic reactions to that.

So, that’s a complex discussion. But, transplant, typically for patients less than 70 years of age who have high-risk myelofibrosis or even up to 75 if they have a good performance status and as we said don’t have a lot of comorbid issues with a goal of cure. So, if you have someone where their goal is to try to maximize their time out of the hospital and they’re not focused on longevity, they’re focused on quality of life, that may not be an appropriate patient for transplantation. So, I think a very upfront, honest and a transparent discussion with the patient about what to expect with transplant, what are the pros and cons, what are the risks involved, and importantly does it match up with their expectations or their desires.

Katherine:

Right.

We have a question from Mike that we received prior to the program. He wants to know, “What does it mean to have high-risk myelofibrosis?”

Dr. Mascarenhas:

So, high-risk can be defined different ways. For example, if you’re using the DIPSS score, it means that you have enough of those points to put you in a category that would suggest that your disease is more likely in a shorter time period to cause significant morbidity and mortality than someone who has low risk disease. So, it’s really, as we said before, we don’t stage myelofibrosis like stage I, II, III, and IV metastatic disease. But we risk stratify patients. We put them in these categories, and that helps to decide what treatments may be more appropriate. And as we were discussing, transplant is a therapy if you’re a high-risk patient and you’re inclined and you don’t have a lot of comorbidities, and you’re not very advanced in age. That may be a treatment that is appropriate for a high-risk patient, a high risk for having a bad outcome of the disease within a shorter period of time.

Katherine:

Right.

And we have another question from Craig that we received earlier. “I’m currently receiving regular phlebotomies for PV, but my doctor is considering switching me to inhibitor therapy. What can I expect and are there side effects that I should be concerned about?”

Dr. Mascarenhas:

So, for some patients, therapeutic phlebotomy is all that they need, and they do very well with it, and they don’t need to take a therapeutic like a JAK inhibitor or hydroxyurea, which is a non-specific treatment.

But some patients do. So, some patients where if their risk score is higher and their risk for thrombosis, that may be an appropriate indication. And some patients have a lot of symptoms with their PV. So, not all PV patients present and behave the same way. Some patients have a very low symptom burden. Some patients have a very significant symptom burden. Itching, for example can be a very annoying and very troublesome symptom for patients with PV.

And, if you don’t have PV or you don’t know someone with PV, you may not understand or realize the negative impact of having intractable itching, often associated with taking a shower or warm water.

And, that can really detract from quality of life and cause a lot of anxiety. So, that’s an example of where sometimes a JAK inhibitor like ruxolitinib can be really lifesaving in terms of restoring quality-of-life and functionality to a patient.

Usually, drugs like ruxolitinib are very well-tolerated too, which we’re fortunate about. There’s not a lot of toxicity associated with them. So, for example, nausea, vomiting, diarrhea, hair falling out with chemotherapeutics, you really don’t see with ruxolitinib or Jakafi. Easy bruising, headaches and some dizziness up front sometimes may be seen. They’re usually low-grade and they’re usually fleeting. And usually, the benefit, the feel-good aspect of it outweighs toxicity that can be seen with the drugs. They are immunomodulatory drugs. So, ruxolitinib or Jakafi may increase, to some small extent, but likely, real extent, infectious complications like shingles, urinary tract infections, upper respiratory infections. So, sometimes there is this increased risk. It’s often outweighed by the benefit of the drug.

But, there are risks that are associated, and of course the results are not guaranteed. So, I always warn patients, be careful when you look at the package inserts or talk to the physicians. Risks are risks. They’re not guaranteed. So, most patients don’t have these toxicities, but one is at risk for toxicity whenever they take any medication.

Katherine:

Yeah. Before we close, Dr. Mascarenhas, let’s talk about research. Are there new developments that you’re excited about?

Dr. Mascarenhas:

Absolutely. So, what I’m happily interested in and involved in is clinical investigation and moving the field forward, and there are many people out there that are similarly involved and they’re doing really excellent work. So, I am super jazzed and enthusiastic and optimistic, and it’s what gets me work every day and inspires me is all of the effort that is happening. And, it’s a continuum. So, it’s not just one person trying to try a different drug here and there. It’s really a bringing together of many different people because these are rare diseases.

Many different people from many different institutions that have different areas of expertise, but have a common goal of translating from laboratory informed data, so, not just taking a dart and throwing it at the dartboard and hoping it sticks. But actually taking data that we learned from the lab and leveraging that information to develop therapies that are informed, that are targeted, that are personalized and going through a process of evaluating them to get them into the clinic, with the goal of, and I would say ambitiously, our goal these days is moving beyond trying to make patients feel better, which is an important goal, but it’s really can we really target the disease in a more effective way to induce remissions, to, dare I say, cure patients. So, I think the ambitious goal of the clinical investigators and laboratory investigators that are active in MPN research today is really one looking for an understanding at the basis of the biology of the disease to develop curative therapies. And, I am optimistic that that will happen.

And, I don’t mean happen in a hundred years from now. I mean happen in our lifetime. So, that’s where we’re going. There’s a lot of very exciting drugs, oral and intravenous drugs and they target very different types of aspects of the disease, and I think patients and physicians will see that maybe those drugs are used best in combination. So, the idea of using one drug, waiting for it to fail and using another drug is really old news, and much of oncology is combination therapy. So, taking drugs that have different targets or mechanisms of action and non-overlapping toxicity to try to better target and delete what’s called the myelofibrosis stem cell that’s the basic issue here, which we don’t effectively delete other than transplant. So, our goal would be to put bone marrow transplanters out of business.

Katherine:

Well, that’s a great plan. I hope that that can happen one day. Thank you so much for joining us today, Dr. Mascarenhas. We appreciate you taking the time.

Dr. Mascarenhas:

My pleasure.

Katherine:

And thank you to all of our partners.

To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for joining us today. Thank you so much.

 

Multiple Myeloma: Danielle’s Clinical Trial Profile

Multiple Myeloma: Danielle’s Clinical Trial Profile from Patient Empowerment Network on Vimeo.

Multiple myeloma patient Danielle was a very active person – and even went on vacation – right before receiving her diagnosis. Her myeloma journey unfolded with her myeloma symptoms, diagnosis, treatment, and participation in a life-altering clinical trial. “I decided to do the study trial because I also wanted to help individuals. If it wasn’t going to help me, then my data that they collect from the study trial will definitely help the scientists, researchers, the doctors. It would help them try to find a cure.” 

See More from Patient-to-Patient Diverse Myeloma Clinical Trial Profiles

Transcript:

Danielle:

Hello, everyone. My name is Danielle.

My myeloma story began in 2011. I was experiencing pain in my hip and my back area, and it was the pain that would come and go.  I was also very lethargic in 2011 and couldn’t understand why I was so extremely tired, so I thought the pain in my hip and back area was due to sciatic nerve, and I just didn’t do anything about it, ignored the pain. My husband and I went on our first trip without our sons in October of 2011, and two days before the trip, I developed this really bad nasty pain in my hip and leg area, which actually altered my walk, but I had no idea what the heck was going on, and so I was so frustrated that I… As soon as we got home, I went to see an orthopedic doctor because at that time I was working out like five times a week, so I thought maybe I pulled something, a pinched nerve or something. So I went to see him, he took X-rays, I believe it was an MRI, couldn’t be sure, but when I went back to get my test results, he sat me down and said, “Mrs. Spann, there’s a mass here in your fibula, and I’m going to recommend you to an orthopedic oncologist.” So, that was the very beginning of my diagnosis, initial diagnosis. Of course, I was in denial because I’m like, I knew what an oncologist was, but he must not be talking to the right person, but I went ahead and I met with the orthopedic oncologist. He ran a bunch of tests and mentioned to me that I had myeloma, which is concentrated in one area, which was my fibula, and then he recommended that I have my fibula removed on my right leg. Two days before surgery was scheduled, I received a phone call from his office, saying, “Mrs. Stann, you have lytic lesions all throughout your skeletal structure, and we’re recommending that you go see a bone marrow transplant oncologist.” So now it’s becoming real. The diagnosis is what it was, and I just wanted to know how I could basically fight this. I’m the type of person where you tell me one thing and let’s try to find a solution, so I met with the bone marrow transplant specialist, the oncologist, and then we formulated a plan, and that plan was for me to go on my first study trial. And so that was my introduction into my having multiple myeloma.

I made the decision to participate in a trial, because I trusted my doctor. He had the expertise to understand where my myeloma was, the counts, how aggressive it was, and he recommended that I go on the study trial. He also told me that if the study trial was not going to work for me, or if it wasn’t helping me, that he was going to take me off the study trial. So, I was on the study trial from like January to March…to the end of March, and he sat me down and said that it was not working, my numbers weren’t really moving, and that he was taking me off the study trial. And he took me off the study trial, there were some other treatments that were involved, and then I had two stem cell transplants. After the transplant in 2012, I went ahead and started another treatment regimen, and I was on that for several years, which worked well. My numbers were coming down, but then unfortunately they started going back up, so he mentioned that I should go on another study trial.  I weighed the odds, and I knew that he would not lead me down the wrong path. So, I went ahead and I participated in the study trial that I’m still on today, and I’ve been on it for about three, four years.

I decided to do the study trial because I also wanted to help individuals. If it wasn’t going to help me, then my data that they collect from the study trial will definitely help the scientists, researchers, the doctors. It would help them try to find a cure. And so that’s what I wanted to help in some form or fashion, and when I first was diagnosed going to the Winship Cancer Center twice a week, there was a quote that was posted in the cancer center, and that quote was by Dr. Martin Luther King Jr., the quote read, “Life’s most important and persistent question is, ‘What have we done to help others?’” And I would go into the center and I’m like, “Yeah, what have I done to help others?” And me participating in the study trial, I felt like I’m helping others indirectly, and it wasn’t always just about myself, it was, “Okay, yes, the study trial gives the data, and it’s helping me, but it’s also helping that next person as well.”

So, I always look at my life as before diagnosis and after, and my after does not look like my before, I can’t do the same things, I can’t do the same things that I used to do. And one of those things is going to the mall and being in there like 10 hours, that’s so remedial, but it just goes to show like I cannot exert myself the same type of energy that I could before diagnosis. And again, that’s my new normal.  I stay positive with everything in life, things happen, but you just have to do what you can to make it better, no matter what it is.

I am happy and proud and so grateful and thankful to mention that as of January 2021, my myeloma is 0% detectable, which means there’s no presence of multiple myeloma in my blood, in my urine, nor in my bone marrow. And so I’m still on a study trial, and I have two different chemo meds that I have to take, and I just act accordingly if I know that one of the chemo meds that I have to take twice a week gives me an upset stomach. I just accordingly in finding different ways to push through it. It is what it is, and my motto when I was having my bone marrow transplants was, “This too shall pass.” And so no matter what I’m going through in life, no matter how down I get. This moment will pass. And so tomorrow, you’ll look back on today and say, “You know what, I did it, I made it.” And you’ll do that for the next day, until you realize that you’re just constantly defeating that previous day, and you’re moving forward.

So, I’ve heard the terminology of a clinical trial, never really paid attention to it because I never had to…I had an idea what the clinical trial was. But once it really came home to me, I realized that, in my words, the clinical trial is collecting the data necessary, they’re going to give you the trial medication, because they’re looking to get this, this medicine approved to put on the market. These medications would not get approved by the FDA, acetaminophen (Tylenol) at one point had to go have a study trial and then get approved by the FDA and then can be distributed to the masses. And so it’s the same with these other drugs. We need individuals to participate positively, knowing that if this is not helping me right now, it will help someone in five years, in two years, in 20 years. The advice that I would give is to trust your doctor, your doctor would not recommend a study trial if he felt that there was a medication that’s already on the market that would help you better. If the study trial you’re on is keeping you with your family, and at the same time is…the scientist, the researchers they’re gathering all this data, it could come to be an actual medication in three, five, seven years. And so just think of it as something that you’re helping society…and your fellow…and your fellow man.  

How to Play an Active Role in Your Myeloma Treatment and Care Decisions

How to Play an Active Role in Your Myeloma Treatment and Care Decisions from Patient Empowerment Network on Vimeo.

How can you actively participate in your myeloma care and treatment decisions? Engaging with your healthcare team is essential and may lead to better overall outcomes. In this program, Dr. Rafael Fonseca provides tips for how best to advocate for yourself or a loved one, as well as tools for making treatment and care decisions.

Dr. Rafael Fonseca is the interim director of Mayo Clinic Cancer Center and serves as the director for Innovation and Transformational Relationships at Mayo Clinic in Arizona. Learn more about Dr. Fonseca here.

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Myeloma Treatment Decisions: What Should Be Considered?

What Standard Testing Follows a Myeloma Diagnosis?

Which Myeloma Patients Should Consider a Stem Cell Transplant?


Transcript:

Katherine Banwell:    

Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today we’re going to explore how to engage with your healthcare team when diagnosed with myeloma, and we’ll discuss the patient’s role in care decisions. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Let’s meet our guest today. Joining me is Dr. Rafael Fonseca. Dr. Fonseca, welcome, and would you please introduce yourself?

Dr. Rafael Fonseca:   

Yes, of course. Happy to do that. Thank you very much, Katherine.  

I am a hematologist/oncologist, but I specialize in the area of multiple myeloma. I work at the Mayo Clinic in Arizona. I currently serve also as interim executive director for the Mayo Clinic Cancer Center that is at large across the Mayo Clinic enterprise. But at heart, I’m a myeloma doctor and I love to take care of myeloma patients. I devote my research and the rest of my academic activities to the field of myeloma.

Katherine:                  

Excellent. Thank you so much for joining us today. Let’s start with a question that’s on the mind of many of our audience members. We’re hearing that the COVID-19 vaccine is safe, but how effective is it for myeloma patients?

Dr. Fonseca:               

Thank you. I think that’s a fundamental question. It’s hard to know precisely how to gauge effectiveness when it comes to vaccination because historically, we know that is done by measuring antibodies and there’s a number of publications that are addressing this.

The concern has been two-fold. One is that because the disease itself is something that starts from the person’s immune cells become cancerous, that perhaps that would prevent them from having a very good response. Number two, and perhaps more importantly, will the treatments that are used for myeloma, etc. or lymphoma, can they interfere with our ability to mount an effective immune response? I think the response is mixed right now. I think I tell all my patients the upside is much better than the downside. I think we have a good record now of the safety of this product. I encourage everyone to get their vaccination.

I think it’s important to discuss this with your healthcare provider because sometimes people say, “Should I stop a little bit so that I can get a better response?” While it’s theoretically possible, we don’t want people to stop treatment if they don’t have to do that. Just my very last quick comment, the good news is that the community transmission is clearly going down as more and more people have participated in the vaccination.

We have more people who now have participated in this level of immunity that we have in the community. Hopefully, for patients as well as for their families, the risk of contracting this will continue to decrease.

Katherine:                  

Yeah. We can only hope. Well, let’s learn a little bit more about the disease itself. Dr. Fonseca, to level set with our audience, can you help us understand myeloma?

Dr. Fonseca:               

I’m happy to do so. Multiple myeloma is a cancer form of the bone marrow that arises when the cells that under normal circumstances protect us by the formation of antibodies. These are called the plasma cells. They become malignant. Myeloma is the last stage of a process where a plasma cell can go through a benign tumor or benign phase, if you may, something we call the monoclonal gammopathy, which by the way is quite common. About two percent of people over the age of 50 have this abnormality. Think of it like the colon polyp, a precursor condition.

There’s an intermediate stage that we call smoldering multiple myeloma, which is just more growth, but not quite at the level that it creates problems for the individual.

Then lastly, what we just simply call multiple myeloma, and that is when the growth of those cells becomes of such magnitude that a person starts having problems or starts having symptoms related to that. These cells live predominantly inside the bones in the space we call the bone marrow. They can do a number of things that actually lead to the symptoms and to the clinical presentation. As they grow in the bone marrow, they take some of that real estate.

A person may experience fatigue and that is because they have anemia.

The myeloma cells are also very characteristic because they can erode into the structure of bones, so destruction of bone is another feature that we see in patients with myeloma. That can be either seen on x-rays or sometimes people will present with symptoms related to bone pain or discomfort with movement or weight bearing. Those are signs that we look for.

Lastly, the myeloma cells product proteins and some of the fragments of those proteins can be damaging to the kidneys. Occasionally, people will present with decreased kidney function and sometimes outright failure of the kidneys. Those are the common presentations. It is a disease that mostly affects people in their 70s. It is not something that you can detect through routine testing; it’s just indirectly we start seeing abnormalities and then we do the right testing. If anyone is hearing this, of course, they need to have a detailed discussion with their own provider.

Katherine:                  

Of course, yeah. When a person is diagnosed with myeloma, they usually have a whole healthcare team. Who is typically on that team?

Dr. Fonseca:               

Absolutely. Let me start by saying the key to the successful management of myeloma is to have a well-organized team. It’s a disease that requires an integrated approach that usually brings around the patient a physician.

As part of my team, we also have advanced practice providers. We work with nurse practitioners that help us do the longitudinal care of patients. We have the nursing team. Every time I meet a new patient, I make it a point to bring my nursing team into the room so they can put a name and a face together, as patients will be interacting, of course, with a nursing team through the portal and the various visits. We have a team that is in charge of the chemotherapy administration. That is usually a separate a nursing team that is in charge of the administration of the medications. But we really don’t stop there.

We have pharmacists who help us review the medications for our patients. Very importantly, we have social workers that help us address psychosocial needs, as well as some of the practicalities that become inevitable when one deals with a serious diagnosis like multiple myeloma.

Katherine:                  

Yeah. Lately, we’ve been hearing this term, “shared decision making,” which basically means that patients and clinicians collaborate to make healthcare decisions, and it can help patients to take a more active role in their care.

I’d like to get your thoughts, Dr. Fonseca, on how best to make this process work.

Dr. Fonseca:               

We are very fortunate to live in this time of medicine, where ultimately, we recognize that the patient is the person expert. It is the patient decisions that should drive what is to be done in a situation. Whenever I interact with patients, I tell them, “Listen, I’m going to be like your counselor. I will provide you with options of what I think is reasonable. I will go to different degrees of effort in trying to convince you one way or another for a particular intervention. But at the end of the day, I only do a good job if I present you with the options and the pros and cons of those various approaches.”

I weave that into my language on every single conversation we have with patients. I think we’re way past the time where a physician would come and say, “This is what you’re going to do,” or “This is what will happen.” My language always includes, “I would recommend this.”

“I think the next best step for you to consider would be X, Y, or Z.” But ultimately, I look at patients and not infrequently at the person next to them, a family member or a close friend, and I say, “You’re the boss and with the person next to you providing additional support, comment, and guidance, we can together reach the best decision of what should proceed.” I think we’re incredibly fortunate because patients have access to sophisticated information, especially patients that have serious conditions such as would be cancer and, in my case, myeloma.

As an example, when I work with general internal medicine residents that work with me learning about hematology, I sometimes tell them, “You’re gonna walk into a room. Are you gonna be seeing what I say, this is like a tennis match between professionals. Are you gonna see the level of questions that patients are going to be asking me? They’re going to be asking me about the latest study that was presented at this meeting and the P value and this and that.”

“I can guarantee you that you would not have the tools to be able to address all those questions, simply because there’s such an in-depth understanding of the disease.” I realize this is not everyone. I’m giving you an extreme example. There are individuals that need additional support, more resources. But just to interact with someone who has such commitment to understand their disease and to help us by that understanding make the right decision makes my job so much more rewarding.

Katherine:                  

What do you think is the role of a patient then in their care?

Dr. Fonseca:               

I think it needs to be … I’m describing in some detail and there’s a lot to unpack there. Of course, patients are dealing with a very serious diagnosis. It’s okay to have periods where they are in a pause moment and they’re reflecting of what their facing, and that they can gather information from close family members.

I think we, as providers and the medical team, need to deliver a message that provides clear options for them as far as what the best next phase of their treatment or their management might be, including observations or supportive care. But the patient ultimately is a person who has to make that decision. I frequently get the question, and this is not surprising, and it happens all the time. A patient tells me, “What would you do if this was a family member?” I always tell them, “I always talk to you as if you were my family member, as if you were my brother, my mother, my father.

So, I try to live deeply to that fiduciary responsibility I have to your well-being. I recognize that there are circumstances, and that’s part of the finesse and the art of medicine, that I have to help a little bit more walk you through that step. Sometimes, it’s just human that one may want to say, I just want to disconnect. Maybe I’m not the person that wants to go and read in detail. But perhaps I have my daughter or my son who are helping me and understand better where things are.”

I think one of the key aspects of my role is to make sure that I have a sense that the person has a good understanding to be able to make an informed decision. At the end of it all, if the person decides to proceed in such way that doesn’t necessarily align with what I’m trying to do, I’m deeply respectful of that choice. I will go to extra lengths. So, if someone is foregoing treatment, when I know their treatment has a high likelihood of improving their quality of life, relieve a symptom, or improve survival, I don’t think I would do a good job if I don’t present why that’s so important. But ultimately, it is the patient’s decision.

Katherine:                  

Related to what you’ve just been speaking about, we have a question from the audience. This one is from Sarah. Her question is, “What advice do you have for caregivers? How can I be supportive during appointments?”

Dr. Fonseca:               

That’s a great question.

I have experienced this both as a physician, as well as a caregiver myself to someone who has had a cancer. I think I’m gonna say that there are several roles that caregivers play. Some of them are obvious and I’m gonna call them practical or perhaps even pedestrian, you know, organizing the activities of every day. That’s important, but a lot of people can do that. The second role is to be in assistance for the knowledge that is needed for some of this decision making. Sometimes patients can be overwhelmed, and we need some support and some vetting and peer process from a trusted and loved person so you can go through that.

That is very helpful, but what is essential, and the number one thing is you are first and foremost the loving family member or friend of that individual who is living through a very profound human experience. I think the first role of a caregiver has to be to express that role.

I, myself, reflect on moments where perhaps in a quick, reactive way I wanted to solve some of the immediate practicalities and what was needed most was a direct support. Even if I face a situation today, if I was, again, a caregiver for someone with a serious diagnosis with cancer, I would start with that priority. Number one, you are the support and the loving person. Number two is I will try to provide information. And number three, hopefully you can help with meals and the driving and what have you. But there’s many more people who can come and help in that regard. Not a lot can do the first part.

Katherine:                  

Right, absolutely. Yeah, those are excellent points. Let’s talk about treatment goals. What are the goals of myeloma treatment from a clinical perspective?

Dr. Fonseca:               

I’ve been very fortunate, also, to live through this era when we have seen a plethora of studies and new drugs being approved for the treatment of myeloma.

When I first started, I used to say no one wanted to do myeloma because we didn’t have good treatments. People wanted to study leukemia, lymphoma. It just turns out that this is probably one of the most vibrant areas of hematology from a science and from a clinical research perspective, of course. If I see young patients who have multiple myeloma, I have essentially two goals. The first one is to induce the deepest possible response I can do so in a safe manner. I also repeat, “in a safe manner.” But I really have the goal to try to induce the deepest response possible because that has translated and continues to translate, and in many ways proven to be associated with an improvement on their longevity and the time we can control the disease.

And it leads me to second goal, and that is that I firmly believe there is a subset of myeloma patients that are cured from their disease.

Now, this is possible because of the availability of these new treatments. I will only be able to say that in 10 and 15 years from now, when we have monitored patients for a long period of time, and we have been able to see that became true. But by all indicators, we have patients that are living many, many years without the disease coming back. I think that would be important. Now, we have patients that with more advanced age sometimes it’s difficult to propose some of the most intense form of treatments like stem-cell transplants.

We don’t do a lot of that in individuals over the age of 72 just because the toll that it takes on a person is very high, and the risks become higher. But still, in that population, providing the best treatment possible becomes a goal because I think more and more, we’re seeing patients in that age category that can start to get close to what normal life expectancy would be. It’s not there. It’s not perfect, but you start to get close. Lastly, if someone asked me, I have that balance between quantity and quality, the good news in myeloma, if you do it right, quantity and quality go hand in hand.

So, effective treatment provides symptom relief and provides durability of responses.

Katherine:                  

That’s excellent. What other factors do you consider when determining a treatment approach?

Dr. Fonseca:               

The human experience that comes to the bedside as we consider treatments is so multi-factorial and multi-complex that all that needs to be brought into consideration. Whenever I walk into the room, I tell residents usually the medical part can be resolved pretty quick, but we’re reading how much we can communicate? What’s the level of understanding? What do I understand about the support system for this person? Is there someone who can drive to the treatment center? Is there someone perhaps whose other medical conditions would create certain challenges in how they’re gonna be treated?

This person is telling me they do daily hikes for four miles. Well, that’s different from someone who I see comes into the clinic and has to use a cane. We try to integrate all of that information to make the right decisions. I’ve made a lot of my career in the early years working and showing how, for instance, genetic factors are important. I’ve come to realize later in my career and through some of the very elegant work that other colleagues have done, that these factors are just as important in determining the ultimate outcome of patients. Whenever I talk about that clinical experience, there’s two things I always tell the residents.

I use the residents a lot because I think it’s a good example of how we aspire to interact with patients. Number one is every single encounter is a final exam. You have to put your best foot forward. Every single encounter should be considered a final exam. Number two is when I walk into that room, there are three things I do, particularly the first time I meet a person.

Number one is connect, right? We cannot have a conversation and I’m not gonna be able to move forward unless we have a human connection and I have gained the trust of the patient and the family members that are there. That’s number one. The second point is decide. That is usually okay, we’re gonna do this treatment or that. That is a small part. Most of the time for me, that’s a very small fraction of the time and of the mental energy that I consume. There’s cases that are more complicated, but most of the time it’s pretty straightforward. So, it’s connect, decide do very small, and then on the other end is explain.

So, that’s how I can connect. I propose we do this, and then why we are gonna do it and what can you expect. If you can do those three things, I think that goes a long way in establishing a fruitful and a productive relationship with a patient and their families.

Katherine:                  

I would suspect that you also take into consideration the patient’s health, their age, maybe test results, side effects, things like that?   

Dr. Fonseca:               

Of course. So, we look at the medical record and with the advent, of course, of the electronic record and all the tests that we do, our consideration is quite complex. We have to look at all those factors, and the age, and comorbidities. It’s rare that we would take one factor alone that would trump everything else. We usually have to integrate the information. The same is true when we manage myeloma patients and we’re monitoring their protein levels and their response to treatment. I tell patients, they ask me, “What would you do? What’s the magic number for this or that?”

I say, “It’s a little bit like you’re flying a Cessna plane and you have all these dials in your dashboard, and that’s how we manage the situation is the integration of all of that information.”

Katherine:                  

Right. Can you help us understand, Dr. Fonseca, how test results may affect treatment options?

Dr. Fonseca:               

Sure. Happy to do that. In myeloma, we are very fortunate in that we have, and it’s not the topic for today, but we have the best biomarker that exists for any cancer. That is that we can measure the proteins that are associated with the growth of the cells. We have multiple tests that we can do. We do them in the blood and we do them in the urine. They’re simple tests that have been done for decades now that allow us to monitor how a person is doing with regards to their disease. I use the following analogy. Myeloma cells live inside the bones, as I mentioned, in the bone marrow.

They don’t come out into the blood. So, we cannot measure them. Indirectly, we can measure how many they are and how they are behaving by measuring this protein. I use an analogy of imagine you’re walking in a street, and you see smoke coming out of a building. There are two things you can do. First is you diagnose that there is a fire inside the building, right? We see that with myeloma by measuring these abnormal proteins.

Then as a firefighting team comes on, you can gauge whether they’re making progress or not by the amount of smoke that comes out. That’s exactly what we do when we monitor myeloma. We monitor the M-Spike, the serum free light chain, the urinary proteins. That’s how we make those determinations.

At the same time, we do that, we have to look indirectly at the rest of the body. We have to look at the kidney function. We have to look at the blood counts. We have to look at the hemoglobin and the red cell count because that can (A) start on the wrong foot because of the myeloma itself, but (B) can also suffer as a consequence of our treatment.

It is, again, that idea of having the multiple dials in the dashboard that allow us to reach our practice. We have to be adjusting. So, if we measure the proteins and we’re doing great, but then at the same time we see we’re suffering in blood counts, and we may need to adjust those as we provide supportive treatment. If we don’t see the proteins go down, then that may mean we need to change to a different form of treatment or that the person is unfortunately a refractory or relapsing to something.

So, that’s how we integrate the test results into our management.

Katherine:                  

What sort of questions should patients consider asking about their treatment plan?

Dr. Fonseca:               

I think it’s important that patients understand a few things. They can be described in multiple ways. Number one is, of course, what? What is it that is being used? I think that includes a description of what to expect, the practicalities, the names of the medications, their side effect profile, and what to report when you use those medicines. I think that’s very important because if you’re empowered with that information, you’re gonna be better off as you react for symptoms that may come along. I always tell patients when you have a cancer diagnosis, your self-awareness goes through the roof because we’re gonna be paying attention to everything, every skin change, every pain we have.

So, I think having a bit of that proactive discussion becomes important as they think about the treatments that they want. I think the how-to on the practicalities are very important. The best where the nursing team and the pharmacists help us a lot too. Do you take the medicines at night? Do you take them with meals? Is there something that you shouldn’t be mixing? How much time would it take for me to get a refill? It’s different to get a medication from a specialty pharmacy versus your down-the-street Walgreens. So, all of those things are important that patients, again, participate in the understanding.

If not them, at least the caregivers that are a part of this team. I think it’s important that patients ask also some brief descriptions of (A) the biology of the disease. If I have myeloma, what type of myeloma do I have? Does that matter as far as what treatments I’m going to be using? What treatment options may be available to me because of my specific subtype? We have subsets of myeloma that have options that are not available to others.

Also, I think it’s important that patients also ask a sense from the physicians as to where they are. I’d like to describe this a little bit more. Sometimes, patients ask us specific questions about, am I in a complete response? Am I in a very good partial response? What is a PFS? Those terms work very well when we talk about clinical trials, but they don’t necessarily describe in a great way the situation for an individual patient. I’d use a lot more objectives than I’d use technical terms when I describe where patients are. I say, “You have an excellent response. You have a very deep response.”

Then I’d provide more details if they want. “Yes, you’re MRD-negative at 10 to the -6.” But sometimes I find that it’s harder for patients to understand where they are if they completely focus on the staging system or the response criteria, etc. Because maybe a VGPR, a very good partial response, doesn’t sound very good.

But then you can be in a very good partial response for 15 years and it doesn’t matter. You my want to be in an MRD-negative status, but you still have a good outcome. That’s why the general description of the status by a physician becomes important.

Katherine:                  

Do you think patients should get a second opinion consult with a specialist?

Dr. Fonseca:               

In general, my answer is going to be yes. This is not self-serving. I think myeloma has become so complex that trying to integrate at least once, or if not, in some infrequent basis, an opinion of a myeloma specialist becomes important. This is no one’s fault. If you’re a community oncologist somewhere where myeloma represents only a small fraction of your practice, I can guarantee you, you cannot stay on top of the literature. I cannot stay up with everything that goes on with myeloma, even though that’s what I do 100% of the time.

I get an email every week with all the articles, all the publications, and I have to integrate that. I have to think, okay, does this matter or not? I go to the professional meetings. I see all the abstracts and I still feel like I’m missing out. How could you do that if that is only a small fraction of your practice? I’m sure that the same applies for other cancers, breast and colon. You can’t move. You cannot uproot yourself and leave your community and your family, but I think there should be ways by which patients at least have an opinion from someone who has more expertise. Fortunately, there are many centers across the nation now that have that expertise for the management of myeloma.

Katherine:                  

Dr. Fonseca, we have a question from a newly diagnosed myeloma patient. Barbara says, “I am just about to begin my first myeloma treatment. What can I expect?”

Dr. Fonseca:

Thank you, Barbara, for the question. I think if you start on treatment, first of all I hope they already went through a good description of what the treatments are, the frequency by which you’re gonna have to go to the center, and also what are the toxicities to look out for.

One of the most common toxicities that we face and one of the most challenging parts of initial treatment is the use of steroids. So, we use dexamethasone as part of every single regimen we use for myeloma. I tell patients, “Dexamethasone is a simple drug at first glance, but it’s oftentimes the most complicated part of treatment.”

The human brain works at triple speed when you’re on dexamethasone. So, it’s hard to sometimes be able to sleep properly. People can become anxious and even the sweetest person in the world can become a little bit edgy on dexamethasone.

I always say Mother Teresa on dexamethasone would be an edgy person. Just be patient. Work with the team. Just know that on the other side of treatment there is a return to normal life.

Our goal as we embark on treatments and, for instance, is I see patients that are going to go through transplant, I tell them, “Our goal is you finish, you recover, and you go back to your life. You back to work. You go back to your family, your kids, your sports.” That’s really what we strive for when we treat patients with myeloma.  

Katherine:                  

Yeah. Once on therapy, how is the disease monitored and how do you know if the treatment is working?

Dr. Fonseca:               

Well, fortunately, we use the same markers. Once a person is in therapy, we will be monitoring. We monitor at least on a monthly basis of those myeloma protein markers. Once a person reaches a great level of response, sometimes we complement that with an analysis of the bone marrow. Of course, it’s more invasive, so we don’t like to do a lot of them, but we do them as needed. As we go forward and monitor patients, we will be looking for signs that those proteins remain in a low level as stable as an indicator that the disease is under control.

Now, if I saw someone and then I start seeing that there’s an increased concentration of those proteins or we see something else clinical, we might need to do a little bit of a regrouping and test again in great detail to determine if the person is experiencing regrowth and the disease is so-called relapsed.           

Katherine:                  

Why is it so important for patients to speak up when it comes to symptoms or treatment side effects?

Dr. Fonseca:               

Well, that’s a great question. If you don’t speak about them, we don’t know about them. It seems very obvious, but then we cannot make the proper adjustments. I’ll give you a couple of examples. I already talked about dexamethasone, but a common drug we use is something called bortezomib. Bortezomib is a proteasome inhibitor.

That’s a mouthful, but it’s one of the key type of drugs we use. It’s given as an injection under the skin. Not to be confused, by the way, with daratumumab. Faspro is the name of that medication, so not to be confused with that is bortezomib, which we have been using for many years.

Bortezomib has a potential toxicity that is called peripheral neuropathy. If patients have peripheral neuropathy, that can go from very mild where you have some numbness and tingling, to the more extreme cases that it’s associated with pain, discomfort, even weakness and disability.

Well, if we don’t know that’s happening, then we can’t react to it and we can’t adjust doses or switch to something different altogether. You can imagine now we have more options, but in the old days, I always tell patients, “You might be tempted not to say anything about this because you might be thinking, boy, this is working. I don’t want to interfere with my treatment. I can live with the peripheral neuropathy.” But if it gets worse, despite the fact that the treatment is working, the person might have a very significant impingement on their quality of life.

More so now that we have so many alternatives, it’s important not to get us into a path that we might reach a point of an irreversible chronic complication from treatment.

Katherine:                  

No, and that would be awful.

Dr. Fonseca:               

Absolutely.

Katherine:                  

Before we end the program, Dr. Fonseca, have there been any recent developments in myeloma treatment in research that make you hopeful? 

Dr. Fonseca:               

Absolutely. I would say that the one area of work that makes me most hopeful is what we’re seeing with immunotherapy. We have seen that both as the ASH meeting, as well as the ASCO meeting in this year, where people are presenting updates with the various clinical trials with either bi-specific antibodies or CAR T cell therapy as a new avenue for the treatment of myeloma.

In fact, at the last ASH meeting, we had 14 presentations of different compounds or different constructs that are active. I think the future is bright in that regard. We’re seeing their application right now. A lot of these updates have also been made as ASCO.

We’re seeing the update of the treatment of treatments with fairly advanced and aggressive disease where we can still show very significant responses. I participate in some of these trials. I can tell you in my institution, using some of the bi-specifics, I see patients who have previously exhausted all of their options and now are MRD negative at 10 to the -6.

If we’re seeing that in the very advanced disease, I cannot wait to see what happens when we start using these treatments in either early relapse and why not in the near future as frontline part of our therapy? I think to me, that whole field of T-cell engagers, where there’s bi-specifics or the CAR T cells remains one of the most exciting areas for future research.

Katherine:                  

How can patients stay up to date on information like this?

Dr. Fonseca:               

I think what we alluded to before is very important to work with groups like yours and other patient support organizations that can keep them up to date. I think they’re doing a very good job at also providing updates post some of the large meetings. I know there’s a lot of patients out there that are very sophisticated that will even join the medical meetings. That happens with some frequency; that they want to learn, and patients that go and ask me details about the statistics of the trial. That’s a whole spectrum, right?

But at the minimum, I would say a strong connection with a support group, or a patient support organization becomes an imperative as you deal with this. Also, that would help you because with this whole concept of the information not always being complete and truthful, that can be scary as well, too.

If someone goes and just looks for, I would say even some of the resources that are out there in a textbook today, just keep in mind that textbook was probably written five years ago, and it represents the studies of about 10 or 15 years ago. How that relates to you, it’s very distant. So, it is because of this continuous process of research that we know better what’s going on at the present time.

Katherine:                  

Dr. Fonseca, thank you so much for taking the time to join us today.

Dr. Fonseca:               

Oh, it’s my pleasure. Thank you for the opportunity.

Katherine:                  

And thank you to all of our partners. To learn more about myeloma, and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for joining us.

Who Is on Your MPN Healthcare Team?

Who is on Your MPN Healthcare Team? from Patient Empowerment Network on Vimeo.

Who are the key members of the myeloproliferative neoplasm (MPN) healthcare team? Dr. Naveen Pemmaraju discusses the team members he recommends in the modern era for optimal care for patients with polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF).

Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju, here.

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Transcript:

Katherine Banwell:    

When a person is diagnosed with an MPN, they have a whole healthcare team. Who is typically on that team?

Dr. Pemmaraju:         

Well, it’s interesting. Yeah, that’s evolved over time.

It used to just be patient and their local oncologist, right? And the oncologist office has become a very busy place with mostly solid tumors. So, breast, prostate, colon, and then maybe a few scattered patients in most practices with blood cancers. Obviously, blood abnormalities are common with platelets and anemia and all that, but to actually have an MPN patient in the general hem/onc practice is actually quite rare. Right? These diseases are 4 to 5 out of 100,000 people.

Now, fast forward to the modern era. I think this is important. I think now, what I personally encourage – and obviously I’m biased because I’m here at the academic center. But I really think that patients with rare blood cancers such as MPNs should be co-managed. So, be seen by your local hematologist/oncologist, for sure. They know you the best. But also have a referral, if you’re able to and have the resources and ability to travel, to an academic center where you can see a blood cancer specialist such as me or my colleagues, as I only focus on blood cancer.

So, I’m not seeing patients with a solid tumor. So, local oncologist. If you can have a blood cancer expert as part of your care, it doesn’t have to replace the care. And then to have a member of the nursing allied professions – nursing and APP, advanced practice providers – is really becoming essential to help with acquiring the prescriptions from the specialty pharmacy, prior authorizations, teaching of the injectables, such as Interferon, figuring out enrolling on clinical trials.

So – and then, if a patient, young and fit, with myelofibrosis, you’ll want to be consulted with a stem cell transplant doctor. And then, finally, as if that wasn’t enough, I think a good pharmacist team is important nowadays to go over the drug-to-drug interactions, side effects. It’s not just about the JAK inhibitors but all the other medicines – antibiotics and everything else – that may be a bit unique to the MPN patient compared to the general cancer patient.

What Actions Should Cancer Patients in Treatment Take With COVID-19 Vaccination?

What Actions Should Cancer Patients in Treatment Take With COVID-19 Vaccination? from Patient Empowerment Network on Vimeo.

What actions should cancer patients in active treatment take in terms of COVID-vaccination and other measures? Expert Dr. Shaji Kumar shares information about cancer patients undergoing various types of treatment and advice about precautions for cancer patients after full COVID-19 vaccination.

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Transcript:

Jeff Bushnell:

What would you tell the patients who are in active treatment and who planned to get the vaccine just continue as normal after they get it, with all the appropriate precautions?

Dr. Shaji Kumar:

I think there’s one other important aspect which is, what is the right timing to get vaccinated, the vaccine, and that is a question that often comes up. So patients who are not getting active treatment, there is obviously no concern whenever the name comes up, go ahead and get the vaccine. And the second is what if someone is actually getting active treatment for their cancer, is there any role in terms of trying to find the vaccination, with respect to the doses of the medications and for most of the treatment we are using for cancer, there are no clear guidelines in terms of the when they can get the vaccine, that having several guidelines that have been put out by different organizations. The bottom line is, if there is an ability to space out or give some time between the vaccine and the dose of the medication, do that, don’t modify your treatments, just so that you can get the cross at a particular time. The only place where we would recommend specific guidelines within the context of somebody who may have had a bone marrow transplant or had some other kind of cellular therapies, in those contexts, we often recommend that you wait for a couple of months after the stem cell transplant, before we get the vaccines. But for all the other treatments that we are getting right now, we want to just within the schedule of the treatment that’s already on going, try and get the vaccine in between two doses.

Mary Leer:

For those who have been vaccinated and are living with cancer, you spoke to that in great depth, but I’m also wondering about people that are perhaps in post-treatment and let’s look at social distancing measures or other restrictions, are those different for patients versus the general population?

Dr. Shaji Kumar:

No, I think the proportions are the same, I think the social distancing and the masking should continue to be observed the same way, and I think the only other word of caution I think may be particularly relevant for the cancer patients would be, again, trying to avoid again those kind of being outdoors and larger groups of people, even if when you maintain the social distancing, try and not do that. The outdoors are probably a little better than smaller indoor gatherings, and it’s mostly the common sense proportions, and I think the cancer patients are probably more tuned to this because they have been following some of those things even before the COVID came on and post-vaccination, I would recommend that these steps don’t change at all, partly because we gain for a given person, we don’t know how robust the immune response that those patients have after the vaccination and the lack of good testing to say that, okay, now you’re fully vaccinated, your response is great, you don’t need to worry about getting infected.

Empowered Patient and Care Partner Ask the Expert: Addressing COVID-19 Concerns

Empowered Patient and Care Partner Ask the Expert: Addressing COVID-19 Concerns from Patient Empowerment Network on Vimeo.

With COVID-19 infection and vaccine concerns, what are the key points for cancer patients and care partners to know? Expert Dr. Shaji Kumar from Mayo Clinic shares valuable information about protective measures against COVID-19 infection, vaccine side effects and effectiveness, working toward herd immunity, and cancer research benefits that have emerged from the pandemic. 

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Transcript:

Mary Leer:

My name is Mary Leer. I’m the Breast Cancer Network Manager.

Jeff Bushnell:

And I’m Jeff Bushnell, the MPN Network Manager at the Patient Empowerment Network. I’m a caregiver.

Dr. Shaji Kumar: I

’m Shaji Kumar, a hematologist at Mayo Clinic.

Mary Leer:

Jeff and I are proud to be part of a strong team of compassionate volunteers, helping health communities adapt to the realities of living with a serious illness, living with cancer during a pandemic certainly presents another layer of challenges. So, Jeff and I will drill down to ask the important questions from the community. For this production, Empowered Patient and Care Partner Ask the Expert, we are very lucky to be joined by noted expert, Dr. Shaji Kumar, a consultant in the division of hematology at Mayo Clinic. Thank you for taking the time to join us, Dr. Kumar.

Dr. Shaji Kumar:

Thank you for having me, Mary.

Mary Leer:

Let’s start with the top of mind questions for so many of us right now, what should every patient and care partner facing a cancer diagnosis know during the pandemic?

Dr. Shaji Kumar:

I think it’s a challenging time for everyone, and it’s obviously more challenging for patients dealing with cancer at the same time, thankfully, we have a vaccine at hand that will certainly make the situation a lot better, but I think from a cancer standpoint, I think what we need to keep in mind all the precautions we talk about in terms  of social distancing, masking, hand washing and all those measures apply equally to everyone, even more so to patients with cancer. And the reason why we say that it’s even more important for several reasons, one, and we continue to learn more about the pandemic and its impact on cancer, one thing that has become clear is that patients with underlying conditions including cancer are to other folks were more affected by the infection, more likely to have more severe interactions and poorer outcomes. Now, patients with cancer appear to be at a higher risk of getting the infection and then they get the infection having a more serious disease. Now, it’s hard to know how much of this is also related to the fact that patients with cancer often have to go into the hospital or the clinic, and hence are more likely to get exposed to the infection than someone who is able to just stay at home.

So that’s one thing. And second, we know that the ongoing treatment for cancer definitely suppresses the immune system, and hence places people at a higher risk of the infection itself. Now, even patients who have their past history of cancer, this appears to be some increased risk, even though this is a little bit, unclear how much more it impacts those individuals. But I think the bottom line is keep the awareness that you might be at a higher risk of getting the infection, more serious infection, and the need to take those precautionary measures in a more strict fashion, and getting the vaccination when you can get it is all things that one needs to keep in mind.

Jeff Bushnell:

Well, that’s wonderful, Dr. Kumar, you mentioned the vaccinations, I am a strong proponent of that, I happen to have been involved in the Moderna vaccine trial, which is and still enrolled, they’re doing the follow-up. I guess they’re checking the last time I was in last week, they took 8 vials of blood, I think they’re checking to see whether I have the antibodies and how long it will last, but I was very happy with the way it was conducted, they were very forthcoming with information.

It was very interesting. And out here in San Diego, where I am, we have done pretty well as a county in vaccinating people and Summer got the vaccine as well with myelofibrosis and she feels a lot better. But for cancer patients who have tested positive for COVID, are there notable consistencies amongst that group of people, and have we learned anything from those patients yet about maybe their chances of getting it more, or their reaction to it? That kind of thing.

Dr. Shaji Kumar:

We know that there’s a wide spectrum of reaction to the vaccine. The majority of the people would not notice any symptoms related to that except for some pain at the injection site.  Not there are some folks, number of people who might have more or just myalgia, muscle pains, just feeling fatigue, some low-grade fevers, just feeling blah for 24-48 hours, and it seems to be not too uncommon. The reactions to the vaccine in terms of the side effects or the symptoms, there doesn’t appear to be much of a difference between cancer patients and normal individuals. Now, in terms of the efficacy of vaccination, you just mentioned Jeff, about you being checked for the antibodies, obviously, that is something that we hope will happen to all individuals who get the vaccine, but we know that is not going to be the case, there’s going to be a wide variation in terms of how strong an immune response one might develop against vaccines. Now We know from, not necessarily the COVID vaccine, but the vaccinations that have been used in the past, whether it be flu vaccines or pneumococcal vaccines, that we all get patients with cancer or patients going through treatment for cancer that can suppress the immune system, tend to have a lower response. But again, that varies quite widely from patient to patient now, there are some vaccines where we can clearly look at the antibody response and say, “Oh, this is not adequate, and we need to maybe give an extra shot.”

We just don’t have that information for COVID vaccines yet. So the way I would look at it is, even though the response to the back in a given person might be less than what we eventually would identify to be optimal, it’s likely to be better than not having to see the vaccine, so I would encourage obviously, everybody to get the vaccine. Now, what about someone who has already had an infection, what would be the response? Should we vaccinate those people? We certainly should. Again, we don’t know the immunity from a natural infection, how long would that last? That is still something that is unknown, and the vaccination dose is likely to make the responses more relevant and more durable, so I would recommend the vaccines for everyone. We don’t think one vaccine is any different from another in terms of your underlying cancer or lack thereof. So in terms of assessing for the antibodies, there is no clear guideline in terms of what one should anticipate from  the vaccine, so there is really no way to say, check the antibody, and they can go ahead and get one more dose or you’re fully vaccinated. So I think the bottom line is, get the vaccine, you don’t need to necessarily test for a response, and then we continue with the usual measures for prevention.

Jeff Bushnell:

And so what would you tell the… I guess that’s pretty much the answer to the next question I had. What would you tell the patients who are in active treatment and who planned to get the vaccine just continue as normal after they get it, with all the appropriate precautions?

Dr. Shaji Kumar:

Yeah, no, I think there’s one other important aspect, Jeff, to that question you just raised, which is, what is the right timing to get vaccinated, the vaccine, and that is a question that often comes up. So patients who are not getting active treatment, there is obviously no concern whenever the name comes up, go ahead and get the vaccine. And the second is what if someone is actually getting active treatment for their cancer, is there any role in terms of trying to find the vaccination, with respect to the doses of the medications and for most of the treatment we are using for cancer, there are no clear guidelines in terms of the when they can get the vaccine, that having several guidelines that have been put out by different organizations. The bottom line is, if there is an ability to space out or give sometime between the vaccine and the dose of the medication, do that, don’t modify your treatments, just so that you can get the cross at a particular time. The only place where we would recommend specific guidelines within the context of somebody who may have had a bone marrow transplant or had some other kind of cellular therapies, in those contexts, we often recommend that you wait for a couple of months after the stem cell transplant, before we get the vaccines. But for all the other treatments that we are getting right now, we want to just within the schedule of the treatment that’s already on going, try and get the vaccine in between two doses.

Mary Leer:

For those who have been vaccinated and are living with cancer, you spoke to that in great depth, but I’m also wondering about people that are perhaps in post-treatment and let’s look at social distancing measures or other restrictions, are those different for patients versus the general population?

Dr. Shaji Kumar:

No, I think the proportions are the same, I think the social distancing and the masking should continue to be observed the same way, and I think the only other word of caution I think may be particularly relevant for the cancer patients would be, again, trying to avoid again those kind of being outdoors and larger groups of people, even if when you maintain the social distancing, try and not do that. The outdoors are probably a little better than smaller indoor gatherings, and it’s mostly the common sense proportions, and I think the cancer patients are probably more tuned to this because they have been following some of those things even before the COVID came on and post-vaccination, I would recommend that these steps don’t change at all, partly because we gain for a given person, we don’t know how robust the immune response that those patients have after the vaccination and the lack of good testing to say that, okay, now you’re fully vaccinated, your response is great, you don’t need to worry about getting infected.

Mary Leer:

Wow, thank you so much. That’s so helpful. I’m going to shift to vaccine hesitancy. This is an important topic for many. Drug development takes years, sometimes decades. Can you speak to those who might be hesitant about the speed of vaccine development around COVID. I’ve heard this often from other people saying, “Well, they develop this so quickly, how can we trust it?”

Dr. Shaji Kumar:

Yeah, no, I think those concerns are quite valid, I think vaccines have always been a very controversial topic and not just COVID vaccination but even for childhood vaccinations. There have been long-standing concerns that some of those vaccinations may be responsible for some of the issues that we see in the children and even in the late adulthood. I think what we really want to get across is, again, taking that question apart, and there are multiple different aspects to it, one is the whole concept of how we created the vaccine so quickly, we kept telling everyone from the time that it started that it takes five to 10 years to develop a good vaccine, and now we have something in a year, so obviously that raises concerns amongst people. I think it’s just a testament to how far technology has come. In the past, we had to isolate the protein and use that protein to develop the immune response, and what has been really unique about the COVID situation has been the Pfizer vaccine and the Moderna vaccine, both of which uses a new technology called the mRNA-based technology. And this is something that has been developed over the past decade to decade-and-a-half, and I would say this is a platform that was perfect, just waiting for the right opportunity to come along.

And the COVID situation really presented that. And even though it was the speed with which this was developed, is just because the technology has come along so much and we can actually do that, and the second is how fast the clinical trials have been done, and I think that speaks to, again, the infrastructure that they have been developed over the years to rapidly develop and implement a clinical trial. So the clinical trials, both Pfizer and Moderna trials had 40 to 50,000 people enrolled in a quick phase and the community transmission that was happening at a very high rate. We could get these trials done in a very rapid manner, so the patients or the people who enrolled in this clinical trial the fact that they were not getting infected could be determined in a much, much faster fashion than what you would have done in the past with any of the other vaccines. So I think the technology is robust. The [COVID]  trials are very well-conducted and the end point in terms of efficacy has been very well-determined or very accurately determined.  And given the size of these trials and the number of people who have been a goal, I think we can feel fairly confident that the risk associated with this vaccine is pretty low, so you can argue that one of the risk of a particular side effect is only 1 in 80,000. So maybe to the 40,000 people enroll in the trial, they may not have adequate numbers of that and that was certainly a concern when they started vaccinating. And we just know a couple of days ago, there was a publication that looked at almost like 63 million vaccination doses that have been given, and overall the risk of vaccine related side effects have been very, very minimal.

So I think that should also boost our confidence.

But on the other hand, we all heard about what would happen with some of those vaccines and the blood clots, and I think that even though…yes, it is, as it is a risk. It is a very, very small risk. And the fact that you were able to identify them right away again, I think tells us that should there be rare side effects, you’re going to find it, and we are going to figure out the mechanics of why those side effects happen. And we’re going to figure out how to avoid those things.

So, I think the information flow is so fast and all the data related to vaccines and the side effects are being captured in a real-time fashion that we would be… You’d immediately be of avail of side effects should that happen.

Mary Leer:

Wow, that’s so reassuring. Thank you.

Jeff Bushnell:

Another question kind of along the same lines, doctor is the last few days, especially, it’s Vaccine hesitancy has really become sort of the issue to the potential of achieving herd immunity, and how can everybody in the medical community, you guys are facing those stuff in a different way, but the average person, how can we help overcome hesitancy and increase the people’s trust in the vaccine, and also increase the equitable distribution amongst all populations? Some populations are hesitant to take it, others have distance problems for being able to get it. What can we do to sort of push ourselves over the hill to get to that herd immunity?

Dr. Shaji Kumar:

Yeah, no, You bring up a very important point, and I hope we are in a much better place than many parts of the world right now because we have one of the few countries where a significant proportion of the people have been vaccinated, but we are not quite at the point where we can claim herd immunity, I think we still need to continue to pursue this, and I think the ideal goal is to get everyone who’s eligible to get a vaccine vaccinated. Now, you bring up some of the very important points, because even though vaccine hesitancy is a real problem, the underlying reason behind this is manifold, and the only way to tackle that is we have a multi-front approach that will take into account what is the reason behind it.

So for the people where it’s hard to get to populations which can live in far from the areas, it may be more the ability to use those vaccines, which does need the complicated storage, for example, the J&J vaccine. You only need one dose. It’s easy to store. So that may be one of the approaches to be taken. And people who believe that this is a vaccine is going to create side effects, or it’s part of some grand scheme to introduce a variety of things. I think it’s a person of education, and I think they really need to tell them what can happen with. Not really just to them, but the fact that if you continue to allow these infections to proceed on stuff, there are going to be increasing numbers of mutations, and that in turn is going to make the pandemic much more difficult to control in the long run. So it’s totally an individual benefit, but it’s on to the society’s benefit to have everyone be vaccinated. And then definitely, I think knowing that should anything unto it happen, there’s going to be medical care that’s going to be available to these individuals, and I think that’s also an important point, so who are near and dear to them is going to be the key thing.

Mary Leer:

Here’s a question many cancer patients are unclear about if antibodies are present or if I have tested positive before, there’s a wondering, “Should I still get the vaccine?”

Dr. Shaji Kumar:

Yeah, I know the recommendation right now is to go ahead and get the vaccine, partly because we don’t know the natural immunity from the infection, how long does it last. So it seems like the antibodies can start to wane off the infection. And again, we don’t have a lot of data on it, but it looks 3 to 6 months, it might start waning at least to the level that they can detect. Now, whether that is sufficient or even the undetectable levels is protective against a future infection, we don’t know. There have been some reports of people getting a second infection even though they have been infected before again, scattered reports, we don’t know how widespread that phenomenon is going to be, so given all these, I think the current recommendation would be to go ahead and get vaccinated. We generally tell people to wait for two to three months after the infection to go ahead with the vaccination.

Mary Leer:

Alright, thank you

Jeff Bushnell:

Should people… Is the idea of pre-screening, especially for cancer patients, maybe who may be at risk, I guess, to see whether they have antibodies or whatever, be an effective thing to decide which vaccine they should get? or I know, as I say, I was in the trial and they were very forthcoming to the participants with what the numbers were, and I was flabbergasted at how effective the vaccine was, it was just amazing to me, and that kind of information that I guess is not available publicly maybe it should be. Does it help to decide which vaccine you get? All I hear on the TV is get the first one you can. What are your thoughts on that?

Dr. Shaji Kumar:

Yeah, no, I completely agree with you. I think even those numbers may mean… You look at the Moderna and the Pfizer trials, and they said, now over 90 percent effective. Look at the AstraZeneca trials, you know, it’s like they recorded 70 to 80, 85 percent, and the J&J about 80 to 90 percent effective. Do these numbers mean much? It’s really hard to know, I think, partly because they have been tested in, again, different countries, different times, as the virus was continually changing its characteristics. So it does it mean… So one could argue that maybe the vaccines that were tested later on when this will be some of the mutants were already there might be more effective, but we don’t know.

I think at the end of the day, 80 versus 90 is not something we would decide a vaccine on. The fact that, yes, if something was only 10 percent effective versus 90 percent, it’s a probably different story. So based on the numbers we have seen, I would say whatever you can get to first, if you don’t want to get jabbed twice, maybe you go with something that goes, it’s only one dose, but that may be the only distinguishing factor here, but nevertheless, I think we have to just get the vaccination, the first vaccine that we can get our hands on.

Mary Leer:

So let’s hope there is some good that comes from the bad. Are there any noticeable trends born out of the pandemic that will be or could be a benefit to the future of cancer care or research?

Dr. Shaji Kumar:

Mary, That’s a very important question, and I think we always learn from adversity, and I think this is going to be no different. I think, especially when the pandemic hit back in the spring of last year, we all had to think fast on our feet to figure out how best to continue to tell about the best care for the cancer patients without compromising the care in any way. And we knew that bringing the patients back into the clinic at the same rate we did before the pandemic would expose them to significant risk for infection, so how do we continue with treatment? There have been very different things people have tried…one of them is to try and get the medications to patients at home. If they are on IV medications, they can be changed to something that’s comparable that can be given by mouth. We already did that for some patients. For some patients who used to come to the clinic very often, so we figure out is there a way for them to get some of those testing done in a clinic much closer to home, so they can avoid the travel, they can avoid being in a bigger city, they can avoid being in a bigger institution, again, reducing the risk of exposure, and then you look at those numbers and then decide on the next course of treatment. We converted many of the clinic visits to video visits. Nothing is as good as having the patient right in front of you, but this is the best we could do under the circumstances.

And I think that helped. So I think the clinical trials was a big problem because in many of those trials were done in a very rigid fashion with very little variability allowed within the protocols. And everybody loosened from the clinical trial sponsors, the pharmaceutical companies, the institutional review board, the investigators to try and build flexibility into those clinical trial structures to allow patients to continue to be on those trials, many of which are important and both helping. So what does that mean for the future? I think the video visits are here to stay, I think we will continue to utilize that and bring patients back to the clinic only when it’s absolutely needed. I think the clinical trials will have in-built flexibility so that patients can enroll on clinical trials remotely, they can potentially be given some of those medications at home, maybe it would be something where we would check into the patients on a regular basis to make sure things are proceeding in the right way. I think there are increasingly technologies that will allow the patients to communicate in real time with the care team and also provide many of the data that we need through iPads or iPhones, Apple watches, whatever we end up using.

So that is that I think that technology will rapidly take off in the next few years, I think. So I think a lot of the care of the patients with cancer in general, and particularly cancer patients, I think is going to look very different five years from now, because of all these things that we have always thought of and we thought, “Yeah it will take time to implement, it’s difficult.” Now we figure it out in a year. We can do a lot of those things.

Mary Leer:

Yeah, thank you.

Jeff Bushnell:

For the final question, you’ve given tremendous information here, Dr. Kumar w What’s the final takeaway for the average cancer patient and caregiver, how to get through this? What’s your bottom line for us all?

Dr. Shaji Kumar:

Bottomline is, I think Your cancer treatment comes first, let’s not compromise on it, let us do it as safe as we can by observing all the instructions in terms of social distancing, masking, avoiding gatherings, getting vaccinated, and make sure you keep connected with your care team. You don’t have to be in the clinic to do that. There’s a variety of different tools, I think every hospital has options to either through their medical records to message their care team, or set up video visits and so forth.

So we want to be in a state where it was before the pandemic in terms of your communications, but use the technology, so we can decrease the risk of exposure without compromising the quality of care.

Mary Leer:

Alright, well, thank you so much, Dr. Kumar, that you have just given us such valuable information, and I want to thank Jeff as well, and the Patient Empowerment Network for putting this together.

Jeff Bushnell:

Thank you, Dr. Kumar, appreciate it.

Dr. Kumar:

Thank you, Jeff.

How to Play an Active Role in Your MPN Treatment and Care Decisions

How to Play an Active Role in Your MPN Treatment and Care Decisions from Patient Empowerment Network on Vimeo.

How can you play a role in your MPN care and treatment decisions? Engaging with your healthcare team is essential and may lead to better overall outcomes. In this program, Dr. Naveen Pemmaraju provides tips for how best to advocate for yourself or a loved one, as well as tools for making treatment and care decisions.

Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju, here.

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Transcript:

Katherine Banwell:    

Hello and welcome. I’m Katherine Banwell, your host for today’s program.

Today, we’re going to explore how to engage with your healthcare team when diagnosed with a myeloproliferative neoplasm, and we’ll discuss the patient’s role in care decisions.

Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you.

All right. Let’s meet our guest today. Joining me is Dr. Naveen Pemmaraju. Dr. Pemmaraju, would you please introduce yourself?

Dr. Pemmaraju:         

Well, thank you for having me, Katherine and team. I’m Dr. Naveen Pemmaraju. Associate Professor of Leukemia and the Director of the Rare Disease Program with Blastic Neoplastic Cell Neoplasm (BPDCN) here at MD Anderson, and I’m happy to be here with you guys.

Katherine Banwell:    

Thank you so much. We’re glad to have you with us today. As we move through this conversation, we’ll talk about the classic myeloproliferative neoplasms: essential thrombocythemia, polycythemia vera, and myelofibrosis.

But before we get into our discussion, let’s start with the question that’s on the minds of many of our audience members. We’re all hearing that the COVID-19 vaccine is safe, but how effective is it for MPN patients?

Dr. Pemmaraju:         

Well, I believe that this is one of the most important issues of our time.

I think the way I would approach the COVID-19 question is, one, is we know that if our patients contract the virus, that can be deadly in actually many of our patients. So, I think it’s actually important to remember that the virus is still out there and that getting the virus is potentially very life-threatening, not only for the general population but for our patients.

For the vaccines, I kind of have two stories to tell you. So, one is my own anecdotal experience in the clinic where it has been surprisingly and remarkably well-tolerated in most of our patients. This is both the mRNA vaccines and as well as the J&J vaccine. And so, overall, we’ve seen a very minimal amount of allergic or other reactions.

I think the most important part, as you said at the top, is for specific medical advice, we need to be talking to our own providers. But I think for our MPN patients, we’re giving some caution, looking at the blood counts, what chemotherapy folks are on. But, in general, I’ve been happy with that rollout.

Now, for the effectiveness of them, sure. That’s a question of ongoing research. There are some data that’s coming out, particularly in CLL and other leukemias that – correct – maybe some of our immunocompromised patients, as you would expect, may not be able to mount the appropriate response. But all that data is moving and fluid, so we’ll see.

And then I think the other point here is with this question of the virus itself and maybe some of these vaccines having a signal for increased blood clots or coagulopathy.

This is something I think we have to follow in our MPN community only because our patients are already at a high risk for both bleeding and clotting. So, the virus itself, COVID-19, post-syndrome coagulopathy, possible side effects – idiosyncratic and rare, for sure, from these vaccines that can lead to a vaccine-induced thrombotic state. I think these are some of the factors that we have to watch out for. So, in general, we don’t yet know the exact answer for each patient, PV, ET, MF, how effective the vaccine may be. But we are encouraging everyone to go for it unless there’s an obvious contraindication. Katherine?

Katherine Banwell:    

Okay. Good. Thank you. Let’s learn a little bit more about the disease itself. Dr. Pemmaraju, do a level set with our audience. Can you help us understand the differences between ET, PV, and MF?

Dr. Pemmaraju:         

Yeah, this is very important because we toss these words around as if there’s some big definition that was given, and oftentimes, that never happens. So, let’s pause to do that. So, this goes back to the 1950s when William Dameshek, who really postulated the modern MPDs at that time as they were known – myeloproliferative disorders – really thought that there were four diseases that were similar at some level and then presented differently. So, that’s polycythemia vera, essential thrombocytosis, myelofibrosis, and CML, chronic myeloid leukemia.

Then, as the modern era comes in, CML is divided off because of the Philadelphia chromosome, BCR-ABL, which is present in 100 percent of those patients.

So, now we know CML is its own thing. And now we have the big three, sort of non-Philadelphia chromosome MPNs, as they’re now known, because neoplasm – cancer – instead of disorder. Within the subtype, and this is important, the subtypes that you mentioned are the most common.

So, polycythemia vera – poly meaning many, cythemia, cells, vera is Latin for true. This is the designation for the patient who has a higher than expected blood red cell mass or hematocrit. And it actually, interestingly, Katherine, most patients with p. vera have an increase in all three of their blood lines, so the red cells, hemoglobin, hematocrit, platelets, and white count. Those patients with PV are especially at risk for both bleeding and clotting, transformation to myelofibrosis, and even transformation to acute leukemia in maybe 5 to 7 percent of patients.

So, the usual treatment there, Katherine, is to bring off the blood mass. That’s the phlebotomy.  And then in the patient who is above the age of 60 or has a prior blood clot, to give some form of chemotherapy, hydroxyurea (Hydrea) or interferon, for example.

Now, the second grouping is ET, essential thrombocytosis. Again, this word vera or essential, meaning not reactive, not benign, not from a regular cause like a surgery or a trauma or an inflammation. So, it means a cancerous cause, an autonomous cause, something that’s coming on its own.

Thrombocythemia or thrombocytosis, meaning too many platelets. So, usually, patients with ET have too many platelets as their predominant manifestation. But again, as with p. vera, patients can get into problems with that. Very, very high platelets, usually a million and a half or higher, can actually lead to bleeding. Not necessarily clotting, but extra bleeding. And then patients with any platelet levels, because the platelet level doesn’t exactly correlate, can have either bleeding or clotting. So, that’s usually the predominant factor. And again, the underlying problem with these MPNs is that they can transform to the other ones – PV, MF, even acute leukemia.

And then, finally, myelofibrosis, which we could spend the whole hour on just by itself, is the more advanced state out of these.  So, it can either arise out of the PV or ET or stand alone. And really here, this is an advanced bone marrow failure state with bone marrow scarring or fibrosis. And now, usually, most patients, their blood counts, rather than high are now low because the bone marrow is unable to produce enough cells. And then, therefore, the sequela of the disease – anemia, thrombocytopenia. So, low blood, low platelets.

Then you need transfusions. The liver and the spleen get larger because they remember how to make blood cells. People can have a wasting away appearance. And then here, more than the PV or ET, this is more of an acute disease for many where if you have intermediate to high stage, these patients can transform more readily to leukemia and have a decreased overall survival.

Katherine Banwell:    

When a person is diagnosed with an MPN, they have a whole healthcare team. Who is typically on that team?

Dr. Pemmaraju:         

Well, it’s interesting. Yeah, that’s evolved over time.

It used to just be patient and their local oncologist, right? And the oncologist office has become a very busy place with mostly solid tumors. So, breast, prostate, colon, and then maybe a few scattered patients in most practices with blood cancers. Obviously, blood abnormalities are common with platelets and anemia and all that, but to actually have an MPN patient in the general hem/onc practice is actually quite rare. Right? These diseases are 4 to 5 out of 100,000 people.

Now, fast-forward to the modern era. I think this is important. I think now, what I personally encourage – and obviously I’m biased because I’m here at the academic center. But I really think that patients with rare blood cancers such as MPNs should be co-managed. So, be seen by your local hematologist/oncologist, for sure. They know you the best. But also have a referral, if you’re able to and have the resources and ability to travel, to an academic center where you can see a blood cancer specialist such as me or my colleagues, as I only focus on blood cancer.

So, I’m not seeing patients with a solid tumor. So, local oncologist. If you can have a blood cancer expert as part of your care, it doesn’t have to replace the care. And then to have a member of the nursing allied professions – nursing and APP, advanced practice providers – is really becoming essential to help with acquiring the prescriptions from the specialty pharmacy, prior authorizations, teaching of the injectables, such as interferon, figuring out enrolling on clinical trials.

So – and then, if a patient, young and fit, with myelofibrosis, you’ll want to be consulted with a stem cell transplant doctor. And then, finally, as if that wasn’t enough, I think a good pharmacist team is important nowadays to go over the drug-to-drug interactions, side effects. It’s not just about the JAK inhibitors but all the other medicines – antibiotics and everything else – that may be a bit unique to the MPN patient compared to the general cancer patient.

Katherine Banwell:    

Right. Lately, we’ve been hearing this term “shared decision-making,” which basically means the patients and clinicians collaborate to make healthcare decisions. And it can help patients to take a more active role in their care. So, I’d like to get your thoughts on how best to make this process work.

Dr. Pemmaraju:         

This is a passion area to me. I think this is so important that  you bring this out. I think a generation or two ago, Katherine, it may have been common for there to be more of a one-way monologue, if you will, doctor to patient, and that may have been the majority of the conversation before.

I don’t see it that way anymore, and most of my colleagues don’t either. I think it should be a dialogue, as you said. It should be a back-and-forth communication, one that learns and evolves over time as any real relationship would, right? Outside in the real world. So, I think that’s important. Number two, I think trust needs to be earned, not just given.

So, that means patient and physician, and really the physician team – so, all the other members of the team – building that trust over time through frequent communication, visits, all of this. And then, finally, I think the key here is that a lot of patients always ask, “Hey, what can I do on my own?” I’ll tell you what you can do. You can be involved and read and empower yourself if you’re able to, if you’re able to and you can. Many may not be able to due to their illness or for other reasons.

But if you are able to, I think it’s great to read online. There, I just said it. Let me repeat it to make sure everyone heard that. I want you to read. I think it’s fine. Consult Dr. Google. What’s the worst that happens? The worst that happens is you find misinformation. Well, don’t keep it to yourself. right? So, Google, look up things, go to social media, see what experts in your area are talking about, go to Facebook, go to the patient groups. But remember, everyone’s case is different. Someone else’s is different from yours, and yours is different from the next. So, gather information like a sponge.

Formulate it, synthesize it in the way that only you know how to do, bring some notes, and then talk about it with me at the next visit, “Hey, I saw this on the Internet.” “Okay, great, let’s talk about that.” Or, “Hey, this new formulation of interferon is coming.” “Great, let’s talk about it.” So, gather information, sort out signal from the noise with your healthcare team. Sort that out and then move on, move on, move on. So, I think these are some of the aspects of what’s called shared decision-making. No longer a monologue, one-way street. Let’s have a dialogue, let’s have a partnership, let’s figure out a way to empower each other in this journey.

Katherine Banwell:    

We’re going to talk in a few moments about online research and how well that works or how well it doesn’t. But let’s talk about treatment goals first for ET, PV, and MF. What are the goals of treatment from a clinical perspective?

Dr. Pemmaraju:         

Well, I think the goals are divided up into three factors. So, I think for the MPN patient, goal number one has to be what the patient themselves want to achieve.

Oftentimes, that’s different than what’s on the numbers with the labs and what the physician wants. So, I think a lot of our patients correctly are suffering from – or mentioning to us that they’re suffering from quality of life issues. So, fatigue is the most common manifestation of all the MPNs, followed by bone pain, night sweats, inability to concentrate, etcetera, etcetera.

So, I think quality of life is the goal of most people, and I think that’s an admirable goal. And some of the medicines can help that. Some can actually hurt that in the short term. So, let’s put that as bucket number one. What does the patient want to achieve? Usually, it’s the alleviation of fatigue, itching, bone pain, etcetera.

Number two, I think, is the sort of on-paper game, if you will, right? So, what do the labs show, what does the bone marrow biopsy show, what does the spleen show? I think all of that is good, too, in that bucket. And clearly, if someone has transfusion dependent anemia, two times a week needing blood transfusions, and whatever treatment you can do can alleviate that down to once a week, once a month never – okay, that’s a win for the patient.

And then I think, finally, our goals. You’re right. You asked me specifically “What are my goals for our patients?” Well, I want to see that your overall survival has improved if I can. So, your length of life, your quality of life has improved, minimization of side effects from whatever therapy we’re doing. If we’re going on a clinical trial or combining therapies in a novel way, that you’re not experiencing some brand new or idiosyncratic toxicity or side effect.

And then, finally, I think the key is to monitor for, let’s say, other things. Are you developing a second cancer, a second blood cancer? Are you having another problem that’s outside of your MPN, such as iron deficiency anemia or thyroid disease? Something that’s extremely common, has nothing to do with the MPN, but is also happening. And then do you have a healthcare team?

I failed to mention in your earlier question the primary care doctor, right? Let’s mention that person as well. If our patients have the general practitioner who they had already been seeing before the MPN diagnosis, or at least established one after, then some of these important aspects, like cancer screening, cholesterol checks, some of these other important things can be done in parallel to the MPN therapy and then, of course, combined at different points.

So, these are kind of my benchmarks for goals of therapy. They will vary from patient to patient and, of course, from case to case. The patient with advanced intermediate to high-risk myelofibrosis going to transplant, well, that’s markedly different from the patient who’s young with ET with no blood clots and relatively controlled blood counts. So, different goals there, Katherine.

Katherine Banwell:    

Right. Right. So, you just mentioned a couple of factors that you take into consideration, but there are others as well, I think. What about the patient’s age and overall health, for instance?

Dr. Pemmaraju:         

Could not be more important. You’re right. I think age – and let’s use that as a surrogate for what we call ECOG performance data. So, the overall kind of fitness of a patient, as you said, may be the most important factor. And then followed by these other conditions, so-called co-morbidities. I’d like to talk about that for a second because that’s a lot of the program here. Depending on a patient’s age, performance status, fitness, and other organs that are involved, that actually leads to a couple of important points.

One, it may limit or reduce the number of treatment options that a person has based on their ability to even tolerate it in the first place. Both oral chemos that are available, some of these clinical trials that need to use an IV drug.

Number two, it may predict how your overall survival is going to be. So, perhaps your MPN, as we used in the other example, you have an earlier stage MPN that really doesn’t require treatment. It requires active observation.

But then on the other hand, you have advanced heart disease or kidney disease. That may actually do you more harm in the end. So, that’s actually very important that you bring that up.

And then, finally, right, is this concept that you have the co-morbidities and then you have the MPN, and then they kind of change and morph over time where one is the dominant issue, the other isn’t. And so, you do need that decision care team as you were mentioning earlier. So, let’s definitely say that out loud that that matters. And I think it also reminds us that nothing is in a vacuum. The MPN doesn’t exist in an isolated space, right? So, your MPN co-exists with your heart disease, your kidney disease, your lung disease, your past, your present habits, anything.

Katherine Banwell:    

Exactly.

Dr. Pemmaraju:         

So, I’m really glad you brought that up. And I think also, to your point with the shared decision-making model, I think sometimes, as physicians, we may not ask, and as patients, we forget to mention, “Oh, X, Y, Z in my history,” or “Oh, I’m taking this herbal supplement.” Sometimes these things are important to mention.

So, when in doubt, bring up everything to your care team so that you can make decisions together.

Katherine Banwell:    

Right. It might help to make notes before you go in to talk to your doctor.

Dr. Pemmaraju:         

Sure. Sure, absolutely. That doesn’t hurt, and it could help you at least organize your own thoughts even if you don’t use them in the visit.

Katherine Banwell:    

Exactly. Yeah. Dr. Pemmaraju, let’s talk about biomarker testing. Can you help us understand what biomarkers are and how they may affect treatments?

Dr. Pemmaraju:         

Yes. Biomarkers – I think that word gets mentioned a lot with really no definition, because it’s one of those words that can be whatever someone wants it to be. So, you’re right. For us, it’s a very important word in MPN. Bio meaning of life, scientific, and then marker meaning some kind of a measuring stick that has a value.

Well, there are two ways to look at biomarkers. One is the obvious, which is we have the defined big three molecular mutations. So, that’s JAK2V617F, followed by CALR mutation, followed by MPL. Those are the big three. Those make up about 90 percent of all patients with MPNs. You’re technically not born with them, although new data suggests that you may acquire these mutations right after birth. So, those markers are important, because they can be used to diagnose the disease, right? Particularly in the challenging patient. They have high platelets, you can’t tell if it’s reactive or ET. Okay, so they’re helpful with diagnosis.

Maybe some studies have shown that some of these markers can be predictive, Katherine, of blood clots. Let that research be ongoing. And then, obviously, some of these may be helpful in terms of designing the future treatments, particularly targeted therapies. So, I think biomarkers are part of our field, if you look at it that way, at diagnosis and risk stratification prognosis. But there are other factors that are starting to come out. One is there are molecular mutations outside of these big three.

So, outside of JAK2, CALR, and MPL, that are very important actually. Not everyone is checking for them. They are ASXL1 mutations, EZH2, IDH1 and 2, so on and so forth.

So, these are extended molecular markers that can be checked at some doctors’ offices that now, in the latest scoring systems, if you have one of those or more than one or two, they can elevate your risk score. So, if you have low-risk or intermediate-risk myelofibrosis, they may make you intermediate or high risk.

So, that may be a bit more complicated than what most people are aware of. But just so you know, there are markers that can be readily checked that can tell if your disease may be a bit higher risk than we though, say, 10 years ago.

I think other biomarkers that we look at are some of the labs that are just the regular labs that are on almost every panel, but they can tell a lot about the disease. There’s the LDH, lactate dehydrogenase. There are several markers, such as CRP and sed rate.

So, anyway, there are a lot of labs that we can check depending on where you are in your disease state that can kind of tell us a lot about how inflamed you are, how active your disease is at the moment, and then that will lead to further confirmatory tests. So, I think, yeah, in general, this is an active, developing area of research in our MPN field.

Katherine Banwell:    

It seems like results could really influence therapy choices then. So, do you think patients should ask for these tests specifically?

Dr. Pemmaraju:         

I’m a big fan of patients being empowered to ask anything that comes to mind. And again, that’s why I love this discussion because maybe there might be some people out there who are shocked, frankly, at what we’re talking about here. I think it’s great to do what you said. Yes. I think do your research, online or otherwise. Come up with a list of questions. Bring – if you’re able to, of course – if you have the ability to, bring one person with you. Or nowadays, on the telehealth, we put one person on through the phone during the pandemic time.

And then – yeah. I mean, yeah, sure, just you hear about something, ask about it. The worst thing that your doctor says is, “Hey, that’s only a research test. That’s not available.” It doesn’t hurt to ask. And it may help to lead to other discussions. I think it’s also a good idea to get a second or a third opinion if you need to. There, I said it. It’s your body, it’s your life, it’s your choice. I think, yes, advocate for yourself, because at the end of the day, who else is going to do that?

Katherine Banwell:    

Absolutely. Dr. Pemmaraju, are there other questions that patients should consider asking about their proposed treatment plan?

Dr. Pemmaraju:         

You know, I think the biggest thing that I think is getting left out in the rare blood cancers that I spend all my time in is the ability and access to clinical trials. And that’s one thing I wanted to discuss with you this morning, which is you’re seen in your local doctor’s office. They’re doing the heroic work, and I really think it is, of seeing breast cancer patients, prostate, lung, colon, PV.

It’s just the difference between the frequent, common tumors that get chapters dedicated to them in the board review testing and whole months dedicated in the oncology fellowship compared to the patient with the rare blood cancer that, really, you may only encounter once or twice in your career. As compared to, say, me, where I’m a specialist in only that area.

I think that we – look, here’s the deal. Even today, only 5 to 7 percent of all oncology patients are ever referred to – are ever enrolled on a clinical trial. Clinical trials oftentimes are seen as last resort, last ditch. And I understand that. In fact, I even thought that before I went into medical school.

Then once you get to this point where I am, you realize, wait a second, clinical trial, oftentimes, are frontline programs. Yes, sometimes they’re randomized, sometimes they’re not. Very few times does anyone get placebo, right? Which is what a lot of people are worried about. Or if they do, it’s two drugs versus one plus placebo. Anyway, so there are a lot of different things that are clinical trials.

But we realize now that oncology has so little known information still in 2021 and beyond. So the ability to enroll in a clinical trial, be referred to it, travel to it, know about it, get on it, you’re contributing so much information to not only yourself and patients in your cohort but possibly for the future. So, that’s my only plug, is I wish that we would all ask each other more about “What clinical trials are available, how do I look them up, do you recommend this for me, or can I figure out with you how to travel and do all that stuff?”

So, I think clinical trials in rare blood cancers such as MPNs are underutilized, under-referred to, under-thought of. And then even when we are able to get people there, Katherine, it’s difficult to keep people traveling, particularly vulnerable people.

Katherine Banwell:      

Yeah. We have a question from a newly diagnosed PD patient. Sharon says, “I’m just about to begin Jakafi. What can I expect?”

Dr. Pemmaraju:         

Yeah, great question, right? So, with ruxolitinib or Jakafi, I think the biggest couple of points here is, for what’s known, this is the first-in-class JAK inhibitor that we have the most experience with.

So, now we have over a decade-plus of experience. I guess general things are general, right? This is not specific medical advice. That’s not the intention of this program. But, in general, I would stick with what’s on the package label insert, and there are a couple things we know.

One is this is a highly effective drug. This drug, which we have tested now in multiple, multiple, multiple different trials in myelofibrosis, polycythemia vera, now approved in a form of graft-versus-host disease, different doses. So, I would say check the dose for your particular disease and indication. Double-check it with your pharmacist. Make sure there are no drug-to-drug interactions.

Number two, I think what’s important is that some patients on this drug can experience immunosuppression. So, that means that you may be at risk for some infections, and there’s some nice literature about that.

So, check with your doctor about that, particularly reactivation of old infections, looking out for viral infections, such as herpes zoster or shingles. And then I think the other key here is to watch out for the modulation of your disease. So, a lot of folks have big spleens, Katherine. Those shrink down. Then patients get their appetite back, they’re able to eat, and so some people can have weight gain that then goes the other way. So, these are some of the things you want to watch out for.

But, in general, read the package insert. If you have the ability to, it’s worth reading the – if you can, read the paper, right? Go read the New England Journal paper or – if you can look at that. And then make sure you talk to your local pharmacist and ask the same question there. You might be surprised at some tidbits and pearls you can pick up.

Katherine Banwell:    

Right. Once on therapy, how is the disease monitored, and how do you know if the treatment is working?

Dr. Pemmaraju:         

Yeah. So, it differs from each disease, but let’s take polycythemia vera for a good example. So, let’s suppose you have polycythemia vera. I think there are three markers here that you can check. One is the blood counts, right?

So, you want to make sure that the blood counts are controlled. New England Journal, five or six years ago now, our Italian colleagues published a very seminal paper which shows that the goal of therapy should be that the hematocrit should be below 45. So, that’s actually a very nice number to have. So, not just waiting for symptoms of the disease but keep the number low. And if you do that, that correlates with decreased cardiac events, thromboembolic events.

Number two, I think that, besides the blood count, the spleen. The spleen and liver size also is a nice surrogate for how the disease is doing. So, if that’s enlarging or getting out of control, that may be time to stop what you’re doing, reassess. The disease may be progressing to myelofibrosis, for example.

And then I think, lastly, the absence of stuff actually helps, too. So, the absence of major bleeding, the absence of blood clots, the absence of transformation to MF. I think if the quality of life is good, you’re decreasing blood clots and bleeding, you’re not going to a more advanced disease state, these are all wins for us with p. vera.

Katherine Banwell:    

You touched on this briefly, but I’m wondering when a patient should consider changing treatments.

Dr. Pemmaraju:         

Yeah, changing treatments is more art than science, I would say. So, it does – that’s one of those that is kind of specific from patient to patient. In general, what we just talked about gives you that guidance. So, in polycythemia vera, since we brought that up earlier, uncontrolled blood counts despite maximum medication intervention, the phlebotomy requirement being untoward and impossible to keep up with, the spleen size growing out of control, the quality of life being impossible – these are some aspects to look into changing therapy and/or clinical trial.

But remember, it’s not a one-size-fits-all, right? So, some patients, the counts – some of these things may or may not actually play out. So, it has to be more of a gestalt, more of a total picture there.

Katherine Banwell:    

Yeah. Why is it so important for patients to speak up when it comes to symptoms or treatment side effects?

Dr. Pemmaraju:         

Well, I’m going to be that magician who you watch the TV show, they give away all the secrets. So, this is the big secret. Your doctor cannot read your mind. I hate to say that, Katherine. I just said it here, and it’s going to surprise some people. No, I mean, seriously. Right. So, I think the problem with the MPNs – not the problem, the caveat, the difficulty – is if you are a patient, you have this war that’s suffering inside of you. I know that as an expert person. You know that as a patient. But whoever you’re sitting in front of is not going to know that.

And there are two reasons for that. One is you don’t look like that. Most of our patients – whatever this is, I’m going to put this in big air quotes, so in case someone’s not watching this and they’re only hearing, I’m putting air quotes. People say to my patients, “Wow, you don’t look like a cancer patient.” Whatever that means, right? So, most of our patients don’t have their hair falling out, etcetera, etcetera. So, there’s that aspect of it, the visual education part of it.

Then there’s also the part, which is a lot of these symptoms burdens are not obvious on the physical exam. You cannot tell by talking to someone or looking at them if they have night sweats, bone pain, even itching, any of these things. Fatigue. You can’t tell if someone has fatigue most of the time unless you ask them. So, this is one of those where shared partnership in decision-making is not just a generic phrase. This is important.

I would say that for a patient with an MPN, the MPN symptom burden – the questionnaire, the 10 questions that we now have settled on – that can tell so much more or as much as the physical exam or the blood counts.

So, it’s imperative. It’s not just a luxury. It’s imperative. And if the patient themselves is unable to speak up, then if the advocate or caregiver or loved one can, if that person is available.

The other point I would say to this is that oftentimes the symptoms can precede – they can come before laboratory changes, physical exam changes, all these things. So, a constant, constant communication, “Hey, I was playing 18 holes of golf last year.”

“Now I can’t even get out of bed.” Hello? That tells you more than almost anything you can read on a piece of paper. So, you, as always, are spot-on with what you said. And this is the case where people say, “What can I do to help my care?” This is it. Speak up, speak out. It’s your body, it’s your life, make sure you feel empowered to do that.

Katherine Banwell:    

Right. It’s important to have that dialogue. Dr. Pemmaraju, you’re very active on social media, and patients often share information with one another. So, what advice do you have for patients to ensure online sources are actually credible?

Dr. Pemmaraju:         

Wow, great question. First thing I would say is I encourage everyone to get out there, so that’s key opinion leaders, local physicians, nurses, pharmacists, patients, caregivers, everyone. But Part two is what you said is true. Most everything out there is noise. It could be garbage. It could be background. It could be misinformation. So, you do have to have some way to filter it.

I call it signal from the noise. That’s a common phrase that a lot of people on social media use. I guess three things that I would give as tips. One is don’t be afraid to read and get on there, but I would just say whatever you read, take it with a grain of salt, as you said, and just write everything down where you have it organized.

Number two, tend to gravitate towards known experts and known sources. So, for example, you mentioned that I’m on there. That’s great. Ruben Mesa, our great friend and colleague, etcetera, etcetera. So, if you know who the 10 or 15 thought leaders are on Twitter or social media, see what they’re saying directly. That’s nice, because it’s straight from them to the public.

And then three is stick with the organizations and entities that are trusted sources. New England Journal of Medicine, ASCO, ASH, programs such as yourself, etcetera, etcetera, who are trying to put out there the latest and honest information.

Okay. So, now the fourth part, though, I think is the most important, which is what we said earlier, which is whatever you look up, discuss it with your doctor and your physician team. Period. Because no matter what research you did, no matter what patients groups you join, there might be something that either doesn’t apply to you, or worse, as you said, it could be actual misinformation, and it’s a red herring.

So, maybe find information, figure out a way to filter it, cross-check it, and then bring it up to your doctor team. I think that’s a winning way for success with information nowadays.

Katherine Banwell:    

Yeah. That’s really helpful information. Well, we have another audience question. This one is from Richard. He wants to know, what advice do you have for caregivers, and how can he be supportive during appointments?

Dr. Pemmaraju:         

Yeah. Richard’s question really is so important. Really, before the pandemic and now with the pandemic this extended time, this is the most important question that’s coming up. This is a challenge. I think a lot of our patients who are older, frail, live alone, they don’t even have the option to do that. That may be 25 percent of our patients right there,

And that’s very heartbreaking and difficult, and clearly, their care – it may not be compromised, but it’s certainly limited in some ways without getting that other perspective, right? So, I think that’s important.

Now, out of the 75 percent of the people who may have someone that can be a part of their life, a lot of these folks, Katherine, are limited because of the pandemic. Most hospitals, smartly, I think, still have restrictions on not allowing every single person in the building just for health and safety protocols. So, telehealth has had to be a substitute, I would say, for that, and in a lot of cases, has been helpful. In some cases, frustrating, obviously, with technical difficulties, etcetera, etcetera.

I would say that the key is – and I really want this to be very specific. It would be easy to just say, “Yep, bring a loved one to your visit.” No, it’s not that easy, right? So, now, during the pandemic, I think two things are very important and what I’ve noticed. One is, if the patient is able to, if their health allows them to, prime the loved one or caregiver, “Hey, I’m going to be in the doctor’s office from this time.”

And I always say make it like the cable person visit, right? From 8:00 to 5:00. So, “Hey, today, on Tuesday, if you can have your cell phone on you, that would be nice, because I’m going to patch you in, and you can listen in the background.” This is actually a key pearl I can give to people. You’d be surprised how helpful that is. Because most people, if they’re not living in the same household or whatever – “Oh, I didn’t even know you were going to be,” – okay.

Number two, when the loved one or caregiver is involved, which I encourage for everyone, try to discuss with them the night before, if your health allows you to, to go over some of the key questions. Say, “Hey, guess what? I only understand about 7 to 10 percent of what goes on in these visits, but I need you to ask this.” So, you can kind of prime your loved one to do that.

And then, lastly, you had mentioned earlier to have this list of questions. Well, that’s a great thing to give to the caregiver, right? So, if you’re able to use email and your family member is in California and you’re in Texas, maybe a quick email the night before.

“Hey, here’s what I’m thinking. In case I forget, will you ask this to the doctor?” A lot of these visits may only be five or 10 minutes, but you’d be surprised, if you have a list of two or three questions – boom, boom, boom – and then it’ll alleviate those worries there.

Lastly, I would also say don’t feel – I want to tell this to the viewers out there. Don’t feel pressured when you’re in the visit with us that you have to get every single thing out. And what I mean by that is now with email and the electronic medical record portal systems, there is some ability to contact people during – I’m sorry, after and between visits. So, maybe that might help you to not feel so much pressure in the visit.

Katherine Banwell:    

Yeah. Very good advice. Thank you. Before we end the program, have there been any recent developments in MPN treatment and research that make you hopeful?

Dr. Pemmaraju:         

I have a lot of optimism and hope. It really blossomed over the last two years. I’m happy to report to our viewers – and this is incredible, I never would have predicted this. There are over 10, maybe 13, Phase 3 clinical trials that are in the clinic now or opening soon.

Phase 3 meaning sort of the latest – one of the latest stages of clinical trial development for new drugs. And a lot of these drugs are combinations. So, a lot of them, you’re on your ruxolitinib, for example, you’re on your JAK inhibitor, and then you add in the second agent. This is very exciting. I, myself, am leading or a part of several of these. I mean, we could have never envisioned this five years ago. So, not only these drugs have shown encouraging activity in the Phase 1 and 2 – so, the earlier stages – so, now we have to confirm and test them in larger trials. So, stay tuned for that, whether you can participate directly or at least follow along with the information.

And then the other piece of excitement is that there’s a lot of beyond JAK inhibitor drugs. So, that means novel pathways, new drugs that have nothing to do with JAK that can either stand alone by themselves or be combined. That’s another exciting area. We have multiple classes of drugs emerging from the lab into the clinic now that I hope will have a lot of benefit for our patients.

So, tons of optimism and excitement, frankly, that just wasn’t there five years ago.

Katherine Banwell:    

That’s wonderful. Dr. Pemmaraju, thank you so much for joining us today.

Dr. Pemmaraju:         

Thank you, Katherine. I just love the chance to spend time with you guys. I hope to do this soon one day.

Katherine Banwell:    

And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell, thanks for joining us.

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.

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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.

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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.

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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.

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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.

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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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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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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.