Myelofibrosis Clinical Trials | The Benefits of Patient Participation

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How do clinical trials fit into a myelofibrosis treatment plan? Dr. Idoroenyi Amanam discusses the benefits of clinical trial participation and advice for patients who may be hesitant to join a trial. 

Dr. Idoroenyi Amanam is a specialist in myeloproliferative disorders and is an Assistant Professor in the Division of Leukemia at City of Hope. Learn more about Dr. Amanam.

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Transcript:

Katherine Banwell:

Dr. Amanam, where do clinical trials fit into a myelofibrosis treatment plan?  

Dr. Idoroenyi Amanam:

When you think about where we’re at right now in myelofibrosis, I think  from the scientific standpoint, it’s really exciting.  We’re understanding this disease so much better.  We have  a much clearer picture of how this disease works.  

But with that said, unfortunately, when you look at the drugs that we have available to us, we don’t really have that many still,  and so clinical trials play a big part in this disease space. Because there’s patients who are  very advanced when we diagnose them, and our FDA-approved therapies may not really make a big dent in their symptomology, may not change  what happens with the disease moving forward. And so, clinical trials do give patients such as that an opportunity to actually  get the disease under control. 

Katherine Banwell:

Well, as you mentioned, patients participating in trials are key to moving research forward, right? Can you talk about the benefits of clinical trial participation?  

Even though it – 

Dr. Idoroenyi Amanam:

Yeah – 

Katherine Banwell:

– might be scary for a lot of people. 

Dr. Idoroenyi Amanam:

Right, right. 

Katherine Banwell:

I think a lot of people still think, “Oh, I’m just going to get a placebo, and it’s not going to help me in any way,” but that isn’t really the case anymore, is it? 

Dr. Idoroenyi Amanam:

It’s not the case.  And I think the way that you can think about trials are, we use trials to give therapies that we view as very promising to patients who fall in a gap. And that gap may be that, even though we have FDA-approved drugs, we know that those drugs really may not work for that specific patient. And so, we have an understanding from either a different type of disease that’s very similar to myelofibrosis, or  our  scientific experience that there is a therapy that will work. And we want to use trials to be able to give that patient a chance to, kind of, get over that hump. 

And so, in today’s era, we don’t structure trials around therapies that we don’t believe actually work or  getting patients in trouble down the line. And so, we really, really do believe that our trials are, kind of, an extension of what we have currently  on the market, and so it is a part of our everyday armamentarium to fight this tough cancer. 

Katherine Banwell:

What would you say to patients who are hesitant of participating in a trial?  

Dr. Idoroenyi Amanam:

One, I’d really want to get a clear sense of what their hesitance is.  Is it that the trial would put them at higher risk for some sort of toxicity?  Is it that they’re worried that there’s some other therapy that’s out there that, potentially, would give them a better chance at fighting this disease? the third part of it, the unknown, is always scary for many people.  

And I think my job is to be a part of a patient’s team.  Talk through those concerns, get a sense if this is something that really is the best thing for them, or do we have something else that might be a better option? And unfortunately, there are some instances where I do believe the trial is the best option for a patient, but the patient does not feel comfortable, and we have to go down another path.  

And all of us, we will never force a patient to go down a path that they don’t feel comfortable with. We all believe in patient autonomy, but there are some instances where   the trial may be the best opportunity, and I will do my best job to advocate for that and come to a decision together with the patient. 

Why Myelofibrosis Patients Should Be Engaged in Their Care Decisions

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Dr. Idoroenyi Amanam, a myeloproliferative disorder specialist and researcher from City of Hope, shares expert perspective on the importance of patient participation in care and treatment decisions. Dr. Amanam emphasizes the necessity of having a care partner and utilizing all members of the healthcare team. 

Dr. Idoroenyi Amanam is a specialist in myeloproliferative disorders and is an Assistant Professor in the Division of Leukemia at City of Hope. Learn more about Dr. Amanam.

Download Resource Guide

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Transcript:

Katherine Banwell:

Well, how do you encourage patients and care partners to be involved in decision making? Do you have any advice for them?  

Dr. Idoroenyi Amanam:

That’s a very important question, and it’s a tough one. And I will tell you, personally, I have family members who have medical  disorders that I think do require a lot of support from other family or friends. And  based off my experience, I encourage patients to involve their families in these discussions, because I don’t think  we’re at a space where things are very binary.  

I think the decisions that we make when we’re treating patients with myeloproliferative disorders and myelofibrosis, there are some nuances there, and I think family can really help the providers, in addition to the patients, in coming to the right decision about how we’re going to move forward.  

So, I think my advice is, involve your family, involve your friends.  I think having a community of support is very important when you have a type of disease such as this. 

Katherine Banwell:

And it’s important to have somebody there with you, a care partner or a friend, as you say, who may be able to ask questions that you, as the patient, haven’t thought of. Somebody there to take notes, just in case you need to refer to something after. 

Dr. Idoroenyi Amanam:

Absolutely. Absolutely, I agree. I think it’s a team from both sides  to, kind of, extend what you’ve said. To the medical side, the pharmacist may give me some input about  some things I may have missed with the patient,  the nurse practitioner in clinic, the RN in clinic,  the other staff.  

And  I think it’s one of those situations where the more people involved can help us, kind of, draw that picture better. I think we’re trying to get a sense of how we can move forward in the best way, and having all of those parties being active and offering  the best that they can is really helpful for everyone. 

Promising Advances in Myelofibrosis Research | Optimism for Patients

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 Dr. Idoroenyi Amanam from City of Hope discusses how the availability of newer JAK inhibitor therapies is providing better options for myelofibrosis patients. Dr. Amanam also shares how researchers are working to reduce the risk of disease progression and providing curative options for myelofibrosis.

Dr. Idoroenyi Amanam is a specialist in myeloproliferative disorders and is an Assistant Professor in the Division of Leukemia at City of Hope. Learn more about Dr. Amanam.

Download Resource Guide

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Transcript:

Katherine Banwell:

Dr. Amanam the JAK inhibitor class of therapies often have side effects associated with them. What advancements are being made to manage these side effects? 

Dr. Idoroenyi Amanam:

Yeah. That’s a great question. And I would think about when these JAK inhibitors first  came out, I think we were all willing to accept the side effects because of the fact that you only had one JAK inhibitor, and then we now have more than one. We have a couple of JAK inhibitors, and I think the idea is that the biggest problem that we had with some of the JAK inhibitors were that  patients’ counts couldn’t tolerate higher doses of the JAK inhibitor.  

And we know that for some JAK inhibitors, you have to be at a baseline dose that will help with shrinking your spleen, or improving symptoms, and if you’re below that dose, it doesn’t work very well. And so,  we had a tough time   making adjustments based off of patients’ counts. And so, we have some newer JAK inhibitors that    patients tolerate    these drugs even though they have lower counts. And so, I think that’s one big change that we’ve all seen over the past five years.   

Katherine Banwell:

Is there anything else you’d like to add about advancements in myelofibrosis care?  

Dr. Idoroenyi Amanam:

I would say if we think about early stage, a patient who’s diagnosed, and they’re told that they’re early myelofibrosis, or what we really truly define as low risk myelofibrosis, we traditionally did not have any therapies for those patients, and I think that it’s exciting that we are in a space now where we are thinking about therapies for those patients, and therapies that will reduce the risk of progression to a more advanced stage of myelofibrosis.   

For patients who are in the middle, I think we’ve had a rapid expansion of therapies that are available to patients, and I think a lot of our clinical trials that are currently in play are really directed towards that group, and so that’s really exciting. And then for the high-risk patients, City of Hope is a bone marrow transplant center, cellular therapy center, and we have a lot of experience in performing allogeneic stem cell transplants for myelofibrosis patients.  

And I think across the country, we have gotten better at performing allogeneic stem cell transplants for myelofibrosis, and as of right now, it’s one of the few curative therapies that we have.  

And we have been able to understand how to get these patients through a very intense regiment and get them to the other side, and I think that’s also very exciting. We still have a lot of clinical trials in allogeneic stem cell transplant space as well. And so, I think we are in a place now where we have a therapy for everyone, if they choose to want one.  And not only a therapy just to bridge them, or treat their symptoms, but therapies that, potentially, will get rid of this disease.  

And I think for a patient, when you go to sleep at night, or when you talk with your family, the biggest worry is having something  that there’s no clear sense of we can get rid of it. And I think we’re getting to a better place where I can confidently tell patients we’re getting better at getting rid of this, and I think that’s the most exciting thing that we have right now.  

Katherine Banwell:

Yeah. It’s a promising field. 

Dr. Idoroenyi Amanam:

Very promising.  

Katherine Banwell:

If we look at care and treatment, why should a patient with myelofibrosis consider a second opinion with a specialist?  

Dr. Idoroenyi Amanam:

The field is rapidly changing, and we have very few FDA-approved therapies even in 2024 for myeloproliferative disorders and myelofibrosis. And because of some of the changes in understanding of the biology of this disease, I think that there are many patients that will benefit from physician or a center that specializes in treating patients with this very rare disorder. And I believe that   even though we have very few FDA-approved therapies, I do believe that we can give patients an opportunity to get this type of disorder under control, and actually offer curative approaches that may not be available to all facilities. 

Recent Advances in Myelofibrosis Research | Disease-Modifying Therapies

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Where is progress being made in the field of myelofibrosis? Dr. Idoroenyi Amanam discusses how disease-modifying therapies and cellular therapies are advancing patient care.

Dr. Idoroenyi Amanam is a specialist in myeloproliferative disorders and is an Assistant Professor in the Division of Leukemia at City of Hope. Learn more about Dr. Amanam.

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 Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors 

Transcript:

Katherine Banwell:

What are your areas of focus in myelofibrosis research? 

Dr. Idoroenyi Amanam:

So, I think the way that I would break it down, when you think about myelofibrosis patients, or patients with myeloproliferative disorders, patients who are diagnosed, and they’re really pretty much asymptomatic, and then you have patients who have some symptoms, then you have some patients who are very symptomatic or transitioning into a more severe disease and predominantly, that disease is acute leukemia. And so, I have an interest in trying to identify new treatments for all of these types of patients.  

Katherine Banwell:

In your opinion, what new myelofibrosis advances are most promising? 

Dr. Idoroenyi Amanam:

So, traditionally, for a lot of myeloproliferative disorders, including myelofibrosis, we had a watch and wait approach, so we typically actually did not really have very good therapies. And I think all of that changed with the approval of the first JAK inhibitor, ruxolitinib (Jakafi).  

And we have transitioned to understanding the signaling pathways that are involved in myelofibrosis and myeloproliferative disorders. And we understand certain driver mutations that are involved in these signaling pathways and involved in other areas that help drive these diseases.  

And I think what’s exciting for us right now is, we’re transitioning from having a Band-Aid approach, or a watching and wait approach, to actually having interventions that are what I would call disease-modifying drugs. And so, these drugs target some of these drivers that drive the disease.  

They target some of the inflammation that’s associated with the disease, and, in fact, they’re also targeting some of our own immune cells that may help us protect us against these diseases progressing into more aggressive disorders. 

Katherine Banwell:

How are innovations in technology accelerating myelofibrosis research?  

Dr. Idoroenyi Amanam:

You think about how we understood these diseases 20 to 30 years ago. I think we understood the clinical presentation. We understood that some patients had big spleens, we understood that those patients’ counts didn’t do so well, we understood when we would look at their marrow, how the cells looked under the microscope. And we’ve transitioned to now understanding how some of those proteins that are   in these  signaling pathways are either turned on or turned off.    

We have a better understanding of the genetics of this disease, and how it changes over time, and what that means for patients prognostically, and how they will actually respond to our current therapies.  

And obviously, it’s driving how we are setting up clinical trials and other therapies  for the future. And so, I really would say our  genetic, genomic understanding of these disorders have really opened up many opportunities for us to treat these patients better.  

Katherine Banwell:

Well, are there other research developments showing promise that patients should know about? 

Dr. Idoroenyi Amanam:

So, traditionally, we’ve thought about  these disorders as disorders where, maybe if we improved symptoms – so we give you, maybe, a pill that would help with your symptoms, or we’ll give you another medication that will help with keeping your counts under control or reducing your risk for clotting and stroke.  

And we are currently in a space where cellular therapy has exploded across all areas of oncology. We have many clinical trials that are using  therapies that  take your own cells,  or other donor cells from healthy  people, and we are giving them to patients with the hope that it will  get rid of these  bad cells that are driving myelofibrosis.  

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

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Dr. John Mascarenhas discusses the evolving landscape of myelofibrosis research, emphasizing the concerted effort among researchers, pharmaceutical companies, and advocacy organizations to advance care and treatment options for patients.

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

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Transcript:

Katherine Banwell:

Is there anything you’d like to add about the evolution of myelofibrosis care? What are you excited about?  

Dr. John Mascarenhas:

I always make the comment they don’t feel rare to me because I see so many patients with myelofibrosis, and it’s what I do. But I recognize in the context of lung cancer, breast cancer, other more common cancers, these can be forgotten diseases. But what has been encouraging is, between the NIH funding, for example, our research consortium, which is really geared to translating the biology into clinical trials across the country.  

Pharmaceutical interest, which is essential to providing drugs and finance to run trials. Young investigators that are coming to the field that want to make a difference, institutions that continue to support the programs, and then foundations. Whether it’s MPN Research Foundation, Leukemia & Lymphoma Society, it really takes a village. And we’ve been working with the FDA to try to better understand how to develop trials that are meaningful that can get drugs approved and to the patients. 

So, it’s a concerted effort, and I’ve been enthusiastic. I remain optimistic that everyone is trying to do the same thing, achieve the same goal, and work together, and that’s the only way we’re going to be able to do it. 

Katherine Banwell:

Yeah. How long does it take for the FDA to approve a drug? 

Dr. John Mascarenhas:

Forever. It feels like forever. It’s a long process, and for patients, it can be quite frustrating, because there’s so many steps involved. From just the original, initial steps to allow you to introduce a drug into humans, there are IND-enabling studies, so there’s a lot that goes into this.  

And then the initial studies are safety studies. They often can take quite some time. There’s a lot of scrutiny on safety, obviously, because the FDA is really charging to making sure that we do no harm, that we maintain safety for patients, so that can take a long time. And then the ultimate testing and comparison to a control arm is essential to get a drug approved. 

So, we’re looking at a timeline that can easily be a decade from the time we have a great idea, and have a drug available to us, to the time that we can prove that through the different stages, and then demonstrate that to the FDA and negotiate what a label will look like. And that is both a time-consuming process, a very expensive process, and a laborious process, but obviously an important process. 

Katherine Banwell:

Yes, and as you say, so many new drugs and therapies have become available in the last 10 years or more that really have advanced myelofibrosis care enormously. 

How Can You Learn More About Myelofibrosis Clinical Trials?

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Dr. John Mascarenhas shares advice for patients looking to learn more about clinical trials starting with consulting a specialist. Dr. Mascarenhas also emphasizes key questions to ask, including a discussion of the benefits versus risks of participating in a potential trial. 

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

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Transcript:

Katherine Banwell:

What about clinical trials? How can patients learn more? 

Dr. John Mascarenhas:

Well, clinical trials is definitely a confusing area because there are many clinical trials, and some of them are relevant to some patients may be not relevant to other patients.  

So, I think two ways is, see someone who does this. So, not everyone has as their primary care provider hematologist an expert, or someone who’s dedicated to doing this. They may be in a practice where they’re in the community setting; it’s not reasonable for them to be seen at a tertiary care center. 

But if you can get there even for a consultation or an initial visit to get plugged in, I think it’s really critical to see someone who is really invested in this with research opportunities, and clinical trial availabilities. And then the other resource would be clinicaltrials.gov.  

You can go in there, you can put in your diagnosis myelofibrosis. You can even manipulate it for geography to understand what trials are in your area. 

But it is a good way of looking, just to understand what’s there. And then the MPN Research Foundation, which is very supportive of the patient community and engages them, is often a very good resource to go to, to either learn about trials or join webinars where physicians that do this will discuss, and I think that’s another excellent resource. 

Katherine Banwell:

What questions should patients ask their team about clinical trials?   

Dr. John Mascarenhas:

Well, are there clinical trials for what I have? Because again, they can be very particular to where you are in the disease process, what medications you are on, what your kidney function is, things that may really influence decision-making. So, are there clinical trials, what are the clinical trials evaluating, what should I expect from them, both potentially from a positive angle, but also from a negative. What are the known toxicities, what would be the time commitment? Trials are more involved, and they’re more involved for a reason. 

I actually personally think patients do better on clinical trials than standard of care. The reason why I think that is because they are more involved. There’s a lot more oversight and eyes on you. Not just a physician perspective, but maybe more importantly, from a research nurse and research coordinator perspective. There’s a lot of regulatory burden which translates, I think, to a lot of attention to safety and assessment for advocacy.  

So, I think understanding what that looks like at any given institution, and how it will affect the patient and their caregivers from a time perspective, and obviously toxicity. But also, what is this trial trying to achieve? Does it make sense for what I’m trying to achieve? 

Katherine Banwell:

I suppose another question might be where this trial is taking place? As a myelofibrosis patient and a caregiver, are we going to have to travel to get to this clinical trial? 

Dr. John Mascarenhas:

I think that’s a major obstacle for a lot of patients. We’re talking about a disease that typically affects people that are in there sixth, seventh, eighth decade of life. Patients don’t all live, as I’ve said, right around a cancer center or a tertiary care center, so travel, the logistics of it, the ability to have that support available. Sometimes it’s loved ones, and adult children having to take time off of work to be able to help in that process.  

It’s a lot, and I’m particularly sensitive to it, because I work in a metropolitan area, and I realize getting in and out is not easy. And a lot of times, these trials understand that, and they build into the trials stipends and support for transportation and/or lodging which helps. It’s not perfect, but it can help at least financially, and sometimes logistically, so I would definitely inquire about what those resources are.  

And sometimes foundations also help bridge the gaps for patients to help link them to trials and facilitate that. So, it’s a super important part of engaging in a trial. 

Katherine Banwell:

And who is on the health care team that might be able to answer questions like this? 

Dr. John Mascarenhas:

Well, for sure, the physician should be able to. But I think the most valuable resource often is the nurse and the nurse practitioner. There are usually research coordinators. These are often young people, but very bright young people, that are very invested in this that will sometimes show up at the clinic to talk to the patient or will be a phone resource that you could reach out to. So, it can really be, I think, three levels. The coordinator, the nurse or nurse practitioner, or physician assistant, and the physician. So, it really shouldn’t be one person, but a team of people that are available to you. 

Expert Outlook | New Myelofibrosis Therapies Showing Promise

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What myelofibrosis therapies in development show promise? Dr. John Mascarenhas, a myelofibrosis researcher, reviews innovative treatments that are being combined with JAK inhibitors as well as single agent therapies that are making headway for patients with myelofibrosis. 

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

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Transcript:

Katherine Banwell:

The JAK inhibitor class of therapies has been around for over a decade now. What new therapies are showing promise when being studied in combination with these therapies? 

Dr. John Mascarenhas:

So, I think the ones that are really exciting, and there are a number. We don’t know which one is the best, but I’ll tell you the ones that I think really have potential would be drugs like pelabresib, the pan-BET inhibitor, and the MANIFEST-2 study. Even a drug called navitoclax, that isn’t going to move forward, taught us a lot. We know that pathway is important, we just have to improve upon how we’re doing this.

Drugs like selinexor (Xpovio), the XPO1 inhibitor, is ongoing in the SENTRY study. A drug called navtemadlin is a very active drug, and that’s been shown as a single agent after ruxolitinib (Jakafi) failure. But now, it’s going after those patients who are not having an optimal response with ruxolitinib, adding it on on the backend.  

So, what I really love about the way we’re doing this is, I think it’s a very thoughtful approach trying to use these really active drugs that exploit non-redundant pathways in the disease, both either up front, to really get the biggest bang for your buck, to really try to reduce the diseased burden earlier on, or to try to add on as a strategy if patients aren’t enjoying the maximum benefit from ruxolitinib. So, we are really trying to tackle it from different angles and some of these drugs really look promising. 

Katherine Banwell:

Yeah, yeah. Are there other single agent therapies that are being studied for myelofibrosis?  

Dr. John Mascarenhas:

There are. So, I’ll name two that I also think really deserve some attention. One is called TP-3654, and it’s a drug by Sumitomo that’s a PIM 1 kinase inhibitor. So, this also goes after a very specific pathway – inflammatory pathway – a signaling pathway – that is known to be an important driver of disease and has very nice data, particularly from a symptom-burden perspective. But also, again, this concept of disease modulation and reduction in cytokines in patients who’ve previously been on ruxolitinib.  

So, there’s data there where they’re going to add it on to ruxolitinib that really looks like an interesting approach forward. And then the drug I think many of us are very anxious to see results in which is ongoing, is the IMpactMF study.   

This is the randomized phase three study of imetelstat (Rytelo), which is a telomerase inhibitor and infusional agent that goes after a very important enzyme that keeps malignant cells alive and really is one of the drugs that I think has the true potential to go after the stem cell, the origin of the disease, and improve survival. It’s the only study we have had, and currently have, where the endpoint for the registration phase we’ve studied is survival. It’s patients who have failed ruxolitinib and are getting this drug as a single agent, versus best available therapy.  

A very exciting trial and really important. Whether you’re on the trial or you’re a candidate for it, it really helps us move the field forward, because it gives us essential insights into the disease and how to do better. 

Katherine Banwell:

Yeah. When it comes to the latest research and treatment, what question should patients ask their health care team about new or developing treatment options? 

 Dr. John Mascarenhas:

Well, I think every patient is different, and truly different since their biology is different, the way they present is different, their course is different. So, really, the treatment options, including the trial options, really need to be tailored to the patient. It has to make sense for that patient. It has to meet their expectations, be aligned with their goals of therapy, and balance. Balance risk with potential benefit. Patients have to understand. The physicians have to present very clearly that some trials are randomized studies, and you could get a placebo. 

And it’s often blinded, so the patient doesn’t know, the physician doesn’t know. But importantly, in some of these studies, there’s crossovers, so even if you don’t get the drug up front, you can get it in the backend. All of these things really have to be disclosed very carefully and thoughtfully, so the patient’s really making an informed decision that makes sense for them and is meeting their expectations. 

Updates in Myelofibrosis Research From an Expert

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Dr. John Mascarenhas shares updates on myelofibrosis research. Dr. Mascarenhas highlights the shift towards combination therapies, particularly the use of JAK inhibitors alongside novel agents, with the goal of improving disease response and patient outcomes.
 
Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

See More from Evolve Myelofibrosis

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Expert Outlook | New Myelofibrosis Therapies Showing Promise

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Transcript:

Dr. John Mascarenhas:

My name is John Mascarenhas, I am a professor of medicine at the Icahn School of Medicine here in New York City. I direct the Center of Excellence for Blood Cancers and Myeloid Disorders, and I lead the adult leukemia program. But my real passion and interest is in myeloproliferative neoplasms and translational research, trying to understand the biology of the diseases and helping translate that into effective therapies in the clinic. 

Katherine Banwell:

Dr. Mascarenhas, from your perspective, what are the highlights so far this year in myelofibrosis research? 

Dr. John Mascarenhas:

So, I think myelofibrosis research – I’ve been in this field for about 20 years, and I’ve watched it go from a field where we had very little insight into the biology of the disease, which meant very little targeted or informed therapies to the era of JAK inhibitors.  

The first being 2011 with ruxolitinib (Jakafi), then 2019 with fedratinib, 2022 with pacritinib, and then 2023 with momelotinib (Inrebic), has really afforded us a significant advantage in trying to tailor the treatments for different patient niches to improve spleen and symptom benefit.  

And I do think that translates to a survival benefit in our patients with myelofibrosis. So, outside of bone marrow transplant, really these treatments are not curing patients, but they are addressing certain aspects of the disease. 

What I’m most excited about is the new era; the next generation of approaches that we’re seeing, and we have been seeing, and will continue to see emerge, and these include combination therapy approaches up front. So, taking those JAK inhibitors, the benefit they have, and trying to improve upon that with the addition of informed therapies, rational drugs that have pre-clinical evidence. 

Meaning, in the lab with cells from patients with animals that are engineered to have myelofibrosis, so that when we take them into the clinic, we are more confident, more informed in our decision-making, that we’re not exposing patients to drugs that really don’t have rationale.

Katherine Banwell:

What do these research advances mean for myelofibrosis patients? 

Dr. John Mascarenhas:

Well, I think what we’re seeing is a shift towards more combination therapy. So, what I think it means for a patient is deeper responses from not just spleen and symptom, but what we’re looking at very intently are biomarkers of disease modulation and disease response, hopefully, disease course changes.  

So, things like reductions in their driver mutation. These are gene mutations like JAK2, CALR, MPL, reductions in inflammatory markers, reduction in bone marrow fibrosis in the bone marrow.  

All of these things suggest that we’re really starting to modulate the disease in a more significant way. What we’re trying to show is that that actually matters to a patient, that these findings actually translate to better progression-free survival, better overall survival. So, I’m really enthusiastic and excited by what is happening now, because I do think it pays off. 

It’s incremental benefits, but things that are now more targeted, like mutant CALR antibody approaches, or BiTE approaches.  

To those patients who have this abnormal CALR protein expressed on the surface of the cell transformative with at least the potential to be JAK2 selective inhibitors, really going after that mutant JAK2 in a very selective way, or a Type II JAK2 inhibitor. Really, the potential to have very molecularly defined targeted therapies that will, hopefully, get us much deeper responses; that patients will see even greater benefits, better improvements in symptom burden, spleen, but ultimately survival.  

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

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What can be helpful for myeloproliferative neoplasm (MPN) symptom management and disease progression awareness? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses education tools and interventions, support resources, and the value of both community oncologists and academic centers in MPN care. 

[ACT]IVATION TIP

“…I think there’s a distinct role for community oncologists and specialists, and really this should be something that works really well together.”

See More From [ACT]IVATED MPNs

Related Resources:

What's Next for Improving Quality of Life in Polycythemia Vera?

What’s Next for Improving Quality of Life in Polycythemia Vera?

 Advancing Therapies for Polycythemia Vera: Enhancing Control and Quality of Life

Advancing Therapies for Polycythemia Vera: Enhancing Control and Quality of Life

 Can Monitoring Albumin Levels Help Track Myelofibrosis Treatment Success?

Can Monitoring Albumin Levels Help Track Myelofibrosis Treatment Success?

Transcript:

Lisa Hatfield:

Dr. Kuykendall, can you discuss any specific interventions or educational tools that have proven effective in improving symptom management and disease progression awareness for patients facing PV, myelofibrosis, and/or essential thrombocythemia? 

Dr. Andrew Kuykendall:

Yeah, so I mean, I think when we’re talking about symptom management, disease progression, awareness, one of the things I think is very helpful is to have an expert or specialist in your corner. And that doesn’t mean that’s the person you’re seeing every month or every two months or every three months. It means you’ve seen them one time at least, right? And you’ve been able to sit down and ask every question you have, right? It is very important as medicine gets very, very specialized.

Just the amount of information that’s out there on every single one of these disease states is impossible to keep up with. If you’re a generalist, if you’re treating everything, you just can’t be completely up to date on everything that’s going on in myeloproliferative neoplasms when you’ve got a colon cancer patient, a breast cancer patient, a pancreatic cancer patient, anemic patient coming into your clinic.

And so having that specialist in your corner really gives you that resource of asking some of these challenging questions. And I think that more than specific medications, I think what a specialist can provide is that education and that lifeline. So beyond having that specialist in your corner, I think that it’s also helpful to have a network of kind of colleagues or patients that you have as a support group or as a resource group to bounce things off of.

And so there’s a number of patient networks, whether it’s Patient Empowerment Networks or MPN Advocacy & Education International or Facebook groups or whatever it is, right? There’s a lot of different resources where patients can reach out and touch base with other patients or you know look for programs, educational awareness programs that are out there and really become an advocate for themselves and really drive their own care.

So when we’re talking about interventions, educational tools for symptom management, disease awareness, I would say seek out and have a specialist that you see at least one time that you can reach out to with any questions. And also build a network of some sort of patient group where you can access real-time education and resources and also talk with other patients about their experiences.

Lisa Hatfield:

Okay, thank you. So you mentioned having a specialist, and I also have a specialist for my particular blood cancer. I live in an area where we don’t have a multiple myeloma specialist, so I’ve had to go out of state for that. So one of my biggest fears when I did that, I have a very good oncologist locally. I did not want to offend him if I said I wanted to go seek out an expert opinion. Do you have any suggestions for patients who might be afraid to mention that to their community oncologist if they’re seeing a community oncologist? 

Dr. Andrew Kuykendall:

The first thing I’d say is that there’s probably nothing to be scared of. I think that community oncologists generally understand what specialists are there for. Honestly, it takes a bit of weight off their plate. If we spend an hour, hour-and-a-half with our patients talking about everything that comes with a diagnosis of myeloproliferative neoplasms, I think that’s time that, they can spend on other things in their clinic.

And they usually have very busy clinic schedules. And at the same time, I think that this is usually a kind of symbiotic or mutualistic relationship where both people involved or both physicians involved can really play a role and benefit the others. So I’m in Florida, and this is a big state, right? 

For me to get to Key West is going to take a while, for me to get to Tallahassee is going to take a while. Miami is a long way away, but I have patients from Key West and Tallahassee and Miami. And now with virtual medicine, we could do a little bit more virtual, but it still doesn’t replace the seeing the patient in person. And so I would say 80 to 90 percent of my patients have a community oncologist that they see that has my cell phone number or my email address, and is encouraged to reach out to me with any questions, concerns, thoughts. And when we see patients and we come up with treatment plans, I’m usually kind of reaching out to their community oncologists to say, hey, this is what we’re trying to execute, this is the plan.

Do you want us to help with that? Are you able to take it? Let’s work on this together. And so typically this isn’t something to worry a lot about if you really are concerned, I think one way is say, hey, I’d like to see a specialist to talk about clinical trials. And honestly, that’s one of the things that community oncologists are like oh, okay. Absolutely. That’s a great reason to see them. The two most common reasons for a community oncologist to refer someone to an academic center is probably clinical trials or discussion of transplant, right? And so you could say, hey, I want to talk about transplant, or I want to talk about clinical trials, and typically that’ll be a good reason to get in the door.

So yeah, my [ACT]IVATION tip for this is, I think there’s a distinct role for community oncologists and specialists, and really this should be something that works really well together.

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What’s Next for Improving Quality of Life in Polycythemia Vera?

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What are key challenges in myeloproliferative neoplasm (MPN) care, and how do symptoms evolve over time? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses constitutional MPN symptoms, strategies to manage fatigue, neurovascular symptoms, and symptom management. 

[ACT]IVATION TIP

“…we have a variety of symptoms that can be associated with kind of myeloproliferative neoplasms as a whole, as well as each one of these distinct disease entities. And the therapy for each of these differs based on the particular symptom.”

See More From [ACT]IVATED MPNs

Related Resources:

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

 Advancing Therapies for Polycythemia Vera: Enhancing Control and Quality of Life

Advancing Therapies for Polycythemia Vera: Enhancing Control and Quality of Life

 Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Transcript:

Lisa Hatfield:

Dr. Kuykendall. I’d like you to speak to some of the key challenges in managing MPN symptoms. So based on the available treatment options, what are the most challenging symptoms associated with polycythemia vera, PV, or myelofibrosis and essential thrombocythemia, ET? And what are we learning about how these symptoms evolve as the disease progresses? 

Dr. Andrew Kuykendall:

When we think about myelofibrosis, that’s probably the disease we associate with the most clear, distinct symptoms. And patients often have what we call “constitutional symptoms.” And this could be fevers, chills, night sweats, bone pain, weight loss. These are classic symptoms of a disease that is really causing a lot of inflammation, right? And driving a lot of these inflammatory pathways, and these types of symptoms are quite well-addressed with JAK inhibitors, these disease specific anti-inflammatories, of which we now have four that are approved for myelofibrosis in different capacities. But there are more symptoms beyond those. I think when we think about polycythemia vera, we get less constitutional symptoms, although that certainly can be seen in a subset of patients.

But we see more itching is probably the classic polycythemia vera symptom. This itching that is quite challenging, doesn’t necessarily respond to antihistamines, and can be something that’s exacerbated by like taking hot showers or being in hot water. There’s a fancy name for it called aquagenic pruritus. And patients may not even be aware this is related to their disease. I’ve met many patients who’ve come in, who’ve been diagnosed with PV who complain of this challenge with showering or being in hot water, who really never put two and two together.

And sometimes the itching isn’t even described as itching. It feels like fire ants all over their body. And you have patients that are really avoiding right, showering. And so they’re doing it maybe once a week or once every two weeks. And so again, this is a symptom that responds quite well to ruxolitinib (Jakafi), which is approved in the second line here.

Beyond that, I think the biggest symptom across myeloproliferative neoplasms is fatigue. And I don’t have great magic tips for fatigue other than to say what we found out is probably non-pharmacologic interventions are better than pharmacologic interventions for fatigue. Things like just making sure you have good sleep hygiene, getting good sleep, healthy diet, exercise, yoga, mindfulness, resting, these probably are more successful in treating fatigue than any specific drug or agent that we have. And I think that speaks to really a failure on our part to develop better therapies. But certainly it’s something that we’re all very well aware of. And so it’s something we monitor in any of our clinical trials when we are developing agents is how does fatigue change over time? And lastly, I’d say for ET I think you can start to see some symptoms that are more kind of neurovascular.

So things like headaches, migraines, ringing in the ears, or tinnitus, or tinnitus. These can be unique to ET and may predate or preempt the actual diagnosis. So a lot of our young patients that are diagnosed with ET may come to attention of physicians because they’ve been dealing with migraines or headaches or fatigue plus migraines and headaches for a long time. Then lo and behold, blood work shows that they have a very high platelet count.

And so over time, I think when we look at these symptoms, certainly there can be waxing and waning of some symptoms, response to therapies, lack of response to therapies. But if the symptoms really do change rapidly, which we don’t see that often, oftentimes this can accompany a change in the disease, right? And that’s the time to go in and see if something’s changed. Sometimes reevaluate the disease status. And so my [ACT]IVATION tip for this is that we have a variety of symptoms that can be associated with kind of myeloproliferative neoplasms as a whole, as well as each one of these distinct disease entities. And the therapy for each of these differs based on the particular symptom.

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Advancing Therapies for Polycythemia Vera: Enhancing Control and Quality of Life

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How are emerging polycythemia vera (PV) treatments addressing quality of life? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses disease control versus quality of life issues for PV patients and shares updates about rusfertide and hepcidin mimetic clinical trials. 

[ACT]IVATION TIP

“…there’s a lot of things that factor into a suboptimal quality of life for patients with PV. And we need to think about all of those as we try to chip away and make patients’ quality of life as good as possible.”

See More From [ACT]IVATED MPNs

Related Resources:

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

 What's Next for Improving Quality of Life in Polycythemia Vera?

What’s Next for Improving Quality of Life in Polycythemia Vera?

 Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Transcript:

Lisa Hatfield:

Dr. Kuykendall, since current treatments may help control polycythemia vera but don’t significantly improve symptoms, what steps are being taken to develop new therapies that not only control PV, but also improve patients’ overall quality of life? 

Dr. Andrew Kuykendall:

Yeah, so right now the therapies we have for polycythemia vera essentially the main goal is to reduce as I tell patients, to reduce the things that could kill you, right? To reduce the risk of thrombotic events, cardiovascular events, strokes, blood clots, heart attacks, things like that. As we know that that is a huge issue for these patients that are at increased risk for cardiovascular events.

So we do that with a variety of different strategies, but I think increasingly what we’re realizing is we need to make a primary focus of treatment being on improving and maximizing quality of life. And this is something that really we should be thinking about across all malignancies. But specifically polycythemia vera (PV), where this is a disease that if we are able to avoid some of these cardiovascular events, patients have a very good quantity of life expected with their disease.

So they may live with their disease for 15, 20, 30 years. So if that’s the case, we really need to be thinking about how we can do that as best as possible, right? We’re talking about a third of your life living with this disease. And so we need to do that in a quality way. So agents like ruxolitinib (Jakafi) that have been approved for polycythemia vera in the second-line setting.

Ruxolitinib is known that it is improving some of the disease-related symptoms that come with PV, fevers, chills, itching, night sweats, bone pain really does help with those things. But I think that we can move beyond that. And we’re developing agents like rusfertide. Rusfertide is the hepcidin mimetic that is aiming to reduce the amount of phlebotomies patients need. And for me, that’s important in a variety of ways.

One if you don’t need to get phlebotomies all the time, you’re not tied to the healthcare system, right? Nearly as much. And that could be a huge dissatisfactor. And so at the same time, getting a phlebotomy is not that fun either. It requires going in and sitting there getting blood drawn, you may get lightheaded, fatigued that comes with that.

So eliminating that aspect of negative quality of life. At the same time, we’re starting to see with rusfertide whispers that it may help with some of these symptoms that may be related to iron deficiency. Things like brain fog, concentration issues, fatigue. And so if we can help a little bit with that aspect of things too, man, we could start to kind of, you know, chip away at some of the quality of life issues that are ongoing.

And then down the road, I think some of these JAK2-specific inhibitors may have the continued ability to modify the underlying disease. And certainly that’s a huge goal, right? If we can actually start to get true responses really get at the core of the disease to get the disease to go away. And I think that ultimately that will hopefully result in better quality of life as well. So, at the same time, I think my [ACT]IVATION tip for this question is that there’s a lot of things that factor into a suboptimal quality of life for patients with PV. And we need to think about all of those as we try to chip away and make patients’ quality of life as good as possible.

Lisa Hatfield:

Okay. Thank you. And is there any hope of any of these newer therapies being of limited duration, or are all of them continuous therapy? Because I know as a patient myself, that quality of life is impacted by knowing that I’ll be on therapy forever, some kind of therapy forever. Any hope for that? 

Dr. Andrew Kuykendall:

Yeah, so certainly with some agents there is hope for that. So some of the agents I referenced, ruxolitinib, rusfertide, these are agents that probably are, are going to be continued therapy. We always call it indefinite, right? As long as we think that the benefits outweigh the risks, we continue that. If we stop those, typically the reasons we’re using them, those come back quite quickly.

But I would say it’s not necessarily the case with agents like interferon. So interferon is an agent that’s less associated with symptomatic improvement, although we do see it in a subset of patients, it’s more associated with the ability to potentially modify the underlying disease. And so what we’ve seen with interferon is that we can measure patients’ JAK2 allele burdens, the number of cells that have the JAK2 mutation that drives the disease.

And in patients that are on interferon for 2, 3, 4 years, we see the number of cells that have the JAK2 mutation go down over time quite consistently. And even in the case that in some patients it goes less than 10 percent or to a level, we really can’t pick it up with our standard testing. And my experience with that is we can actually stop interferon in some of these patients for a pretty extended period of time.

So we have patients where we stopped for one or two years with blood counts that remain quite well-controlled, patients feeling well. In time, we might have to restart it as things start to to pop up, but I think that we are starting to get to these kind of at least treatment reductions, dose reductions where we can spread things out, but also kind of brief treatment interruptions where we get this kind of treatment-free period that certainly can be attractive to some patients.

Lisa Hatfield:

Okay. Thank you. And I’ll just do a quick shoutout to physicians like yourself who deal primarily with MPNs or work a lot with MPNs, that if a patient is watching this and doesn’t have somebody who really specializes in MPNs, everything you’re talking about, clinical trials, it might be helpful even to just get a consult or what I call an expert or second opinion on how to manage your MPNs. So anyway, thought I’d throw that out there. Thank you.

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Can Monitoring Albumin Levels Help Track Myelofibrosis Treatment Success?

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How might albumin levels help guide myeloproliferative neoplasm (MPN) care? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses research on albumin monitoring and treatment for myelofibrosis and polycythemia vera, treatment response, spleen size, and proactive patient advice. 

[ACT]IVATION TIP

“...monitor your lab work. Use everything at your fingertips to kind of get a sense for how something’s working. So whether it’s albumin level or spleen size or symptoms…really take everything into account to know if the treatment’s working for you.”

See More From [ACT]IVATED MPNs

Related Resources:

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

 Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

 How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

How Can Specialists and Support Networks Improve Myeloproliferative Neoplasm Care?

Transcript:

Lisa Hatfield:

Dr. Kuykendall, your research shows that changes in serum or blood albumin levels are linked to better survival in myelofibrosis patients treated with ruxolitinib (Jakafi). How could this information help doctors monitor patient progress and adjust treatment plans over time? 

Dr. Andrew Kuykendall:

Yeah, so this is a bit of a passion project for me that I spent a few years looking at. Want to give a little bit of a backstory. Ruxolitinib is a JAK inhibitor that is approved for myelofibrosis as well as polycythemia vera. And what it’s able to do, the way to think about,  it’s like a disease-specific anti-inflammatory. And so the disease itself kind of ramps up these inflammatory pathways, ruxolitinib blocks those inflammatory pathways, and it improves patients’ symptoms.

It’s also been able to show kind of reduction in splenomegaly or enlarged spleen for patients, but it doesn’t really get rid of disease, right? So we’d like it to be better than it is. We’d like it to get rid of the bone marrow disease and get so-called complete response or complete remission. But we don’t see that.

However, we know that it helps a lot of patients. And I think that the challenge is knowing when it’s helping someone and when to continue it versus when should you consider alternative options? And we’ve really struggled with kind of coming up with an objective definition of how to kind of define treatment success or failure with this. And so the best data we have supports patients that have a spleen response tend to have better survival than those patients that don’t have a spleen response.

However, we’re not routinely imaging patients’ spleens in the clinic, and many patients it’s difficult to monitor their spleen kind of growth or reduction in size on exam. And so this not isn’t always the most feasible way to monitor kind of response to therapy. And symptoms…they can be variable.

Certainly if patients are feeling better, that’s a great thing, but a lot of things factor into symptoms. And so kind of on a week to week, month to month, visit to visit basis, that may be challenging. And so I was very interested in looking at albumin, which I think kind of factors into a lot of things regarding health. So patients that are more nutritionally optimized have better albumin levels, patients that have less inflammation just in general have better albumin levels and patients that are eating well have better albumin levels.

And actually I kind of harkened back to a trial that was published on ruxolitinib-treated patients that showed that patients who got ruxolitinib, their albumin levels rose over time. And that was intriguing to me, which was the thought was, okay, well if that happens, if this is something specific to ruxolitinib, does this… Is this something that that can actually define those patients that do well? 

Does this represent someone who’s able to eat better and be more nutritionally optimized? Does this represent someone who’s getting a really good anti-inflammatory benefit from ruxolitinib? And so we looked at our patients and actually combined our data sets with an Italian data set as well, and showed that those patients on ruxolitinib whose albumin either stayed the same or improved, actually derived a survival benefit compared to those whose albumin levels stayed the…or whose albumin levels decreased over time.

And that was unique to ruxolitinib when we looked at patients who weren’t treated with ruxolitinib, who had myelofibrosis, we didn’t see the same pattern. And so the reason I think this is interesting and potentially clinically relevant, is that we’re always looking at albumin levels. The albumin is involved in kind of the complete metabolic panel or the CMP that we routinely are getting on patients.

And so this is something that’s very available to physicians as they’re watching someone on ruxolitinib. And so they can look and say, oh, look from when they started till now that albumin level has gone up by a certain amount. I think this is someone who actually I do feel comfortable that they’re doing well. And alternatively, maybe it’s someone who’s on ruxolitinib whose albumin level continues to decline. And you may say, think, you may think, hey, this is maybe symbolic of a treatment that may not be doing enough right now.

Lisa Hatfield:

Okay. Thank you. And do you have an [ACT]IVATION tip for that question? 

Dr. Andrew Kuykendall:

I think my [ACT]IVATION tip for this one is monitor your lab work. Use everything at your fingertips to kind of get a sense for how something’s working. So whether it’s albumin level or spleen size or symptoms. I think the [ACT]IVATION tip here is really take everything into account to know if the treatment’s working for you.

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Holistic Health Strategies for MPN Patients: Integrating Nutrition, Exercise, Mental Health, and Preventive Care

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Myeloproliferative neoplasm (MPN) expert Dr. Abdulraheem Yacoub discusses ways for MPN patients to make efforts toward optimal MPN, overall health and patient well-being, and proactive patient advice. 

[ACT]IVATION TIP

“…as they are seeking the best care for their MPNs, they should also seek the best care for their global health like in nutrition, exercise, psychological health, cardiovascular risk reduction, primary cancer screening, and prevention, all the preventative healthcare vaccination. So all the global health interventions that improve your health are absolutely necessary for patients with MPNs.”

See More From [ACT]IVATED MPN

Related Resources:

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

 Exploring New Frontiers: Innovative Drug Combinations and Clinical Trials in Myelofibrosis Care

Exploring New Frontiers: Innovative Drug Combinations and Clinical Trials in Myelofibrosis Care

 Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Transcript:

Lisa Hatfield:

Can you discuss any specific interventions or educational tools that have proven effective in improving symptom management and disease progression awareness for patients facing PV, myelofibrosis, or ET?

Dr. Abdulraheem Yacoub:

Patients who live with MPNs are in this for the long run, and this is a chronic health challenge they would have to endure over the rest of their lives. And having access to tools that improve their health in general is instrumental. So as we advocate always for good nutrition in any other disease all the concepts that apply to healthy living apply here very vividly. So healthy nutrition is important. We like to involve a dietician early on in our patients, although there is no specific diet that is uniquely specific for MPN, but there are certain dietary interventions that are globally of benefit to patients to be healthier. We also advocate for mental health and psychological health, and we involve our oncology psychologists to be partners with us on patients’ care and to tackle the challenges that they have to cope with as they live with a chronic cancer.

We also endorse exercise as a method of improving functionality, improving strength, improving emotional well-being, and also as a tool to battle fatigue and musculoskeletal pains. So really many of the concepts that stand correct to everybody with any chronic disease stand correct here, but the impact in MPN is a lot more profound, because those patients will live with the diseases for a long time. And all the tools that you have to improve your global health will also improve your cancer health. We’re also very strong advocates of primary prevention. So patients with MPN are at an adverse cardiovascular risk and interventions that improve cardiovascular health such as exercise, maybe seeing a cardiologist management of cardiovascular risk factors like hypertension, diabetes, hyperlipidemia can also improve the patient’s risk and reduce their MPN risks by lowering their other cardiovascular risks.

We also advocate for primary cancer prevention and screening. So patients with MPN should be also undergoing more meticulous cancer screening and prevention in order to be able to manage their…in the case of second primary malignancies to be able to address that a lot earlier in the course of those diseases and improve the patient’s odds of living a longer and healthier life. So really my [ACT]IVATION tip for patients is that as they are seeking the best care for their MPNs, they should also seek the best care for their global health like in nutrition, exercise, psychological health, cardiovascular risk reduction, primary cancer screening, and prevention, all the preventative healthcare vaccination. So all the global health interventions that improve your health are absolutely necessary for patients with MPNs.

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Navigating Disease Progression in MPNs: Strategies for Patient and Care Partner Awareness and Monitoring

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Myeloproliferative neoplasm (MPN) expert Dr. Abdulraheem Yacoub explains MPN disease progression, the difficulties with assessing MPN progression, factors that play into determining progression, and proactive patient advice for when MPN re-evaluation might be needed.

[ACT]IVATION TIP

“…be able to be aware of the baseline and any change of baseline and when do you draw the line where you actually need to re-stage or re-evaluated the disease all together and decide if the patients have closed the line or have transformed or progressed that they need different care.”

See More From [ACT]IVATED MPN

Related Resources:

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

 Exploring New Frontiers: Innovative Drug Combinations and Clinical Trials in Myelofibrosis Care

Exploring New Frontiers: Innovative Drug Combinations and Clinical Trials in Myelofibrosis Care

 Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

Transcript:

Lisa Hatfield:

Dr. Yacoub, how do you and your colleagues enhance patient and caregiver understanding of disease progression in all of the main MPNs, polycythemia vera, myelofibrosis, and essential thrombocythemia, and what strategies can be implemented to monitor and respond to changes in the disease?

Dr. Abdulraheem Yacoub:

The concept of disease progression is an evolving field and even among experts is still something we debate a lot on how to better communicate that and how to better define that. So it is a challenge even for the most skilled physicians who manage patients with MPNs. However, we all understand what progression is or, and we all understand when things are going great. It’s very much obvious that patients are doing well. And when patients are not doing well there often it’s because they’re progressing.

So we have a vague understanding of the concept of what is going well, what is not going well, but to actually be able to be granular and describe what exactly that means. There’s a lot of uncertainty and vagueness in the field. But my two cents on this is that patients should be aware of what is their normal and was, is their usual abnormal symptoms, their usual abnormal findings in the blood and the trends in how their blood, and their symptoms are evolving over time.

And when there is a sudden change in an adverse or unfavorable way in the symptoms or the blood numbers, that this is definitely a trigger to evaluate for progression. I think being self-aware and being educated about what to expect with your disease allows you to be more capable of detecting when disease is progressing. We also try to explain to patients what the range of progression could sound like. It could be a change in symptoms, could be a change in labs, a change in physical exam, a change in how the bone marrow biopsy looks like, acquisition of new DNA errors and mutations.

So there are many different forms of progression. But as long as patients understand the science, as long as we can communicate to patients what is the usual path of normal or expected outcome of the disease and what’s not expected and what’s not normal and what’s above normal, and the patients and their physicians can pick that up as it happens, that’d be the best way to the best [ACT]IVATION tip for those patients and providers is to be able to be aware of the baseline and any change of baseline and when do you draw the line where you actually need to re-stage or re-evaluate the disease all together and decide if the patients have crossed the line or have transformed or progressed that they need different care.

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Empowering Patients: Enhancing Shared Decision-Making in Myeloproliferative Neoplasm Care

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Myeloproliferative neoplasm (MPN) expert Dr. Abdulraheem Yacoub discusses how he approaches shared decision-making with patients, benefits of shared decision-making, and how to be proactive in elevating your own care.

[ACT]IVATION TIP

“…you have to understand that your cancer is a disease you’re going to partner with for the rest of your life. And the more skilled and knowledgeable you are, the more you can get the best care you deserve and advocate for yourself and be able to communicate your challenges with your doctors and be a participating partner in your own care.”

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Related Resources:

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

Prioritizing Quality of Life: Addressing Symptom Management Challenges in MPNs

 Holistic Health Strategies for MPN Patients

Holistic Health Strategies for MPN Patients: Integrating Nutrition, Exercise, Mental Health, and Preventive Care

 Navigating Disease Progression in MPNs: Strategies for Patient and Care partner Awareness and Monitoring

Navigating Disease Progression in MPNs: Strategies for Patient and Care partner Awareness and Monitoring

Transcript:

Lisa Hatfield:

Dr. Yacoub, how can patients engage in shared decision-making with their healthcare providers to determine the most appropriate treatment approach for their myeloproliferative neoplasm? Whichever one that may be.

Dr. Abdulraheem Yacoub: 

Thank you. So I perceive my relationship with my patients as a partnership. I try to teach them, but I also learn a lot from them. Some patients keep up with newsletters and FDA approvals and press releases as much as, or better than any of the physicians that we work with. So, I think the more informed the patient is, the more able they are to contribute to their own well-being and to the improvement in healthcare. Many healthcare projects in the U.S. are led by patients and patient advocates. So the more involved patients are, the more aware they are of the moving parts in the field, the more they can contribute to their own improvement and their own health.

So my advice to patients is to be as involved as they can. And these are chronic cancers they will live with for the rest of their lives. So my [ACT]IVATION tip for these patients is that you have to understand that your cancer is a disease you’re going to partner with for the rest of your life. And the more skilled and knowledgeable you are, the more you can get the best care you deserve and advocate for yourself and be able to communicate your challenges with your doctors and be a participating partner in your own care.

Lisa Hatfield:

So when it comes to shared decision-making, is it very common for you to work with the patient and their local general oncologist for shared decision-making, or do you typically have a patient come talk with you, and then the patient takes the information back that they’ve learned from you to their local oncologist?

Dr. Abdulraheem Yacoub:

There are many phases and many methods of how we can collaborate with patients and their caregivers and their local providers and so forth. And this carries different forms. We see this a lot recently with the FDA approval of interferons and physicians who are in practice have not been trained to use that, and they all are interested in applying the new technology and using the new medicines in their patients. And they seek us. They actually send patients for us to co-manage so that they can learn from the process. So they’re very involved and they’re very curious and they want to learn the new medicines and how to use them and how to apply the new knowledge and how to interpret molecular results and so forth.

Everybody has a role to play. Community physicians who treat patients have a key role at delivering care to patients, patients also have a role at learning this. And our job is to teach patients and their doctors how to raise their levels to be able to to speak the same language, to be able to understand the same knowledge and to be able to contribute and make informed decisions. The more informed the patient, the more they can contribute and the more they can be active partners in the healthcare.

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