Tag Archive for: MF
Thrive | What You Should Know About MPN Symptoms & Treatment Side Effects
Thrive | What You Should Know About MPN Symptoms & Treatment Side Effects from Patient Empowerment Network on Vimeo.
How are MPN symptoms and treatment side effect managed? In this animated explainer video, an MPN specialist and myelofibrosis patient discuss the importance of clear communication with your healthcare team, the process for assessing common issues, and advice for advocating for yourself.
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How Molecular Markers Affect MPN Treatment | Advances in Research |
Transcript:
Brian:
Hi, I’m Brian. Nice to meet you! I’ve been living with a condition called myelofibrosis for many years. While there have certainly been ups and downs, I’ve been able to navigate care for my condition and to live a full life.
So how have I been able to do that? First and foremost, I have a great relationship with my care team, whom I communicate with regularly. Meet, Dr. Liu – my doctor.
Dr. Liu:
Hi! I’m Dr. Liu, and I’m a hematologist and a specialist in myeloproliferative neoplasms or MPNs. The three types of MPNs are essential thrombocythemia, or ET, polycythemia vera or PV, and myelofibrosis, or MF. This group of blood cancers is characterized by the bone marrow overproducing a certain type of cell.
Maintaining a good relationship with your healthcare team, coupled with finding a treatment approach that works for you, can help you live a full life and to thrive with an MPN.
Brian:
Exactly, Dr. Liu. Over the years, I’ve experienced periodic issues with my condition. I’ve had symptoms and treatment side effects that have been bothersome and interfered with my life. But, communication with my team has been essential to feeling well.
Dr. Liu:
That’s right, Brian. When symptoms or treatment side effects are bothering you, it’s important to let your healthcare provide know how you are feeling.
Brian:
For example, recently I felt tired beyond general sleepiness. And when I shared this with Dr. Liu, we discussed potential causes of the fatigue, and we talked in-depth about my options to manage it, including changing therapy and some simple changes to my diet and lifestyle.1 Over time, my energy levels improved, but having the open dialogue with Dr. Liu was essential to tackling this symptom head-on.
Dr. Liu:
That’s a great example. When I first hear from a patient that they are having an issue, we go through several steps to find a solution.2
We start by ensuring that the disease is well-controlled, so we check blood counts. Next, we try to determine if it is a symptom of the MPN or a side effect of the treatment. Once we’ve done those steps, we come up with potential solutions which may include, but are not limited to:
- A dose reduction or a treatment holiday.
- Changing therapy to find something that is more well-tolerated.
Other considerations are dependent upon the specific symptoms and side effects but may include:
- Supportive care options, including diet and exercise.
- A visit to your primary care doctor to see if there is something else going on physically.
Brian:
That’s good to know, Dr. Liu. Something you brought up with me, which I feel is important to mention, is mental health. Often, emotional symptoms can take a toll on the body, causing fatigue or other issues.
Dr. Liu:
Great point, Brian. Seeking care for your mental health is crucial, particularly if you are in active treatment.
Brian:
Of course, we know that the symptoms and treatment side effects for MPNs can vary widely, so what advice do you have for patients who may be afraid to speak up?
Dr. Liu:
The most important thing to remember is that we have options to help you, no matter what you are going through. It’s your body and if you don’t let your provider know what you’re going through, they can’t help you.
Brian:
So true. It’s also a good idea to bring a care partner along to appointments, sometimes a spouse or friend can you help you communicate what’s going on.
Dr. Liu:
That’s great advice, Brian. Bringing someone along to take notes is a great idea. Also, be sure to write down any questions or concerns you have in advance to make the most of your appointment.
Brian:
OK, Dr. Liu, let’s recap your advice for MPN symptom management:
Dr. Liu:
Good idea! First, remember that everyone’s MPN is different, so managing symptoms and side effects can be tricky. Communicating with your healthcare team is critical to your overall care – report any and all concerns to your team immediately.
And, do your part. Make sure you see your primary care physician regularly and do your best to maintain a healthy lifestyle.
Brian
And, most importantly, remember you are at the center of your care. Never hesitate to share your opinion and to advocate for yourself.
To learn more, visit powerfulpatients.org/MPN to access a library of tools. Thanks for joining us!
How Molecular Markers Affect MPN Treatment | Advances in Research
How Molecular Markers Affect MPN Treatment | Advances in Research from Patient Empowerment Network on Vimeo.
Are there new molecular markers being discovered that could affect myeloproliferative neoplasm (MPN) care? Dr. Lucia Masarova explains common MPN driver mutations and what researchers are learning about recently discovered molecular markers, such as ASXL1.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
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Expert Perspective | Disease Modification in Polycythemia Vera |
Transcript:
Katherine Banwell:
Dr. Masarova, molecular testing is important for people diagnosed with MPNs and may help provide insight into effective treatment approaches. What are some new areas of research related to molecular markers?
Dr. Lucia Masarova:
Molecular markers are very relevant in our designs or thinking about myeloproliferative neoplasms. Not only treatments, but also the disease qualification or prognostication wherever since the discovery of the so-called driver mutations, which are the mutations responsible for the overproduction of the blood counts and disease pathogenesis.
Among them we have the most common, JAK2 mutation, then also calreticulin, MPL, or in some instances we don’t even understand and call it triple-negative.
There we have learned, over the years, that the amount of the expression, or allele burden, does correlate with the disease behavior outcome. And then our ability to reverse that. So, a chief decrease of the burden is also relevant to the outcome of the patients. So, developing therapies or even putting these as an endpoint for clinical trials is important for our decision-making and moving towards eradication of the disease.
Then there are additional molecular changes, which include non-drivers, which are additional mutations that we have learned and even implemented in the latest prognostic models, some of them are very unfavorable, such as ASXL1, Ezh2, IDH mutations, certain splicing factors.
And those play additional roles, a lot of it we still do not understand, in how the disease is going to ultimately behave. What is their interplay, and how we can interfere with that?
So, learning about the impact of these mutations and the drivers and the other effects that cause the disease evolution will probably become the landmark of this decade and in facing myeloproliferative neoplasms.
And I’m hoping we will develop medications, or we will be able to focus our efforts and our decision-making based on molecular definition, as it’s currently very broadly seen across all cancers. We call it precision medicine where we really define, “How does this look like,” not how we box it in based on morphology. What is it driving? What is it not responding? And what can we do to improve that?
So, I totally see here a big potent and powerful tool to allow us to make the most individualized and customized decisions for our patients to offer them the best outcomes.
Expert Perspective | Disease Modification in Polycythemia Vera
Expert Perspective | Disease Modification in Polycythemia Vera from Patient Empowerment Network on Vimeo.
Is it possible to change the course of disease in polycythemia vera patients? MPN specialist and researcher Dr. Lucia Masarova shares an overview of the research in disease modification, discussing her work as the coauthor in an article entitled Moving Towards Disease Modification in Polycythemia Vera, recently published in the journal Blood.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
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How Molecular Markers Affect MPN Treatment | Advances in Research |
Transcript:
Katherine Banwell:
Dr. Masarova, you are a coauthor in an article entitled Moving Towards Disease Modification in Polycythemia Vera, which was recently published in the journal Blood. Can you share some of the highlights of the article and what it means for PV patients?
Dr. Lucia Masarova:
Disease modification in polycythemia vera. I’m so excited finally being talking about this because we’ve been really, really, really so hungry for this term, although we still don’t know what it means.
So, we group together with lots of experts in the myeloproliferative neoplasm field and try to brainstorm and put together, “What does it actually mean?” And to me, and to all of us, it was to offer our patients the normal or not-normal lifespan without the consequences of the disease that they face. Because we historically divided polycythemia vera into high-risk or low-risk disease based on the age or previous history of thrombosis or clotting complications.
However, there is a huge area of patients that wouldn’t have either, and still suffer tremendously a bad quality of life, and ultimately also face the disease progression to myelofibrosis, which is the most actual complication of long-term polycythemia vera duration.
So, the concept of disease modification would be to actually prevent the complications to even occur. To allow our patient to live free of having the fear of living with a thrombosis or clotting complication or ultimately progress into myelofibrosis. We have to learn how to get there. What are the relevant endpoints of tools for us to utilize to really understand? We have learned a lot from seeing what we call molecular remissions, or control of the JAK2 mutation with certain medications, for example, interferons or latest ruxolitinib (Jakafi), the JAK inhibition, where the decrease of the allele burden, which represents the disease, is correlated with better outcome.
So, that is something that we have to be learning down the road with a longer follow-up. But that basically triggered us to focus on what can we do better? How do we prevent this from even happening rather than only controlling the historically main points of the disease which are presented by the blood counts symptoms and display? And where we are actually failing quite a lot of patients because despite them having a control count, they still don’t have a happy life, and lots of them suffer and complain.
So, this is something to be learned, and this is opening the disease modification not only for polycythemia vera, but also for all patients with myeloproliferative neoplasms, which have a little bit of a different feeling in the whole myeloid malignancies field. Because it is a very long disease, and it could evolve and change, and only now we starting to understand what does actually happen there. Why some people could live for 30 years, and never face any consequences, and the others would progress very fast?
So, disease modification would normally allow us to develop and learn more tools and better biomarkers, but also focus on drugs that are really needed to help with these long-term outcomes of our patients.
Myeloproliferative Neoplasm News and Research Updates
Myeloproliferative Neoplasm News and Research Updates from Patient Empowerment Network on Vimeo.
Dr. Lucia Masarova, a myeloproliferative neoplasm (MPN) specialist and researcher, discusses the latest updates from a recent MPN Congress. Some of the highlights include new learnings in hematopoiesis, JAK inhibitor comparisons, interferon therapy, and the potential for combination treatments in the future.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
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Advances in Research | Emerging MPN Therapies on the Horizon |
Transcript:
Katherine Banwell:
Dr. Masarova, you were in New York recently for the MPN congress. Can you share some highlights from that meeting?
Dr. Lucia Masarova:
Yeah. Sure. That was a very interesting and very loaded conference full of experts and great data. I really liked the overall excellent update of all the therapies that currently exist in the MPN space, including polycythemia vera, essential thrombocythemia, myelofibrosis. So, really a broad breadth of JAK inhibitors, their current sequencing, combinations, interferon update. I very much like also the focus of the novel therapies, which actually talked about, for example, the development of the antibody against mutant calreticulin, PIM inhibitors, and a couple others. They are very promising in the space.
There were also very, very relevant clinical data. I think I really, really enjoyed the radiation in hematopoiesis topic. It spurred lots of discussions in the room. And also, fantastic talks about clonal hematopoiesis and its role in patients with myeloproliferative neoplasms and cancers.
Overall, very great data on artificial intelligence because that will be a very needed tool, but also a very worrisome tool, at this point, until you learn how to use it to help our patients. But that showed a very promising effect and ability for us to, for example, predict thrombosis risk in polycythemia vera patients or to distinguish patients with ET versus prefibrotic myelofibrosis, which is still subject to lots of basically subjective analysis from hematopathologist.
And also, the poster section was quite striking and really excellent. You could walk around and see so many interesting data. The match and direct comparisons of JAK inhibitors, particularly the latest approved, momelotinib (Ojjaara), as it compared to safety data which do currently exist in fedratinib (Inrebic) or pacritinib (Vonjo).
Katherine Banwell:
When it comes to MPN research and emerging treatment options, what are you excited about specifically?
Dr. Lucia Masarova:
There is a lot of excitement in the field currently, and it really depends how we put these patients in, as I would call, boxes, but I don’t like the term. We have these less aggressive diseases, such as polycythemia vera and essential thrombocythemia.
Where I’m really excited about the role of interferon, with the approval of ropeginterferon (Besremi), or ropeginterferon in United States as well as Europe, we have opened a door for learning how can we do better.
It is approved for polycythemia vera patients. There are currently clinical trials running in essential thrombocythemia patients, within patients with prefibrotic myelofibrosis. That’s an agent that has an ability to go after the disease clone and hopefully, hopefully eradicate or prevent it. Especially, especially exciting in the terms of preventing it for progression.
Then iron metabolize modifier, hepcidin mimetics, other agents impacting this. It’s very important we finally learn how iron plays the role in these patients and how we can actually improve. Very important area in helping patients requiring phlebotomies and hopefully, hopefully altering the whole disease outcome in the long-term.
For myelofibrosis we live in an era of JAK inhibitors. We are so excited to have four currently approved and we’re looking forward to the combinations where we have now safer and less cytopenic agents that have a role in anemia or thrombocytopenia and hopefully will be able to be combined with others.
So, we could even move the field more into other hematologic malignancies, where in myeloma we use five, six, seven, eight drugs. For myelofibrosis, we still have one. So, I think we have still a lot to do.
And then non-JAK inhibitor combination. Non-JAK inhibitor, a compounds or mechanism of action really tailored to the disease pathogenesis. Calreticulin, excellent topic, which I’m saying maybe in couple years we will be really classifying myeloproliferative neoplasm calreticulin-mutated, but also JAK2-mutated, and we will not be calling them one because hopefully we will find a tool to eradicate calreticulin and to really be able to offer ultimate – what I call ultimate cure.
So, that’ll be something really exciting to come and all of these investigators in MPN fields are so eager to see what – whether the preclinical data we have seen are going to stand in our patients. And that would be really fantastic.
Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask
Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask from Patient Empowerment Network on Vimeo.
When considering therapy for myelofibrosis, where do you start? Dr. Lucia Masarova shares advice and key questions to ask your provider when making myelofibrosis treatment decisions.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
See More from Evolve Myelofibrosis
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Is Stem Cell Transplant the Only Curative Option for Myelofibrosis? |
Myeloproliferative Neoplasm News and Research Updates |
Transcript:
Katherine Banwell:
When considering treatment options, what key questions should patients ask about their proposed treatment plan?
Dr. Lucia Masarova:
What’s the goal of my therapy? That is one of the most important things to know. Patients don’t even know how long they have to be on the medicines. What to do and how does it look when the medicine is still working? What do I need to be looking for in this medicine? And then what are we going to do if it fails? And what does it actually mean when it fails? What is the schedule? How burdensome the treatment is? How often do I have to come?
How often and what do I have to pay? Because the financial burden we have to really, really face the truth. It is very, very, very significant and somebody living with this disease predicates. It’s something we cannot take lightly, and we really have to combine our efforts and help with that. There are fantastic patient support organizations, but is not well-known, and is still in the rare – in rarer field. So, there’s more effort that we do.
When do I need more help? Where to be referred to more experts? What is the role of stem cell transplantations, if ever? So, those are really the key things.
Where do I find reliable resources to learn about my treatment, to learn about the disease? How do I connect with people from the same community? It is a disease with a lower age in a lot of circumstances and really facing this disease in the 30s or 40s or 50s is a really challenging thing. Although we have more and more medications currently, we really do have now to start thinking about their durability, about the safety for long-term, about their assessments for not performing, and where do we place the ultimate cure for stem cell transplants?
And how do we make it actually happen in more and more eligible patients? Because we have to face the truth. It is still not utilized to where it belongs. Patients are not being referred.
Patients are not being transplanted. And they may change with novel therapies. But we have to really consider all of our tools to offer the longest life span and to prevent all the disease trouble that comes with living with MPNs.
Katherine Banwell:
When it comes to clinical trials, where do they fit in in choosing treatment?
Dr. Lucia Masarova:
For me, it’s number one., and always number one.
That’s just the academic centers which are dedicated and focused on developing better and novel and up front and just tailored and customized drugs. But I know that the life is out there and it’s a little bit more challenging for everybody to deal with such a rare disease.
I would definitely say any patient that does not respond to current therapy in terms of uncontrolled symptoms or spleen, or other concerns should be referred and evaluated for participation in clinical trials. It is the only way we could understand what is driving that this is not responding and how could we help the best?
For patients with myelofibrosis, which is the most aggressive myeloproliferative neoplasm, I would definitely put it in. If they are not doing well on number one, JAK inhibitor, whatever is being used, they should be highly encouraged to be referred to centers and evaluated for clinical trials.
We have been developing as others and own strategies to potentiate the benefit and efficacy of the current treatments, as well as agents in what we call salvage or refractory setting.
However, I cannot emphasize enough to really focus on the first track that providers choose for their patients and utilize it to the best ability to avoid frequent or quick switching. Because in a salvage or refractory setting we cannot offer the same benefit we could offer upfront. We are pushing the disease, maybe being less responsive, maybe more refractory, if we don’t handle the medication we have currently on the table to the best ability.
Those are excellent medications, fantastic drugs, but there are shortcomings in each and every one of them. And we could do better to really start thinking about what has happened with the medication, why is it failing the patient, and what else could we do? And that’s only possible in the clinical trial setting, especially in such a rare disease as myeloproliferative neoplasms are.
Katherine Banwell:
Why is it important for patients to feel like they have a voice in their treatment options?
Dr. Lucia Masarova:
Because it’s about the patients. I would say, as I always say to my patients, “Nobody’s a better advocate for you than you.” I really, really, really like working with patients. They are educated. They understand where to find resources. They’re not afraid to ask. That challenges all of my team and everybody to really be engaged. They know when to notify me. Not to be quiet when they need something. And really raise their voice when something doesn’t work.
Patients know their bodies more than anybody can. And no data, no boxes, no books can ever tell me how it actually is. It’s not by chance we have two ears to listen and one mouth to talk.
So, we have to really listen what the patient has to say and take all the abilities, the resources, the knowledge, the capabilities to really make the best thing for the patients, because it is ultimately and only about that.
Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?
Is Stem Cell Transplant the Only Curative Option for Myelofibrosis? from Patient Empowerment Network on Vimeo.
Is there a cure for myelofibrosis? Dr. Lucia Masarova explains the role of stem cell transplant for the treatment of myelofibrosis and reviews additional therapies for patients who do not qualify for the procedure, such as JAK inhibitor therapy.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
See More from Evolve Myelofibrosis
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Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask |
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Transcript:
Katherine Banwell:
Dr. Masarova, stem cell transplant is sometimes recommended for people with myelofibrosis. Is this still the closest option to cure for those patients?
Dr. Lucia Masarova:
I would say so, as much as we don’t like it. We would like to develop novel conservative, less aggressive, that we call procedures or drugs. Stem cell transplants still represent a long-term cure for patients that are eligible.
Katherine Banwell:
What about for patients who don’t qualify for stem cell transplant? What are effective long-term treatments for them?
Dr. Lucia Masarova:
That’s a very, very important question and topic. The key point here is the long-term because long-term is a little difficult term in conservative management of myeloproliferative neoplasm, particularly when it comes to myelofibrosis.
With the development of JAK inhibitors, the longest experiences we have with the first one called ruxolitinib or Jakafi, we have seen prolonged outcomes in survival so patients could live longer than expected before.
However, it’s not forever. So, that’s why we are trying to develop novel strategies where I see a lot of roles of combinations of JAK inhibitors and other correlative compounds, such as bromodomains inhibitors or hypomethylating agents or others that would affect the pathways that we are missing currently to cover with the JAK inhibition. And that ultimately leads to medication failures and patients being refractory and then having a shortened lifespan.
So, I’m hoping we will develop something for long-term. Particularly promising a very, very interesting concept is with the calreticulin where we are developing monoclonal antibodies or vaccines because we have seen and discovered calreticulin driver to be a targetable thing that causes immunogenicity.
But I do really hope that we will move forward with these discoveries and the JAK mutate or other drivers causing myeloproliferative neoplasms to offer long-term management.
Myelofibrosis Therapies in Clinical Trials | BET Inhibitors
Myelofibrosis Therapies in Clinical Trials | BET Inhibitors from Patient Empowerment Network on Vimeo.
What are BET inhibitors? Dr. Lucia Masarova, an MPN specialist and researcher, explains what BET inhibitors are and discusses the role these therapies may play in the treatment of myelofibrosis.
Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.
See More from Evolve Myelofibrosis
Related Resources:
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Is Stem Cell Transplant the Only Curative Option for Myelofibrosis? |
Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask |
How Molecular Markers Affect MPN Treatment | Advances in Research |
Transcript:
Katherine Banwell:
We’re starting to hear more about BET inhibitors. Could you explain what they are and how they work to treat myelofibrosis?
Dr. Lucia Masarova:
BET inhibitors are abbreviations for bromodomain inhibition, which is a very relevant regulator of transcription factors that play a significant role for making the blood cells.
So, just differentiation of red cells or platelets, as well as very significant role in cytokines regulation. We know that myelofibrosis is a disease that is defined by overactive JAK-STAT Pathway that ultimately leads to increased cytokines.
However, there are other pathways that play a significant role, and one of the very major ones is NF-kB, where the BET inhibitor come in play because they target it and help us to decrease the cytokine load as well as alter the differentiational block that happens in the red cells or megakaryocytes or platelets in these patients.
So, the combination of bromodomain inhibition, or even using it as a single agent on or after refractory I think is a very promising tool that excludes the only JAK inhibition that we’ve been developing for diseases and opens the door for combination strategies that we were so many years thinking through and trying to find out.
This is really the most promising compound or way of altering the disease background that we can see.
Expert Advice | Living and Thriving With an MPN
Expert Advice | Living and Thriving With an MPN from Patient Empowerment Network on Vimeo.
Is it possible to live well and thrive with a myeloproliferative neoplasm (MPN)? Dr. Naveen Pemmaraju, an MPN specialist and researcher, shares key advice for patients, stressing the importance of taking an active role in learning about their disease and communicating with their team to manage common symptoms and side effects.
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.
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Transcript:
Katherine Banwell:
In your experience, what does it mean to thrive with an MPN?
Dr. Pemmaraju:
Well, I really love that phrase so much because it’s meaningful to me.
You know, you’re talking about something that resonates with me and my patients, which is not just living with the MPN, but you’re talking about thriving with an MPN. That’s so resonant to us. I think really, I would go for three parts to that.
One is that it’s an acknowledgment or a complete understanding of the disease. So, not denial, the opposite of denial, whatever that is, Katherine. So, understanding as much as you can about the disease which is, I encourage people to Google, look up on the internet. I just, what I want you to do is couple that with talking about it in context with your provider. I think the worry that people have is you’re at your home midnight, you’re Googling stuff, it may or may not be right. So, anyway, so just do that, but then bring the information to the next visit. So, fully understanding and learning as much as you can in your own way. Number two is to be able to have a quality of life that is not just living with the disease, but actually being successful at your relationships, your work, whatever it is that brings you meaning and joy in life. And that sometimes has to do with the MPN paradigm, sometimes has to do with the other stuff we said.
But I think, doing that, not despite the fact that you have the MPN, but acknowledging it with that, right? And then I think the third aspect is, if you have some way or some platform to be able to express yourself with the MPN because it’s such a rare disease, we think maybe only four out of 100,000 people worldwide get these. A lot of patients, not for everybody, by the way, but a lot of patients are thriving on support groups.
It used to be you have to be in person, that’s very difficult to do with rare diseases. But now online, social media, a lot of different ways to get involved. Whether someone’s an introvert or an extrovert, whether someone wants to be private or public, all those things are hugely important, so it’s a personal decision. But for many, they want to get out there, and it’s not necessarily this scientific information exchange, although that’s good. But the support and encouragement and comradery of talking to other patients about what we’re talking about.
It is, in fact, a little bit more facile to do it with the more common diseases, breast cancer, all of these things. And it’s much more difficult, social media online has opened that up. So, to me, I think that’s a kind of mix that I’ve been seeing in my patients. And that leads to empowerment. It leads to taking control of the things that can be controlled, leaving the things that can’t be controlled to what needs to happen. And then an understanding and anticipation of things that may happen in the next few visits, in the next few years. I think that’s how people can thrive with these MPNs.
Katherine Banwell:
Dr. Pemmaraju, When it comes to living and thriving within an MPN, managing disease symptoms and treatment side effects is a big part of that. How can symptoms and side effects impact life with an MPN?
Dr. Pemmaraju:
Katherine, I’m glad you asked about that because I think before we get into the science and the pathobiology and all these complex things, it really starts with the patient. And as you and your team and others have really noted, the MPN for many of our patients, it is a chronic, often lifelong journey. And we really need to reemphasize in this modern era, the patient-centered experience and the caregiver experience.
And so I would emphasize a few things. One is that our MPNs are oftentimes so-called invisible diseases to other people. So, this phrase that just really is tough for us to hear for our patients and our loved ones, “Oh, you don’t look like you’re sick. You don’t look like you have cancer.” So, it emphasizes the internal part of the internal medicine, that’s one.
Number two, it reminds you that you cannot tell on the external what kind of a war, a cytokine war that is going on inside of a patient. And so even though the blood counts are normal, the spleen is okay, the treatment paradigm is going okay, we don’t know what’s really going on. So, that’s why our great friend and colleague Ruben Mesa invented and pioneered the MPN symptom burden to really nail down what’s going on.
And then third is our treatments, Katherine, our treatments, while overall halting or stopping or helping the MPN can then introduce a whole other round of toxicity, side effects, and so we need to manage that.
So, both the disease itself and the treatments, two separate entities, and that’s what we need to be monitoring in the clinic.
Advances in Research | Emerging MPN Therapies on the Horizon
Advances in Research | Emerging MPN Therapies on the Horizon from Patient Empowerment Network on Vimeo.
The pace of research in myeloproliferative neoplasms (MPNs) is advancing rapidly, but what do patients need to know? MPN specialist and researcher Dr. Naveen Pemmaraju shares an update on the latest research and his optimism for the future of MPN care.
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.
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Transcript:
Katherine Banwell:
Dr. Pemmaraju, as a researcher, what are new and emerging therapies on the horizon in MPN care?
Dr. Pemmaraju:
Well, Katherine, I’m glad you asked because I’m proud to tell you here, at the end of 2023, that we’ve now entered a new golden era of therapies for MPNs. Your group, and others, have led the way in advocating, but for so many years, honestly, we didn’t have many breakthroughs or new medicines. And now we literally have something we’re hearing about once a month. I think this golden era is divided into four buckets, Katherine, and that’s why I’m so excited for our patients and their caregivers. Number one is novel JAK inhibitors. So, beyond the approved ruxolitinib, fedratinib, and now pacritinib, we have a fourth one that’s under consideration, that’s called momelotinib. Hopefully, we’ll have that approved by the end of the year.
[Editor’s Note: Momelotinib (Ojjaara) was approved by the U.S. Food and Drug Administration (FDA) on Sept 15, 2023 for the treatment of intermediate- or high-risk myelofibrosis, in adults with anemia.]
And there are actually other drugs around the world. So, not just in the U.S. and North America that are being developed as a further JAK inhibitor. So, just like we’ve seen in CML with the TKIs for BCR-ABL after the imatinib (Gleevec) medicine, hopefully, we have seven to 10 choices for our patients.
Number two is the combinatorial approach of a JAK inhibitor plus something else. And that’s a field that I’m personally very involved in and helping to lead. The concept there is you take the known workhorse drug, the JAK inhibitor, use it as the backbone, and then add in the second agent. We started to do those studies in patients who were already starting to lose a response and we added in the second agent, those were called suboptimal studies. And then now we’re moving those drugs into the frontline setting in international global randomized studies. So, stay tuned, let’s see how those go.
But the concept is, can you take a new agent, whether it’s a BET inhibitor, a bromodomain inhibitor, a Bcl-xL inhibitor, PI3 Kinase, et cetera, and combine it with the JAK inhibitor? The third bucket that’s even more exciting to many people is that of novel agents standing alone by themselves. Now you’ve had either a JAK inhibitor or some other therapy for your myelofibrosis. That didn’t work for whatever reason. Now you’re looking for a completely new strategy.
An explosion of research, not just in the lab, which we’ve had for the last 10 years, but over the last three or four years, amazingly, even despite the COVID pandemic. I would say dozens, really dozens of trials that are what you would consider beyond or non-JAK inhibitor therapy. Some of them include telomerase inhibition, with the imetelstat agent, for example.
And so the concept here is, can you now hit the myelofibrosis in a completely different pathway? And the answer clearly is yes. And those results have been tested now in the lower stages, the earlier stages, Phase I and II. And you’re starting to see those drugs enter into the phase two and phase three. We eagerly await those results if there can be a viable beyond JAK inhibitor. And then finally, if that wasn’t exciting enough, there’s a fourth bucket, which is thinking about specifically the anemia myelofibrosis. We’ve never really historically done that. We’ve had older drugs, danazol (Danocrine), steroids, growth factor shots, blood transfusions.
But now here you see both pharmaceutical interest, as well as academic interest, in developing agents that either specifically target the anemia of MF or both, the MF and the anemia. And that could be a game changer for our patients in the next five years. So, Katherine, a wealth of exploding research that I’m personally very excited about that gives me and our field hope, momentum, and enthusiasm going into 2024.
Essential Thrombocythemia Watch & Wait | What Patients Should Know
Essential Thrombocythemia Watch & Wait | What Patients Should Know from Patient Empowerment Network on Vimeo.
What is watch and wait, and what does it mean for essential thrombocythemia (ET) patients? Dr. Naveen Pemmaraju defines this term, helps viewers to understand why it’s beneficial to wait before beginning treatment, and shares advice for managing the worry that can be associated with this time period.
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.
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Advances in Research | Emerging MPN Therapies on the Horizon |
Transcript:
Katherine Banwell:
Stephanie writes, “I have ET, and I’m not being treated. Do you have advice for the watch-and-wait period? I’m anxious about the disease changing and don’t know what I’m waiting for.” So, before you answer the question, Dr. Pemmaraju, would you define this term, watch and wait?
Dr. Pemmaraju:
I will. And to Stephanie and everyone out there, this is a great question. I will say half the folks I talk to actually call it watch and worry, okay. Some people call it watch and wait, and as Stephanie’s saying, some people call it watch and worry.
Yeah, the concept is threefold. One is that there are many cancers, many cancers, including blood cancers, that can be caught so early on that they don’t require treatment. A lot of patients with CLL, chronic lymphocytic leukemia, ET, as Stephanie mentioned, in the solid tumor. It’s very common to be diagnosed with a prostate cancer that’s low grade, early stage that can be observed. Number two is in ET, there is a science behind it.
What we found in our studies, and they can be updated over time and you’ll see those, the traditional is that if you’re below the age of 60 and/or you’ve had no blood clot, thrombotic event, that’s considered low risk. And the treatment can be observation, perhaps adding in a baby aspirin to prevent against blood clots if there’s no contraindication. Now what’s magic about that age 60, obviously as you know, it’s not magic. It’s more of a statistical, continuous variable algorithm that says around that time, the risk of blood clots goes up.
And so then you’d consider cytoreductive therapy at that point. Now there’s exceptions to that. Many of our young patients are on therapy, but there’s usually some reason for that. Some high-risk feature, wildly uncontrolled blood counts, for example, symptom burden, some other high-risk features. So, it’s a suggestion. It’s a guideline, not an absolute. And then the third part of it is, the what do you do in that time? And that’s the frustrating thing. And I think that’s what Stephanie’s getting to.
Again, that’s why I said the watch and worry versus watch and wait. Some of it is, how are you feeling outside of this? Some patients take it as a great news. Hey, you have this blood cancer, that’s not good news. But the good news is it’s probably not going to be active for a long time, we can, “just watch it.” But some people, as Stephanie is saying, take it the opposite way. What do you mean I got a blood cancer? I got something lurking in my body. You’re telling me it’s there, you know it’s there. And so what’s up with that? And the concept there is that some of these situations like low-risk ET, we found that if you treat too early, too aggressively, you can actually do harm.
So, that’s the key. These chemo drugs are not benign as you had me discuss earlier. They have toxicity, side effects, short-term, long-term. So, it’s a risk-benefit thing. If the risk far outweighs the benefit, as in the younger patient with no symptoms, no high-risk features, observation is okay. But at some point, when it turns, that’s the threshold.
So, really the key is, if we believe these are stem cell blood cancer disorders, we need to be thinking about and designing therapies with minimal to no toxicity. Something that actually modifies the disease early on and something that leads to long-term outcomes. And we don’t have that yet in ET. We’re working on that in PV and myelofibrosis. So, stay tuned for that.
And then finally, let me also add, this is an important point, not everybody gets it. This watch and wait versus watch and worry. So, I’m glad Stephanie brought that up because it’s not always good news, uniformly, when you tell someone, good news is you don’t have to do anything bad news, there’s something there.
Increased MPN Symptoms | What Does It Mean for Patients?
Increased MPN Symptoms | What Does It Mean for Patients? from Patient Empowerment Network on Vimeo.
What might an increase in myeloproliferative neoplasm (MPN) symptoms indicate? MPN specialist Dr. Naveen Pemmaraju discusses possible reasons for an increase in symptoms and shares advice for seeking care when experiencing common issues.
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.
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Thriving With an MPN | Advice for Setting Goals and Making Treatment Decisions |
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Understanding and Managing Common MPN Symptoms and Side Effects |
Transcript:
Katherine Banwell:
What might an increase in symptoms mean? Does it mean that the disease is progressing or that maybe it’s time to change therapies?
Dr. Pemmaraju:
Yeah, possibly. So, with all this objective evidence, there are different buckets of disease progression. And some of them are objective and obvious, rising spleen, increasing blasts, or leukemia cells in the peripheral blood. The start of transfusion dependency for either anemia or platelets that weren’t there before. Sometimes, there are obvious things that you can point to, but there are a couple of scenarios where it’s not as obvious. You just named one. One is increasing symptom burden profile. You see, sometimes you have to think about, is it the sequela of the treatment itself or is it disease progression?
I’ll give an example. If you start on an Interferon product and the dose is too high, you may be feeling not so great from the interferon. But maybe in that case, a simple dose reduction was the answer, because then you’re still getting the anti-disease activity, less side effects and all that. So, I’ll answer your question by saying possibly, but it can’t be the whole story. So, increasing symptoms is a harbinger, it’s a red flag.
In the clinic, it means pause. Workup, is this a subject of the treatment itself? Is it because the disease is progressing? Do we need to do a restaging and workup, whether that means a bone marrow biopsy, whatever that means? Or again, let’s put that other in there. What about the other comorbidities? Do you have class one heart failure, that’s now class three and you’re retaining fluid? And that’s why you’re short of breath and you actually need an echo and a cardiologist and an evaluation of your diuresis. So, I think that’s important, but the key is don’t blow it off, right? So, increasing symptom in MPN is telling you something isn’t right, and we need to check it out.
Katherine Banwell:
Right, and for the patient, tell your healthcare team about it.
Dr. Pemmaraju:
Communicate always. I think what we see is people are so proper and so compassionate and so kind and collegial, and that’s beautiful. But actually in the MPNs and all these rare blood cancers where so little is known and so little is obvious, communication is the key.
