Tag Archive for: MPN treatments

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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.

See More From Thrive MPNs

 

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Myeloproliferative Neoplasm News and Research Updates

Myeloproliferative Neoplasm News and Research Updates

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Expert Perspective | Disease Modification in Polycythemia Vera

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

 

Related Programs:

Are There Predictors That an MPN May Be Progressing

Are There Predictors That an MPN May Be Progressing?

Myeloproliferative Neoplasm News and Research Updates

Myeloproliferative Neoplasm News and Research Updates

How Molecular Markers Affect MPN Treatment | Advances in Research

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.

See More From Thrive MPNs

 

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Are There Predictors That an MPN May Be Progressing

Are There Predictors That an MPN May Be Progressing?

Myeloproliferative Neoplasm News and Research Updates

Myeloproliferative Neoplasm News and Research Updates

Advances in Research | Emerging MPN Therapies on the Horizon

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

Related Resources:

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

Thriving With an MPN: Advice for Setting Goals and Making Treatment Decisions

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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Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask

Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask

Myeloproliferative Neoplasm News and Research Updates

Myeloproliferative Neoplasm News and Research Updates

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:

Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask

Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask

How Molecular Markers Affect MPN Treatment | Advances in Research

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.  

Nurse Natasha Johnson: Why Is It Important for You to Empower MPN Patients?

Nurse Natasha Johnson: Why Is It Important for You to Empower MPN Patients? from Patient Empowerment Network on Vimeo.

What are some ways that myeloproliferative neoplasm (MPN) oncology nurses and other MPN care team members can aid in empowering patients? Oncology nurse practitioner Natasha Johnson from Moffitt Cancer Center how she helps patients and their loved ones become more involved in care and treatment decisions.

See More from Empowering Providers to Empower Patients (EPEP)

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Dr. Gabriela Hobbs Why Is It Important for You to Empower MPN Patients

Dr. Ana Maria Lopez Why Is It Important for You to Empower Patients

Dr. Ana Maria Lopez Why Is It Important for You to Empower Patients

Transcript:

Natasha Johnson:

So I’m an encourager, so I will encourage patients to be involved, get their loved ones involved, educate themself on the disease on the common symptoms, on treatments, and then ways to alleviate those symptoms. So I would encourage them too, to keep on living. Let’s do what we can to improve the symptom burden so you can keep on living. And I think this is important, because I believe that empowered patients are very well-informed, and they are the top key player in their care. They’ll share their goals of care, which makes them better prepared to work with the healthcare team to create an individualized plan of treatment for them.

Becoming an Empowered and [ACT]IVATED MPN Patient

Patient Empowerment Network (PEN) is committed to efforts to educate and empower patients and care partners in the myeloproliferative neoplasm (MPN) community. MPN treatment options are ever-increasing with research advancements in treatments and testing, and it’s essential for patients and families to educate themselves with health literacy tools and resources on the latest information in MPN care. With this goal in mind, PEN kicked off the [ACT]IVATED MPN program, which aims to inform, empower, and engage patients to stay abreast of up-to-date information in myeloproliferative neoplasm care.

The [ACT]IVATED MPN program is aimed at newly diagnosed MPN patients, yet it can help patients at any stage of disease. [ACT]IVATED MPN helps patients and care partners stay updated on the latest options for their MPN, provides patient activation tools to help overcome care disparities and barriers to accessing care, and powerful tips for self-advocacy, coping, and living well with  a blood cancer.

MPN Expert

Myeloproliferative Neoplasm Disparities

Race or ethnicity may have an impact on some health conditions, including MPNs. Cancer patient Lisa Hatfield interviewed Dr. Idoroenyi Amanam, Assistant Professor in the Division of Hematology at City of Hope. He explained about MPN risks and outcomes that may be impacted by patient race or ethnicity. “…we know that if you have high blood pressure, you have a higher risk for these complications-associated MPNs. And we know that African American males have a higher risk for that, so I think those things are…that example is a clear indicator that really identifying these basic risk factors that are related to diet, exercise, your weight, and other behavioral, possibly behavior-related factors may put you at higher risk to have complications from MPNs.”

 

Dr. Amanam further shared research findings and what needs further study to draw conclusions. “We have looked at incidences of thrombotic events in patients who have MPNs, and we tried to see if there was a difference between racial groups, and we didn’t. We did see that if you’re younger, you do have a higher risk of thrombosis over time, but there was no difference for if you were white, Hispanic, or African American. What we did find though, in a small single center study is that if you are non-white, there’s a higher risk of death over time. And I think we still need a lot of work to get a better understanding as to why that’s the case.”

Dr. Amanam Background

Solutions for Improved Myeloproliferative Neoplasm Care

Clinical trials are key in moving MPN research forward to improved treatments and care. Dr. Amanam shared his perspective about other players besides patients who can help move the needle forward. “I think that going back to the idea that we want to practice the best science, we want to be able to publish the best data. The responsibility is on the clinicians, the scientists, the clinical trialists, the drug companies, the institutions to really be able to structure clinical trials that are relevant to our real world experience. And so how can we better encourage that? I think from a government perspective, potentially incentivizing drug companies and institutions and the other major players that really are involved in pushing this field forward to practice better science.

Dr. Amanam expanded on how clinical trials can be diversified. “And once we’re clear that being able to have a diverse participant pool will give us the best results and therefore will lead to your drug being approved. I think we will have more participants from all groups.

Some underrepresented patient communities may experience obstacles to MPN care. MPN Nurse Practitioner Natasha Johnson from Moffitt Cancer Center provided advice to help patients. “Patients themselves can research clinical trials by looking at clinicaltrials.gov and see what’s out there and contact the academic center that’s performing those trials. There’s free information online that provides recorded sessions from conferences or speakers or speaking done by the MPN experts that you can just look to and get to easily to help understand the disease, knowing the symptoms, and then guiding treatment.” Natasha Johnson continued with additional advice for optimal care. “.…to try to get into a large cancer center or academic center and see an MPN expert. Many times, this is just by self-referral. Charity is sometimes provided through these. Zoom visits can be done as consults or follow-up visits. So my encouragement would be search these out, find out who the experts are, and contact them directly and see if there is any possibility or a way that you can get in to see an MPN expert for a consult so you can get the best care possible.” 

Signs of MPN disease progression is something that patients and providers must be on the lookout for. Natasha Johnson shared her advice for patients to empower themselves against disease progression. “…monitor your blood cell counts, be your own advocate. Think about if they’re changing, could it be medication, or is it disease progression? Monitor your symptoms. Look at the total symptoms score or write down your symptoms and try to record where you’re at in intervals. Are things getting worse? If they are, don’t wait three months for your next appointment. Contact your healthcare provider and ask to be seen. Ask about getting a repeat bone marrow biopsy to establish where the current disease status is because that can open up doors possibly to more treatments.”

The future of MPN care has an additional approach to address. Dr. Amanam shared his perspective and how patients can help advocate for improved treatments. “I think in the next three to five years, we’re going to have drugs that are going to actually be able to treat the underlying disease before it gets to a point where you may need more aggressive therapy…And so it’s exciting where we’re going, and I think the questions that as a patient that I would ask are, because of the fact that we only have few FDA-approved therapies, are there any clinical trials that are able to target the underlying disease as opposed to just treating the symptoms? I think that’s very important for the patients to ask, especially in this space now.

Rates for stem cell transplant approvals must improve for lower income groups and for African American and Hispanic groups to provide better health outcomes for blood cancers like MPNs. Dr.  Amanam explained what’s involved with transplants and how others can help as donors. “You can donate your bone marrow, or you can donate your stem cells that are not inside of your bone marrow. And typically as a donor, your experience of actually donating is about a day. And the recovery time after you donate your bone marrow or stem cells, it’s typically within about one to three days.  So the benefit of donating your stem cells or bone marrow outweighs the inconvenience of a day or a couple of days of your schedule being altered. So I think that’s really important to understand. And I think if we can get more people to be aware of this, I think we can definitely get more donors.

[ACT]IVATED Program Logo

[ACT]IVATED MPN Program Resources

The [ACT]IVATED MPN program series takes a three-part approach to inform, empower, and engage both the overall MPN community and patient groups who experience health disparities. The series includes the following resources:

Though there are myeloproliferative neoplasm disparities, patients and care partners can take action to empower themselves to help ensure optimal care. We hope you can take advantage of these valuable resources to aid in your MPN care for yourself or for your loved one.

Understanding MPN Treatment Goals and Shared Decision-Making

Understanding MPN Treatment Goals and Shared Decision-Making from Patient Empowerment Network on Vimeo.

Myeloproliferative neoplasm (MPN) treatment goals can vary widely among patients, so how do care providers work with different goal types? Expert Dr. Idoroenyi Amanam from City of Hope explains how treatment approaches can vary, his perspective in shared decision-making, and advice for patients to receive optimal care.

[ACT]IVATION TIP:

“I would recommend that you get a clear expectation, with your diagnosis as to what that means for you specifically and what the treatments will do for you short-term and long-term.”

Descargar Guía|Download Guide 

See More From [ACT]IVATED MPN

Related Resources:

Are There Any MPN Disparities in Subtypes and Genetics

Are There Any MPN Disparities in Subtypes and Genetics?

How Can MPN Clinical Trials Be Diversified?

How Can MPN Clinical Trials Be Diversified?

Graft-Versus-Host Disease Risk for BIPOC Patients

Graft-Versus-Host Disease Risk for BIPOC Patients

Transcript:

Lisa Hatfield:

 So, Dr. Amanam, you probably have patients coming in with a wide spectrum of knowledge from patients who want to be told what to do for treatment to patients who might even bring in abstracts from ASCO and ASH. So how do you work with your patients to make those treatment decisions? And a second part to that question is, with increasing treatment options, what should your MPN patients consider when deciding on treatments?

Dr. Indoroenyi Amanam:

So I believe that it’s very important to understand contextually where the patient’s coming from. And you’re right, I think some patients actually want to receive a presentation on the data. From a randomized Phase III clinical trial, there are some patients who are not interested in hearing about the data. They just want you to tell them what you think. And I think understanding where a patient’s coming from is very important. And so I believe, at least from my experience with the diversity of experiences in my own life, that I have the capacity to really gauge what patients need in order to proceed forward with the treatment that they’re most comfortable with. I think that’s the answer to that question. For with…in regards to increasing treatment options, it’s difficult because I think we’re in a very exciting time for MPN patients, we have a lot of treatment options.

We have a lot of clinical trials, we have a lot of…we have more FDA-approved therapies than we did 10 years ago. And I think it’s important to set the expectations as to what a therapy does. So some therapies will potentially decrease the risk of the disease progressing. There are some therapies that really help improve symptoms. There are some therapies that do both, and I think it’s very important to be very clear as to what each individual therapy does and the side effects associated with those therapies. And it usually, for the most part, patients are pretty…they declare themselves as to what they’re looking for. I think everyone walking into a room who’ve been told that they have cancer, they want to cure, but once you set the expectations that for MPNs this is possibly a chronic disease, and there are some issues associated with the chronic disease that we have to manage. And I think once it’s clear as to what MPN means for the patients, it changes the understanding of wanting a cure.

And I think, I will say I want for us to get to a point where we can cure all patients, but we don’t, we aren’t not there yet. And so ensuring that patients have a good quality of life is the most important thing for me and really being happy with what we’re doing in terms of treatment.

So my activation tip for this question is, I would recommend that you get a clear expectation, with your diagnosis as to what that means for you specifically and what the treatments will do for you short-term and long-term.


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What Does the Future of Myeloproliferative Neoplasm Care Look Like?

What Does the Future of Myeloproliferative Neoplasm Care Look Like? from Patient Empowerment Network on Vimeo.

What does the future of myeloproliferative neoplasm (MPN) care look like for patients? Expert Dr. Idoroenyi Amanam from City of Hope explains how MPN treatments have changed in recent decades, symptoms that are relieved with treatments, and how treatments of the future may help patients.

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Myeloproliferative Neoplasm Basics for Newly Diagnosed Patients

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Transcript:

Lisa Hatfield:

Dr. Amanam, what promising treatments are available for newly diagnosed MPN patients, and what questions should patients be asking? They come into your office scared to death and not even knowing what to ask. Do you have any suggestions for what those patients should be asking when they go in as a newly diagnosed patient?

Dr. Indoroenyi Amanam:

Right, right. Twenty years ago, we really didn’t have any therapies for most MPN patients, aside from performing phlebotomy and using non-specific therapies to try to help control their counts and therefore reduce their risk of clotting and stroke. We are getting to a point which is really exciting, where we actually are treating the underlying disease, meaning that the cells that are causing this cancer, we have been able to identify targets that will help eradicate those cells and, therefore, get rid of the cancer. And so we’re getting there. Unfortunately, we still are not there yet, and so when we look at the FDA-approved drugs in this space, really, they help control symptoms, they help control some of the associated complications with the disease, mainly when your spleen’s enlarged, and that potentially may affect your quality of life, mainly your nutritional status and your physical status, and so we do have drugs that are able to do that, that are FDA-approved right now.

I think in the next three to five years, we’re going to have drugs that are going to actually be able to treat the underlying disease before it gets to a point where you may need more aggressive therapy. Currently, the only defined curative therapy that we have, when I say defined, meaning that we have multiple studies that have shown that that’s the case, is bone marrow transplant. I’m a bone marrow transplanter, I do treat some of my MPN patients with bone marrow transplant to get rid of the underlying…those underlying cells that are driving this disease. But that’s a very intense therapy and it has its own associated complications. But we are…will be having other drugs that potentially we would be able to offer that are not as intense as bone marrow transplant.

And those include immunotherapy, other drugs that can target the signals that drive these cells to divide and multiply. Also there are within the bone marrow for patients that have myelofibrosis, which is one of the MPNs, we will be able to target the environment that allows for these cells to persist and grow. And so it’s exciting where we’re going, and I think the questions that as a patient that I would ask are, because of the fact that we only have few FDA-approved therapies, are there any clinical trials that are able to target the underlying disease as opposed to just treating the symptoms? I think that’s very important for the patients to ask, especially in this space now.


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[ACT]IVATED MPN Resource Guide en español

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Spanish Activated MPN Resource Guide

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[ACT]IVATED MPN Resource Guide

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Activated MPN Resource Guide

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Should All MPN Patients Undergo Molecular Testing?

Should All MPN Patients Undergo Molecular Testing? from Patient Empowerment Network on Vimeo.

Dr. Gabriela Hobbs discusses the necessity of molecular testing for myeloproliferative neoplasm (MPN) patients, including the pros and cons of this in-depth testing for patients with polycythemia vera (PV) and essential thrombocythemia (ET).

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

How useful is having a genetic panel done? Should all patients get molecular or genetic testing? 

Dr. Hobbs:

Great question. And I think that it is very important to have genetic testing.   

And genetic testing involves more than just testing the JAK2 mutation. So, we know that the JAK2 mutation is the most common mutation in patients with MPN. But that being said, there are other mutations that also occur such as the calreticulin mutation and the MPL mutation.   

And so, I think having genetic testing that at least tests for those three mutations is very important so that we can actually help a patient know that they have an MPN. In addition to those three main mutations, many clinicians now have access to what’s called extended next-generation sequencing, where there’s a panel that tests for many different genes at the same time and can test for a variety of other mutations.  

And this is particularly relevant for patients with myelofibrosis. As we know that having other mutations, like, for example, mutations in IDH or ASXL1 and others, can increase the risk of that disease in terms of its risk of transforming to leukemia or how long a patient may live with their myelofibrosis. 

And so, I do recommend having extended next-generation sequencing done at least at diagnosis.  

When I generally think about repeating that, if there’s something that looks like it’s changing within the patient’s disease, to be honest, also on the flipside of that argument, sometimes this next-generation sequencing will mostly contribute to adding anxiety and will not necessarily directly impact how a patient is treated. And this is particularly true in patients with PV and ET, where we’ll sometimes order these tests, and we get a bunch of mutations back, but we don’t know what to do with that information yet.  

And so, as a researcher – not a clinician – as a researcher, I think it’s very important to have that information so that we can then do studies and understand the patterns of mutations and how that affects outcome. But as a clinician, and you as a patient, you need to really be aware of how that’s going to impact the patient in front of you and how that may impact you as a patient. Do you want to know if you have these mutations if nothing can be done about it? So, I would say, take a moment to reflect upon what I said and also to ask your clinician, how is this information going to help me? Do I need to have this information?  

Maybe you want to have it done so that it’s in your record. But maybe you don’t necessarily want to know those results. And everybody’s very different. And I think it’s absolutely wonderful to talk to my patients about all the information. But there may be some patients that really are just, like, do the test but don’t tell me the results, because I know that I’m just going to be very anxious knowing that I have something that I can’t do anything about. So, just take a minute to talk about it with your doctors. I think that’s really important.