Myeloproliferative Neoplasms Archives

Myeloproliferative neoplasms (MPNs) are a closely related group of progressive blood cancers in which the bone marrow typically overproduces one of the mature blood elements. Other shared features include tendencies toward blood clotting/bleeding, organ enlargement, bone marrow scarring (fibrosis) and a possibility of transformation.

More resources for Myeloproliferative Neoplasms from Patient Empowerment Network.

MPN Patient Profile: Robyn Rourick Part 2

Read the first part of Robyn’s MPN journey here…

Picking up after 26 years of watchful monitoring of her myeloproliferative neoplasm (MPN), scientist Robyn Rourick was then referred for an allogeneic stem cell transplant by her MPN specialist, Dr. Gotlib. The transplant team started working through the matching process for a bone marrow transplant donor, which often begins with close biological relatives. Although Robyn’s only sibling wasn’t a transplant match, a person considered a near perfect transplant match for Robyn was found.

At that point in her journey, the possibility of entering a Phase II clinical trial called ORCA-1 was presented by Robyn’s transplant doctor. She discovered that the ORCA-1 treatment had the potential to completely eliminate graft-versus-host disease (GVHD). The clinical trial made sense to her. In Robyn’s trained scientific mind, she agreed that the trial was founded on sound scientific rationale with the potential for clear benefit and signed up for it. She researched other things like whether the transplant team could look at biomarkers to guard against graft-versus-host disease, but she decided to take the clinical trial path as her best option.

As for her feelings about the stem cell transplant, Robyn felt there was likely going to be a positive outcome for her due to the ORCA-1 clinical trial. Her knowledge about the trial really brought her a lot of comfort and put her at ease for the time she’d be around her family post-transplant. Robyn was lucky because her doctor was actually the primary investigator on the study. When he presented the transplant study as an option, that’s when she started to do more searching to find what patient advocacy groups were out there.

Looking back on her MPN journey, Robyn wishes that physicians would provide their patients with more patient advocacy resources, such as those available through organizations like Patient Empowerment Network (PEN). She feels fortunate that she discovered PEN through another patient advocacy website, and she firmly believes in PEN’s mission of empowering patients to gain knowledge to advocate on their own behalf. “I had the realization that in the clinical trial I was in, I was only the sixth patient, and the technology was stellar in terms of what we’re trying to do in terms of cell therapy. I just felt like patients need to know about the treatment advancements, and PEN is an excellent resource for learning about treatment and support options that I wanted to share my knowledge and patient experience with.” 

Robyn was fortunate to have a team of physicians in whose knowledge and treatment recommendations she could trust. She’s  tremendously grateful, because she knows it’s not always the case, and so offers this advice for others, “Make sure that you’re comfortable with your physicians. And if not, then move on. Don’t be afraid to reach out and to make other connections to other doctors, even across the globe. You shouldn’t hesitate to request a conference call with another provider to see if they’re aligned with your diagnosis and your watchful waiting or treatment recommendations. Patients must have the utmost confidence going through their cancer journey.”

As for the scientists who handled her sample in the ORCA-1 trial, Robyn was able to meet the scientists and saw the analytical data of her sample. She was highly impressed with the protocols that they used with the samples. Robyn was just the sixth myelofibrosis patient to join the trial. To have spent her life working on medicines for patients and then to be on the receiving end of this cutting-edge treatment for transplants made her feel very privileged. 

In her life post-transplant, Robyn has continued periodic blood work for routine monitoring and has been doing well. Two years following her transplant, Robyn’s myelofibrosis is in remission, and she has no evidence of fibrosis in her bone marrow. Her test numbers have been progressing nicely, and she hasn’t needed any additional treatment since undergoing the transplant. “I don’t have a single regret. I haven’t had a pimple, an itch, a scratch, absolutely nothing. My life has resumed exactly how it was before the transplant.”

In reflecting on her patient experience, Robyn offers this additional advice to other cancer patients, “Take a deep breath and give it some time to play out. The moment that I heard the word cancer and the risks with rapid progression, I had myself dead and buried. In my mind, what I needed to plan for was death. Prepare my family. Get everything in order. And to me, that was going to be the ultimate outcome. But then as things unfolded, I had conversations, did a little bit of research, and found out I did have some options. Things weren’t so negative in terms of progression and mortality. Don’t jump to the most negative outcome possible.”

MPN Patient Profile: Robyn Rourick Part 1

Though Robyn Rourick is a scientist by training and works for a biotechnology company, she took a mind-body approach to her myeloproliferative neoplasm (MPN) journey. The time that passed between Robyn’s initial MPN diagnosis and when she finally needed treatment was incredibly – and nearly shockingly – long. She was diagnosed with essential thrombocythemia (ET) 26 years after elevated platelets were shown on a routine blood test. After she saw a hematologist, they performed a bone marrow biopsy and concluded she didn’t have myelofibrosis and received the ET diagnosis. Robyn recalls of the time of her diagnosis, “I didn’t know about myeloproliferative disorders. Not many people did at the time. Nobody mentioned that I could potentially have an MPN.” 

Robyn’s blood levels were monitored over the years, and her platelets started to decrease. Though she didn’t realize at the time, her platelets were decreasing because her bone marrow was becoming more fibrotic. She was also tested for the early gene mutations (JAK2) that were discovered as more MPN research occurred but tested negative . She later switched to another hematologist who was very tuned into the gene connections. He looked at Robyn’s medical data comprehensively and was extremely attentive to any minor changes. As her blastocytes began shifting, he urged her to go see MPN specialist Dr. Gotlib. Dr. Gotlib did further analyses and classified her as having myelofibrosis, noting that when she was diagnosed with ET that her original healthcare team also couldn’t have  ruled out pre-fibrotic myelofibrosis at that time. Fortunately, Dr. Gotlib stated if he had diagnosed her with her original blood test 26 years prior, he would have recommended to simply watch and wait while monitoring Robyn’s blood levels on a regular basis. 

Although Robyn felt healthy and had no symptoms besides an enlarged spleen, as Dr. Gotlib dug deeper into her genetic profile, he found a unique mutation that suggested she was at risk for an escalation into acute myeloid leukemia mutation. He recommended Robyn for an immediate allogeneic stem cell transplant for her MPN treatment.    

Robyn then learned that graft-versus-host disease (GVHD) was a major concern for the transplant process, which can be debilitating. So she began to seek patient advocacy resources to inform her MPN journey. “I felt desperate and wanted to meet people who had myelofibrosis who successfully came through transplant. I didn’t want to just talk to a transplant person with a different disease.” Robyn went through some patient connection programs – including Be the Match, Caring Connections Program, and Patient Power – and was able to meet a few people and became quite close with one patient. 

She learned that even though transplant will cure your disease, doctors don’t always elaborate with patients on the potential for a compromised lifestyle due to  graft-versus-host disease. Sometimes patients will come through transplant in worse condition than before the treatment. Robyn had major fears about going through transplant and being able to work and do her extracurricular activities post-transplant. “I felt like I was going to be a letdown for my family and colleagues and didn’t tell my work until I was preparing to go out on leave, which in retrospect was silly.” After telling her manager, Robyn was given complete support, and realized she could have avoided carrying so much anxiety.

“For me, self-education and advocacy are important to enable yourself to have conversations about what’s possible in terms of your treatment. You don’t have to develop an in-depth understanding, but enough to have the ability to be conversational. If you’re proposed a certain pathway, it’s good to know enough to ask why. And if you’ve done some research on your own, then you can ask why not an alternate treatment approach. I think it’s really important to have some knowledge, because it builds your confidence to be able to move forward with what’s being proposed.” 

“Give it time, allow yourself to digest the information, have conversations about it, and develop your own understanding. At first, I was very closed about my diagnosis. I told my immediate family, and I told one very close friend who had gone through autologous transplant. The more that I began to talk about it and the more that I included people in the story, the easier my journey became.” Robyn also saw a cancer therapist who made some really good points to her. “She told me that ‘we’re all going to die of something, but most of us don’t know what that really looks like.’” In Robyn’s case, she had the opportunity to learn more about her disease, guide it, and direct her journey. And that opened up a whole new perspective.

The cancer therapist walked Robyn through some exercises: “What is it you’re afraid of? What do you have control over? Allowing yourself to gain control over some things will build your confidence that you can do this.” Robyn also encourages other patients to engage their network of friends and family and realize that it’s okay to depend on people. It’s not your fault that you have this diagnosis. Getting over the apprehension of telling people about your diagnosis and embracing help from others are key pieces of advice.

Robyn views patient empowerment as essential to the patient journey. She discovered Patient Empowerment Network (PEN) through another patient advocacy website and felt it brought her MPN patient experience full circle in terms of learning what’s available. “As I’m learning more about PEN, I’m just dazzled by the different forums they have to enable knowledge transfer, support systems, and advocacy.” 

Read the second part of Robyn’s MPN journey here…

Roles Reversed: Taking Care of Your Care Partner

Roles Reversed: Taking Care of Your Care Partner from Patient Empowerment Network on Vimeo.

MPN Empowerment Leads Summer and Jeff are experiencing a bit of a change. Jeff is Summer’s care partner, however the roles have been reversed. Jeff recently had a knee replacement and is unable to do many tasks he was before. Summer has jumped in taking care of Jeff, but admits it’s harder than it looks. Watch and hear Summer’s comedic take on switching roles and stepping into the care partner role.  

Want to connect with Jeff and Summer? Email them at question@powerfulpatients.org or text EMPOWER to (833) 213-6657. 

Transcript:

Jeff:

Summer! Bring me a snack.

Summer:

Yes, I’m bringing you a chocolate bonbon.

Jeff:

I don’t want a Ghirardelli, I want a Walker’s shortbread.

Summer:

Ugh, yes sir.

Being a caregiver is not a day in the park.

Jeff:

Hi, I’m Jeff.

Summer:

I’m Summer, hi.

Jeff:

We’re your MPN Network Managers for the Patient Empowerment Network. We’re here today to continue talking about caregiving. In the last video, we talked about me being Summer’s caregiver and or different roles and what I bring to that and so on. We had a chance in the last month to actually turn the tables.

In mid-December I had a knee replacement and since then, Summer has been acting as my caregiver. Tell us how it’s been, Summer.

Summer:

Ugh, it’s been exhausting. I’m driving, I’m doing dishes, I’m emptying the garbage, I’m cooking all the meals, I’m getting everything exactly the way you want. You’re lot more of a perfectionist than I am…

Jeff:

Yup, I should be able to drive in another week or so and Summer hates driving, so I really appreciated that. And pretty soon I’ll get back to my role of doing the driving anyway. She’s done a wonderful job, really been very helpful and I’ve been extremely appreciative of it. What’s been the hardest thing for you, Summer?

Summer:

Thinking about all the little things you take for granted that you couldn’t do, like mailing your letters and emptying your garbage. That’s everything, you really have to be on the ball and think of what the needs of the other person really are.

Jeff:

Very true. We stressed that in the last video. Needs of the patient. We have a real supportive relationship in our normal marriage in general, so for us it’s not difficult, but some people may have a difficult time adjusting to being a caregiver or even being a patient.

Summer:

Right.

Jeff:

One thing you have to remember, give the caregiver time for themselves. I think I did a pretty good job trying to give you time for yourself.

Summer:

Yeah, I did my aerobics, I visited friends, I rehearsed for the play, I did my stand-up comedy, yeah, I did.

Jeff:

So, it’s worked well for us. So, as you enter into this relationship of patient and caregiver, be aware of each person’s needs. And, you should have a good experience with it.

Summer:

Darling, I have an urge for a chocolate cookie. Could you bring me one? I gotta take a nap.

Jeff:

Certainly. I’ll get it after we say goodbye to the people. Goodbye, ’til next time.

Summer:

Bye, ’til next time.

Patient and Care Partner Address the Mental Aspects of an MPN

Patient and Care Partner Address the Mental Aspects of an MPN from Patient Empowerment Network on Vimeo.

MPN patient Summer emphasizes how important it is to have goals. One of her personal goals is to walk 10,000 steps each day! As a care partner, Jeff shares his main goal is to do whatever he can to help Summer achieve her goals. Some of their shared goals are to live life to the fullest, always live in the moment and don’t let the disease you have control your life.  

Want to connect with Jeff and Summer? Email them at question@powerfulpatients.org or text EMPOWER to (833)213-6657. 

Transcript:

Jeff:

Hi, I’m Jeff.

Summer:

Hi, I’m Summer. And hi, I’m Zelda.

Jeff:

And we’re your MPN Network Managers for the Patient Empowerment Network. Here to talk to you today about emotional and metal aspects of dealing with a severe or very serious disease. Summer has some real strong thoughts about that, so I’m going to let her tell you about it.

Summer:

Well, I think it’s really important to always have goals. And one of my first goals, this is my Apple Watch right here to keep track of my steps so I can get 10,000 steps a day. That’s really important. Of course, I’m still teaching my classes. That’s very inspiring. We’re putting on a show pretty soon. And my goal right now too is I’m getting ready for my next comedy show. I’m going to talk about the cloud and about those little people that are in the traffic signs that tell you when to stop or go. And of course I’ve got Zelda and I don’t know what her goal is, just to be a good dog. So, that’s what my goals are. To stay positive.

Jeff:

And Summer has those goals, but one serious and important thing that we decided when she got this disease was we were going to live every life, every moment of our lives to the fullest, in the moment. And that took some adjusting because I’m a real planner. We pay attention to enjoying everything that we do, each day that we do it. And, we decided early on not to change our lifestyle, not to let the disease control us, and just to keep going. And, that alone plus Summer’s fantastic exercise regimen has really done a lot to keep her in good shape and to keep her strong.

As a caregiver, my goals are do whatever I can to help her achieve her goals. So, it’s really important to live life in the moment and keep a positive attitude. Don’t let the disease control you. That’s our advice.

‘Til next time, I’m Jeff.

Summer:

I’m Summer and I’m Zelda.

Expert Perspective: Hopeful MPN Research and Development

Expert Perspective: Hopeful MPN Research and Development from Patient Empowerment Network on Vimeo.

MPN expert and clinical researcher Dr. Abdulraheem Yacoub shares excitement about the future of MPN treatment and research, including an optimistic outlook for new approvals in the coming year. 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You?

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?


Transcript:

Katherine:

I wanted to get your take on the future of MPN research and treatment. Are there new developments that you’re excited about and that make you hopeful?  

Dr. Yacoub:

Absolutely. So, again, I would like to take the last few minutes to advocate for the future. And the future can only come when doctors and patients and advocates work together to advance the science. We have few tools to treat patients and to help patients. We have a lot of unanswered questions. And the only way to answer them is by designing quality clinical trials, enrolling patients on trials, taking the risk, and trying to find new answers and new therapeutics. So, I always would like to advocate for patients to seek clinical trials whether with their doctor or whether they have to travel for it, and for doctors to consider that for their patients. That’s the only way to advance science.  

There are very important national and international studies going on right now. One of the – and first, I would like to emphasize is that we have had ruxolitinib (Jakafi) as the only therapy, or the first-line therapy for myelofibrosis for a decade now.  

Not everybody responds to it, not everybody responds to it for a long time. So, now we’re designing combination trials. So, there’s a few studies that we are trying to redefine, “Is ruxolitinib alone enough, or should we have a combination first-line therapy?” So, these are some of the more important questions being asked right now.  

And this is definitely one of the bigger moves in the field, is trying to redefine what is the first-line therapy for myelofibrosis. For polycythemia vera, we’re also exploring therapeutics that would reduce phlebotomy with things you can add to your medical care to reduce phlebotomy.  

So, that’s also going on. And it’s definitely a big leap forward for many of our patients. For ET, when we don’t have any actual drugs approved other than hydroxyurea (Hydrea) and anagrelide (Agrylin), we actually have trials with interferon going on.  

So, I would like to advocate for that. So, interferon succeeded and now approved for PV, but not yet for ET. We’re working on that. So, again, in every disease, we’re trying to design clinical trials to redefine what is the best treatment today.  

We’re also doing studies to understand the cancer. So, studies where patients donate their samples for research. These are very helpful, very important. And contributing to that always advances the science, and it’s low effort to the patients. So, if there’s a clinical trial that is offered to patients, I would strongly urge everybody to consider that favorably and contribute to science. 

That’s the only way we can help future patients and ourselves immediately, at moving the field forward.  

What Are the Signs of MPN Progression?

What Are the Signs of MPN Progression? from Patient Empowerment Network on Vimeo.

Dr. Abdulraheem Yacoub, an MPN specialist, explains how essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF) may progress from one disease to the next, including potential signs and symptoms of MPN progression. 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 

Related Programs:

 
What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Myelofibrosis?

What Are Treatment Options for Myelofibrosis?


Transcript:

Katherine:

We have a couple of questions from the audience. This one is from Sarah. She writes, “I’ve been living with essential thrombocythemia for three years, and have been relatively stable. Of course, I’m worried about progression to PV or MF. What is my chance of progression, and what are the signs of progression?” 

Dr. Yacoub:

That’s a very good question. And unfortunately, we’re very good at describing those numbers. Unfortunately, our tools at interfering are not as good. So, in general, patients with ET, statistically speaking, have a life expectancy that is not different from their age match peers. And Sarah’s story will be not too indifferent from her sisters and her mother, in terms of what’s going to happen to her long care and her health, provided she gets good medical care. The exception to that is that there is a transformation risk. For ET we caught around a 4 percent every 10 years in which ET will actually change into a different cancer, a higher risk cancer.   

Could be MF, could be MDS, could be acute leukemia. And that will be a much more serious diagnosis. So, it’s about 4 percent in 10 years. We do have a – or we extrapolate some of the data from other cancers. So, certain mutations are more favorable, certain mutations are more risky. And we try to forecast that but worried it’s really hard to predict that since it’s such a long journey with disease. The first symptoms or the findings, when patients start suspecting that their disease has changed, is that the pattern of symptoms that they have are different.   

They often become worse. So, they have more constitutional symptoms, more tiredness, more fevers, more night sweats, losing weight, not being able to eat a full meal, abdominal distension, the spleen gets bigger.  

So, these are some of the feelings that patients can experience that lead to this. Other objective things is when the blood tests change in a less favorable way. So, for patients with ET who always run at 800,000 platelet count, if they’re suddenly 200, and that’s in the normal range, but that’s actually not good news, because the cancer changed. And this change is not favorable. So, as the doctors run routine labs, if they see the sudden change in labs, that’s also abnormal.  

If the doctor can feel that the spleen gets bigger every time, that’s also concerning. If the patients suddenly have anemia or very high white cell count or immature white cells in the blood, that’s also a concern.  

So, that’s why it’s great or important to establish a baseline symptom burden.  A baseline spleen, a baseline bone marrow biopsy with mutation analysis, so that patients have a clear reference point to where they started, and if things change, they can always go back to that point and compare.  

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions? from Patient Empowerment Network on Vimeo.

Dr. Abdulraheem Yacoub, an MPN specialist, shares advice for patient self-advocacy and provides tips for participating in care and treatment decisions.

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You?

Expert Perspective: Hopeful MPN Research and Development

Expert Perspective: Hopeful MPN Research and Development


Transcript:

Katherine:

Dr. Yacoub, what is the role of the patient in their care? When does shared decision-making come into play?  

Dr. Yacoub:

Absolutely. Patients are the drivers and the centerpiece of their health care. And patient self-advocacy is the most important tool. So, many of our patients are young, and they will live with their cancers a lot longer than many cancer doctors will practice oncology. And they will have many doctors. Statistically, each MPN patient will have multiple doctors throughout their career. And they will hear different derivatives. And the science will change. And they will be given different counseling over the time. And their disease will change.  

And they will have different needs as they go further. So, patients being involved in their well-being and their cancer care is important from the first day. And I always tell patients, “You need to start building your village from day one.” It is not just the patient, it’s your caregivers, it’s who else can help you.  

Who else can advise you? You might want to also invest in a friend or a spouse or a child, to come to you and listen to some of those discussions so that they can advise you later on, “Why are you making different decisions?” So, we encourage patients to be very involved early on, to build their own village, and to seek care. We routinely ask for second opinions. We want patients to always hear the story and hear the same story from another doctor so that they hear the range of how we word the truth and how we word the facts.  

And this way, they can have a better perspective. So, this is now a standard. Almost all patients should have two doctors, at least, the treating doctor and one doctor who’s an MPN specialist, who would give them another twist or another perspective to their health.  

So, and that is always important. And then there are very good references and online resources for patients to tackle in, such as this seminar and other good places where patients can seek more information. They also can go to a clinical trial to find out what are the ongoing clinical trials and advancements.   

There are structured patient symposiums nationally and regionally. So, and we strongly recommend that patients seek more opinions and more help and more resources and be very engaged with this disease, especially that it is a chronic cancer, and it’s not going to – 

Katherine:

It’s not going away. 

Dr. Yacoub:

It’s just a new lifestyle. And they need to be as engaged with it as they can.   

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You? from Patient Empowerment Network on Vimeo.

MPN specialist Dr. Abdulraheem Yacoub reviews factors that determine which treatment is most appropriate for your essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF). 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?

Expert Perspective: Hopeful MPN Research and Development

Expert Perspective: Hopeful MPN Research and Development


Transcript:

Katherine:

As with most conditions, all patients are different, of course, and what might work for one person might not be appropriate for another. So, how do you choose which treatment is right for a patient?   

Dr, Yacoub:

Excellent. So, and that’s actually the heart of what we define as the art of practicing medicine and being patient-centric and focused. And patients already have their own wishes and their needs. And everything should start with having a discussion with patients on what is their priority, and what are they trying to achieve.  

And we do have to explain to them the tools we have, the interventions that can help them. But we also need to make sure they’re compatible with what they actually want and their goals in life. And sometimes what we doctors want is not exactly what the patients want. So, we always have to remind ourselves that patients are the drivers of their care. And they have the absolute right to be informed and to make informed decisions based on the options we advise them about.  

So, that is always a centerpiece of healthcare. And then patients – basically, we defined four pillars of care. We want to control their symptoms, we want to prevent complications, we want to modify the disease so it doesn’t transform, and we want our therapies not to have toxicities, not to have side effects that are worse than the disease.  

So, we bring that up to the table. And we also look at the patient. What are their symptoms? What did the disease cause them to be complications?  

What is the risk that their cancer is actually going to progress quickly to hurt their lives? And how serious is the therapy we’re recommending? And we need to make sure that there is a good match between what we’re offering and what the disease is manifesting. So, for example, for patients who have a lot of symptoms, but they have low-risk cancer that they can live with for a long time, we focus on symptoms. We focus on treatments that improve their symptoms.  

While with patients who have more serious diseases that are eminently life-threatening, we focus on an expedited path to a more aggressive therapy and a bone marrow transplant.  

And then we also try to match those therapies with the other patient’s wishes and needs and so forth. So, all these factors are important. We have more tools to try to prognosticate. So, prognosticate is the medical word that we use as forecasting.  

We like to forecast the disease or the cancer. We try to predict the patient’s future. Fortunately, we actually have good tools to prognosticate now. We have models or calculators that factor in patients’ features, their symptoms, their age, their blood counts, their bone marrow findings, and their DNA mutations. And it gives us a score, a risk score that can correlate with their life expectancy or their outcomes.  

And we use those tools to guide us. So, there’s actually a tool we use to help patients reach that decision. It’s an objective tool to decide how serious is this disease and how seriously we should tackle it. It’s very applicable for patients with myelofibrosis, more sort of the other lower-risk cancers.  

Katherine:

What about comorbidities? How do they fit into the treatment plan?  

Dr. Yacoub:

Very important.  

So, again, it also goes back to finding the balance between how serious is the disease, how serious is the treatment, and how will the patient’s general health tolerate and factor in the choices patients make accordingly. So, myeloproliferative neoplasms do happen in a broad range of ages. And we have children, minors with MPNs, and we have elderly patients with MPNs. And it’s a continuous spectrum. And each individual patient will have their own health concerns and their own health comorbidities and their own wishes. And we always have to make sure that we match our therapies, the disease seriousness, and the patient’s wishes, which is also stemming from their own other health battles, too.  

We cannot turn a blind eye to the other health issues going on. That plays a major factor as we choose to discuss bone marrow transplantation with patients. Because that’s when the medical comorbidities are often the first barrier to go through.  

Katherine:

Are there specific biomarkers that may affect prognosis or treatment?  

Dr. Yacoub:

Yes. So, and we’re glad that actually myeloproliferative neoplasms are actually the model in medical oncology on how predictors can tell us a lot more about the patient’s future about the prognosis. So, early on in MPNs, we’ve developed models, like the International Prognostic Scoring Systems in many different iterations. And more recently, the Molecular Based International Prognostic Scores.  

They factor in patient’s age, they factor in blood numbers, they factor in DNA abnormalities, they also factor in DNA mutations, including the common driver mutations JAK2 and CALR and MPL, as well as more novel mutations that we call higher risk mutations.  

So, based on these models, we use these tools to predict how the cancer will behave, and how to approach it. This advancement has been an application for our MPN patients for a while, way ahead of all other fields of oncology. So, we’re proud that we can give our patients this tool before all other doctors were able to. Yeah.   

What Are Treatment Options for Myelofibrosis?

What Are Treatment Options for Myelofibrosis? from Patient Empowerment Network on Vimeo.

MPN specialist Dr. Abdulraheem Yacoub shares and overview of treatment options, including therapies in development, for patients living with myelofibrosis (MF).

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.

 
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You?

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?


Transcript:

Katherine:

And since myelofibrosis is a progressive condition, I imagine it’s more difficult to manage. So, what else is available for patients with myelofibrosis?  

Dr. Yacoub:

Correct So, myelofibrosis is the higher end of this spectrum of cancers.  

It is a cancer that is associated with much higher symptom burden and impact on daily life. It is also associated with low blood counts, and some patients will require transfusions. It’s a major morbidity to our patients. And in addition, it’s a cancer that is associated with shortened life. So, patients with myelofibrosis will not live as long as their health would have allowed them. And some of them will live actually a much shorter life than they want or deserve.  

So, myelofibrosis treatment requires a lot more considerations. So, for patients who are in good health, who have a cancer that is more aggressive, that would be imminently impacting their longevity, we start a discussion about a curative role of allogeneic stem cell transplantation very early in their course.  

Because bone marrow transplantation can be curative, and those patients can live a long life after a successful transplant. So, this is a treatment modality that should be brought up very early for patients with higher risk myelofibrosis. There are approved JAK inhibitors, ruxolitinib (Jakafi) and fedratinib (Inrebic). And we know that ruxolitinib which has been approved for over 10 years can improve symptoms, can improve the spleen volume, can actually prolong lives for patients on it, and also makes the transplant more successful.  

So, we should be offering that to the appropriate patients also early in their diagnosis, in a strategy where, in addition to that, we get them to a transplant. Fedratinib is approved in that setting. And we are very optimistic that by the end of this calendar year, we will have two other JAK inhibitors approved.  

[Editor’s Note: As of February 28, 2022, pacritinib (Vonjo) has been approved for the treatment of myelofibrosis patients with severe thrombocytopenia.] 

So, we look forward to those two drugs. Momelotinib and pacritinib for patients with special disease features. And hopefully, by the end of this year, we will have a list of JAK inhibitors that we can choose from, which is great news for our patients.  

Katherine:

Oh, we’re still fighting. 

Dr. Yacoub:

Yes, absolutely.  

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Polycythemia Vera? from Patient Empowerment Network on Vimeo.

MPN specialist Dr. Abdulraheem Yacoub provides an overview of available treatment options for patients living with polycythemia vera (PV). 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You?

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?


Transcript:

Katherine:

You mentioned using interferons for ET. That’s something that you would also use for polycythemia vera. Yes?   

Dr. Yacoub:

Absolutely. So, the same principles will apply to polycythemia vera. We would like to treat the higher-risk patients more aggressively. Hydroxyurea (Hydrea) and interferon are also the first-line therapies in these patients.   

The good news in 2022 is that we actually finally have an FDA-approved interferon for our patients. Finally, after 50 years of using interferon, now, we have an FDA approval. So, the new interferon, ropeginterferon alfa-2b is a medication that was studied prospectively in Europe, and it has been approved and in clinical use in Europe under the brand name Besremi.  

And this year, it was approved in the U.S. for patients with polycythemia vera, which is a great achievement for the medical field and a great tool to help our patients. We have used other brands off-label in the past, but it’s glad now to get this confirmation from the FDA that this is a standard of care for all patients.  

And then beyond that, ruxolitinib or Jakafi, is also approved as a second-line option in patients who have had hydroxyurea as their first line.  

So, these are the medicines we use for polycythemia vera. We also use therapeutic phlebotomy. And the goal in high-risk polycythemia vera, or actually in all patient polycythemia vera, is to reduce their hematocrit.  

And we want it under 45 percent every day of the year. And we use the tools that we just discussed phlebotomy and medicines to achieve that, in addition to aspirin. So, that’s how PV is more unique than ET. Yes. 

What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Essential Thrombocythemia? from Patient Empowerment Network on Vimeo.

MPN specialist Dr. Abdulraheem Yacoub reviews the various treatment approaches available to patients living with essential thrombocythmia (ET).

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 
 

Related Programs:

 
How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

Advice for Choosing MPN Therapy: What’s Right for You?

Advice for Choosing MPN Therapy: What’s Right for You?

How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?


Transcript:

Dr: Yacoub: 

So, let’s talk about essential thrombocythemia. This is among the other MPNs, the cancer with the lowest risk. Patients with essential thrombocythemia can have clots and can have bleedings. And they also often have symptoms because of their cancer.  

But they also enjoy a long life expectancy that is almost indifferent from patients who don’t have cancer provided they get good care. So, our emphasis is on focusing that their life quality is not touched by their cancer, and focusing on treating patients with symptoms, to ameliorate the symptoms and allowing them to have a decent and good quality of life. At the same time, we would like to reduce the risk of clotting and bleeding.  

And we have tools and medicines that are very effective at doing that in select patients who we define as high risk. And now there is a more clear definition of that. So, high-risk patients are patients who are over age 60 and have a JAK2 mutation, or patients who have already had a clot.  

That is not the majority of ET patients actually. The majority are not high risk. And those patients might not require therapy to reduce their platelet count. 

But for high-risk patients, we have tools to help them. So, hydroxyurea (Hydrea) is the most commonly used medicine in this setting.  

The goal of hydroxyurea is to reduce the platelet count. And we’d like to keep it under 400, sometimes under 600 under different circumstances. And that will reduce the risk of clotting and bleeding for our patients. The other option, which I also feel passionate about is interferon.  

Interferons are drugs that we’ve used for decades. They’re very effective. They’re safe in the right hands. And they do have advantages over hydroxyurea in terms of long-term safety. These are medications we can give to young patients, we can give to pregnant patients, we can give for long term without concerns of toxicity, and also they have a higher ceiling. Patients with interferon can achieve a disease control that we cannot achieve with hydroxyurea.  

And this will be beneficial long term treating those patients. So, these – yeah, and then aspirin therapy is always something we would like to include in this regimen.  

Katherine:

I was going to ask you about that. So, aspirin is still being used as a treatment?  

 Dr. Yacoub:

Absolutely. So, the standard of care is to use aspirin. Usually, one baby aspirin once a day, preferably in the morning is what we recommend. And that’s probably all the aspirin they need. We do not want them to take more than that either.  

How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions from Patient Empowerment Network on Vimeo.

Dr. Abdulraheem Yacoub, an MPN specialist, reviews treatment goals for patients with essential thrombocythemia (ET), polycthemia vera (PV), and myelofibrosis (MF). Dr. Yacoub goes on to explain factors that may impact treatment decisions, including the management of symptoms and side effects. 

Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 

Related Programs:

 
What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Essential Thrombocythemia?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Polycythemia Vera?

What Are Treatment Options for Myelofibrosis?

What Are Treatment Options for Myelofibrosis?


Transcript:

Katherine:

To give our patient audience some context before we get into the specifics of MPN treatment approaches, how would you define treatment goals?  

Dr. Yacoub:

Thank you, thank you. And I always like to highlight and emphasize that unlike many of the cancer syndromes that patients deal with, myeloproliferative neoplasms are unique. 

These are chronic cancers. There’s no finish line. And this is a disease you live with. It affects every day of your life, every activity of your future life. You plan your life events accordingly. Pregnancies and marriages and trips and all of that. So, this is a chronic cancer. And as we plan therapy, we always factor that in. We would like the cancer to have the least or almost no impact on your daily life.  

Whether it’s symptoms, whether it’s disability and dysfunction and inability to perform your daily functions, whether it’s actual physical symptoms that you’re having from the cancer, or whether it’s affecting complications that are hurting your health. So, we would like to focus on all of these, the medical aspect as well as the impact of the disease to everyday symptoms.  

This is a unique feature of these cancers. And it doesn’t really exist much in other diseases.  

So, as we approach our patients, we would like to get a good assessment of the disease burden to their lives. These can be symptoms. So, we actually have very good objective tools to measure symptoms, such as the MPN-SAF. It’s an objective tool to calculate the symptoms. So, we would like to get an objective baseline of symptoms. 

Because we do want to address the symptoms, regardless of the MPN subtype. We do want to master actually the symptoms because that is what patients feel every day, and we want to affect that early in the treatment. We also would like to get a good assessment of the disease complications. Have the patient suffered a clot or a hemorrhage or symptoms because of an enlarged spleen? Or were they unable to perform certain activities? Are they able to eat? Are they losing weight?  

So, we would like to see how is the cancer also causing them immediate morbidity, and we also would like to tackle the future. So, cancers tend to get worse with time. They tend to transform into a higher risk cancer. So, as we approach any of the MPN patients, we also talk about the future risk of the cancer turning into a more aggressive form of cancer.  

So, we would like if we can, for every patient to focus on these three pillars of their care: their immediate quality of life and symptoms, their immediate complications, and their future disease progression.  

And we would like to factor in that our treatments does not add more side effects to their lives. So, that’s the fourth pillar of how we take care of patients. So, these are the basic concepts that will apply today for all patients with all three diseases.  

Some patients will have more emphasis on one or the other. But this is something in our mind as doctors who treat MPN patients.  

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Thriving with an MPN: What You Should Know About Care and Treatment

Thriving with an MPN: What You Should Know About Care and Treatment from Patient Empowerment Network on Vimeo.

 MPN specialist and researcher, Dr. Abdulraheem Yacoub, reviews factors that help guide care decisions for MPNs – essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). Dr. Yaboub discusses the goals of treatment, shares tools for taking an active role in your care, and provides an update on promising new therapies for MPNs.

 
Dr. Abdulraheem Yacoub is a hematologist oncologist at the University of Kansas Cancer Center. Dr. Yacoub is an active researcher and is an Associate Professor of Hematologic Malignancies and Cellular Therapeutics. Learn more about Dr. Yacoub, here.
 
 

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Expert Advice for Finding an MPN Clinical Trial


Transcript:

Katherine:                  

Hello, and welcome. I’m Katherine Banwell, your host for today. Today’s program is about how to live and thrive with an MPN. We’re going to discuss MPN treatment goals, and how you can play an active role in your care.

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

All right, let’s meet our guest. Joining me today is Dr. Abdulraheem Yacoub. Welcome, Dr. Yacoub. Would you please introduce yourself?

Dr. Yacoub:               

Hello, Katherine. And thank you very much for inviting me to participate in this very important and near and dear topic to my heart and to everything I do every day.

I’m a hematologist-oncologist at the University of Kansas. I practice hematology 100 percent of my time, and I dedicate it to patients with MPNs. I’m an active researcher through clinical trials at my own institution, as well as part of many national and international collaborations. We all strive to provide the best care and the updates for our patients. I’m also a Director of our hematology clinics in cancers at the University of Kansas, and I’m an Associate Professor of Medicine at the University of Kansas.

Katherine:                  

Well, thank you so much for taking time out of your very busy schedule to join us today. We appreciate it.

Dr. Yacoub:               

Absolutely, my pleasure.

Katherine:                  

To give our patient audience some context before we get into the specifics of MPN treatment approaches, how would you define treatment goals?

Dr. Yacoub:               

Thank you, thank you. And I always like to highlight and emphasize that unlike many of the cancer syndromes that patients deal with, myeloproliferative neoplasms are unique.

These are chronic cancers. There’s no finish line. And this is a disease you live with. It affects every day of your life, every activity of your future life. You plan your life events accordingly. Pregnancies and marriages and trips and all of that. So, this is a chronic cancer. And as we plan therapy, we always factor that in. We would like the cancer to have the least or almost no impact on your daily life.

Whether it’s symptoms, whether it’s disability and dysfunction and inability to perform your daily functions, whether it’s actual physical symptoms that you’re having from the cancer, or whether it’s affecting complications that are hurting your health. So, we would like to focus on all of these, the medical aspect as well as the impact of the disease to everyday symptoms.

This is a unique feature of these cancers. And it doesn’t really exist much in other diseases.

Katherine:                  

That’s helpful to understand as we move through today’s program. And we’re going to cover the three classic MPNs, polycythemia vera, essential thrombocythemia, and myelofibrosis.

So, for the person who has one of these conditions, can you help us understand the treatment approaches for each? Let’s start with essential thrombocythemia or ET.

Dr. Yacoub:               

Excellent. So, I’m going to start with some general concepts. So, as we approach our patients, we would like to get a good assessment of the disease burden to their lives. These can be symptoms. So, we actually have very good objective tools to measure symptoms, such as the MPN-SAF. It’s an objective tool to calculate the symptoms. So, we would like to get an objective baseline of symptoms.

Because we do want to address the symptoms, regardless of the MPN subtype. We do want to master actually the symptoms because that is what patients feel every day and we want to affect that early in the treatment. We also would like to get a good assessment of the disease complications. Have the patient suffered a clot or a hemorrhage or symptoms because of an enlarged spleen? Or were they unable to perform certain activities? Are they able to eat? Are they losing weight?

So, we would like to see how is the cancer also causing them immediate morbidity, and we also would like to tackle the future. So, cancers tend to get worse with time. They tend to transform into a higher risk cancer. So, as we approach any of the MPN patients, we also talk about the future risk of the cancer turning into a more aggressive form of cancer.

So, we would like if we can, for every patient to focus on these three pillars of their care: their immediate quality of life and symptoms, their immediate complications, and their future disease progression.

And we would like to factor in that our treatments does not add more side effects to their lives. So, that’s the fourth pillar of how we take care of patients. So, these are the basic concepts that will apply today for all patients with all three diseases.

Some patients will have more emphasis on one or the other. But this is something in our mind as doctors who treat MPN patients, we try to balance all these three pillars for every patient. So, let’s talk about essential thrombocythemia. This is among the other MPNs, the cancer with the lowest risk. Patients with essential thrombocythemia can have clots and can have bleedings. And they also often have symptoms because of their cancer.

But they also enjoy a long life expectancy that is almost indifferent from patients who don’t have cancer provided they get good care. So, our emphasis is on focusing that their life quality is not touched by their cancer, and focusing on treating patients with symptoms, to ameliorate the symptoms and allowing them to have a decent and good quality of life. At the same time, we would like to reduce the risk of clotting and bleeding.

And we have tools and medicines that are very effective at doing that in select patients who we define as high risk. And now there is a more clear definition of that. So, high-risk patients are patients who are over age 60 and have a JAK2 mutation, or patients who have already had a clot.

That is not the majority of ET patients actually. The majority are not high risk. And those patients might not require therapy to reduce their platelet count.

But for high-risk patients, we have tools to help them. So, hydroxyurea (Hydrea) is the most commonly used medicine in this setting.

The goal of hydroxyurea is to reduce the platelet count. And we’d like to keep it under 400, sometimes under 600 under different circumstances. And that will reduce the risk of clotting and bleeding for our patients. The other option, which I also feel passionate about is interferon.

Interferons are drugs that we’ve used for decades. They’re very effective. They’re safe in the right hands.

And they do have advantages over hydroxyurea in terms of long-term safety. These are medications we can give to young patients, we can give to pregnant patients, we can give for long term without concerns of toxicity, and also they have a higher ceiling. Patients with interferon can achieve a disease control that we cannot achieve with hydroxyurea.

And this will be beneficial long term treating those patients. So, these – Yeah, and then aspirin therapy is always something we would like to include in this regimen.

Katherine:                  

I was going to ask you about that. So, aspirin is still being used as a treatment?

Dr. Yacoub:               

Absolutely. So, the standard of care is to use aspirin. Usually, one baby aspirin once a day, preferably in the morning is what we recommend. And that’s probably all the aspirin they need. We do not want them to take more than that either.

Katherine:                  

And you mentioned using interferons for ET. That’s something that you would also use for polycythemia vera. Yes?

Dr. Yacoub:               

Absolutely. So, the same principles will apply to polycythemia vera. We would like to treat the higher-risk patients more aggressively. Hydroxyurea and interferon are also the first-line therapies in these patients.

The good news in 2022 is that we actually finally have an FDA-approved interferon for our patients. Finally, after 50 years of using interferon, now, we have an FDA approval. So, the new interferon, ropeginterferon alfa-2b is a medication that was studied prospectively in Europe, and it has been approved and in clinical use in Europe under the brand name Besremi.

And this year, it was approved in the US for patients with polycythemia vera, which is a great achievement for the medical field and a great tool to help our patients. We have used other brands off-label in the past, but it’s glad now to get this confirmation from the FDA that this is a standard of care for all patients.

And then beyond that, ruxolitinib or Jakafi, is also approved as a second-line option in patients who have had hydroxyurea as their first line.

So, these are the medicines we use for polycythemia vera. We also use therapeutic phlebotomy. And the goal in high-risk polycythemia vera, or actually in all patient polycythemia vera, is to reduce their hematocrit.

And we want it under 45 percent every day of the year. And we use the tools that we just discussed phlebotomy and medicines to achieve that, in addition to aspirin. So, that’s how PV is more unique than ET. Yes.

Katherine:                  

And since myelofibrosis is a progressive condition, I imagine it’s more difficult to manage. So, what else is available for patients with myelofibrosis?

Dr. Yacoub:               

Correct So, myelofibrosis is the higher end of this spectrum of cancers.

It is a cancer that is associated with much higher symptom burden and impact on daily life. It is also associated with low blood counts, and some patients will require transfusions. It’s a major morbidity to our patients. And in addition, it’s a cancer that is associated with shortened life. So, patients with myelofibrosis will not live as long as their health would have allowed them. And some of them will live actually a much shorter life than they want or deserve.

So, myelofibrosis treatment requires a lot more considerations. So, for patients who are in good health, who have a cancer that is more aggressive, that would be imminently impacting their longevity, we start a discussion about a curative role of allogeneic stem cell transplantation very early in their course.

Because bone marrow transplantation can be curative, and those patients can live a long life after a successful transplant. So, this is a treatment modality that should be brought up very early for patients with higher risk myelofibrosis. There are approved JAK inhibitors, ruxolitinib and fedratinib (Inrebic). And we know that Ruxolitinib which has been approved for over 10 years can improve symptoms, can improve the spleen volume, can actually prolong lives for patients on it, and also makes the transplant more successful.

So, we should be offering that to the appropriate patients also early in their diagnosis, in a strategy where, in addition to that, we get them to a transplant. Fedratinib is approved in that setting. And we are very optimistic that by the end of this calendar year, we will have two other JAK inhibitors approved.

So, we look forward to those two drugs. Momelotinib and pacritinib for patients with special disease features.

[Editor’s Note: As of February 28, 2022, pacritinib (Vonjo) has been approved for the treatment of myelofibrosis patients with severe thrombocytopenia.]

And hopefully, by the end of this year, we will have a list of JAK inhibitors that we can choose from, which is great news for our patients.

Katherine:                  

Oh, we’re still fighting.

Dr. Yacoub:               

Yes, absolutely.

Katherine:                  

As with most conditions, all patients are different, of course, and what might work for one person might not be appropriate for another. So, how do you choose which treatment is right for a patient?

Dr, Yacoub:               

Excellent. So, and that’s actually the heart of what we define as the art of practicing medicine and being patient-centric and focused. And patients already have their own wishes and their needs. And everything should start with having a discussion with patients on what is their priority, and what are they trying to achieve.

And we do have to explain to them the tools we have, the interventions that can help them. But we also need to make sure they’re compatible with what they actually want and their goals in life. And sometimes what we doctors want is not exactly what the patients want. So, we always have to remind ourselves that patients are the drivers of their care. And they have the absolute right to be informed and to make informed decisions based on the options we advise them about.

So, that is always a centerpiece of healthcare. And then patients – Basically, we defined four pillars of care. We want to control their symptoms, we want to prevent complications, we want to modify the disease so it doesn’t transform, and we want our therapies not to have toxicities, not to have side effects that are worse than the disease. So, we bring that up to the table. And we also look at the patient. What is their symptoms? What did the disease cause them to be complications?

What is the risk that their cancer is actually going to progress quickly to hurt their lives? And how serious is the therapy we’re recommending? And we need to make sure that there is a good match between what we’re offering and what the disease is manifesting. So, for example, for patients who have a lot of symptoms, but they have low-risk cancer that they can live with for a long time, we focus on symptoms. We focus on treatments that improve their symptoms.

While with patients who have more serious diseases that are eminently life-threatening, we focus on an expedited path to a more aggressive therapy and a bone marrow transplant. And then we also try to match those therapies with the other patient’s wishes and needs and so forth. So, all these factors are important. We have more tools to try to prognosticate. So, prognosticate is the medical word that we use as forecasting.

We like to forecast the disease or the cancer. We try to predict the patient’s future. Fortunately, we actually have good tools to prognosticate now. We have models or calculators that factor in patients’ features, their symptoms, their age, their blood counts, their bone marrow findings, and their DNA mutations. And it gives us a score a risk score that can correlate with their life expectancy or their outcomes.

And we use those tools to guide us. So, there’s actually a tool we use to help patients reach that decision. It’s an objective tool to decide how serious is this disease and how seriously we should tackle it. It’s very applicable for patients with myelofibrosis, more sort of the other lower-risk cancers.

Katherine:                  

What about comorbidities? How do they fit into the treatment plan?

Dr. Yacoub:               

Very important.

So, again, it also goes back to finding the balance between how serious is the disease, how serious is the treatment, and how will the patient’s general health tolerate and factor in the choices patients make accordingly. So, myeloproliferative neoplasms do happen in a broad range of ages. And we have children, minors with MPNs, and we have elderly patients with MPNs. And it’s a continuous spectrum. And each individual patient will have their own health concerns and their own health comorbidities and their own wishes. And we always have to make sure that we match our therapies, the disease seriousness, and the patient’s wishes, which is also stemming from their own other health battles, too.

We cannot turn a blind eye to the other health issues going on. That plays a major factor as we choose to discuss bone marrow transplantation with patients. Because that’s when the medical comorbidities are often the first barrier to go through.

Katherine:                  

Are there specific biomarkers that may affect prognosis or treatment?

Dr. Yacoub:               

Yes. So, and we’re glad that actually myeloproliferative neoplasms are actually the model in medical oncology on how predictors can tell us a lot more about the patient’s future about the prognosis. So, early on in MPNs, we’ve developed models, like the International Prognostic Scoring Systems in many different iterations. And more recently, the Molecular Based International Prognostic Scores.

They factor in patient’s age, they factor in blood numbers, they factor in DNA abnormalities, they also factor in DNA mutations, including the common driver mutations JAK2 and CALR and MPL, as well as more novel mutations that we call higher risk mutations.

So, based on these models, we use these tools to predict how the cancer will behave, and how to approach it. This advancement has been an application for our MPN patients for a while, way ahead of all other fields of oncology. So, we’re proud that we can give our patients this tool before all other doctors were able to. Yeah.

Katherine:                  

That’s excellent. Dr. Yacoub, what is the role of the patient in their care? When does shared decision-making come into play?

Dr. Yacoub:               

Absolutely.

Patients are the drivers and the centerpiece of their health care. And patient self-advocacy is the most important tool. So, many of our patients are young and they will live with their cancers a lot longer than many cancer doctors will practice oncology. And they will have many doctors. Statistically, each MPN patient will have multiple doctors throughout their career. And they will hear different derivatives. And the science will change. And they will be given different counseling over the time. And their disease will change.

And they will have different needs as they go further. So, patients being involved in their wellbeing and their cancer care is important from the first day. And I always tell patients, “You need to start building your village from day one.” It is not just the patient, it’s your caregivers, it’s who else can help you.

Who else can advise you? You might want to also invest in a friend or a spouse or a child, to come to you and listen to some of those discussions so that they can advise you later on, “Why are you making different decisions?” So, we encourage patients to be very involved early on, to build their own village, and to seek care. We routinely ask for second opinions. We want patients to always hear the story and hear the same story from another doctor so that they hear the range of how we word the truth and how we word the facts.

And this way, they can have a better perspective. So, this is now a standard. Almost all patients should have two doctors, at least, the treating doctor and one doctor who’s an MPN specialist, who would give them another twist or another perspective to their health.

So, and that is always important. And then there are very good references and online resources for patients to tackle in, such as this seminar and other good places where patients can seek more information. They also can go to a clinical trial to find out what are the ongoing clinical trials and advancements.

There are structured patient symposiums nationally and regionally. So, and we strongly recommend that patients seek more opinions and more help and more resources and be very engaged with this disease, especially that it is a chronic cancer, and it’s not going to –

Katherine:                  

It’s not going away.

Dr. Yacoub:               

It’s just a new lifestyle. And they need to be as engaged with it as they can.

Katherine:                  

Absolutely. We have a couple of questions from the audience. This one is from Sarah. She writes, “I’ve been living with essential thrombocythemia for three years, and have been relatively stable. Of course, I’m worried about progression to PV or MF. What is my chance of progression, and what are the signs of progression?”

Dr. Yacoub:               

That’s a very good question. And unfortunately, we’re very good at describing those numbers. Unfortunately, our tools at interfering are not as good. So, in general, patients with ET, statistically speaking, have a life expectancy that is not different from their age match peers. And Sarah’s story will be not too indifferent from her sisters and her mother, in terms of what’s going to happen to her long care and her health, provided she gets good medical care. The exception to that is that there is a transformation risk. For ET we caught around a 4 percent every 10 years in which ET will actually change into a different cancer, a higher risk cancer.

Could be MF, could be MDS, could be acute leukemia. And that will be a much more serious diagnosis. So, it’s about 4 percent in 10 years. We do have a – or we extrapolate some of the data from other cancers. So, certain mutations are more favorable, certain mutations are more risky. And we try to forecast that but worried it’s really hard to predict that since it’s such a long journey with disease. The first symptoms or the findings, when patients start suspecting that their disease has changed, is that the pattern of symptoms that they have are different.

They often become worse. So, they have more constitutional symptoms, more tiredness, more fevers, more night sweats, losing weight, not being able to eat a full meal, abdominal distension, the spleen gets bigger.

So, these are some of the feelings that patients can experience that lead to this. Other objective things is when the blood tests change in a less favorable way. So, for patients with ET who always run at 800,000 platelet count, if they’re suddenly 200, and that’s in the normal range, but that’s actually not good news, because the cancer changed. And this change is not favorable. So, as the doctors run routine labs, if they see the sudden change in labs, that’s also abnormal.

If the doctor can feel that the spleen gets bigger every time, that’s also concerning. If the patients suddenly have anemia or very high white cell count or immature white cells in the blood, that’s also a concern.

So, that’s why it’s great or important to establish a baseline symptom burden.                                   

A baseline spleen, a baseline bone marrow biopsy with mutation analysis, so that patients have a clear reference point to where they started, and if things change, they can always go back to that point and compare.

Katherine:                  

Right. We have another question. This one from Victor. He says, “I was diagnosed with PV in 2018. And I’ve been treated with hydroxyurea. Recently, I’ve been very fatigued. I want to exercise, but I don’t have the energy to do much. Do you have any advice for boosting my energy?”

Dr. Yacoub:               

That is a very good question and very common question. So, the causes for fatigue in adults, in general, so many. And adding PV to that adds a few other reasons why one would be more fatigued. So, assuming that Victor follows with his doctor, and his primary care doctor has systematically went through all the possible causes for fatigue, and those were addressed.

Now that PV specific causes, A). Hydroxyurea can cause fatigue. So, maybe it’s the hydroxyurea dose. And that’s a side effect. And maybe that’s not the best medicine for him. B). Polycythemia vera can cause fatigue. Maybe we’re not controlling it enough. Maybe we need to dial up the dose of the medicine or dial down the dose of the medicine accordingly. And then there’s also the iron deficiency which we induce with PV and phlebotomy.

And whether we actually have taken Victor to become very low on iron, and that can cause fatigue. So, we have to evaluate the treatments, the disease, and the side effects of the interventions we’ve done. And those are the polycythemia vera specific factors that can add to the fatigue.

Katherine:                  

Here’s another question from the audience. This is from Sandy. She writes, are MPNs hereditary? Should my children or siblings be aware of their risk?

Dr. Yacoub:               

All right. Well, the answer to that question changed many times over the last 10 years. So, the answer changed from absolutely not, to very possibly maybe over the years. So, although we don’t think of cancers as inherited, it’s not passed from one parent to their children. But MPNs tend to run in families. And for 11 percent of patients with MPN, and that number has also increased over the years, have actually a first-degree family member with MPN. That is a big coincidence, it’s almost too high to be a coincidence. So, we are realizing that there is genetic makeup or clustering that can cause MPNs to happen more often in certain families.

So, how does this apply to patients? So, if a patient has MPN, that does not mean that their children or siblings will get MPN, it just means they’re more likely than the other people to have MPN, just because they all share the same genetic makeup. And they should be made aware. And they should maintain good health care and maintain the relationship with a primary and have routine labs and all that. But not necessarily that they will get cancer. This still is a very rare disease, and 11 percent of a rare disease still is a small number.

Katherine:                  

Thank you for answering those patient questions. I appreciate it.

Dr. Yacoub:               

My Pleasure.

Katherine:                  

And to our patients, please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them on future programs.

So, Dr. Yacoub, as we close out our program and our conversation, I wanted to get your take on the future of MPN research and treatment. Are there new developments that you’re excited about and that make you hopeful?

Dr. Yacoub:               

Absolutely. So, again, I would like to take the last few minutes to advocate for the future. And the future can only come when doctors and patients and advocates work together to advance the science. We have few tools to treat patients and to help patients. We have a lot of unanswered questions. And the only way to answer them is by designing quality clinical trials, enrolling patients on trials, taking the risk, and trying to find new answers and new therapeutics. So, I always would like to advocate for patients to seek clinical trials whether with their doctor or whether they have to travel for it, and for doctors to consider that for their patients. That’s the only way to advance science.

There are very important national and international studies going on right now. One of the – And first, I would like to emphasize is that we have had ruxolitinib as the only therapy, or the first-line therapy for myelofibrosis for a decade now. Not everybody responds to it, not everybody responds to it for a long time. So, now we’re designing combination trials. So, there’s a few studies that are trying to redefine, “Is ruxolitinib alone enough, or should we have a combination first-line therapy?” So, these are some of the more important questions being asked right now.

And this is definitely one of the bigger moves in the field, is trying to redefine what is the first-line therapy for myelofibrosis. For polycythemia vera, we’re also exploring therapeutics that would reduce phlebotomy with things you can add to your medical care to reduce phlebotomy.

So, that’s also going on. And it’s definitely a big leap forward for many of our patients. For ET, when we don’t have any actual drugs approved other than hydroxyurea and anagrelide (Agrylin), we actually have trials with interferon going on.

So, I would like to advocate for that. So, interferon succeeded and now approved for PV, but not yet for ET. We’re working on that. So, again, in every disease, we’re trying to design clinical trials to redefine what is the best treatment today.

We’re also doing studies to understand the cancer. So, studies where patients donate their samples for research. These are very helpful, very important. And contributing to that always advances the science, and it’s low effort to the patients. So, if there’s a clinical trial that is offered to patients, I would strongly urge everybody to consider that favorably and contribute to science.

That’s the only way we can help future patients and ourselves immediately, at moving the field forward.

Katherine:                  

Seems like there’s a lot of progress in the field.

Dr. Yacoub:               

A lot of progress. I look forward to future events. I’m going to have a lot more tools to discuss. Hopefully, by this time next year, we’re going to have four JAK inhibitors, injectables for PV, interferon for ET, and a lot more things to go over.

Katherine:                  

That’s wonderful. Dr. Yacoub, thank you so much for taking the time to join us today.

Dr. Yacoub:               

You’re welcome. And it’s my pleasure. I feel passionate about this. And I’m happy to help.

Katherine:                  

Thank you. And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a productive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us today.

Updates from ASH: How Biomarker Testing Has Changed MPN Care

Updates from ASH: How Biomarker Testing Has Changed MPN Care from Patient Empowerment Network on Vimeo.

MPN specialist, Dr. Andrew Kuykendall, discusses how the identification of specific biomarkers in myeloproliferative neoplasms (MPNs), such as the JAK2 mutation, have moved research forward. Dr. Kuykendall shares promising findings that were released at the 2021 American Society of Hematology (ASH) annual meeting and how this may impact MPN care in the future.

Dr. Andrew Kuykendall is an Assistant Member at Moffitt Cancer Center in the Department of Malignant Hematology. Dr. Kuykendall’s clinical and research efforts focus on myeloproliferative neoplasms (MPNs), MDS/MPN overlap syndromes and systemic mastocytosis (SM). Learn more about Dr. Kuykendall, here.

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Transcript

Katherine:

How has molecular or biomarker testing changed the field of MPN care and treatment?

Dr. Kuykendall:

Well, I think, first and foremost just understanding – going back to 2005 and knowing that we have JAK2 mutations. I think that gave really a lot of clarity to the diagnosis and really understanding the biology of how the disease acted through the JAK-STAT pathway. And certainly, that led to the understanding of MPL mutations and then calreticulin mutations.

We’re still figuring out exactly how calreticulin mutations work. There was a great abstract, a preclinical abstract, this year talking about the impact of interferon on calreticulin mutations and how that may differ from what we see in the impact of interferon on diseases that are driven by JAK2 mutations.

Clinically, we see a little bit of difference in how those diseases respond and we may understand a little bit better about why that happens. Additionally, that’s kind of gone down to looking at these big next generation sequencing panels where we identify high-risk mutations and that can certainly change our understanding of the prognosis of these diseases.

We’re starting to get, at least in the AML world, we’re getting targeted agents that can potentially target some of these mutations such as IDH1 and IDH2 mutations that have specific inhibitors.

Those are mutations that occur in myeloproliferative neoplasm patients and convey a worse prognosis, so there are ongoing trials looking to see if we can use those IDH inhibitors in myeloproliferative neoplasms either in the chronic phase or maybe in the more accelerated advanced phase.

You know the big thing, this meeting, was actually looking at polycythemia vera patients and what’s the relevance of the JAK2 mutant allele burden. I think this is something we’ve talked about a lot as far as how significant this is. We know in chronic phase myeloproliferative neoplasms that that JAK2 mutation tends to be associated with more thrombotic complications.

There are more blood clots in the veins and the arteries. There were a couple great abstracts that looked at the really the implications of the JAK2 mutation and the fact that it is associated with more thrombosis, but maybe more venous thrombosis. That might be a big risk factor for venous thrombosis and it may be that cardiovascular risk factors, such as diabetes, hyperlipidemia that’s really what’s driving the arteriole thrombosis. It also looked at the variant allele fraction, the number of cells that have that JAK2 mutation.

One abstract showed that if you have over a 50 percent allele fraction, if more than 50 percent of the alleles have the mutation – a higher burden of that mutation that’s associated with an increased thrombotic risk even in low-risk polycythemia vera patients. Whether or not that’s enough evidence to really change the paradigm of how we treat low-risk patients is to be determined, but I think very interesting and provocative work.