MPN Treatments and Clinical Trials Archives

When it comes to treatment, MPN patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for Myeloproliferative Neoplasms (MPN) Treatments and Clinical Trials from Patient Empowerment Network

Three Reasons MPN Patients and Their Families Should Continue Telemedicine

What are some reasons why MPN patients and families should continue to use telemedicine visits? In the “Should MPN Patients and Their Families Continue Telemedicine?” program, expert Dr. Kristen Pettit from Rogel Cancer Center shares three ways myeloproliferative neoplasm (MPN) patients and families can benefit from continuing to use telemedicine for care.

1. Access to Care

One of the most impactful benefits to patients from the COVID-19 pandemic has been the emergence of telemedicine. Telemedicine has brought improved access to MPN experts, care teams, and specialty cancer centers via smartphone, tablet, or computer to broaden expert care for patients and care partners. Many family members or friends can now join telemedicine visits to help ask questions and to take notes for more engaged patient care.

2. Safety and Travel Benefits

The use of telemedicine has also brought safety and travel benefits. Patients and care partners have reduced the amount of time they spend in clinics and waiting rooms. Their risk of infection with viruses, bacteria, and other illnesses has been decreased when some of their MPN care appointments can be carried out remotely. And reduced travel to appointments helps patients and care partners in lowering  travel costs and stress related to commuting or scheduling rides.

3. Remote Monitoring

MPN patients must also empower themselves to keep track of their health concerns to ensure their remote monitoring is reported accurately. Patients and care partners can monitor any weight changes, enlargement or feeling of fullness with their spleen, feeling overly tired or fatigued, and other symptoms their care provider has mentioned as symptoms to be aware of. If you have any questions about symptoms, make sure to ask your doctor.

By continuing to take advantage of telemedicine, MPN patients and care partners can reap the benefits of remote care paired with in-person visits as advised by your doctor. If you’d like to expand your knowledge, check out our MPN information.

Thriving With MPNs: Your Role in Managing Your Treatment and Care

Thriving With MPNs: Your Role in Managing Your Treatment and Care from Patient Empowerment Network on Vimeo.

 How can patients thrive with a myeloproliferative neoplasm (MPN)? Dr. Jeanne Palmer discusses treatment approaches, strategies for managing disease symptoms and treatment side effects, and advice on how patients can be proactive in their care.

Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.

 

Related Programs:

 
How Should You Participate in MPN Care and Treatment Decisions?

How Should You Participate in MPN Care and Treatment Decisions?

What Are the Signs of MPN Progression?

What are the Signs of MPN Progression

Expert Perspective: Hopeful MPN Research and Development


Transcript:

Katherine Banwell:

Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to talk 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. 

Well, joining us today is Dr. Jeanne Palmer. Dr. Palmer, welcome. Would you please introduce yourself? 

Dr. Jeanne Palmer:

Thank you so much. I am so happy to be here and to help participate in this. My name is Jeanne Palmer. I am a hematologist at Mayo Clinic in Arizona. I specialize in MPNs as well as bone marrow transplant, and I am thrilled to be here. 

Katherine Banwell:

Thank you for taking the time out of your busy schedule to join us today, Dr. Palmer. We start all of the webinars in our Thrive series with the same question and that is, what does it mean to you to thrive with an MPN? 

Dr. Jeanne Palmer:

I think living with an MPN can be very difficult. I think there is a number of things. First of all, there’s always the worry of what’s going to happen in the future. Many of these MPNs can start as fairly, for lack of a better term, as benign issues and can convert to something much more serious. So, I think living with that sort of timebomb in the back it can be extremely stressful. So, figuring out how to live with the fact that there is some degree of uncertainty. 

I think the other thing is making sure to understand your disease. These are very rare disorders and even if you go to a hematologist-oncologist specialist, a lot of times they don’t have all the information because they don’t see a lot of them every year. So, it’s really important to make sure that above and beyond that you understand what’s going on in your body so that when new things happen, new symptoms happen, you’re able to really address them as opposed to sort of living with something that may make you feel poorly that’s not being addressed.  

So, again, I think the biggest piece of this is seeing how do you live with uncertainty, and how do you make sure you understand your disease well enough that you know what’s going on in your own body. 

Katherine Banwell:

Yeah. That’s helpful to understand, especially as we move through today’s program, and we’re going to cover the three classic MPNs, polycythemia vera, essential thrombocythemia and myelofibrosis. One part of thriving with an MPN is managing the symptoms of the disease. Would you walk us through the common symptoms of each of the MPNs? Let’s start with essential thrombocythemia. 

Dr. Jeanne Palmer:

Right. So, there are a number of shared symptoms throughout all the diseases and when we start to figure out how to categorize them, they call into several different categories. The first one is inflammation-related symptoms. We know that the inherent pathway that’s dysregulated or that causes these diseases to happen can also result in significant inflammation in a person, that can result in things like fevers, night sweats, weight loss, and overall feeling really fatigued and poorly, which is something that it seems to be much more prevalent in patients with MPNs, all sorts of them, actually. 

The next set of symptoms is related to microvasculature, so all the little blood vessels. And sometimes we think, oh, maybe that’s because there’s too many red blood cells or platelets and the blood become viscous. It’s probably more related to the actual dysregulation of that JAK2 pathway, which is inherent to all the myeloproliferative diseases and as a result, the little blood vessels can clamp down and that can give people headaches, visual changes, numbness and tingling in the hands and feet, and even can cause sort of a painful rash called erythromelalgia in the body. 

So, these are things that can happen that are probably less appreciated side effects of the disease. And finally, there’s spleen-related symptoms. The spleen is in the left upper quadrant of the abdomen and it’s an organ that generally is about 12 centimeters in length, 10 to 12, but in patients with myeloproliferative diseases it can be enlarged. And as a result of an enlarged spleen people can have feeling like they get fuller early. So, if you’re eating a meal, all of the sudden you can only eat half of that meal versus the whole meal.  

Discomfort or pain in the left upper quadrant. Sometimes it’s much more noticeable when you like bend over to tie your shoes. And then sometimes people can actually, when the spleen gets really big, the blood flow can be impaired towards the end of it which can cause some of the spleen tissue to die, and that can be painful. So, these are things that if somebody does start to notice that they’re having fullness in the left upper quadrant, pain, stuff like that, that that may be related to spleen symptoms. 

Katherine Banwell:

What about PV or polycythemia vera, what are the symptoms? 

Dr. Jeanne Palmer:

So, all of these sorts of relate to all of the myeloproliferative diseases. So, one other one that I didn’t mention, and this is actually more in PV than others, is itching. Itching can be absolutely unbearable when somebody has PV. It’s particularly noticeable after taking a shower. So, a lot of times I’ve met patients who are like I haven’t been able to take a shower in years, because it causes such a high degree of itching. 

Katherine Banwell:

Why a shower? Is it different from having a bath?  

Dr. Jeanne Palmer:

Water on the body that can cause the problem. So, if people take hot showers, it’s even worse. Although I think that people sort of react to it differently. Usually what patients end up doing is more like sponge bath type of things, rather than actually being exposed to the water. 

Taking colder showers or cooler showers can sometimes help mitigate that. But the itching, and even in the absence of a shower, people can have pretty severe itching, and that can also be one of the major side effects. 

Katherine Banwell:

Much of the time the chosen treatment for MPNs manages the symptoms of the condition. I’d like to review the different types and classes of treatment for the three MPNs. So, let’s start with essential thrombocythemia again. When is it time to treat, and what are the options available?  

Dr. Jeanne Palmer:

Right. So, with essential thrombocythemia, that’s the disease that sometimes we don’t need to treat. 

So, we basically have a risk stratification system and this risk is based on age, history of a blood clot, the presence or absence of a JAK2 mutation. So, for example, if somebody is 28, does not have a JAK2 mutation, which is again one of those driver mutations, and never had a blood clot, they actually don’t necessarily need to do anything and just be monitored.  

Somebody who is less than 60 and has a JAK2 mutation or who is greater than 60 and does not have a JAK2 mutation, in that setting, a lot of times you can use aspirin. Now, it gets a little bit gray in terms of that over 60 without the JAK2 mutation with regards to whether at that point you really should start taking some medicine to lower the platelets. 

Now, if somebody has a JAK2 mutation, is greater than 60 or has had a blood clot, hands down they need to take medicine to lower the platelets, in addition to aspirin or whatever blood thinner they may need. So, for example, if you have a blood clot in a vein, a lot of times you need to take a blood thinner and that will be a lifelong thing. And again, we do these risk stratifications because we know there is a certain risk of clotting associated with the risk of essential thrombocythemia. 

So, for example, somebody who is less than 60 and does not have a JAK2 mutation, never had a clot, their risk of clotting is probably very close to that of the normal population. Whereas if you’re higher risk and have a JAK2 mutation and greater than 60 or have had a history of a clot, the risk of clot is probably about 4 percent per year. So, this is something that can vary quite widely, and even though that 4 percent per year on the short-term doesn’t sound like a lot, if you take it additive over years, that’s why we generally try to be aggressive about lowering the platelets.  

In lowering the platelets, the goal is to get less than 400 and doing that can be done through several different medications. The most commonly used medications is a drug called hydroxyurea, which has been around for a number of years, and a drug called anagrelide which is probably a little less commonly used, because it has some more GI side effects and headaches associated with it. 

In some cases, especially in younger patients with this disease, we can consider using interferon, which is an injection of a cytokine, which are one of the chemicals that regulates the immune system within the body. But this interferon can actually help lower the platelets and there is a question of whether it may affect the biology of the disease as well. 

Katherine Banwell:

Let’s turn to polycythemia vera or PV, what are the different options available for treating it? 

Dr. Jeanne Palmer:

So, for polycythemia vera, everyone needs to be on aspirin. 

And additionally, everyone needs to make sure to keep their blood count low, to manage their hematocrit, which is one of the measures of red blood cells. So, in men it’s generally recommended to keep below 45 and in women it’s recommended to keep below 42 percent.  Now, the studied number was 45 percent and that was a study that was done, I don’t know, it was probably about 10 plus years ago, that actually showed that by keeping the blood hematocrit less than 45 percent you reduce the risk of having negative events like cardiovascular events and heart attacks. Because women tend to run with a lower blood count than men, it’s been extrapolated that 42 percent should be the number used for women. 

Now, this can be done by phlebotomy, which essentially is bloodletting. It’s kind of like donating blood except for that the blood unfortunately can’t be donated to anybody, it has to be discarded. But the phlebotomy is one way to do that, and the reason that works is because it makes somebody iron deficient. So, whereas if this is normal, if you’re iron deficient you become anemic. If your baseline hematocrit is here, making you iron deficient brings you back to normal. So, even though we always associate iron deficiency with anemia, iron deficiency in the setting of polycythemia vera is actually kind of a treatment of sorts. 

Now, once somebody gets above 60 and 60 seems to be sort of the magic age in these diseases, once somebody gets above 60, it is recommended that cytoreductive therapy is used, which means therapy or treatment that will bring down the red count. And again, for this one, hydroxyurea is an option as well as interferon. And there is recently an approval, actually FDA approval for a newer interferon called ropeginterferon or Besremi, which can help just bring down the red blood cells but it is the first interferon that’s actually been FDA approved for this indication. 

Katherine Banwell:

Are JAK inhibitors used as well? 

Dr. Jeanne Palmer:

They are. So, if somebody doesn’t respond well to hydroxyurea, the approval for ruxolitinib is actually for patients who have failed hydroxyurea. Although it’s something that we often consider especially in people who have a lot of symptoms. So, the itching, one of the things that can really help itching actually is Jakafi. If people have night sweats, they have weight loss, spleen related symptoms, those are the patients that will benefit from Jakafi. Additionally, if they are on hydroxyurea and can’t seem to get control of their blood count, Jakafi is a good option to help control the blood counts as well. 

Interferon is a very nice option because there’s great data that shows that you may actually be able to lower the percentage of JAK2 burden. 

So, we’d look at something called an allele burden, which is the percentage of cells that are involved – have the JAK2 mutation. Now, we don’t know whether lowering this percentage necessarily translates to long-term better survival, but I think there is enough data out there, and there is a good biologic underpinning for saying that this actually can help. But yes, Jakafi is another thing. 

And the really exciting thing is that there is a newer agent called rusfertide, which is a hepcidin mimetic, which is basically taking a protein in your body that helps metabolize iron and by making it externally and giving it to somebody that it can actually help bring down the hematocrit without having some of the other side effects we know with some of the other medications. That is currently in Phase III studies, so hopefully in the next couple of years we’ll see approval for that. 

Katherine Banwell:

Oh, that’s great news. And finally, how is myelofibrosis treated? 

Dr. Jeanne Palmer:

So, myelofibrosis is a little bit of a different animal. When you have something like essential thrombocythemia or PV, a lot of this is managing symptoms, preventing blood clots, but if you do appropriate treatment and management of these diseases you could probably live close to a normal life expectancy.  

So, I never typically pin a survival on it. With myelofibrosis, it’s a little bit different because there is a survival. Instead of saying you can live close to normal life expectancy, it backs up to saying how many years do I think you can live with this disease. Now, of course, we are horrible at predicting how many years anyone can live, so we have to take that all with a grain of salt. But we can at least sort of risk stratify people. 

And the first thing that’s really important is to figure out whether somebody is a transplant candidate or not and if, based on age, disease risk features, stuff like that, or whether we think they ever will be a transplant candidate. So, that kind of helps us sort of think about what your path moving forward is.  

Now, the current FDA-approved treatment for myelofibrosis, there are three JAK inhibitors approved, which is like Jakafi, which was the first approved one but there is also Inrebic or fedratinib and Vonjo or pacritinib and these have all been approved over the years. 

The role of JAK inhibitors and treatment of myelofibrosis is symptoms-based. So, for example, a lot of patients with myelofibrosis will have weight loss, night sweats, big spleens, really feeling fatigued and poorly and in this setting, the JAK inhibitor can be very helpful. And you don’t have to have a JAK2 mutation, a lot of times people say, well, I don’t have the JAK2 mutation so how can a JAK inhibitor help. So, the JAK inhibitor works on this pathway, which is called the JAK/STAT pathway, irrespective of mutation. 

So, if you are having symptoms and you have myelofibrosis, JAK mutation, excuse me, the JAK2 mutation does not predict who is going to have a response. And people who, regardless of which mutation you have, may actually benefit from it. 

So, the JAK inhibitors, though, are extremely effective at reducing symptom burden as well as reducing the spleen size. And we know that if a spleen is big and we can make it shrink that, that probably is a surrogate marker for living longer, and I think it’s because inflammation does a lot of wear and tear on the body. So if you can reduce the inflammation and the spleen shrinks, which generally go hand in hand, then you might help somebody live longer. It is not changing the biology of the disease, though, however, it doesn’t change the pathway and that this disease is kind of projecting ahead in terms of creating – it changes, as it goes along, may acquire new mutations or something like that which makes the disease become more serious. 

Right now, the approved therapies for it are JAK inhibitors and the Jakafi, ruxolitinib was the first one approved. Inrebic was approved several years back, or fedratinib. 

And then the most recent one that was approved is Vonjo or pacritinib and that’s a drug that is a JAK inhibitor that is actually very good for people with low platelets. The reason I bring that up is because if we think of what’s the biggest limiter of JAK inhibitors, JAK inhibitors bring down red blood cells, and they bring down platelets. So, when somebody has low platelets it’s very hard to use a JAK inhibitor, because we’re not really able to increase the dose well enough to get that inflammatory reduction because of the fact that the blood counts will drop too low. 

So, now drugs like Vonjo exist which, due to several other mechanisms associated with the drug are actually much more tolerated in somebody with low platelets. So, if you have low platelets, you can actually take the Vonjo, hopefully get the same degree of JAK inhibition to help the spleen shrink, help the symptoms get better without necessarily making the platelets substantially worse. A lot of times they do drop, it doesn’t help bring up the platelets, but it does help people tolerate more JAK inhibition, which ultimately will help with symptoms.  

Dr. Jeanne Palmer:

So, one thing I also wanted to add about myelofibrosis treatment is sometimes people present, they don’t have a lot of symptoms, they don’t have a lot of spleen related problems but they have anemia or low blood counts and these can be incredibly hard to treat. 

Even with symptoms and low red blood cell count or anemia or low platelets, it can be challenging to treat because many of these medications lower that. To treat the anemia there are several things that we can do. One of the first ones is using erythropoietin, and so there are many agents, they go by the names of like Procrit or darbepoetin alfa, that actually stimulate red blood cell growth by – like we give a recombinant hormone that helps red blood cells grow. This is normally something produced by the kidney. 

So, one thing that’s important before going on one of these injections is to make sure that the kidney is not already producing enough. So, for example, if the kidney said, oh geez, I really need more red cells and is making lots of this hormone, erythropoietin, giving more of it is not going to help the system. But in people who don’t have a really high level it can be very beneficial. 

The other thing that can help with anemia, specifically, is a drug called danazol (Danocrine).  

It’s been around for a very long time. There are multiple presumed mechanisms of action, but one of them is that it is kind of a testosterone derivative. So, this is a medicine that can often help increase red blood cells in probably about 40 percent of people, and it’s a pill that you take twice a day. 

Another option, sometimes we use thalidomide or lenalidomide (Revlimid). These are medications that have been used quite frequently in the setting of multiple myeloma and even a little bit in myelodysplastic syndrome, so some other blood disorders.  

But in the setting of myelofibrosis, they can be helpful with anemia and sometimes are combined with prednisone or a corticosteroid. And then finally, in terms of drugs that are being tested and hopefully will be approved at some point in the future. There is a drug called momelotinib, which is another JAK inhibitor that actually has some mechanisms that may also help improve hemoglobin. 

So, this is something I’m really looking forward to and we anticipate may be approved by the end of the year. And finally, there is another drug called luspatercept (Reblozyl). Luspatercept may work in the setting where your kidneys are already producing enough erythropoietin. So, the luspatercept is an injection that you receive once every three weeks.  

It is currently FDA-approved for the treatment of myelodysplastic syndrome but this is something that has been shown to have some efficacy in myelofibrosis as well. So, this could be another therapeutic option for patients with myelofibrosis. 

It is also important, especially for people who have polycythemia vera myelofibrosis to make sure that your iron has been checked and B-12 has been checked, because just because you have a bone marrow disorder doesn’t necessarily mean you don’t have a nutrition deficit that may be able to help improve your hemoglobin somewhat. But these are important things to talk to your doctor. I do not recommend just starting to take iron or B-12, however, if you’re anemic because in many cases you are not deficient and taking too much iron can actually be damaged. 

Katherine Banwell:

Yeah, that’s great advice.  

When would you consider a stem cell transplant? 

Dr. Jeanne Palmer:

So, the stem cell transplant is based on disease risk. There is a number of ways we assess disease risk. The first two ones that were published a number of years back were the DIPSS score, which is Dynamic International Prognostic System Score, or the DIPSS Plus, which basically is the DIPSS and then you add to it a few other clinical features. This symptom score is based largely on things that we can see without even a bone marrow biopsy, so things like symptoms, age, number of white blood cells, whether somebody has anemia. And then the number of something called blasts, which is very immature white blood cells. The DIPSS Plus takes into account low platelets, need for transfusions, and chromosome abnormalities, which is the only test among that that needs to be from a bone marrow biopsy. 

Now, these were created prior to Jakafi being commercially available. So, we have to take a little bit of a grain of salt with those because of the fact that Jakafi probably has changed how long people can live with this disease. 

Now, more recently they’ve tried to account for these other molecular changes. So, when we take the genetic landscape of these diseases, we have the known driver mutations, so the JAK2 mutation which I have talked about, also calreticulin and MPL.  

These three mutations all affect that one pathway, the JAK/STAT pathway, so they all affect the pathway that drives the disease and they are known to be kind of mutually exclusive and definitely contribute to the formation of the disease.  

Some of these other mutations are called somatic mutations. They could be checked by things next generation sequencing or genetic analysis. There’s a number of different names that people use for this testing, but we look for mutations that are present and these mutations, number one, can sometimes tell us risk. So, there’s certain mutations that are high risk. Other times it can actually give us other opportunities for therapy, especially of the disease progresses. But these mutations are important to know for risk stratification. For example, if somebody has DIPSS score that is maybe not super high risk, but then they have one of these mutations, we know that that probably makes their disease a little bit more aggressive. 

And that’s when we think about transplant, is when we know that the disease probably has an average life – when somebody gets to the point in their disease where we estimate their life expectancy is around five years, recognizing that we’re not very good at this. That is the type of point when we start to think about transplant. But the timing of transplant is something that’s extremely difficult and a very personalized decision. It’s something that it’s really important to understand the disease risks, how we assess them and the caveats of these disease risk assessments as we move forward planning and timing of transplant and that’s something that is, again, a very, very important discussion to have at length with your physician. 

And I always recommend, there is quite a few of us out there who actually specialize in transplant for myelofibrosis and having discussions with somebody who really understands the biology of the myelofibrosis and important because it’s very different than a lot of the other diseases that are transplanted.   

Katherine Banwell:

Yeah. Well, speaking of that, patients can sometimes feel like they’re bothering their healthcare team with their comments and questions. Why do you think it’s important for patients to speak up when it comes to symptoms and side effects?  

Dr. Jeanne Palmer:

Well, there is a lot of things. This is a disease, again, that we can direct our therapy many times towards symptoms, and so when we think about how do I direct my therapy, so how do I treat somebody, symptoms are an incredibly important part of it. And there is nothing worse than having a patient come and see me who I see every six months, because they’ve been pretty stable and they’re like, “Oh, for three months I’ve been feeling awful.” And you’re like, well, “Why didn’t you let me know, we could do something about this?” 

So, if there is something that doesn’t feel right, it’s very, very important to talk to your healthcare provider. I would much rather be bothered and handle something earlier on than miss something and really have a lot more catch-up to do afterwards. 

The other thing is symptoms may indicate a blood clotting event. We know that patients will have a higher risk of blood clotting. These are extremely important to identify early on because if they go unchecked, they can cause more damage. 

Katherine Banwell:

With many of the treatments available as pills now, patients have a role in self-administering their treatment regimen. What happens if a patient forgets to take a medication? Does it impact its effectiveness? 

Dr. Jeanne Palmer:

Generally no. I think the ones that would are certain blood thinners you really don’t want to miss and you don’t want to miss the doses on it. With drugs like Jakafi, if you miss one dose you probably won’t notice it, but if you miss multiple doses you can actually get very sick from that. So, some of these medications are really important to be consistent on. 

Now, I know this could be a challenge. I mean I don’t take very many medications and I sometimes have a hard time keeping track of what I take, so I know that this can be a difficult thing to do. So, one thing is if you really find you’re struggling with it, setting an alarm on your phone or your Apple Watch or whatever… 

Katherine Banwell:

…device. 

Dr. Jeanne Palmer:

Device you have can be a really helpful way of doing it. Also having a pill box. They make pretty amazing pill boxes these days that can account for taking drugs once a day, twice a day, three times a day. I’ve even seen them up to four times a day, although generally the most you’ll probably have to take a medicine for a myeloproliferative disease is twice a day. But those are different ways that can really help make sure you’re consistent about taking your medication. 

Katherine Banwell:

And if a patient misses a dose, do they need to call their healthcare team and let them know? 

Dr. Jeanne Palmer:

Not just for one missed dose. If like, for example, they’re run out and they say, “Oh, geez, I don’t have any and many of these drugs are specialty pharmacy,” so they need to be mailed, and you know that you’re going to be missing it for a while. Or let’s say you look at your pill bottle and go, “Oh shoot, I only have so many pills left,” it is helpful to call because a lot of times, for example, if somebody is on Jakafi and they know they’re going to run out of their pills four days before they’re going to get their next shipment in, then what I sometimes do is I lower the dose a little bit to make sure they maintain a dose throughout that time. 

But this is something you definitely want to do under the advice of a healthcare provider. You don’t want to just all of the sudden go, “Oh, well I’m going to run out so I’m just going to change my dose,” and kind of do that. 

Katherine Banwell:

Yeah, yeah. We received some audience questions prior to the program today. This one is from Jacqueline, “What can I do to minimize pruritus or itching due to PV? A typical histamine blocker like Claritin or Zyrtec has done nothing whatsoever.” 

Dr. Jeanne Palmer:

Yeah. Unfortunately, the itching of this is not as much mediated by an allergic type reaction or histamine. It’s a lot related to that microvasculature, those tiny little blood vessels. Things like avoiding hot showers, as we talked about, taking cooler showers or not even taking showers, just like cleaning yourself with a washcloth can be helpful. There are certain medications that we can use sometimes that help. 

Now, first of all, Jakafi is extremely effective for itching. Of course, it does have side effects. It’s not always approved for your disease, so for example, it’s not approved for essential thrombocythemia. But JAK inhibitors can be helpful in that setting. There are also medications like Gabapentin, which is a medicine that we use to treat peripheral neuropathy and that can actually be helpful because actually the itching, a lot of it is related to nerves not functioning right, so gabapentin can be helpful. 

And a really old-school medicine that I sometimes use, especially if the itching is most prevalent at night, is a drug called Doxepin and that’s been around for a very long time, but it can be extremely sedating and has to be used with caution, especially in patients who are older. 

Katherine Banwell:

Here is a question from Daniel. “How often should a person with PV have hematology appointments, and how often should you have blood tests?” 

Dr. Jeanne Palmer:

Well, that is something that you need to discuss with a provider, because everyone’s a little bit different. If I have somebody who I’m managing on a medication, they’ve been rock solid stable, it may be every few months that I check blood and maybe every six months that I see them.  

If I have somebody who have been particularly difficult to control and I’m sort of adjusting medications or they’re having symptoms, then I try to check blood more regularly, like on a monthly basis. But again, this is something that – I have checked blood as frequently as every two weeks, especially in somebody who has an extremely high red blood cell count that I’m trying to lower. I have checked blood as infrequently as every three months. Again, in somebody who is not undergoing treatment, say, for example, who has essential thrombocythemia, sometimes I check blood even less. So, it really is something that can vary from every two weeks to every six months. 

Katherine Banwell:

Okay. Katie had this question. “What are the signs of progression from PV to MF or AML, both clinically and in blood tests, and when do you need a new bone marrow biopsy to check for this happening?” 

Dr. Jeanne Palmer:

So, in terms of progression, there are several things that we see happen. 

I think most importantly is, let’s say you have PV, and you’ve always been on medication, and it’s been hard to control. And all of a sudden, you don’t need medication to control it anymore, or the same thing for essential thrombocythemia. You have been taking medication, and all of a sudden your platelets go down, and you don’t need to take drugs anymore. A lot of times people are like, “Oh, that means I’m fixed and I’m well,” not necessarily, you really need to make sure to talk to your healthcare provider and potentially get a bone marrow biopsy. 

Now, the other thing – sometimes the blood counts will actually drop too low, so you’ll have somebody who has PV, who has always been too high and then all of the sudden they come in, and their hemoglobin is very low, and they’re anemic, and that’s another situation where you do that. So, anytime the blood counts start to drop is concerning.  

Now, it’s a continuum, so the blood counts may drop as you’re at the point of transitioning but it doesn’t – it’s not like if your blood count is dropping you say, “Oh my God, I have myelofibrosis, I need a bone marrow transplant tomorrow.” That’s not necessarily the case. This is generally a transition type process. 

Also when the spleen starts to get enlarged. Now, the spleen can be enlarged even in the setting of just ET or just PV, so spleen enlargement does not necessarily mean you’re transforming, but it can be one of the things that we would see that would indicate that. 

Katherine Banwell:

Okay. 

Dr. Jeanne Palmer:

And then finally white blood cell count increasing can often be a sign of that. Now, in terms of progression to AML, that is generally something we’ll see in the blood. AML or acute myeloid leukemia, is indicated by the presence of blasts at greater than 20 percent. Now, many patients with myelofibrosis, in particular, but even PV and ET, may have blasts in their peripheral blood. Blasts are normal. If I did a marrow on every healthy person out there, they are going to have some blasts, because these are the first part of the development of white blood cells. So, they’re like baby white blood cells. But what the problem is, is when they start to grow too much. 

And so in the setting of myelofibrosis and even sometimes with these other diseases, the blasts will be in the peripheral blood primarily because the bone marrow is damaged and doesn’t hold them in very well. It becomes AML when it gets greater than 20 percent, so that blasts of greater than 20 percent in the peripheral blood or in the bone marrow but a lot of times we find it in the peripheral blood is where we indicate this has progressed to AML. 

Katherine Banwell:

Yeah. 

Dr. Jeanne Palmer:

Blasts of greater than 10 percent are also something that we really want to pay attention to, because that would suggest that the disease is starting to become more aggressive. Now, blasts vary, so for example, I’ve had patients go up to 11 and then drop back down to 3 or 4, and then they say around 3 or 4 or 5. So, you always want to make sure to double-check because one blast count at 11 percent, whereas it’s very important to address, may not necessarily reflect that you need to change in treatment at that time. Again, these blood tests, I always tell people, do not freak out over one blood test.  

Make sure you get at least a couple of them to really confirm what you are looking at. 

Katherine Banwell:

Thank you, Dr. Palmer. And to our viewers, please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them answered on future webinars.  

Dr. Palmer, was we close out this conversation I wanted to get your thoughts on where we stand with progress in helping people live longer and truly thrive with MPN. What would you like to leave the audience with? 

Dr. Jeanne Palmer:

So, I think that the first thing is make sure you understand your disease. Don’t hesitate to ask for a second opinion. It’s always good to make sure you talk to someone who can really explain so you feel like when you go home you understand what’s going on in your body. Make sure you understand what symptoms to look for, what things to be aware of, because a lot of times people come in and they have no idea that, oh, these symptoms are actually related to their disease. 

The other thing to make sure is that you’re very honest with your provider on how you’re feeling. A lot of times people come in and they say, “Oh, how are you feeling?” “I feel fine,” but then they start to ask very specific questions and they’re like, “Oh yeah, I’m really tired, my fatigue is an 8 out of 10,” or something. 

So, make sure you’re really honest with your provider. When they ask you how they’re doing, this is not a social visit, this is a visit where they need to know your symptoms, so you don’t need to say I’m fine like you normally would if you were walking down the street. 

The next thing is to always make sure to know where there’s clinical trials because we are making enormous great leaps and bounds in this field. It’s a really exciting time for myeloproliferative diseases, and there’s a number of new drugs that are being tested and coming out. So, it’s always important, if the opportunity is available and you can do it, clinical trials are a great way to get treatment. 

Plus, you are giving back, because these are things that help us learn whether something works or not. So, you’re not as much a guinea pig, you never get a sugar pill. It’s one of those things you will always get the treatment you need and then they may add something to it or you may be in the situation where there is no treatment, so they try something. 

But clinical trials, I have to emphasize, are a great way to get therapy and really are how we know everything that we know about treatment for these diseases. 

Katherine Banwell:

Yeah. It sounds like there’s a lot of progress and hope in the field.  

Dr. Jeanne Palmer:

Oh, absolutely. 

Katherine Banwell:

Thank you so much, Dr. Palmer, for joining us today.  

Dr. Jeanne Palmer:

You are very welcome, my pleasure, and it’s always fun to do these things, so thank you for having me.   

Katherine Banwell:

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

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A Patient’s Perspective | Participating in a Clinical Trial

A Patient’s Perspective | Participating in a Clinical Trial from Patient Empowerment Network on Vimeo.

Colorectal cancer survivor Cindi Terwoord recounts her clinical trial experience and explains why she believes patients should consider trial participation.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.

Cindi Terwoord is a colorectal cancer survivor and patient advocate. Learn more about Cindi, here.

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Transcript:

Katherine Banwell:    

Cindi, you were diagnosed with stage IV colorectal cancer, and decided to participate in a clinic trial. Can you tell us about what it was like when you were diagnosed?

Cindi Terwoord:        

Yeah. That was in September of 2019, and I had had some problems; bloody diarrhea one evening, and then the next morning the same thing. So, I called my husband at work, I said, “Things aren’t looking right. I think I’d better go to the emergency room.”

And so, we went there, they took blood work – so I think they knew something was going on – and said, “We’re going to keep you for observation.” So, then I knew it must’ve been something bad. And so, two days later, then I had a colonoscopy, and that’s when they found the tumor, and so that was the beginning of my journey.

Katherine Banwell:    

Mm-hmm. Had you had a colonoscopy before, or was that your first one?

Cindi Terwoord:        

No, I had screenings, I would get screenings. I had heard a lot of bad things about colonoscopies, and complications and that, so I was always very leery of doing that. Shame on me. I go for my other screenings, but I didn’t like to do that one. I have those down pat now, I’m very good at those.

Katherine Banwell:    

Yeah, I’m sure you do. So, Cindi, what helped guide your decision to join a clinical trial?

Cindi Terwoord:        

Well, I have a friend – it was very interesting.

He was probably one of the first people we told, because he had all sorts of cancer, and he was, I believe, one of the first patients in the nation to take part in this trial. It’s nivolumab (Opdivo), and he’s been on it for about seven years. And he had had various cancers would crop up, but it was keeping him alive.

And so, frankly, I didn’t know I was going to have the option of a trial, but he told me run straight to Cleveland Clinic, it’s one of the best hospitals. So, I took his advice. And the first day the doctor walked in, and then all these people walked in, and I’m like, “Why do I have so many people in here?” Not just a doctor and a nurse. There was like a whole – this is interesting.

And so, then they said, “Well, we have something to offer you. And we have this immunotherapy trial, and you would be one of the first patients to try this.”

Now, when they said first patient, I’m not quite sure if they meant the first colon cancer patient, I’m not sure. But they told me the name of it, and I said, “I’m in. I’m in.” Because I knew my friend had survived all these years, and I thought, “Well, I’ve gotten the worst diagnosis I can have, what do I have to lose?” So, I said, “I’m on board, I’m on board.”

Katherine Banwell:    

Mm-hmm. Did you have any hesitations?

Cindi Terwoord:        

Nope. No, I’m an optimistic person, and what they assured me was that I could drop out at any time, which I liked that option.

Because I go, “Well, if I’m not feeling well, and it’s not working, I’ll get out.” So, I liked that part of it. I also liked, as Dr. Funchain had said, you go in for more visits. And I like being closely monitored, I felt that was very good.

I’ve always kept very good track of my health. I get my records, I get my office notes from my doctor. I’m one of those people. I probably know the results of blood tests before the doctor does because I’m looking them up. So, I felt very confident in their care. They watched me like a hawk. I kept a diary because they were asking me so many questions.

Katherine Banwell:    

Oh, good for you.

Cindi Terwoord:        

I’m a transcriptionist, so I just typed out all my notes, and I’d hand it to them.

Katherine Banwell:    

That’s a great idea.

Cindi Terwoord:        

Here’s how I’m feeling, here’s…And I was very lucky I didn’t have many side effects.

Katherine Banwell:    

In your conversations with your doctor, did you weigh the pros and cons about joining a trial? Or had you already made up your mind that yes, indeed, you were going for it?

Cindi Terwoord:        

Yeah, I already said, “I’m in, I’m in.” Like I said, it had kept my friend alive for these many years, he’s still on it, and I had no hesitation whatsoever.

I wish more people – I wanted to get out there and talk to every patient in the waiting room and say, “Do it, do it.”

I mean, you can’t start chemotherapy then get in the trial. And if I ever hear of someone that has cancer, I ask them, “Well, were you given the option to get into a trial?” Well, and then some of them had started the chemo before they even thought of that.

Katherine Banwell:    

Mm-hmm. So, how are you doing now, Cindi? How are you feeling?

Cindi Terwoord:        

Good, good, I’m doing fantastic, thank goodness, and staying healthy. I’m big into herbal supplements, always was, so I keep those up, and I’m exercising. I’m pretty much back to normal –

Katherine Banwell:

Cindi, what advice do you have for patients who may be considering participating in a trial? 

Cindi Terwoord:

Do it. Like I said, I don’t see any downside to it. You want to get better as quickly as possible, and this could help accelerate your recovery. And everything Dr. Funchain mentioned, as far as – I really never brought up any questions about whether it would be covered. 

And then somewhere along the line, one of the research people said, “Well, anything the trial research group needs done – like the blood draws – that’s not charged to your insurance.” So, that was nice, that was very encouraging, because I think everybody’s afraid your insurance is going to drop you or something.  

And then the first day I was in there for treatment, a social worker came in, and they talked to you. “Do you need financial help? We also have art therapy, music therapy,” so that was very helpful. I mean, she came in and said, “I’m a social worker,” and I’m like, “Oh, okay. I didn’t know somebody was coming in here to talk to me.” 

But that was all very helpful, and I did get free parking for a few weeks. I mean, sometimes I’d have to remind them. I’d say, “It’s costing me more to park than to get treated.” But, yeah, like I said, I’m a big advocate for it, because you hear so many positive outcomes from immunotherapy trials, and boy, I’d say if you’re a candidate, do it. 

Katherine Banwell:

Dr. Funchain, do you have any final thoughts that you’d like to leave the audience with? 

Dr. Pauline Funchain:

First, Cindi, I have to say thank you. I say thank you to every clinical trial participant, everybody who participates in the science. Because honestly, whether you give blood, or you try a new drug, I think people don’t understand how many other lives they touch when they do that.  

It’s really incredible. Coming into clinic day in and day out, we get to see – I mean, really, even within a year or two years, there are people that we’ve seen on clinical trial that we’re now treating normally, standardly, insurance is paying for it, it’s all standard of care. And those are even the people we can see, and there are so many people we can’t see in other centers all over the world, and people who will go on after us, right?  

 So, it’s an amazing – I wouldn’t even consider most of the time that it’s a personal sacrifice. There are a couple more visits and things like that, but it is an incredible gift that people do, in terms of getting trials. And then for some of those trials, people have some amazing results. 

And so, just the opportunity to have patients get an outcome that wouldn’t have existed without that trial, like Cindi, is incredible, incredible. 

What Are the Risks and Benefits of Joining a Clinical Trial?

What Are the Risks and Benefits of Joining a Clinical Trial? from Patient Empowerment Network on Vimeo.

Why should a cancer patient consider a clinical trial? Dr. Pauline Funchain of the Cleveland Clinic explains the advantages of clinical trial participation.

Dr. Pauline Funchain is a medical oncologist at the Cleveland Clinic. Dr. Funchain serves as Director of the Melanoma Oncology Program, co-Director of the Comprehensive Melanoma Program, and is also Director of the Genomics Program at the Taussig Cancer Institute of the Cleveland Clinic. Learn more about Dr. Funchain, here.

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Transcript:

Katherine Banwell:

Why would a cancer patient consider participating in a clinical trial? What are the benefits? 

Dr. Pauline Funchain:

So, I mean, the number one benefit, I think, for everyone, including the cancer patient, is really clinical trials help us help the patient, and help us help future patients, really.  

We learn more about what good practices are in the future, what better drugs there are for us, what better regimens there are for us, by doing these trials. And ideally, everyone would participate in a trial, but it’s a very personal decision, so we weigh all the risks and benefits. I think that is the main reason.  

I think a couple of other good reasons to consider a trial would be the chance to see a drug that a person might not otherwise have access to. So, a lot of the drugs in clinical trials are brand new, or the way they’re sequenced are brand new. And so, this is a chance to be able to have a body, or a cancer, see something else that wouldn’t otherwise be available.  

And I think the last thing – and this is sort of the thing we don’t talk about as much – but really, because clinical trials are designed to be as safe as possible, and because they are new procedures, there’s a lot of safety protocols that are involved with them, which means a lot of eyes are on somebody going through a clinical trial.  

Which actually to me means a little bit sort of more love and care from a lot more people. It’s not that the standard of care – there’s plenty of love and care and plenty of people, but this doubles or triples the amount of eyes on a person going through a trial. 

Katherine Banwell:

Yeah. When it comes to having a conversation with their doctor, how can a patient best weigh the risks and benefits to determine whether a trial is right for them? 

Dr. Pauline Funchain:

Right. So, I think that’s a very personal decision, and that’s something that a person with cancer would be talking to their physician about very carefully to really understand what the risks are for them, what the benefits are for them. Because for everybody, risks and benefits are totally different. So, I think it’s really important to sort of understand the general concept. It’s a new drug, we don’t always know whether it will or will not work. And there tend to be more visits, just because people are under more surveillance in a trial.  

So, sort of getting all the subtleties of what those risks and benefits are, I think, are really important. 

Katherine Banwell:

Mm-hmm. What are some key questions that patients should ask? 

Dr. Pauline Funchain:

Well, I think the first question that any patient should ask is, “Is there a trial for me?” I think that every patient needs to know is that an option. It isn’t an option for everyone. And if it is, I think it’s – everybody wants that Plan A, B, and C, right? You want to know what your Plan A, B, and C are. If one of them includes a trial, and what the order might be for the particular person, in terms of whether a trial is Plan A, B, or C. 

How Can MPN Patients Become More Proactive in Their Care?

How Can MPN Patients Become More Proactive in Their Care? from Patient Empowerment Network on Vimeo.

How can MPN patients become more empowered and active in their care? Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares advice for patients to gain confidence to become a more active participant for optimal care.

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Transcript:

Dr. Nicole Rochester: 

So what advice would you give for patients so that they can really take a proactive approach to their healthcare and feel more confident in talking about their concerns and communicating with their healthcare team, you’ve shared with us how important that is. Do you have maybe two or three specific tips or maybe questions that every MPN patient should ask their healthcare provider?

Dr. Claire Harrison: 

I think the first thing to say is, in my personal view is you do not have to be under an MPN expert to get the best care. I know some people differ with regard to that, but these are chronic conditions, there are national and international guidelines, clinicians are connected. We all talk about patients over time, as we like to do that, we like to get the best for our patients, so a local center with a clinician who you trust, who you get on with…where you can get there easily. You trust their team, you know their logistics work for you, maybe it’s a nurse who work who you get on with, well, who comes to the appointment with you, that is just as good as being under the best professor in the state, where you might not actually see them  when you turn up and go to the unit, so that’s really important, understanding your condition, and if you don’t understand being empowered to ask questions, and if you’re in a position where you can’t ask a question, something’s wrong. So don’t be afraid, take somebody with you, write it down. 

Sometimes it can be a mistake to do a troll on the Internet, so I wouldn’t always encourage that because what’s on the Internet is not always accurate, but go to a trusted website as the clinician…where can I go to find out more information? Some patient advocacy groups run buddy systems that can also be very helpful and it can be very empowering to meet another patient with the same or similar condition, so I think those are all helpful tips from my perspective, also don’t expect to get all the answers all the time, it can be really tricky as a clinician, maybe you get a patient who comes with a big long list of questions, and say What is your top question that you really want answers to. 

Dr. Nicole Rochester:

Those are awesome, awesome tips. I’m just going to repeat a few of them, just to highlight, you mentioned prioritizing your concerns which is incredibly important, and acknowledging that the clinician doesn’t have unlimited time, and so really focusing on the things that concern you the most, you mentioned bringing a buddy to appointments, which is something I fully endorse, so that there’s someone else that’s taking notes or…it can be your eyes and ears during that appointment, things that you may have missed either because of anxiety or stress, and you mentioned writing things down, taking notes, even as the patient asking questions, which is so incredibly important, and really the way that I feel patients demonstrate their involvement in their disease and being an active member of the team, so I really, really appreciate those tips, Dr. Harrison, I think that you have given us so information, so much information about how to empower MPN patients and their families so that they can really get the best care at the outset. 

Advice for Hesitant MPN Clinical Trial Participants

Advice for Hesitant MPN Clinical Trial Participants from Patient Empowerment Network on Vimeo.

What should MPN patients know about clinical trials? Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares information about the varying degrees of clinical trials and advice to those who are hesitant about clinical trials.

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Transcript:

Dr. Nicole Rochester: 

It’s said that clinical trials are tomorrow’s medicine today, and you’ve already kind of alluded to the importance of clinical trials as it relates to MPN. What would you say to an MPN patient who is on the fence or may be concerned or afraid of participating in a clinical trial?

Dr. Claire Harrison: 

It’s right to be cautious and, you know, careful because ultimately it’s a huge privilege as a clinician that involves patients in clinical trials that my patients trust me and trust my team to look after them with something that is experimental, but remember there are varying degrees of experimental. Most clinical trials are not first in man, you’re not a complete guinea pig, it may be a drug, for example, navitoclax is in clinical trials mainly for myelofibrosis also ET and PV but that is a drug that has been used for thousands of patients, for another indication so talk to your healthcare team, if you don’t find the answer from the primary person that you’re used to dealing with, find someone else, be linked to somebody you trust and that you have a good relationship with, take someone with you to the consultation, write down the questions I’m so sure you say this all the time, don’t you Nicole to the people that you talk to, but write down your questions, don’t be afraid to ask them again, there is no stupid question in this context, you will be given a 30-plus page booklet to read, and I lost count of the number of times, my patients go, yeah, I’ve got this, or I trust you.

Actually, you know, you need to read it…we are experimenting on you, and you need to read that and understand. And you need to understand, what happens if I go on the control arm, will I be able to cross over? How many visits will I have, will I have to pay for those visits, etcetera. It’s all really important. But ultimately the relationship with your healthcare provider is important, and using an advocate is really important too.

Dr. Nicole Rochester: 

I agree 100 percent. So important, these are things that I talk about all the time, so I really appreciate that you highlighted that, and just the importance of patients taking an active role in their medical care and also the trust that is required between the patient and their treating providers. So I really appreciate that. Do you have any examples, Dr. Harrison, in your own practice of successes with MPN patients who have participated in clinical trials? 

Dr. Claire Harrison: 

Oh yes, I think I started doing clinical trials, well golly, a long time ago. I think my first clinical trial, probably the records were written parchment to be honest, but we’ve still learned a lot from that, so that was an ET study. It was from that study we understood about the JAK2 mutation, and we understood how patients behave differently. I think probably the most gratifying thing for me was being involved in the JAK inhibitor studies in myelofibrosis and being involved in delivering ruxolitinib and Jakafi to patients and seeing the benefits for those patients. 

Big things, you know, there are patients who are alive because they took part in that trial today, I think, but there are also patients for whom small things were also really important, so as a patient, that’s important to define what is the benefit you want to get. So one of my first patients, you haven’t been able to have a bath or a shower for years, because he had terrible what we call aquagenic pruritus, itching induced by contact with water, we called him two days after he started ruxolitinib (Jakafi), and he was in tears, he could take…or you can take it out.

These things are really important. Like myself, I can imagine not being able to dig it out, I would either be very tough for another patient, it was, well, I looked really skinny because I’d lost loads of weight and I put weight on, and body image was really important as well, but then the small things like being able to be…participate more in family activities is really, really important too. 

What Are the Unmet Needs in Access to MPN Care?

What Are the Unmet Needs in Access to MPN Care? from Patient Empowerment Network on Vimeo.

Which areas of MPN care still need improvements to access? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London explains patients who still experience barriers to care and what can be done to reduce access issues.

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Transcript:

Dr. Nicole Rochester: 

What would you say are the unmet needs in access related to MPN and care, specifically as it relates to clinical trials, and what can we do to address those unmet needs?

Dr. Claire Harrison: 

Well, I think there is a problem with rare diseases in terms of geographical access to trials, and we often find patients have to travel a long way. I know that’s true in North America as well as in Europe. And we’re very lucky in our geographical locations, but in some parts of the world, some companies or doing not open clinical trials, so I think there’s an access issue. 

I think also there is something about patients have to meet rigid entry criteria for clinical trials, and so oftentimes in myelofibrosis, for example, commonly patients who fail ruxolitinib (Jakafi) have a lower platelet count, and that is often an exclusion criteria. Those criteria are there to try to get a uniform population of patients in a trial, but it can feel like you’re excluded as a patient, and it can feel very tough and for your health care team that we can’t include you in a clinical trial. We also have to remember that it is there for safety purposes, so if there is a lower limit for platelet count, that’s often because the drug might affect platelet count. It is really important that we have a broad spectrum of trials available and that we try to increase the availability of trials for patients. 

I also want to say a word about inequality of access and thinking about accessing some different ethnicity, so often non-white MPN patients are under-represented in clinical trials, and I know that a focus in the UK and also in North America as well. And it is really important that patients have access to a clinical trial if they need it, and also that we understand how investigational products will work in people of different backgrounds. So for example, we know that probably, Nicole, your blood count assuming it’s a healthy, normal blood count may well be different from mine for background, racial genetic differences, so drug metabolism might be different, so this is really important, and we need to work hard as a community, the clinical community and the patient community to raise awareness and improve access for patients. 

Dr. Nicole Rochester: 

Well, as someone who does a lot of work in health equity, Dr. Harrison, I really appreciate you pointing that out. It’s certainly an issue here in the United States, as you mentioned, differential access to clinical trials, and we’ve learned that not only our patients, often not aware, but often the providers, at least here in the U.S., are not offering clinical trials as an option for patients from marginalized and minoritized communities. So I really appreciate you bringing that up. 

MPN Treatment Strategies for Patients Who Have Failed Traditional Therapies

MPN Treatment Strategies for Patients Who Have Failed Traditional Therapies from Patient Empowerment Network on Vimeo.

What can be done for MPN patients who have failed on traditional therapies? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London explains some treatments under study as options for some patients.

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Transcript:

Dr. Nicole Rochester: 

What about for patients who have failed therapies, are there any treatment strategies for MPN patients who have failed traditional therapies?

Dr. Claire Harrison: 

Yes, in fact, actually, that’s where we’re evaluating new therapies across all of these entities, so if you’re a PV or an ET patient and you failed a therapy, then this is where, for example, in ET, we would be looking at bomedemstat or we’re looking at the bromodomain inhibitor pelabresib, and there’ll be other agents that we’ll be looking at, or we might be looking at vaccination. And for MF patients that while there are a bunch of different therapies for patients who you have not tolerated or progressed through standard therapy. So actually, there are a lot of options, some of them are already approved, and some of them are in clinical trials. 

How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients?

How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients? from Patient Empowerment Network on Vimeo.

How do MPN treatments vary for low-risk and high-risk patients? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London explains how treatment differs for these patients and changes that she would like to see for care of some patients. 

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Transcript:

Dr. Nicole Rochester: 

How are treatment strategies changing for low-risk and high-risk patients with MPN?

Dr. Claire Harrison: 

It’s complicated because we need to think across the entities, and we don’t have an answer to that for patients with MPN unclassified. And we don’t actually have a good answer to that for this entity called pre-fibrotic myelofibrosis, which does appear and is strongly recognized in the new diagnostic criteria. But for ET, for example, low-risk patients I mentioned triple-negative, calreticulin, M-positive, young patients, platelets less than 1500, not too much changing their queries about aspirin or not, and then for PV patients, we haven’t really changed all kind of high-risk criteria and for both ET and PV, the questionnaire is, should we use the treatment above aspirin or above aspirin and venesection. 

And for the most part, that would be hydroxycarbamide, hydroxyurea (Hydrea), which is the commonest treatment used worldwide or interferon, and these are the right treatment for some patients and not the right treatment for other patients, so some patients can be very fixated on interferon is the absolute best, but there is no clear evidence of that, and there are some patients who interferon is not the right treatment, but low versus high risk becomes even more important for myelofibrosis patients.

And here, we’re thinking about using a risky strategy like transplantation for those patients who have higher risk disease, and we’re using, as I mentioned to you, these molecular markers and newer prognostic tools to stratify patients. And it is important to remember as a patient if someone puts your data into a prognostic tool and that comes up with five years, but it doesn’t mean to say five years on the dot your time’s up, that’s an average. And if we put your data into a slightly different tool, we might get something else. So for the most part, we make decisions like transplants, we are learning more about transplantation and outcomes from that, and then in some countries, some treatments are used for patients who fall into intermediate or high-risk categories, and some clinical trials are based on that as well. I would want to say about myelofibrosis, and something I think I would really like to see changed, not changing yet, but changed, is that we should be able to intervene for patients with a low-risk disease. If my myelofibrosis patients have breast cancer, we would not be going there, then you’ve got low-risk disease, we’ll put you on watch and wait, watch and wait is really hard for our patients, we know that I can see you nodding.

You know that too, right? So if these were patients with breast cancer we would not say, we’ll just watch and wait. So I would really like to see in the next five to 10 years a treatment that we could use earlier in the disease course, but there is nothing at the moment, but we’re looking at that. The other thing we’re looking at, if we’ve got a minute or so is the different endpoint, so we’re trying to understand what does it mean if your allele burden, so the amount of abnormal genes you’ve got goes down, the amount of bone marrow fibrosis you’ve got goes down. And again, this is something we’ll collect in a clinical trial, but also from real-world data. 

How Can MPN Patients Stay Up to Date With New Treatments?

How Can MPN Patients Stay Up to Date With New Treatments? from Patient Empowerment Network on Vimeo.

What are some ways for MPN patients to stay updated on new treatments? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares resources for MPN treatment news and the benefits that data sharing brings for all MPN patients.

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New Developments in MPN Treatment Landscape

What Is in the Treatment Pipeline for Patients With MPNs?

How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients?


Transcript:

Dr. Nicole Rochester: 

Dr. Harrison, how can patients best keep up with the new treatments and communicate with their doctors in a way that makes sure that they have access to these new therapies?

Dr. Claire Harrison: 

Well, I think patient advocacy groups are really important here, and use of social media and the Internet, you’re only a few clicks away from updated data, programs like these, but you also have to trust your team and trust your doctor. We all have to keep up to date. That is a professional requirement, and we also are all networked, so I’d probably get 10 to 15 emails a day from colleague saying, “Hi Claire, can I talk to you about this?” There was an email just the other day about a patient in Washington, actually a very young child, we are all connected. 

We all want the best for our patients. But do you remember that you can contribute as a patient to advance this in your field, and I know many patients are really interested in this, if you are asked to submit in a blood sample, giving permission for us to use your data. So if we can touch maybe on the field of real-world data and real-world data collection here would be good, so what on the real world data? What does that mean? And so this is becoming a really important way that’s recognized by the FDA and other approval agencies in the world, so in Europe, we have EMEA, for example, and in the UK, we have NHRA as a way of collecting data on agents, so once they are approved, we collect data with regard to how the patients do. 

We’ve traditionally done this, but increasingly, as we use electronic data for our patients, we’re more able to collect real-world data, how does my patient who is with myelofibrosis on ruxolitinib (Jakafi) in my clinic inside East London do? So if we can pull that data, we learn a lot more about how these agents are working in patients outside of clinical trial, so you will be contributing if you allow us to collect that kind of data, we discovered JAK2 mutations, CALR mutations, etcetera from samples collected from patients and data. If you want to be part of a clinical trial, then by all means, ask your healthcare team, many MPN centers have lists of trials, and you can always look at clinicaltrials.gov, but boy you throw up a lot of different options when you search in that…on that website. 

Dr. Nicole Rochester: 

Thank you. I think it’s important to talk about real-world data because in this day and age, many of us are very protective about our personal health information and we should be, but as you stated, having access to that data is really a key way to advance the science and technology in the treatment of some of these conditions, so I really appreciate you sharing that.

Dr. Claire Harrison: 

I think just to point out, and I’m sure the audience is acutely aware of what did we learn about COVID? We learned an awful lot about COVID from real-world data from all you MPN patients who gave samples, who told us about how you did with COVID, that’s how we learned about what happened to patients during COVID with MPN, how they responded to vaccination, etcetera. It’s really powerful. And your data will be anonymized, it won’t be linked back to you, Nicole Rochester, or me, Claire Harrison it will be completely anonymous. 

What Is in the Treatment Pipeline for Patients With MPNs?

What Is in the Treatment Pipeline for Patients With MPNs? from Patient Empowerment Network on Vimeo.

What does the MPN treatment pipeline hold for patients? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares insight about future treatments and the outlook for care.

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New Developments in MPN Treatment Landscape

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How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients?


Transcript:

Dr. Nicole Rochester: 

Can you share a little about what’s in the robust pipeline of potential therapies for patients with MPN, what is it that you’re excited about? And is the future bright in this area?

Dr. Claire Harrison: 

I think the future is really bright from the point of view of de-escalating treatment as well as newer treatment, so I think it’s important to point that because all treatments have potential complications inside of it, so as we understand that some of these conditions may have very low risk for patients, it’s important to understand that, and de-intensify, I call that calreticulin (post ET?), I would also call out ET, essential thrombocythemia, which lacks a known driver mutation, so-called triple-negative ET, emerging data suggests that may have very low risk for patients, but what you all want to hear about, of course, is what’s new treatment-wise. 

So I think just to call out, I’m really excited that there will be a new trial this year for ET patients with Bomedemstat, which is an LSD-1 inhibitor, new target, new molecule. We’ve been testing it in myelofibrosis and we’ve tested it now in a bunch of patients with ET, and it seems to be very efficiently reducing the platelet count not affecting hemoglobin and patients appear to get a good benefit with regard to fatigue, which we know is the number one symptom for patients with MPN, so I’m excited about that because it’s been a long time since we’ve had a new treatment for patients with ET. And then for patients with PV, increasingly across the globe, the availability of this newer formulation of interferon, Besremi is becoming more available and the latest data with that agent suggests that it may be superior to standard therapy such as hydroxyurea, hydroxycarbamide in terms of clotting, et cetera, is really important.

Interestingly, and we may both have to think back to our med school days on this, we’ve been targeting the iron pathway for patients with PV. So I always tell my patients with PV, do not let anyone give you iron tablets without speaking to one of our team because that’s like putting oxygen on the fire, it’s like feeding the red cell production. So there is a new agent called Rusfertide PTG-300, which targets the iron pathway and allows iron to build up in the body, but it doesn’t allow it to get to the bone marrow and so, this is a new treatment for PV patients, which might reduce the need for iron removal by phlebotomy or venesection, and has been also shown to give symptomatic benefit, and then of course, there’s a bunch of new treatments for patients with myelofibrosis, that’s probably the busiest part of the portfolio at the moment. 

We’ve just seen positive data with me momelotinib, which is one of the fourth JAK inhibitors, very strong data from the MOMENTUM  study, good results in patients even with low platelet counts down to 25, and then I’m really excited to see strong data coming with navitoclax and pelabresib, which are other agents targeted at are the pathways in myelofibrosis. And then finally, in Denmark, they’ve been looking at vaccination strategies, and I know my patients are really interested in vaccination and gene editing. I don’t have anything new to say on gene editing), but I do have something new to say on vaccination.

So in Denmark, they’ve been looking at producing a vaccination against the calreticulin mutation, Nicole, because it’s expressed on the surface of the blood cells, so antibodies can find it. So this is ongoing, no positive result as yet, but it’s still ongoing and there are newer taking off with regards to vaccination structures, and I think that’s really exciting.  

New Developments in MPN Treatment Landscape

New Developments in MPN Treatment Landscape from Patient Empowerment Network on Vimeo.

For the MPN treatment landscape, what are the latest developments? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares how the treatment landscape is changing and diagnostic criteria to be published on how each specific diagnosis should be used to optimize care.

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What Is in the Treatment Pipeline for Patients With MPNs?

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How Can MPN Patients Become More Proactive in Their Care?


Transcript:

Dr. Nicole Rochester: 

Dr. Harrison, we know that the treatment landscape for MPNs is rapidly changing and keeping up with the pace of the developments can be challenging both for healthcare professionals and certainly for patients and their families, so I was wondering if you can give us a general overview of the treatment landscape and maybe highlight anything that’s new and in development that you think would be helpful for the audience.

Dr. Claire Harrison: 

Well, sure, I think this is obviously really interesting, very important. It’s a fast-moving field. And one of the first changes actually completely hot off the press is a brand new diagnostic criteria that has been produced by two separate groups that are shortly about to be published kind of just seeing pre-published and those of us that have been privileged to be involved, have seen them. So that’s going to really focus the mind of clinicians on how do we achieve the diagnosis, and that’s the first thing that’s really important, get an accurate diagnosis, don’t just accept well, you’ve got an MPN, actually, it’s really important to know what type you’ve got. Maybe as much do you tell as possible in terms of the genetic mutation or change that underlies the disease, because that is increasingly important in what we call prognostication, understanding what the risk of events happening due to the disease actually is. 

When we make a diagnosis, we sometimes can’t be accurate, and we might have to go back and revisit them, so some patients don’t get a PV, ET, or MF diagnosis, they might get an MPN unclassified. That’s okay, but it’s important to keep revisiting that. Once we’ve made a diagnosis, then it’s really important to think about what the risk is to the patient, and we’ve had some changes to our risk classifications recently, right from the ET end of the spectrum where we’re thinking more and more actually, those patients who are under the age of 60 with a lower count and the CALR mutation, do we even need to give you aspirin, because we might be increasing the risk of bleeding. 

To the other end of the spectrum for patients with myelofibrosis, a more aggressive disease, we want to know more about your mutational profile, so we’re doing more powerful genomic tests and assessing them your prognosis, and then what the features of your disease are that need treatment. And there are lots of changes, which I think we’ll get into later in our conversation here and lots of new options, which are really important,  I just also don’t to leave this segment without saying to all of you who are listening, it’s important that you understand your disease, it’s important that you understand the diagnosis, prognosis, etcetera, and you get the best care.

But that’s maybe not enough, you need to know that you’re taking good care of you, and that’s something that’s really important to all of us, so you need to know that you’re managing your vascular risk, don’t just think about your blood. Think about the fact that you know if you’re smoking, quit smoking, if you’re drinking too much, cut it down, if you’re not walking enough,  walk more, lose weight. The majority of patients with MPN actually have a problem with a blood clot, not a further complication of their disease, so maybe we’ll stop there and then we can dive in a bit more deeply.

Participating in a Clinical Trial: What You Need to Know Resource Guide

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Five Tips to Participate in MPN Care and Treatment Decisions

How can myeloproliferative neoplasm (MPN) patients become more active in their care? In the “How Should You Participate in MPN Care and Treatment Decisions?” program, expert Dr. Abdulraheem Yacoub of the University of Kansas Cancer Center shares five key tips MPN patients can take for a more active role for optimal health outcomes.

1. Become a Patient Self-Advocate

It’s vital to have the ability to advocate on your own behalf no matter your age at diagnosis. And some MPN patients will be diagnosed at a relatively young age and will have different MPN care providers over the course of their disease. These patients need to get accustomed with the idea of care approaches changing over time.

2. Get Involved and Build Your Village

Being involved in your well-being as a patient is of utmost importance, and thinking about your support network is recommended as one of your early steps as a patient. Think about who among your friends, family, co-workers, and spiritual community might be able to help support you – and ask your MPN care provider about support resources if you need some additional help.

3. Bring a Friend or Loved One to Appointments

It’s important to have someone else at your appointments with you to help understand the information you receive and to also take notes and to ask questions if it’s helpful for you. Having a second set of ears is especially important with your early visits about treatment options, and the use of telemedicine makes it easier for loved ones to help support your appointments.

4. Get a Second Opinion

Second opinions are no longer the taboo that they were once perceived as. Listen to medical facts given to you from your MPN specialist and from your primary treating physician. And if you want a second opinion from another MPN specialist, this practice is easier to carry out now through telemedicine.

5. Seek Out Credible Resources and Research News

Keep yourself informed about the latest MPN research and treatment news by visiting credible online resources. In addition to PEN, check The Leukemia & Lymphoma Society (LLS) and MPN Research Foundation. The annual meetings of expert conferences like the American Society of Hematology (ASH) and American Society of Clinical Oncology (ASCO) bring research updates for MPN online resources to cover.

By taking a more active role in their care, MPN patients can help determine the best care and treatment plan for optimal health outcomes.