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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.

 

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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.  

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 will 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

Myeloma Patient Profile: Jeff Boero

When Jeff Boero shares his multiple myeloma patient journey, it’s clear that self-education has been a vital part of his experience. He was first diagnosed through his primary care physician who referred him to a general oncology group in the San Francisco area. They confirmed it was multiple myeloma. It soon became clear to Jeff and his wife that he perhaps needed a second opinion, and he was connected with the University of California San Francisco (UCSF) to their multiple myeloma specialist. 

The second opinion changed the approach to Jeff’s care rather dramatically. He was quickly scheduled for a stem cell transplant and subsequent maintenance after that. As Jeff recalls, “Through UCSF, I became eligible for a CAR T-cell immunotherapy trial in 2017. That was very successful and kept me disease-free and medication-free for about 2-1/2 years. And then I relapsed and went on another maintenance program. I became eligible for another clinical trial for a bi-specific T-cell engager (BiTE) that I’m on now and am having good results.”

Jeff was almost in complete denial about his diagnosis for the first 6 months. The diagnosis threw him into a world of terminology and treatment that was completely foreign to him. That sense of his diagnosis feeling foreign also started to lead into a certain level of depression — just not knowing what it is, how is it going to be treated, what it meant to his long-term survival. Jeff remembers, “So, with the encouragement of my wife as caregiver, I became more educated as I engaged in various conversations with specialists and participated in some of the PEN webinars. It  became clearer to me about what some of the options are and what they can be. Being engaged with UCSF really opened up the treatment options. With me becoming more educated and able to speak the language of myeloma, I was starting to understand the diagnosis as it was presented by UCSF. And it led to a much richer engagement in conversation with the oncologist and with the nurse practitioners.” 

As a cancer patient, Jeff views self-education as the key to empowering patients toward better care. It was through self-education that he learned about other options. Before becoming more educated, Jeff was mostly just listening and trying to absorb as much as he could and seemed to remember mostly bad news. According to Jeff, “There’s so much good news around myeloma treatment and available therapies. It was through self-educating and those conversations that my outlook brightened too.”

By patients educating themselves, they can start to ask questions about the clinical trial like: “What is it, and why is it going to show better results than my maintenance therapy?” And in conversation, patients can start to better understand the purpose of the clinical trial. “I think it’s important for patients to understand what they’re trying to accomplish through the clinical trial that wasn’t through their maintenance therapy. What is it about this trial that’s different that we haven’t addressed previously?” But patients can’t ask those questions unless they have at least a basic understanding of their cancer and how the various therapies approach the cancer cell. “But if you listen to webinars and things like that, you’re better able to have those conversations. As a matter of education as these opportunities arise, you’re able to have a much richer conversation with your oncologist and your care team about the benefits that could potentially be derived from the clinical trial.” 

Clinical trials have benefitted Jeff, and he recommends seeking an opinion that is dedicated to research of your specific cancer. Learning institutions have more access to emerging research and treatments that likely won’t be FDA-approved until 2 or 3 years later. “So if you as a patient can be at the forefront of some of these trials, that can be tremendous. I’m on therapies now that didn’t even exist when I was diagnosed. Research is moving quickly.”

Jeff senses some hesitancy among patients about clinical trials. “There’s this misconception that if you join a clinical trial, one group is getting the real stuff, and one group is getting the placebo. And the trials that I’ve been in, everybody gets the real thing, and everybody’s progress is tracked on their response to the real thing.” He knows trials can seem intimidating. Jeff went through his initial clinical trial, because he was almost out of options for conventional maintenance therapy. His cancer burden continued to increase, and he’d been through a number of different treatments. “The CAR T-cell program came up and seemed to be a perfect fit for me. So I did the clinical trial partially out of necessity, but I also had extreme confidence in my oncologist that he was promoting something that he thought would be most beneficial for me. I think it’s a matter of putting trust in your oncologist. Maybe I’ve been lucky, but I’ve had good results and good response to both clinical trials.” He also feels that the sponsoring institution will give an honest appraisal of where the program stands and what the progress and success has been up to that point. 

Reflecting on the value of Patient Empowerment Network (PEN) and other resources, Jeff says, “I’ve gotten so much out of the PEN webinars that are provided and some other organizations. I’m a slow learner in this area but am absorbing as much as I can. I need to hear the same thing a few times before I start to absorb it and fully understand it. So I rewatch the PEN webinars, and it works for me.” He also suggests learning as much as one can but was advised early on to stay away from Google. “There’s so much out-of-date information. Whereas websites like Patient Empowerment Network’s and others have updated information that’s far more relevant. And I also find the navigation on the PEN website very easy to use.”

After meeting patients who don’t have the same level of health insurance benefits, Jeff feels a sense of gratitude. “I had tremendous support from my employer who in essence said take the time you need to get yourself well again. So I have a lot of gratitude for that support, my wife as caregiver, family, social support, my faith community, and for my proximity to UCSF that makes treatment very practical and very possible.” It’s opened his eyes in that regard. There are so many benefits that he has that others don’t have. “I’ve joined various support groups initially to gain support. Now things have come full circle, and I find that I’m at the other end of the conversation to give people comfort in what they could possibly be doing to improve their situation.”


Support Resources

Financial Assistance Programs

Financial Resources for Patients and Families

Health & Disability Insurance

Federal & State Benefit Plans

Is It Too Late for a Myeloma Second Opinion?

Naïve T-cell Depletion in Donor Blood Stem Cell Transplant Patients 

Introduction 

T-cell depletion is a strategy to remove T-cells from bone marrow or peripheral stem cells taken from a donor before they are given to a patient. T-cell depletion (TCD) has been around for quite some time, at least since the early 1980s. TCD decreases the risk of graft-versus-host disease (GVHD) and, in particular, deaths from GVHD. The goal is to also have similar survival to non-T-cell depleted transplants (sometimes referred to T-cell replete or T replete). This would be a significant advance. Removing only the naïve T-cells from the graft looks like a promising approach to achieve these goals. The recent article Naive T-Cell Depletion to Prevent Chronic Graft-Versus-Host Disease reports on a trial looking at the use of naïve T-cell depletion and comparing the results to historical controls.  

What are Naïve T-cells? 

I had not heard of naïve T-Cells before. According to an article from the National Cancer Institute (NCI) Can Chronic Graft-Versus-Host Disease Be Prevented? 

T cells are among the many types of immune cells that can attack cancer cells. But there are several subsets of T cells. Naive T cells have never encountered an antigen—a protein or other molecule that can provoke an immune response. For reasons that are not yet fully understood, they are more likely to react to healthy cells in the transplant recipient. 

I don’t know how naïve T-cells can be distinguished from other T-cells, but apparently this is possible. 

Problems with T-Cell Depletion 

TCD does significantly reduce the risk of GVHD and deaths from GVHD, however it is not without drawbacks.    There have been many attempts at T-cell depletion over the years, first removing all T-cells and more recently selectively removing some of the T-cells. However, most of the problems listed below have persisted to some degree: 

  1. Graft failure is generally higher in TCD transplants than in non TCD transplants  
  2. Epstein-Barr virus–associated lymphoproliferative disorders (LPD). This is a rare side effect of T-replete transplants, but is much more common in TCD transplants (as well as solid organ transplants) Generally, the decrease in deaths acute GVHD are about equivalent in the increase in deaths LPD  
  3. There is a much higher rate of disease recurrence after TCD transplants. It is well established that patients who experience GVHD have a lower rate of relapse (this was known at the time of my transplant, almost 30 years ago). This is because of what is known as a graft-versus-leukemia (GVL) effect (or more generally as a graft-versus-tumor, GVT effect). The T-cells are causing GVHD by attacking the host cells in the patients.  Since any residual cancer cells are also recognized as foreign by the T-cells, they are destroyed as well.

Current Study 

For the most part the current study did not show the problems listed above. There were 138 patients with acute leukemia treated in this trial. There were 2 patients who experienced graft failure (< 2%). The incidence of Epstein-Barr virus (EBV) reactivation was rare, only 3 patients (2.3%) experiences this.  Only 1 required treatment with rituximab (Rituxan, which is one of the more effective therapies for EBV related LPD).  The overall incidence of relapse was 23%, which is in line with the expected relapse rate from T-replete transplants. The incidence of acute GVHD was low and most patients who experienced acute GVHD were treated successfully with steroids.  Only 7% of patients developed chronic GVHD (much lower than T-replete transplants), which also largely responded to steroids. 

Future Reading 

  1. Naive T-Cell Depletion to Prevent Chronic Graft-Versus-Host Disease, the full article from the Journal of Clinical Oncology, from April 2022 (registration may be required). 
  2. A blog article from the National Cancer Institute (NCI) Can Chronic Graft-Versus-Host Disease Be Prevented? This is quite interesting and not nearly as technical as the journal articles. 
  3. An older abstract (2020) from the same author, Marie Bleakley: Naive T-cell depletion in stem cell transplantation 
  4. Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults with Blood Cancers Undergoing Donor Stem Cell Transplant, the clinical trial description from the National Cancer Institute 
  5. The history and future of T-cell depletion as graft-versus-host disease prophylaxis for allogeneic hematopoietic stem cell transplantation  from the journal Blood.  A technical and older (2001) article but may be interesting to some. 
  6. Does GVHD Ever Resolve in Acute Myeloid Leukemia Patients?

References

Naive T-Cell Depletion to Prevent Chronic Graft-Versus-Host Disease Marie BleakleyAlison SehgalStuart SeropianMelinda A. BiernackiElizabeth F. KrakowAnn DahlbergHeather PersingerBarbara HilzingerPaul J. MartinPaul A. CarpenterMary E. FlowersJenna VoutsinasTheodore A. GooleyKeith LoebBrent L. WoodShelly HeimfeldStanley R. Riddell, and Warren D. ShlomchikJournal of Clinical Oncology 2022 40:111174-1185

Myeloma Induction and Consolidation Therapy Defined

Myeloma Induction and Consolidation Therapy Defined from Patient Empowerment Network on Vimeo.

Myeloma expert, Dr. Krina Patel, defines the role and purpose of induction therapy and consolidation therapy in myeloma care.

Dr. Krina Patel is an Associate Professor in the Department of Lymphoma/Myeloma at The University of Texas MD Anderson Cancer Center in Houston, Texas. Dr. Patel is involved in research and cares for patients with multiple myeloma. Learn more about Dr. Patel, here.

Related Resources:

Expert Advice for Newly Diagnosed Myeloma Patients

The Role of a Myeloma Specialist on Your Care Team

Relapsed and Refractory Myeloma Defined

Transcript:

Katherine:    

What is induction therapy?

Dr. Patel:      

So, induction therapy is usually referred for newly diagnosed patients who have never had therapy before and we are now trying to give something to knock that myeloma down. A diagnosis usually is when we have the most myeloma for somebody because they really didn’t know necessarily that they had myeloma before. And our goal is to knock that myeloma down, so it doesn’t cause any more clinical problems.

Katherine:  

What is consolidation therapy?

Dr. Patel:     

So, consolidation means that we’ve knocked that myeloma down now and we’re trying to keep it down, or there’s a little bit of myeloma left that maybe that initial induction therapy didn’t kill; that we try to kind of sweep it all up with the consolidation. So, usually the best example that I can give of consolidation therapy is high-dose melphalan (Alkeran) with stem cell rescuer or what we call autologous stem cell transplant. That, for patients who are in some type of response; then we do that transplant to really kill as many myeloma cells as possible. And then, we do maintenance.

The other part of consolidation therapy is, sometimes after transplant for some of our high-risk patients, or for patients that still have some myeloma even after stem cell transplant, we might give them three or four drugs again to knock the rest of that myeloma down for a little while. Sometimes we do four cycles or so. So, that’s another consolidation strategy.

Katherine:   

How long is a cycle?

Dr. Patel:      

So, each therapy is different but most of our different myeloma therapies cycles are either three or four weeks.

How Can Clinical Trials Be Accessed?

How Can Clinical Trials Be Accessed?  from Patient Empowerment Network on Vimeo.

Clinical researcher Dr. Seth Pollack and patient advocate Sujata Dutta explain the benefits of participating in a clinical trial. They review important questions to ask your doctor and share advice for finding a trial.

Dr. Seth Pollack is Medical Director of the Sarcoma Program at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and is the Steven T. Rosen, MD, Professor of Cancer Biology and associate professor of Medicine in the Division of Hematology and Oncology at the Feinberg School of Medicine. Learn more about Dr. Pollack, here.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

See More from Clinical Trials 101

Related Resources:

What Is a Clinical Trial and What Are the Phases? 

Are Clinical Trials Safe?

A Patient Shares Her Clinical Trial Experience


Transcript:

Katherine Banwell:    

Sujata, there’s clearly a lot of hesitation and misconceptions out there. What would you say to someone who’s considering a trial but is hesitant?

Sujata Dutta:  

I would say speak to your provider, speak to your doctor, and get all these myths kind of busted to say, “it’s going to be expensive” or whatever those questions are. And then, through that process also try and understand what is it that the study is trying to achieve? How is that going to be beneficial to you? So, in my instance, it wasn’t the last line of defense, it was just one of the processes or combos that would help me. And so, that was important for me to understand and then a little bit of education as well. So, I was asking, I have questions on my phone every time I meet my provider, and I did the same thing. So, I think that one of the good practices is keep your note of your questions and have those questions ready. And no question is silly, all questions are important. So, ask as many questions as you can and use that opportunity to educate yourself about it.

And maybe you realize, “No. I don’t think it’s working for me” or “I don’t think this trial is good for me.” But it’s good, important, to have that conversation with your provider, that’s what I would recommend highly.

Katherine Banwell:    

Excellent. Thank you, Dr. Pollack, if someone is interested in participating, how can they find out about what trials are even available for them?

Dr. Seth Pollack:       

Yeah. I mean, the best thing to do is to start just by asking your doctor if they know about any clinical trials. And a lot of the times the clinical trials are run at the big medical centers that may be closer to you, so you could ask your doctor if there’s any clinical trials at the big medical center even. Or I always think it’s good to get a second opinion, you could go get a second opinion at the big medical center that’s close to you and ask them what clinical trials are at your center.

And sometimes they’ll be conscious about some of the clinical trials that may be even run around the country. And you can ask about that as well.

Katherine Banwell:    

Would specialists have more information about clinical trials than say a general practitioner?

Dr. Seth Pollack:       

So, I specialize in rare cancers, so a lot of the times the general practitioners they’ve got my cell phone number, and they text me, and they say, “Hey, do you have a clinical trial going on right now?” And that happens all the time, but yeah, the specialists will usually because frankly there’s so much to know. And the general practitioners really have a lot to keep track of with all the different types of diseases that are out there. Whereas at the big centers, the specialists, part of their job is really to keep their tabs on what’s going on with the clinical trials.

So, they’re good people to ask, either your local doctor could reach out to them, or you could go get a second opinion and ask.

Sujata Dutta:  

There’s also a lot of information, Katherine, on sites such as LLS, or PEN, or American Cancer Society that they also publish a lot of information. Of course, I would recommend once you have that information then vet it by your specialist, or whatever. But if you’re interested in knowing more about clinical trials in general and some that would work for you, then those are also some places to get information from.

Katherine Banwell:    

That’s great information. Thank you, I was going to ask you about that Sujata. Well, before we end the program, Dr. Pollack, I’d like to get your final thoughts. What message do you want to leave the audience with related to clinical trial participation?

Dr. Seth Pollack:       

Yeah. I think clinical trials it can be a very rewarding thing for a lot of patients to do, I think patients really like learning about the new treatments. And I think a lot of patients really like being a part of pushing the therapies forward in addition to feeling like sometimes they’re getting a little bit of an extra layer of scrutiny, because there’s a whole extra team of research coordinators that are going through everything.

And getting access to something that isn’t available yet to the general population. So, I think there’s a whole host of advantages of going on clinical trials, but you need to figure out whether or not a clinical trial is right for you.

Katherine Banwell:    

Yeah. Sujata, what would you like to add?

Sujata Dutta:  

Absolutely, I second everything that Dr. Pollack is saying. And in my personal experience I wouldn’t say everything is hunky-dory, everything is fine. I’m going through treatment, I have chemo every four weeks, I started with chemo every week. That’s when the logistics pace was really difficult because going to Mayo every week was not easy. But anyways, as the trial progress itself every four weeks, but as I said the benefits are huge because I have labs every four weeks. I meet my provider every four weeks.

So, we go through the labs and anything amiss, I’ve had some changes to my dosage because I’ve had some changes in the labs. And so, there’s a lot of scrutiny which I like, but the flip side, for maybe some maybe like, “I have to have chemo every four weeks. Do I want to do that or not?” Or whatever. In my case, I knew it, and I signed up for it, and I’m committed to doing that for two years. And so, I’m fine with that. So, I would say all in all, I’d see more benefits of being in a clinical trial. One, you’re motivated to give back to the community. Two, you are being monitored and so your health is important to your provider just as it is to you. And so, I highly recommend being part of a trial if it works for you and if you’re eligible for one.

Are Clinical Trials Safe?

Are Clinical Trials Safe?  from Patient Empowerment Network on Vimeo.

Clinical researcher Dr. Seth Pollack explains the safety protocols in place for clinical trials, including how data is reported and protected. Patient advocate Sujata Dutta goes on to share her experience in a clinical trial.

Dr. Seth Pollack is Medical Director of the Sarcoma Program at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and is the Steven T. Rosen, MD, Professor of Cancer Biology and associate professor of Medicine in the Division of Hematology and Oncology at the Feinberg School of Medicine. Learn more about Dr. Pollack, here.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

See More from Clinical Trials 101

Related Resources:

Is a Clinical Trial a Last-Resort Option?

A Patient Shares Her Clinical Trial Experience

If I Participate in a Clinical Trial, Will I Be a Guinea Pig?


Transcript:

Katherine Banwell:

Some patients feel that clinical trials aren’t safe, is that the case, Dr. Pollack?  

Dr. Seth Pollack:

No. I mean, we go through, as I was saying before, these clinical trials are extensively vetted. So, the safety is, of course, one of the things that we look most carefully about. But as I was saying before, like with any treatment’s cancer treatments have toxicity, that’s a common problem. So, and when you’re dealing with something brand new sometimes there is a little bit more risk. So, when you’re talking about these very early-stage Phase I trials you probably want to talk to your doctor about what sorts of toxicities you can expect and where they are in the Phase I trial. Are you the first ever to receive this new drug? And if you are nobody’s making you go in the clinical trial, so it can only help to get more information. Right? So, you should ask your team about it, you should find out. 

Most of the time there’s going to be a lot of patients that have been treated already, I mean, they can’t give you definitive data about how things are going but they can maybe say, “Hey. I’ve already treated a few patients on it, and they seem to be doing great.” 

Katherine Banwell:

So, you need to weigh the pros and cons of the trial. 

Dr. Seth Pollack:

You do need to weigh the pros and cons. Now, when you’re talking about these Phase IIs and Phase IIIs, I mean, these are drugs now that have really been vetted for their safety and we have a lot of data about it. And even the Phase Is, it’s not like these things are coming out of nowhere, they’ve been scrutinized, we really expect that they’re going to be safe but we’re doing the trial to prove it. So, it’s a good thing to ask about. 

Katherine Banwell:

Yeah, yeah. 

Sujata Dutta:

Yeah. I would also add that it’s so closely monitored that safety is a top priority, it’s front and center. So, the advantage, I think, with being on a trial is the close monitoring of the patient exactly for this reason. 

If something is amiss it’s going to be picked up as quickly as possible and you’re any issues are going to be addressed as soon as. So, I think, safety does get addressed pretty quickly.  

Katherine Banwell:

Good. 

Can data from trials even be trusted? Dr. Pollack, is that the case? 

Dr. Seth Pollack:

Well, of course, I mean, it can be trusted. Because the thing with the clinical trial data is that you really see the data and there’s all kinds of scrutiny making sure that the data is reported accurately. Now, there’s a whole other conversation we could have as to whether we could interpret the data differently. And sometimes that is an issue that comes up, but the data is reported very accurately. 

So, and there are statistics that are very well understood, and the bar is actually pretty high to say one arm of the trial was better than the other arm of the trial. So, if patients have better survival on one arm, if we say that, usually it means they did considerably better. Enough better that it wasn’t a random chance that one extra patient did better on the treatment arm. No. There were enough patients that did better that the statisticians can go through it with a fine-toothed comb. And they can be absolutely sure up to exactly how many percent sure they can tell you, 0.05 percent or less chance of error that this was a real difference between the study arm and the standard of care arm. 

Sujata Dutta:

I think you mentioned too that one is trust, and one is data. So, Dr. Pollack mentioned a lot about the data, I think the trust is also a very important thing. I like to go with positive intent because I do not have a reason to believe my doctor has some ulterior motive to suggest a clinical trial. And so, I trust them wholeheartedly. The first hurdle is you have to trust the system or what is being proposed to you because, as Dr. Pollack said, it’s gone through a lot of vetting. A recommendation to be part of a trial itself is vetted by your doctor when they make the recommendation. So, have faith, trust, that they are making a good recommendation. And then, of course, the data, I don’t know much about that, but as I said, I trust it. So, I would trust the data too. 

Katherine Banwell:

Of course. Of course. Some patients feel like they’re going to lose their privacy. Sujata, did you feel that at all? 

Sujata Dutta:

No. Not at all. 

I mean, with everything else that is also taken care of, my information, or whatever, is not made available to anybody. And so, obviously there’s a lot of people will get those, and I had a huge pile of paperwork to go through, but I think that’s a good thing. For my peace of mind that I knew that my information was not going to be shared outside of the study, the trial, etc., and things. So, no, I don’t think that’s a problem. 

Katherine Banwell:

Beyond these misconceptions is there anything else you hear? Dr. Pollack?  

Dr. Seth Pollack:

No. I mean, look, in our crazy modern world there’s concerns everywhere, but the clinical trial is very, very careful. Whenever possible we use the medical chart.  

And then, we have a very stringently protected database that’s storing people’s information, but it’s deidentified. So, I mean, we have a separate key to figure out who the patients are and then we try to limit the use of the patient’s name or any identifying information about them beyond that. So, and your information is not shared. For example, if there’s a drug company involved in the trial, your information is not shared with the drug company, you have a new identifier that is unique and not traceable back to you that is provided to whoever, if there’s outside groups working on the trial with you. So, your information is very carefully protected, and everyone is very conscious about issues regarding privacy.  

Katherine Banwell:

That’s great to know.  

A Patient Shares Her Clinical Trial Experience

A Patient Shares Her Clinical Trial Experience  from Patient Empowerment Network on Vimeo.

Sujata Dutta, an empowered patient advocate, explains why she felt participating in a clinical trial was the right decision to treat her myeloma.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

See More from Clinical Trials 101

Related Resources:

If I Participate in a Clinical Trial, Will I Be a Guinea Pig?

Are Clinical Trials a Logistical Nightmare?


Transcript:

Katherine Banwell:    

Sujata, I understand that you went through a series of treatments for your multiple myeloma, which is a type of blood cancer, including a stem cell transplant.

At what point did you and your doctor consider a clinical trial might be best for you?

Sujata Dutta:  

Yes, you’re right. I was diagnosed with multiple myeloma in December, and so the line of treatment or the standard protocol is that you go through what is called an induction therapy. Which is like a few cycles of chemotherapy which get you ready for a transplant. And the transplant, the hope is that it kind of washes away, or cleans off all the cancer cells for you, or at least brings the cancer to a very, very minimal level. And I did go through six rounds of chemo which got me ready for the transplant, and I went through the transplant in June of 2020. However, I’m amongst the very few, small percentage of people that just did not respond with the transplant. So, I was at the same point as where I started. So, it was a little bit disappointing, but my doctors were there to help me understand the situation. It was a hard pill to swallow.

But anyways, there were options. And that’s what I feel very hopeful about with multiple myeloma is that there are so many options available today through treat, or to at least bring the disease under control to a very large extent. And I expressed a desire to be in a trial very earlier on, so my doctor did know that I would lend a year or two listening to what the trials were. And it just so happened that there was a trial that was very apt in my situation, somebody who had gone through a transplant. They have some criteria, and I was able to meet that criteria. And so, for me, it seemed to be the right decision to make. And so, that’s how I agreed to be part of the trial.

Katherine Banwell:    

Can you go into some detail about why you thought a clinical trial was a best thing for you?

Sujata Dutta:  

Yeah. So, initially before knowing much about the strain that I’m a part of, I just had the desire to be part of a trial because I was always in awe of patients who had been in trials before me.

And because of whom I was benefiting. But whatever regiments, medications, combos, whatever was happening. And so, from that perspective I always wanted to give back in some way. Unfortunately, we are having more people being diagnosed with cancers, with multiple myeloma, and so I was very motivated to do something for the community that I was now part of. And so, I had my transplant at Mayo, and I knew that they had a whole bunch of trials and had access to different types of trials. So, that was my first motivation and it just so happened that, as I said, my experience with transplant didn’t go the desired way. And so, when I heard that there was a possibility that I could be part of a trial, I kind of leaned into actually agreeing to be part of that.

Katherine Banwell:    

Yeah. It sounds like that was the next step for you.

Sujata Dutta:  

Yup.

How Is Relapsed/Refractory DLBCL Treated?

How Is Relapsed/Refractory DLBCL Treated? from Patient Empowerment Network on Vimeo.

What options are available if a diffuse large B-cell lymphoma (DLBCL) patient doesn’t respond to treatment or relapses? Dr. Justin Kline discusses potential next steps in treatment for DLBCL patients with relapsed or refractory disease. 

Dr. Justin Kline is the Director of the Lymphoma Program at the University of Chicago Medicine. Learn more about Dr. Kline, here.

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Transcript:

Katherine:      

Let’s talk about if someone doesn’t respond to initial treatment or they relapse. Let’s start by defining some terms for the audience. What does it mean to be refractory?

Dr. Kline:       

So, refractory is a term that’s used to describe a situation where a person has received treatment but that treatment hasn’t worked as well as we have expected. And the most – probably the most important scenario is after initial treatment.

Most people, for example, who receive R-CHOP, somewhere between 80 and 85 percent will have a completely negative PET scan after treatment. That’s remission. If the PET scan is not negative and you do a biopsy and it shows that there’s still lymphoma there, that’s what’s called primary refractory. In other words, the person’s lymphoma was refractory to initial or primary treatment. And in clinical trials that are testing agents, drugs or immunotherapies in folks who’ve had multiple treatments, usually refractory is used to define someone who has either not responded or has had a very, very short response to whatever the last treatment they had was.

Katherine:                  

How does relapse then differ from refractory?

Dr. Kline:       

So, right, so relapse suggests that the lymphoma at some point was in a remission, right?

And so for example, a person gets six treatments of R-CHOP, has a PET scan at the end, the PET scan is clean. We say you’re in remission. Eight months later, the person develops a newly enlarged lymph node, and a biopsy shows that the lymphoma has come back, right? That’s what we would call a relapse. There was a period of remission, whereas refractory usually means there was never a period of remission to begin with.

Katherine:                  

Got it. How typical is it for a patient to relapse?

Dr. Kline:       

Well, again, if you look at all comers, if you treated 100 people with DLBCL, most, probably 70 to 75 percent, would go into remission. About 10 or 15 percent would have primary refractory disease and another 10 or 15 percent would have a remission that would end at some point and they would have a relapse. So, it’s not terribly common.

The problem is that once the lymphoma has either demonstrated that it’s refractory to treatment or it’s come back, it’s relapsed, it’s a little bit more difficult to cure the lymphoma at that point.

Katherine:      

How are patients treated then if they’ve relapsed or refractory?

Dr. Kline:       

Well, so for somebody who’s had primary refractory lymphoma or has a lymphoma that’s relapsed after initial therapy, again, say for the sake of argument with R-CHOP, for many, many years, the next line of treatment if you will was to administer what we call salvage chemotherapy, and this is different chemotherapy from the original R-CHOP, that’s meant to put the lymphoma back into remission. In other worse, to salvage a remission. And for folks whose lymphomas were sensitive or responded, shrunk down to that salvage chemotherapy, we would consolidate that remission.

We would make it deeper using high dose chemotherapy and an autologous or a cell, stem cell transplant. And that’s been the standard of care for younger patients for decades.

That paradigm has been challenged, particularly in refractory patients or those who have very early relapses after R-CHOP, by two important clinical trials that have demonstrated superiority of a type of immunotherapy, a cellular immunotherapy called CAR T-cell therapy, which seems to be more effective even than stem cell transplantation in that population of folks.

How Is DLBCL Treated?

How Is Diffuse Large B-Cell Lymphoma (DLBCL) Treated? from Patient Empowerment Network on Vimeo.

Dr. Justin Kline explains what patients need to know about current DLBCL treatment, including R-CHOP, stem cell therapy, and clinical trials.

Dr. Justin Kline is the Director of the Lymphoma Program at the University of Chicago Medicine. Learn more about Dr. Kline, here.

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Transcript:

Katherine:      

From what I understand, treatment really should start right away. So, what types of treatment are currently available to someone newly diagnosed with DLBCL?

Dr. Kline:       

Sure, so for about 20 plus years now, the standard of care for most patients with DLBCL, regardless of whether it’s a germinal center or an activated B-cell type DLBCL, is a combination of what we call chemo immunotherapy, the acronym for which is R-CHOP, and each of those letters stands for a different medication. The R stands for rituximab, which is an antibody that coats the surface of lymphomatous B cells and sort of signals the immune system to come and kill those cells.

The C is cyclophosphamide, the H is hydroxy doxorubicin, and the O is Oncovin. These are each classical chemotherapy drugs, and they each work through a different mechanism to help kill lymphoma sells. And the P is a steroid pill called prednisone, so it’s a little bit complicated, but the reason that we use cocktails of medicines to treat lymphomas is that it really works to prevent the lymphoma cells from gaining the upper hand, from developing resistance to a single type of treatment.

Katherine:      

Right.

Dr. Kline:       

Now, I should say that for certain DLBCLs, particularly those double hit lymphomas that we talked about, sometimes we use a more intensive cocktail called dose-adjusted R-EPOCH. It has largely the same medications with an additional chemotherapy called etoposide.

The difference is that R-CHOP is given – all the drugs are given intravenously, with the exception of prednisone, over a single day. The dose-adjusted R-EPOCH is given over an infusion over the course of about five days. The other point I might make is that there was a recent large clinical study that compared R-CHOP to a new regimen called polatuzumab R-CHP. So, basically the O in R-CHOP was removed and substituted for this new drug called polatuzumab vedotin, and although many, many combinations similar to R-CHOP have been compared to R-CHOP over the past 20 years and failed, this regimen, polatuzumab R-CHP in the study called the POLARIX study actually was shown to improve what we call progression-free survival by about six percent. So, it may become a new standard of care for treating DLBCL, which is exciting, because we haven’t had one in over 20 years.

Katherine:                  

Right. That’s good news.

Dr. Kline:       

Long answer to a short question, sorry about that. Yes, it is good news.

Katherine:      

That is good news. What about stem cell transplants?

Dr. Kline:       

Good question. So, for newly diagnosed patients, in this era, we rarely if ever are recommending stem cell transplant or stem cell transplantation as part of initial therapy. There are rare circumstances, but for the vast majority of patients who are, people who are diagnosed with DLBCL, it’s not recommended.

Katherine:      

Where do clinical trials fit in?

Dr. Kline:       

It’s a really good question. I practice at an academic medical center, and so one of our missions is to advance therapy and make it better. There’s no way to do that without performing clinical trials, so I think for – clinical trials aren’t for everyone. As a matter of fact, most people with lymphoma are not treated in the context of clinical trials.

But certainly I think they are important to consider, and number one, it’s possible that the particular person might be involved with the clinical trial that is very successful and actually improves their outcome. I always tell people that I see that being involved with the clinical trial is also, to some extent, an altruistic endeavor. You’re helping your doctors learn more about how to treat a type of cancer, hopefully better, maybe not, you know? So, there is some altruism that goes into clinical trials as well. So, I do think that most people who are able should consider having a second opinion. Doesn’t have to be at an academic medical center, but at least with another doctor, where clinical trial options can be discussed.

Could a Clinical Trial Be Your Best Cancer Treatment Option?

Could a Clinical Trial Be Your Best Cancer Treatment Option? from Patient Empowerment Network on Vimeo.

Is a clinical trial right for you? Cancer expert and researcher Dr. Seth Pollack is joined by PEN board member and empowered patient, Sujata Dutta, to discuss key information about clinical trials. The guests review clinical trial terminology, debunk common misconceptions about trials, and Sujuta shares her own story of participation in a clinical trial.

Dr. Seth Pollack is Medical Director of the Sarcoma Program at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and is the Steven T. Rosen, MD, Professor of Cancer Biology and associate professor of Medicine in the Division of Hematology and Oncology at the Feinberg School of Medicine. Learn more about Dr. Pollack, here.

Sujuta Dutta is a myeloma survivor and empowered patient advocate, and serves a Patient Empowerment Network (PEN) board member. Learn more about Sujuta, here.

Download Guide

See More from Clinical Trials 101

Related Resources:

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Understanding Clinical Trial Phases

How Could Clinical Trials Fit Into Your Myeloma Treatment Plan?


Transcript:

Katherine Banwell:    

Hello, and welcome. I’m Katherine Banwell, your host for today’s program.

Today we’re going to discuss clinical trials, what they are and how they work, and debunk some misconceptions along the way. 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 guests today. Joining me is Dr. Seth Pollack. Dr. Pollack, welcome. Would you please introduce yourself?

Dr. Seth Pollack:

Yeah. Thanks so much. It’s a pleasure to be here, my name is Seth Pollack. I’m a medical oncologist here at Northwestern University Medical Center.

And I specialize in treating patients with cancer, and I have a specific interest in patients with a type of cancer called sarcomas.

Katherine Banwell:    

Excellent. Thank you for taking the time to join us today. And here to share the patient perspective is Sujata Dutta, who is on the board of the Patient Empowerment Network and is currently participating in a clinical trial. Sujata, it’s a pleasure to have you with us.

Sujata Dutta:

Pleasure to be here Katherine. Hello, Dr. Pollack. And hi everyone, my name is Sujata Dutta, and I was diagnosed with a cancer called multiple myeloma in December of 2019. And I’ve been on a clinical trial since September of 2020.

Katherine Banwell:    

Thank you, for that information. And we’re going to go into that further in just a few moments. Let’s start with a basic question, Dr. Pollack. What is a clinical trial?

Dr. Seth Pollack:       

Yeah. It’s a basic question, but actually, sometimes, it can be harder to answer than you might think.

I think everybody has an idea in their mind about what a clinical trial is, you’re going to test a new approach. But actually, there’s a whole variety of different things that can be a clinical trial, right? A clinical trial a lot of the times is testing a new drug, could be testing something for the very first time, could be testing something in combination with other drugs for the very first time. It could be testing a standard approach but doing it in a new way. It could even be giving less treatments than we usually do. For example, if there’s a very intense, harsh, standard of care treatment we might even have a clinical trial where we try a little bit less and see that patients do just as well. So, all of those things are clinical trials, but really the clinical trial in its heart is a very organized and careful approach to testing a new treatment strategy for patients.

Katherine Banwell:    

Okay. What are the phases of a clinical trial?

Dr. Seth Pollack:       

Yup. So, the Phase I clinical trial is usually when we’re testing something for the first time in however we’re doing it. So, it could be the first time we’re testing a new drug, or the first time we’re testing a drug in combination with other drugs. And the real thing about a Phase I trial is that the main goal of the trial is to look at the safety and tolerability of the regiment. That doesn’t mean that we’re not really trying to figure out if the regiment works, I mean, that’s also one of the most important things. But the most important thing for a Phase I trial is making sure that it’s safe and tolerable. A Phase II trial is where we, sort of, shift and we’re still making sure, and double checking, that the drug is, but now our main focus becomes on the efficacy of the strategy.

So, now we’re trying to really figure out if this is a strategy that seems affective enough to go to a Phase III. And a Phase III is a big multi-center trial. Frequently those will be placebo controlled where a lot of the times there’ll be randomized trial where we really try to absolutely prove, beyond a shadow of a doubt, that, that strategy is affective. And those are most of the types of trials that patients will encounter.

Katherine Banwell:    

Okay. Thank you for providing clarity around the phases. Before we move onto safety and benefits of clinical trials, let’s hear from Sujata. Sujata, I understand that you went through a series of treatments for your multiple myeloma, which is a type of blood cancer, including a stem cell transplant.

At what point did you and your doctor consider a clinical trial might be best for you?

Sujata Dutta:  

Yes, you’re right. I was diagnosed with multiple myeloma in December, and so the line of treatment or the standard protocol is that you go through what is called an induction therapy. Which is like a few cycles of chemotherapy which get you ready for a transplant. And the transplant, the hope is that it kind of washes away, or cleans off all the cancer cells for you, or at least brings the cancer to a very, very minimal level. And I did go through six rounds of chemo which got me ready for the transplant, and I went through the transplant in June of 2020. However, I’m amongst the very few, small percentage of people that just did not respond with the transplant. So, I was at the same point as where I started. So, it was a little bit disappointing, but my doctors were there to help me understand the situation. It was a hard pill to swallow.

But anyways, there were options. And that’s what I feel very hopeful about with multiple myeloma is that there are so many options available today through treat, or to at least bring the disease under control to a very large extent. And I expressed a desire to be in a trial very earlier on, so my doctor did know that I would lend a year or two listening to what the trials were. And it just so happened that there was a trial that was very apt in my situation, somebody who had gone through a transplant. They have some criteria, and I was able to meet that criteria. And so, for me, it seemed to be the right decision to make. And so, that’s how I agreed to be part of the trial.

Katherine Banwell:    

Can you go into some detail about why you thought a clinical trial was a best thing for you?

Sujata Dutta:  

Yeah. So, initially before knowing much about the strain that I’m a part of, I just had the desire to be part of a trial because I was always in awe of patients who had been in trials before me.

And because of whom I was benefiting. But whatever regiments, medications, combos, whatever was happening. And so, from that perspective I always wanted to give back in some way. Unfortunately, we are having more people being diagnosed with cancers, with multiple myeloma, and so I was very motivated to do something for the community that I was now part of. And so, I had my transplant at Mayo, and I knew that they had a whole bunch of trials and had access to different types of trials. So, that was my first motivation and it just so happened that, as I said, my experience with transplant didn’t go the desired way. And so, when I heard that there was a possibility that I could be part of a trial, I kind of leaned into actually agreeing to be part of that.

Katherine Banwell:    

Yeah. It sounds like that was the next step for you.

Sujata Dutta:  

Yup.

Katherine Banwell:    

Yeah. Well, I’d like to address a list of common concerns about clinical trials that we’ve heard from various audience members prior to this program.

And this is probably the most common; I will be a guinea pig. Dr. Pollack, how do you respond to that?

Dr. Seth Pollack:       

Yeah. I know that is a common concern. I mean, I think the thing that people have to understand about clinical trials is there is just so much oversight that happens for these clinical trials. Every document, every procedure, is scrutinized by multiple committees. There’s a scientific review committee, there’s a review board, IRB, that reviews these. Many of these trials are reviewed by the FDA and they’re reviewed by your doctor and your doctor’s colleagues that are also participating in the trial. So, every detail is discussed at length.

In fact, a lot of the times there’s a lot more structure to being on the clinical trial than just routine clinical care because they’ve thought so thoroughly about when everything needs to be done and what the right timing of is for the various procedures.

Katherine Banwell:    

Right. And another concern that people have is; clinical trials are my last resort treatment option. What do you say to that Dr. Pollack?

Dr. Seth Pollack:       

Yeah, no. That’s a common misconception. So, we like to have clinical trials for every phase of the patient’s cancer journey because we’re trying to make every single part of the cancer journey better. So, I think a lot of people think that, okay, when they hit their last resort that’s kind of the time to try something new. Even in the very earliest parts of the cancer journey, even in the diagnosis phase sometimes we’ll have clinical trials where we’ve tried different images, modalities, or look at things in a different way in terms of the biopsies.

But then, in terms of the cured-of treatments, when somebody is in the cured-of setting we don’t usually try something very brand new. But a lot of the times we’ll try something that is very affective for patients at the end, and we want to try and make the cured-of strategy even better. So, a lot of the times for those patients we’ll have new therapies that are very safe and established that we’re trying to incorporate earlier into patients’ treatments because we know they work really well, right? And then, even in patients who have incurable cancer a lot of times it’s better to try a clinical trial earlier on just because sometimes the clinical trials have the most exciting new therapies that are bringing people a lot of hope.

And a lot of the times you want to try that when you’re really fit and when you’re in good shape. So, that’s why I think that you really want to think about doing a clinical trial when the opportunity arises.

Katherine Banwell:    

Yeah. Beause it could be beneficial to you and it’s certainly going to be beneficial to other people. Is this fact or fiction; it will be expensive? Dr. Pollack?

Dr. Seth Pollack:       

That’s fiction because the way the clinical trials work is we go through everything very carefully to figure out what things are standard and what things are unique to the clinical trials. So, if you are getting chemotherapy, you’re going to need blood work, you’re going to need the chemotherapy drugs, you’re going to need some sort of imaging, CT scan, or whatever your doctor would do.

And all those sorts of things are considered standard, so your insurance company is built for those. Then there’s a bunch of things that are considered research. For example, there’s special research bloodwork, maybe there’s an investigational agent that’s being added to standard chemotherapy. Those things are billed to the study, so you don’t actually have to pay anything extra, it’s just like you’re getting the normal treatment as far as you’re concerned. I mean, that’s the way it always is, and I haven’t had any of my patients ever get into real problems in terms of the finances of these things. It always works very straight forward like standard therapy.

Katherine Banwell:    

Okay. That’s good to know. The logistics will be a nightmare and I don’t live close to a research hospital. Sujata, did you have that issue?

Sujata Dutta:  

Yeah. That’s a very interesting one, and actually I’ll share my experience. I did have this concern about logistics, because I got my transplant at Mayo Rochester, which is a two-hour drive from where I live. And so, when I got to know about it literally me and my husband were like, “Oh, my gosh. What are we going to do?” It’s not just me, my husband is my caregiver, he has to take the day off to drive me to Mayo, wait through my treatment, and drive me back. Then we have boys who were distance learning at the time, and so what do we do with them? Do we drop off a friends or take a favor from a friend? And so on and so forth.

So, the logistics was an issue and we literally said, “Thanks but no thanks” and we walked out of the room. And we came downstairs, and my husband was like, “What the heck?” My team understands everything, and I fortunately work for a very good employer, and they understand everything, people first. And so, he was like, “I can figure this out. Let’s do it if this is what’s going to help you, then let’s just figure this out.” And at that time, it was so good, and I have total respect for Dr. Pollack.

You and everybody in this medical community. My doctor who leads the trial at Mayo, she actually said, “Why don’t you check with your local cancer center? Maybe they are also approved by FDA, and they may be able to administer this treatment to you.” Unfortunately, at that time they weren’t but we were like, “We’re going to go ahead with the trial. It doesn’t matter.” My husband was like, “I’ll take the day off, you don’t worry about it.” And then, four months later my institute did get approved by FDA, and so I was able to transfer from Mayo to my local cancer center, Abramson Cancer Center, which is 20 minutes from home. And so, there are options, I know that it can be an issue and it can be overwhelming at the time which was the case with me. But I was able to overcome that, so maybe there are options available that the patients can consider.

Katherine Banwell:    

Yeah. Dr. Pollack, do you have anything to add?

Dr. Seth Pollack:       

No. I think the logistics and the location are real concerns with clinical trials.

Clinical trials do sometimes require you to have an extra visit, sometimes they’re a little bit less flexible in terms of when you can get your medication. If you’re getting a standard treatment your doctor may say, “It’s probably okay for you to wait an extra week.” Whereas sometimes on a clinical trial, not always, but sometimes they could be a little bit more strict about when you’re supposed to get certain things. And likewise, with the travel for some people that can be an issue. I mean, the clinical trial is not available everywhere. I mean, Sujata was very lucky that she was able to do the clinical trial she was doing close to home, but that doesn’t always happen. So, I think that’s an important thing to talk to your clinical team about.

Katherine Banwell:    

Yeah. Some patients feel that clinical trials aren’t safe, is that the case, Dr. Pollack?

Dr. Seth Pollack:       

No. I mean, we go through, as I was saying before, these clinical trials are extensively vetted. So, the safety is, of course, one of the things that we look most carefully about. But as I was saying before, like with any treatment’s cancer treatments have toxicity, that’s a common problem. So, and when you’re dealing with something brand new sometimes there is a little bit more risk. So, when you’re talking about these very early-stage Phase I trials you probably want to talk to your doctor about what sorts of toxicities you can expect and where they are in the Phase I trial. Are you the first ever to receive this new drug? And if you are nobody’s making you go in the clinical trial, so it can only help to get more information. Right? So, you should ask your team about it, you should find out.

Most of the time there’s going to be a lot of patients that have been treated already, I mean, they can’t give you definitive data about how things are going but they can maybe say, “Hey. I’ve already treated a few patients on it, and they seem to be doing great.”

Katherine Banwell:    

So, you need to weigh the pros and cons of the trial.

Dr. Seth Pollack:       

You do need to weigh the pros and cons. Now, when you’re talking about these Phase IIs and Phase IIIs, I mean, these are drugs now that have really been vetted for their safety and we have a lot of data about it. And even the Phase Is, it’s not like these things are coming out of nowhere, they’ve been scrutinized, we really expect that they’re going to be safe but we’re doing the trial to prove it. So, it’s a good thing to ask about.

Katherine Banwell:    

Yeah, yeah.

Sujata Dutta:  

Yeah. I would also add that it’s so closely monitored that safety is a top priority, it’s front and center. So, the advantage, I think, with being on a trial is the close monitoring of the patient exactly for this reason.

If something is amiss it’s going to be picked up as quickly as possible and you’re any issues are going to be addressed as soon as. So, I think, safety does get addressed pretty quickly.

Katherine Banwell:    

Good, good. Okay. That’s good to know. Another concern is; I’ll get a placebo. Dr. Pollack, what is a placebo first of all? And is that true in a clinical trial setting?

Dr. Seth Pollack:       

So, there are clinical trials with placebos, it’s a real thing. And what a placebo is, it’s a pill and it’s made to look just like the real pill, but it doesn’t have any active drug in it. Sometimes people say it’s a sugar pill, but it may or may not be sugar, but it’ll probably be something without a taste. But it’s an inert substance that is not going to affect you at all.

And your doctor won’t know whether you’re getting a placebo or not, so a lot of the times they’ll call these things double-blind because your doctor doesn’t know, your pharmacist doesn’t know. And to unblind you they have to go through special procedures to find out whether you’re on the studied drug or not.

Katherine Banwell:    

Would a placebo be given solely? Or would it be given in addition to this new drug that’s being tested?

Dr. Seth Pollack:       

Yeah. So, it’s unusual for a placebo to be given solely. Usually there’ll be a clinical trial where you’re getting the standard treatments plus the new drug or standard treatment plus the placebo, so no matter what you’re getting the standard treatments. There are still some trials where, and these are usually for patients with very advanced cancer, who there’s not really any treatment options that are good. Where they will randomize people to just be on the standard drug versus the placebo.

Sometimes what they’ll do is if they want to do a trial that’s the standard drug versus a placebo, they’ll do the imaging very frequently and they’ll have a crossover. So, a crossover means that everybody gets to be on the new drug, but some people will have to go on the placebo first. So, and then they watch you very closely. So, if you get randomized to go on the placebo and your cancer starts to grow, they figure it out very quickly and then they give you the opportunity to go on the new drug.

Katherine Banwell:    

I see, okay. I’ll be stuck in the trial forever and I can’t change my mind. Sujata, did that happen to you?

Sujata Dutta:  

No. I mean, when I finally agreed and signed the dotted line it was made very clear to me that it was voluntary, I was volunteering to be part of the trial and I could get out of the trial at any point of time. So, in my case I’m in Phase III of a trial, the first commitment was for two years and then the next was five years.

So, again, it sounds daunting to me right now, two years is coming to an end in July of this year. I’m like, “Wow! Two years are over already?” And then five years, I’m not thinking about that, but again, it was at any point I could just say that I’ve had enough, or whatever be the reason, I could get out of the trial. So, no. Yes. There’s an option.

Katherine Banwell:    

Can data from trials even be trusted? Dr. Pollack, is that the case?

Dr. Seth Pollack:       

Well, of course, I mean, it can be trusted. Because the thing with the clinical trial data is that you really see the data and there’s all kinds of scrutiny making sure that the data is reported accurately. Now, there’s a whole other conversation we could have as to whether we could interpret the data differently. And sometimes that is an issue that comes up, but the data is reported very accurately.

So, and there are statistics that are very well understood, and the bar is actually pretty high to say one arm of the trial was better than the other arm of the trial. So, if patients have better survival on one arm, if we say that, usually it means they did considerably better. Enough better that it wasn’t a random chance that one extra patient did better on the treatment arm. No. There were enough patients that did better that the statisticians can go through it with a fine-toothed comb. And they can be absolutely sure up to exactly how many percent sure they can tell you, 0.05 percent or less chance of error that this was a real difference between the study arm and the standard of care arm.

Sujata Dutta:  

I think you mentioned too that one is trust, and one is data. So, Dr. Pollack mentioned a lot about the data, I think the trust is also a very important thing. I like to go with positive intent because I do not have a reason to believe my doctor has some ulterior motive to suggest a clinical trial. And so, I trust them wholeheartedly. The first hurdle is you have to trust the system or what is being proposed to you because, as Dr. Pollack said, it’s gone through a lot of vetting. A recommendation to be part of a trial itself is vetted by your doctor when they make the recommendation. So, have faith, trust, that they are making a good recommendation. And then, of course, the data, I don’t know much about that, but as I said, I trust it. So, I would trust the data too.

Katherine Banwell:    

Of course. Of course. Some patients feel like they’re going to lose their privacy. Sujata, did you feel that at all?

Sujata Dutta:  

No. Not at all.

I mean, with everything else that is also taken care of, my information, or whatever, is not made available to anybody. And so, obviously there’s a lot of people will get those, and I had a huge pile of paperwork to go through, but I think that’s a good thing. For my peace of mind that I knew that my information was not going to be shared outside of the study, the trial, etc., and things. So, no, I don’t think that’s a problem.

Katherine Banwell:    

Beyond these misconceptions is there anything else you hear? Dr. Pollack?

Dr. Seth Pollack:       

Well, I hear a lot of people really interested in clinical trials. I mean especially, I treat some patients with rare cancers or with unusual presentations and I think people are very excited to be a part of something that could be new, that could be the next wave. A lot of times the clinical trials have new things with the most exciting science that could be the future of treatment.

So, I think a lot of people are excited about clinical trials. And I also hear some of the reservations that you’re expressing. I think usually when patients ask their questions are very straightforward and easy to address so that people can make their own decisions.

Katherine Banwell:    

Dr. Pollack, I’d like to go back to you and ask you the same question about privacy. Do patients need to be worried about that?

Dr. Seth Pollack:       

No. I mean, look, in our crazy modern world there’s concerns everywhere, but the clinical trial is very, very careful. Whenever possible we use the medical chart.

And then, we have a very stringently protected database that’s storing people’s information, but it’s deidentified. So, I mean, we have a separate key to figure out who the patients are and then we try to limit the use of the patient’s name or any identifying information about them beyond that. So, and your information is not shared. For example, if there’s a drug company involved in the trial, your information is not shared with the drug company, you have a new identifier that is unique and not traceable back to you that is provided to whoever, if there’s outside groups working on the trial with you. So, your information is very carefully protected, and everyone is very conscious about issues regarding privacy.

Katherine Banwell:    

That’s great to know. Sujata, there’s clearly a lot of hesitation and misconceptions out there. What would you say to someone who’s considering a trial but is hesitant?

Sujata Dutta:  

I would say speak to your provider, speak to your doctor, and get all these myths kind of busted to say, “it’s going to be expensive” or whatever those questions are. And then, through that process also try and understand what is it that the study is trying to achieve? How is that going to be beneficial to you? So, in my instance, it wasn’t the last line of defense, it was just one of the processes or combos that would help me. And so, that was important for me to understand and then a little bit of education as well. So, I was asking, I have questions on my phone every time I meet my provider, and I did the same thing. So, I think that one of the good practices is keep your note of your questions and have those questions ready. And no question is silly, all questions are important. So, ask as many questions as you can and use that opportunity to educate yourself about it.

And maybe you realize, “No. I don’t think it’s working for me” or “I don’t think this trial is good for me.” But it’s good, important, to have that conversation with your provider, that’s what I would recommend highly.

Katherine Banwell:    

Excellent. Thank you, Dr. Pollack, if someone is interested in participating, how can they find out about what trials are even available for them?

Dr. Seth Pollack:       

Yeah. I mean, the best thing to do is to start just by asking your doctor if they know about any clinical trials. And a lot of the times the clinical trials are run at the big medical centers that may be closer to you, so you could ask your doctor if there’s any clinical trials at the big medical center even. Or I always think it’s good to get a second opinion, you could go get a second opinion at the big medical center that’s close to you and ask them what clinical trials are at your center.

And sometimes they’ll be conscious about some of the clinical trials that may be even run around the country. And you can ask about that as well.

Katherine Banwell:    

Would specialists have more information about clinical trials than say a general practitioner?

Dr. Seth Pollack:       

So, I specialize in rare cancers, so a lot of the times the general practitioners they’ve got my cell phone number, and they text me, and they say, “Hey, do you have a clinical trial going on right now?” And that happens all the time, but yeah, the specialists will usually because frankly there’s so much to know. And the general practitioners really have a lot to keep track of with all the different types of diseases that are out there. Whereas at the big centers, the specialists, part of their job is really to keep their tabs on what’s going on with the clinical trials.

So, they’re good people to ask, either your local doctor could reach out to them, or you could go get a second opinion and ask.

Sujata Dutta:  

There’s also a lot of information, Katherine, on sites such as LLS, or PEN, or American Cancer Society that they also publish a lot of information. Of course, I would recommend once you have that information then vet it by your specialist, or whatever. But if you’re interested in knowing more about clinical trials in general and some that would work for you, then those are also some places to get information from.

Katherine Banwell:    

That’s great information. Thank you, I was going to ask you about that Sujata. Well, before we end the program, Dr. Pollack, I’d like to get your final thoughts. What message do you want to leave the audience with related to clinical trial participation?

Dr. Seth Pollack:       

Yeah. I think clinical trials it can be a very rewarding thing for a lot of patients to do, I think patients really like learning about the new treatments. And I think a lot of patients really like being a part of pushing the therapies forward in addition to feeling like sometimes they’re getting a little bit of an extra layer of scrutiny, because there’s a whole extra team of research coordinators that are going through everything.

And getting access to something that isn’t available yet to the general population. So, I think there’s a whole host of advantages of going on clinical trials, but you need to figure out whether or not a clinical trial is right for you.

Katherine Banwell:    

Yeah. Sujata, what would you like to add?

Sujata Dutta:  

Absolutely, I second everything that Dr. Pollack is saying. And in my personal experience I wouldn’t say everything is hunky-dory, everything is fine. I’m going through treatment, I have chemo every four weeks, I started with chemo every week. That’s when the logistics pace was really difficult because going to Mayo every week was not easy. But anyways, as the trial progress itself every four weeks, but as I said the benefits are huge because I have labs every four weeks. I meet my provider every four weeks.

So, we go through the labs and anything amiss, I’ve had some changes to my dosage because I’ve had some changes in the labs. And so, there’s a lot of scrutiny which I like, but the flip side, for maybe some maybe like, “I have to have chemo every four weeks. Do I want to do that or not?” Or whatever. In my case, I knew it, and I signed up for it, and I’m committed to doing that for two years. And so, I’m fine with that. So, I would say all in all, I’d see more benefits of being in a clinical trial. One, you’re motivated to give back to the community. Two, you are being monitored and so your health is important to your provider just as it is to you. And so, I highly recommend being part of a trial if it works for you and if you’re eligible for one.

Katherine Banwell:    

Yeah. Sujata Dutta, and Dr. Pollack, thank you both for taking the time to join us today.

Sujata Dutta:   

Thank you.

Dr. Seth Pollack:       

Thank you.

Katherine Banwell:    

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

What Is Precision Medicine for MPNs?

What Is Precision Medicine for MPNs? from Patient Empowerment Network on Vimeo.

Myeloproliferative neoplasm (MPN) patients have the option of precision medicine in the treatment toolbox. Dr. Kristen Pettit from Rogel Cancer Center shares insight about some forms of precision medicine, disease-specific factors, and potential future treatments for personalized medicine.

See More From the MPN TelemEDucation Resource Center

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Transcript:

Dr. Kristen Pettit:

Precision medicine or personalized medicine can take many forms in the MPN field, certainly decisions about whether to consider something like a stem cell transplant or personalized based on disease-specific factors such as the prognostic risk of the individual disease and based on person-specific factors, for example, the patient’s symptoms, their quality of life, their goals, and their other medical issues. Similar personalized factors go into other treatment decisions such as whether or not to start a JAK inhibitor or other treatment as well.

In the future, I think MPN care will continue to see more personalization, perhaps even involving information like specific genetic mutations, for example, there are drugs that are in development that may target on mutant calreticulin, so those might be possibly more specific treatments for folks just with those CALR mutations.

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.
 
 

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How Treatment Goals Impact MPN Treatment Decisions

How Treatment Goals Impact MPN Treatment Decisions

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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.
 

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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.
 

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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.