Tag Archive for: low platelets

Thrive | What You Should Know About MPN Symptoms & Treatment Side Effects

Thrive | What You Should Know About MPN Symptoms & Treatment Side Effects from Patient Empowerment Network on Vimeo.

How are MPN symptoms and treatment side effect managed? In this animated explainer video, an MPN specialist and myelofibrosis patient discuss the importance of clear communication with your healthcare team, the process for assessing common issues, and advice for advocating for yourself.

 

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Transcript:

Brian: 

Hi, I’m Brian. Nice to meet you! I’ve been living with a condition called myelofibrosis for many years. While there have certainly been ups and downs, I’ve been able to navigate care for my condition and to live a full life.  

So how have I been able to do that? First and foremost, I have a great relationship with my care team, whom I communicate with regularly. Meet, Dr. Liu – my doctor. 

Dr. Liu: 

Hi! I’m Dr. Liu, and I’m a hematologist and a specialist in myeloproliferative neoplasms or MPNs. The three types of MPNs are essential thrombocythemia, or ET,  polycythemia vera or PV, and myelofibrosis, or MF.  This group of blood cancers is characterized by the bone marrow overproducing a certain type of cell.  

Maintaining a good relationship with your healthcare team, coupled with finding a treatment approach that works for you, can help you live a full life and to thrive with an MPN. 

Brian: 

Exactly, Dr. Liu. Over the years, I’ve experienced periodic issues with my condition. I’ve had symptoms and treatment side effects that have been bothersome and interfered with my life. But, communication with my team has been essential to feeling well.  

Dr. Liu: 

That’s right, Brian. When symptoms or treatment side effects are bothering you, it’s important to let your healthcare provide know how you are feeling. 

Brian: 

For example, recently I felt tired beyond general sleepiness. And when I shared this with Dr. Liu, we discussed potential causes of the fatigue, and we talked in-depth about my options to manage it, including changing therapy and some simple changes to my diet and lifestyle.1 Over time, my energy levels improved, but having the open dialogue with Dr. Liu was essential to tackling this symptom head-on. 

Dr. Liu: 

That’s a great example. When I first hear from a patient that they are having an issue, we go through several steps to find a solution.2  

We start by ensuring that the disease is well-controlled, so we check blood counts. Next, we try to determine if it is a symptom of the MPN or a side effect of the treatment. Once we’ve done those steps, we come up with potential solutions which may include, but are not limited to: 

  • A dose reduction or a treatment holiday. 
  • Changing therapy to find something that is more well-tolerated. 

Other considerations are dependent upon the specific symptoms and side effects but may include: 

  • Supportive care options, including diet and exercise. 
  • A visit to your primary care doctor to see if there is something else going on physically. 

Brian: 

That’s good to know, Dr. Liu. Something you brought up with me, which I feel is important to mention, is mental health. Often, emotional symptoms can take a toll on the body, causing fatigue or other issues. 

Dr. Liu: 

Great point, Brian. Seeking care for your mental health is crucial, particularly if you are in active treatment. 

Brian: 

Of course, we know that the symptoms and treatment side effects for MPNs can vary widely, so what advice do you have for patients who may be afraid to speak up? 

Dr. Liu: 

The most important thing to remember is that we have options to help you, no matter what you are going through. It’s your body and if you don’t let your provider know what you’re going through, they can’t help you. 

Brian: 

So true. It’s also a good idea to bring a care partner along to appointments, sometimes a spouse or friend can you help you communicate what’s going on. 

Dr. Liu: 

That’s great advice, Brian. Bringing someone along to take notes is a great idea. Also, be sure to write down any questions or concerns you have in advance to make the most of your appointment. 

Brian: 

OK, Dr. Liu, let’s recap your advice for MPN symptom management: 

Dr. Liu: 

Good idea! First, remember that everyone’s MPN is different, so managing symptoms and side effects can be tricky. Communicating with your healthcare team is critical to your overall care – report any and all concerns to your team immediately. 

And, do your part. Make sure you see your primary care physician regularly and do your best to maintain a healthy lifestyle. 

Brian 

And, most importantly, remember you are at the center of your care. Never hesitate to share your opinion and to advocate for yourself. 

To learn more, visit powerfulpatients.org/MPN to access a library of tools. Thanks for joining us! 

What Follicular Lymphoma Treatments Are Available?

What Follicular Lymphoma Treatments Are Available? from Patient Empowerment Network on Vimeo.

Follicular lymphoma patients have different treatment options, but what should patients know about them? Expert Dr. Sameh Gaballa shares an overview of available treatment options and research results of treatment versus watch and wait. 

Dr. Sameh Gaballa is a hematologist/oncologist specializing in treating lymphoid malignancies from Moffitt Cancer Center. Learn more about Dr. Gaballa.

See More from START HERE Follicular Lymphoma

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What Exactly is Follicular Lymphoma? An Expert Explains

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Follicular Lymphoma Patient Expert Q&A: Start Here

Newly Diagnosed with Follicular Lymphoma? Start Here

Newly Diagnosed with Follicular Lymphoma? Start Here


Transcript:

Lisa Hatfield:

So, can you speak to the novel pathways and targets that are currently under investigation in follicular lymphoma? And what are the most important highlights to point out to patients and families?

Dr. Sameh Gaballa: 

Yeah, absolutely. So you have to remember, number one, not all patients with follicular lymphoma have to be treated. A fair number of patients can be safely observed initially, because the…so when I was talking about the types of lymphoma, so the aggressive lymphomas, those ones are treatable, but curable, meaning you treat it, goes away, good chance that it goes away and does not come back.

Whereas follicular lymphoma, those are slow-growing lymphomas. They may or may not cause problems. The treatment though, they’re very treatable. There are a lot of treatments available, but the thing is they’re not curable, meaning that they go into remission, they could stay in remission for years, but then eventually they would come back again. So you have to remember that because of that, large trials were done previously where patients who had no symptoms and not a lot of disease, they were randomized, half would get treated.

The other half were on a watch and wait. And the patients who, survival is exactly the same in both groups, there was not really any advantage to early treatment versus treatment as if there’s a reason in the future. And we typically have some indications where we decide, okay, well, it’s time to treat. And those basically have to do if the lymph nodes are big enough or they’re close to an important structure and we don’t want them to grow more and maybe press on an important structure, or if they’re causing some kind of symptom or they’re causing anemia or low platelets. I mean, there has to be one, because there has to be one reason for why you’re trying to treat that patient, because you’re basically trying to fix a problem.

So if there’s no problem initially, it doesn’t make sense to treat it. Now, there are lots of available treatments, it could be only immune therapy, something like rituximab (Rituxan)  or obinutuzumab (Gazyva); these are antibody treatments. There are also combinations with chemotherapies, like bendamustine (Treanda), rituximab for if we have relatively bulky disease. There are options as well that do not involve chemotherapy.

So something like pills like lenalidomide (Revlimid) combined with rituximab, those are also options that can be used in follicular lymphoma. But over the last few years, there have been a lot of changes in follicular lymphoma and a lot of novel targets and a lot of novel treatments available. So, for example, a few years ago now, we’ve had CAR T-cell therapy approved. Right now, we have two products approved, axi-cel and tisagenlecleucel (Kymriah). There’s also data that was presented with liso-cel in follicular lymphoma. So hopefully we might see an approval for that as well. So that’s one class.

There’s also bispecific antibodies, and it’s very exciting times. We had the first bispecific antibody approved in the United States in December of 2022. That’s mosunetuzumab-axgb (Lunsumio). So what is a BiTE antibody? These basically are advanced types of immune therapies where you give the patient an antibody that has two ends to it, one end sticks to the cancer cell, the other end sticks to your immune cells. So it’s basically handholding your own immune cells or your own T cells to go and get attached to the cancer cell and kill it, not chemotherapy. It, of course, can have some immunological side effects like fevers or inflammation initially when it’s done, typically when in the first cycle or second cycle.

But something called cytokine release syndrome rarely can cause neurological toxicity. That’s also very transient usually, and very rare with bispecific antibodies. But those are two up and coming treatments. Right now, they’re approved in patients who’ve had relapsed/refractory disease, meaning they’ve had two or more lines of previous therapies, but they’re…we have them now in trials where we’re looking at those agents in earlier lines of therapy. There are other agents as well.

A few years ago, we had tazemetostat (Tazverik) approved, which is a pill that targets an enzyme in the cells called EZH2 and they basically, this pill tries to ask the cancer cell to differentiate, rather than get stuck and not die. So they differentiate and then they eventually die, so that’s another class of medicine. And we’ve now seen some data with BTK inhibitors. There’s been data presented from the ROSEWOOD Study with zanubrutinib plus obinutuzumab (Brukinsa plus Gazyva); it’s not yet FDA-approved, but the data looks interesting and certainly needs to be looked at further. 


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Follicular Lymphoma Patient Expert Q&A: Start Here

Follicular Lymphoma Patient Expert Q&A: Start Here from Patient Empowerment Network on Vimeo.

The START HERE program bridges lymphoma expert and patient voice, whether you are newly diagnosed, in active treatment or in watch and wait. In this webinar, Dr. Sameh Gaballa provides an overview of the latest in follicular lymphoma, emerging therapies, clinical trials and options for follicular lymphoma progression and recurrence.

Dr. Sameh Gaballa is a hematologist/oncologist specializing in treating lymphoid malignancies from Moffitt Cancer Center. Learn more about Dr. Gaballa.

Download Resource Guide  |  Descargar guía de recursos

See More from START HERE Follicular Lymphoma

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Newly Diagnosed with Follicular Lymphoma? Start Here


Transcript:

Lisa Hatfield: 

Welcome to the START HERE Patient Empowerment Network Program. This program bridges the expert and patient voice enabling patients and care partners to feel comfortable asking questions of their healthcare team.  Joining me today is Dr. Sameh Gaballa, an oncologist hematologist from Moffitt Cancer Center. Dr. Gaballa’s clinical interests are treating patients with lymphoid malignancies. His research focuses on developing novel targeted agents for treating patients with indolent lymphomas, such as follicular lymphoma, marginal zone lymphoma, and lymphoplasmacytic lymphomas. Thank you so much for joining us today, Dr. Gaballa.

Dr. Sameh Gaballa:

Thank you, Lisa. Happy to be here.

Lisa Hatfield:

Thank you. The world is complicated, but understanding your follicular lymphoma diagnosis and treatment options doesn’t have to be. The goal of START HERE is to create actionable pathways for getting the most out of follicular lymphoma treatment and survivorship. 

Before we get started, please remember to download the program resource guide via the QR code. There’s great information there that will be useful during this program and after. So let’s get started. So, Dr. Gaballa, I’d like to talk about what’s on the follicular lymphoma treatment radar. There’s a lot going on in terms of emerging treatment options, clinical trial data, and other learnings for the follicular lymphoma community.  But before we jump into how the armamentarium is expanding, can you provide an explanation of what follicular lymphoma is?

Dr. Sameh Gaballa:

Yeah, absolutely, thank you, Lisa. So, follicular lymphoma is a type of B-cell non-Hodgkin’s lymphoma. What does that mean? It’s basically, so in your body, there are cells that are part of the immune system; these are lymphocytes. These cells normally, their normal function, is to fight infection, they’re part of your immune system. They actually are involved also with fighting cancers, but sometimes they become malignant. But not all lymphomas are the same. Lymphomas are a huge family. So there’s Hodgkin’s lymphoma, there is non-Hodgkin’s lymphoma. Within non-Hodgkin’s lymphoma, there is a type called B-cell non-Hodgkin’s and there’s a T-cell non-Hodgkin’s lymphoma. And then within B-cell non-Hodgkin’s lymphoma, there are two big groups. So one group, they are these aggressive lymphomas that grow quickly, they can make you sick quickly, and these lymphomas we have to treat right away.

And then you have those slow-growing indolent lymphomas that are sometimes very commonly actually diagnosed by chance, or incidentally, that’s usually the most common way these are diagnosed.  And the most common slow-growing indolent lymphoma is going to be follicular lymphoma. Now, where do you find these lymphomas? It’s a blood disease. So, again, we said that those cells are normally borne in the bone marrow, they are in the blood, they’re in the lymph nodes, they’re in the spleen. So usually you would find those malignant cells usually in the lymph nodes, but you could also find them sometimes in the spleen or in the blood or in the bone marrow as well. And the symptoms they cause will be dependent on where they are and how big the, those, the involvement is.

Lisa Hatfield:

Well, thank you for that detailed overview, Dr. Gaballa. We do have follicular lymphoma patients and care partners who are newly diagnosed, in active treatment, watching and waiting, and also living with their disease joining this program. No matter where you are on your journey, START HERE provides easy-to-understand, reliable, and digestible information to help you make informed decisions. Dr. Gaballa, we’re going to dive right into things with a high-level update. So, can you speak to the novel pathways and targets that are currently under investigation in follicular lymphoma? And what are the most important highlights to point out to patients and families?

Dr. Sameh Gaballa:

Yeah, absolutely. So you have to remember, number one, not all patients with follicular lymphoma have to be treated. A fair number of patients can be safely observed initially, because the…so when I was talking about the types of lymphoma, so the aggressive lymphomas, those ones are treatable, but curable, meaning you treat it, goes away, good chance that it goes away and does not come back. Whereas follicular lymphoma, those are slow-growing lymphomas. They may or may not cause problems. The treatment though, they’re very treatable. There are a lot of treatments available, but the thing is they’re not curable, meaning that they go into remission, they could stay in remission for years, but then eventually they would come back again. So you have to remember that because of that, large trials were done previously where patients who had no symptoms and not a lot of disease, they were randomized, half would get treated.

The other half were on a watch and wait. And the patients who, survival is exactly the same in both groups, there was not really any advantage to early treatment versus treatment as if there’s a reason in the future. And we typically have some indications where we decide, okay, well, it’s time to treat. And those basically have to do if the lymph nodes are big enough or they’re close to an important structure and we don’t want them to grow more and maybe press on an important structure, or if they’re causing some kind of symptom or they’re causing anemia or low platelets. I mean, there has to be one, because there has to be one reason for why you’re trying to treat that patient, because you’re basically trying to fix a problem.

So if there’s no problem initially, it doesn’t make sense to treat it. Now, there are lots of available treatments, it could be only immune therapy, something like rituximab (Rituxan)  or obinutuzumab (Gazyva); these are antibody treatments. There are also combinations with chemotherapies, like bendamustine (Treanda), rituximab for if we have relatively bulky disease. There are options as well that do not involve chemotherapy.

So something like pills like lenalidomide (Revlimid) combined with rituximab, those are also options that can be used in follicular lymphoma. But over the last few years, there have been a lot of changes in follicular lymphoma and a lot of novel targets and a lot of novel treatments available. So, for example, a few years ago now, we’ve had CAR T-cell therapy approved. Right now, we have two products approved, axi-cel and tisagenlecleucel (Kymriah). There’s also data that was presented with liso-cel in follicular lymphoma. So hopefully we might see an approval for that as well. So that’s one class.

There’s also bispecific antibodies, and it’s very exciting times. We had the first bispecific antibody approved in the United States in December of 2022. That’s mosunetuzumab (Lunsumio). So what is a BiTE antibody? These basically are advanced types of immune therapies where you give the patient an antibody that has two ends to it, one end sticks to the cancer cell, the other end sticks to your immune cells. So it’s basically , it’s handholding your own immune cells or your own T cells to go and get attached to the cancer cell and kill it, not chemotherapy. It, of course, can have some immunological side effects like fevers or inflammation initially when it’s done, typically when in the first cycle or second cycle.

But something called cytokine release syndrome rarely can cause neurological toxicity. That’s also very transient usually, and very rare with bispecific antibodies. But those are two up and coming treatments. Right now, they’re approved in patients who’ve had relapsed/refractory disease, meaning they’ve had two or more lines of previous therapies, but they’re…we have them now in trials where we’re looking at those agents in earlier lines of therapy. There are other agents as well.

A few years ago we had tazemetostat (Tazverik) approved, which is a pill that targets an enzyme in the cells called EZH2 and they basically, this pill tries to ask the cancer cell to differentiate, rather than get stuck and not die. So they differentiate and then they eventually die, so that’s another class of medicine. And we’ve now seen some data with BTK inhibitors. There’s been data presented from the ROSEWOOD Study with zanubrutinib plus obinutuzumab (Brukinsa plus Gazyva); it’s not yet FDA-approved, but the data looks interesting and certainly needs to be looked at further.

Lisa Hatfield:

Well, thank you for that overview. It seems like as a blood cancer patient myself, it seems like a hopeful time for patients with the treatments that are kind of on the horizon or are in clinical trials right now. So thank you for that.

Dr. Sameh Gaballa:

Absolutely.

Lisa Hatfield:

So it’s that time now where we answer questions, some of which we’ve received from you, the patients watching this. Remember, as patients, we should always feel empowered to ask our healthcare providers any and all questions we might have about our treatment and prognosis. Please remember, however, that this program is not a substitute for medical care. Always consult with your medical team.  So, Dr. Gaballa, let’s start here. How do you explain follicular lymphoma treatment options and prognosis to your newly diagnosed patients? And what does shared decision-making look like in your office?

Dr. Sameh Gaballa:

Oh, absolutely. So follicular lymphoma, you really have to explain to the patient what, how are we coming to the recommendation that we’re currently giving. So if we think this is, this patient is a good candidate for a watch-and-wait approach, for example, we really have to walk them through why that really is the best option and not why should we jump on treatments and vice versa, if we think this patient needs to be treated, how do we really…the patient really has to understand all the other treatment options and why this needs to be treated. Because a lot of patients initially, sometimes when you present them with a watch-and-wait approach, if they don’t know all the background, they might not feel very comfortable because they might think, “Well, I have this cancer in me, and we’re not doing anything about it, and that doesn’t really sound too…something I should be doing.”

But then when you explain to them, “Well, you see, you don’t have a lot of disease, those studies have already been done in the past where patients who were treated or not treated, the survival was the same, so there, you might get side effects from the treatment, but not necessarily have benefits. And in the future, should this need to be treated, we have a lot of things to do.” So, really, so this is kind of the shared decision portion where you just have to walk the patients through why that will be the best situation. There is data with single-agent rituximab, even in patients who are asymptomatic, and we have the UK data, and that’s an option.

And that is also offered to some of the patients, even if they’re not symptomatic and they don’t have a lot of disease, if that’s what really the patient wants, if they’re not really comfortable with a watch and wait. And there’s again some data to help justify that. Again, there’s no advantage in overall survival, but sometimes the patients would kind of feel more in control. They feel like, “Okay, I did something about it.” So that’s the shared approach.

In terms of your other question about prognosis, unfortunately that’s an area of an unmet need. I mean, we have some tools to help us differentiate follicular lymphoma patients from each other, which patient is high-risk, meaning those are the patients who might relapse quickly, or they might not respond well to treatments. Unfortunately, we don’t have great tools. We have something called a FLIPI score, which is, we use a number of parameters including clinical parameters like stage or age and some other parameters as well, and we have a scoring system. But it doesn’t 100 percent predict if this is going to be a high-risk follicular lymphoma or a low-risk.

Unfortunately, the best predictor of prognosis for follicular lymphoma, you would know about retrospectively,  it’s something called POD24, progression of disease in 24 months. Meaning that if you have a patient who’s treated with chemotherapy and immune therapy, and then they go into remission, and then they relapse again in less than 24 months, progression of disease within 24 months, those are the, those represent about 20 percent of follicular   lymphoma patients, and those represent a high-risk group of patients. That’s the best tool that we have. But unfortunately, if you’re diagnosed today, you’re not going to know if you’re in this group or not until you actually need to be treated and not just treated with immune therapy.

It has to be with chemotherapy as well. And then if you relapse within two years, then we know that this is a high-risk entity. There is genetic testing, there is something called a FLIPI-m7 scoring system. But again, these tools are not great to tease out the low risk from the high-risk follicular lymphoma patients. But 80 percent of patients who are not going to be POD24, meaning that they get treated, they’re in remission for two years or more, and actually those patients have very similar survival to the general population. So, yeah, so a lot of times we don’t know right away, but we do have some tools to kind of give us an idea.

Lisa Hatfield:

Great. Thank you for that information. It’s kind of hard for cancer patients to only know what their prognosis is retrospectively, but that’s a great explanation. Thank you. Okay, another patient question, “How does the staging of follicular lymphoma impact treatment choices?”

Dr. Sameh Gaballa:

Yeah, so as you saw, I didn’t really stress too much about staging, because it’s a blood disease. So the vast majority of patients are going to be what we call stage III to IV disease. So, obviously when you see a patient if if they, they might think that, “Oh my God, I have a stage III to IV cancer,” because that’s really what they’re familiar with. But follicular lymphoma is a blood disease, so by default it’s going to be in a lot of lymph nodes, it might be in the bone marrow as well, but stage III to IV disease follicular lymphoma doesn’t, that does not mean that this is a terminal cancer. Patients could live completely in normal life, even with a stage III to IV follicular lymphoma. This is not like a breast cancer or colon cancer where stage is everything.

But why do we have a staging system? Obviously, there’s a need to have staging system for all cancers, but clinically, the only time it makes a difference is there’s a small group of patients who have a truly stage I or II disease, meaning just one group of lymph nodes on one side of the diaphragm that may fit within one radiation field. So if you have someone who’s just coming in with one or a few groups of lymph nodes all in one place, we call that a stage I or II follicular lymphoma, not common, because again, most patients are stage III to IV. The only difference there is you can potentially offer those patients radiation therapy if it’s truly localized, but then you would need to do a bone marrow biopsy and confirm that it’s not in the bone marrow.

And if it is localized within one radiation field, that can be offered and we can sometimes give after radiation therapy, either observe it or consider giving rituximab afterwards. But that’s the only time where we’re going to mention staging, again, uncommon because most, the vast majority of patients are going to be stage III to IV. So why would we do that? Why would we irradiate if it’s only one group of lymph nodes? Because there’s about, I mean, if you irradiated, those lymph nodes will go away, but there’s about maybe a, it’s different. The number is different between studies, but about maybe a third of patients, if you irradiate that group of lymph nodes or one lymph node, it actually might not come again in the future. So you might have very long remissions/possible cure if you…and this is the only situation where we would consider treating someone who does not have symptoms, because you could have very long remissions with radiation.

Lisa Hatfield:

Although follicular lymphoma is a slow-growing cancer, can you speak to the signs that the disease is progressing in the body, what signs that patients might want to look out for?

Dr. Sameh Gaballa:

Yeah, absolutely. So, typically we educate the patients to there are some red flags to look out for, not just for progression,but also for another condition called disease transformation. So, follicular lymphoma does have a, there is a possibility that it can transform from a slow-growing lymphoma to an aggressive lymphoma. Now, this happens at a rate of about maybe 2 to 3 percent per year, but it’s a cumulative risk, so meaning if a patient lives many, many decades, their lifetime risk can be up to as high as 20, 25 percent, 30 percent, depending on the different literature, so there is a chance that these slow-growing lymphomas can transform to an aggressive lymphoma.

And when they do know this, there’s no watch and wait for transformed disease. It has to be treated with chemo immunotherapy because the goal of treatment then is to try to get rid of the aggressive component. What are the signs and symptoms to suggest that you might have transformed disease? This is not something that the patient would typically need to look out for. I tell my patients that, “You don’t need to see, do I have transformed disease or not. This is going to come, and you’re going to know when you have transformed disease. Extreme fatigue, drenching night sweats, the fever sometimes that are not going away.”

The patient might have pain if the lymph node is pressing on some important structure. They may have loss of appetite, loss of weight. So again, something that dramatically happens quickly over a few weeks of time. So if the patient feels sick for one reason or another and they’re not getting better, it can all happen within a few weeks’ time frame. This is the time to get checked early on and go see your oncologist, because then we might need to investigate if there is any potential for transformation. So that’s issue number one.

Issue number two is, which is the much more common scenario, which is the follicular lymphoma is slowly progressing. How would you know? I mean, if you notice a lymph node that in your neck or under the armpits or the groin areas, if they’re growing, then that needs to be evaluated. I mean the patients should expect that those will be growing, they will grow. But they grow over months and years. They don’t grow over weeks.

So anytime you kind of are unsure, if you feel that it’s growing faster than usual, this is, again, something to look out for. And then the B symptoms that I mentioned. So like the sweats, the fevers, the weight, loss of weight, loss of appetite, these are also sometimes things to look out for. Not necessarily, they don’t always mean that it’s transformed disease. It can also be that the follicular lymphoma is also progressing and might need to be treated as well.

Lisa Hatfield:

And then just a quick follow-up to that question. So a patient is watching out for these red flags, but are they going through any kind of regular monitoring in your office? Are you meeting with them on a regular basis? And how frequent might that be for a follicular lymphoma patient who’s watching and waiting?

Dr. Sameh Gaballa:

Yeah. So how does watch and wait look? So, and I tell patients always watch and wait does not mean ignore. Watch and wait means that we’re monitoring the disease, we’re looking at it. How do we do that? So typically we would see the patient maybe every three to six months. And then depending on how do we, when we get a sense or tempo of how their disease is progressing, then we’ll know how often we need to see them. I’ve had, I still have patients where I’m seeing them every three months. And I also have some patients where the disease has been stable for years, I only see them once a year.

In terms of imaging, that’s also sometimes an area of controversy. Typically, initially for the first maybe year or two years, I do like a scan, like a CT scan every six months, just to get a sense of how quick or how slow the disease is progressing. If there’s absolutely no change at all, then sometimes we either don’t do scans and just go by the patient’s symptoms and blood work and physical exam, or we do maybe once a year scan but not more than that. So this is how we would monitor the patients in a watch-and-wait approach.

Lisa Hatfield:

And we have another question about treatment profiles, “What can I do to reduce side effects during active treatment?”

Dr. Sameh Gaballa:

So it depends on what the treatment that you’re getting. If it’s immune therapy, like rituximab alone, those typically don’t really have a lot of side effects. I mean, sometimes with the first one or two treatments, you might get an allergic reaction, an infusion allergic reaction, which is very common, but subsequently it shouldn’t really cause a lot of side effects. If the patient is getting chemotherapy, well, it depends on which chemotherapy they’re getting. But in general, it’s always good to stay hydrated and to stay physically active. So if the patient goes in with a healthy body, well-hydrated, you eat fresh fruits and vegetables, walking 30 to 60 minutes a day, your body is going to handle the side effects much better than if you’re going in, you’re very weak, and your general health is not adequate.

Lisa Hatfield:

Another patient is asking if you can speak to emerging treatment options for patients with relapsed/refractory follicular lymphoma?

Dr. Sameh Gaballa:

Yeah. So the field of follicular lymphoma is changing rapidly. I always tell patients that sometimes the best treatment is actually on a clinical trial because those are going to be the next generation of treatments that are going to get approved in the next few years. But right now we have the most effective therapy really is CAR T-cell therapy. CAR T-cell therapy by far is the most effective treatment we have at this time. It’s approved for patients who have had two or more lines of prior therapies. We also are investigating this.

I actually have a trial here at Moffitt where we’re looking at CAR T-cell therapy as early as in the second line, in patients who have what we call the high-risk ones, the POD24. So a patient with POD24 follicular lymphoma relapsed in less than two years. We have a trial to investigate the role of CAR T-cell therapy in this setting. The other very promising group of treatments, again, is bispecific antibodies, again, currently approved in the third line, mosunetuzumab.

But there are others coming up and have data on epcoritamab-bysp (Epkinly), as well as a lot of other bispecifics, as well as combinations. I mean, epcoritamab-bysp has also data presented with combination with lenalidomide. And right now, the follow-up duration is not very long, but so far, it looks extremely promising with very high response rates. So those also might be coming very soon. And, of course, once something works in the relapsed/refractory setting, we start looking at earlier lines of therapy.

And actually, we’re now looking at trials in the first-line setting with some of these agents as well. Tazemetostat is a pill. It’s also approved in the third-line setting, but we’re also investigating it. We have a trial here where we’re looking at combining it with standard rituximab, lenalidomide, so tazemetostat plus rituximab, lenalidomide as early as in the second line. So that also is interesting. And as I mentioned before, BTK inhibitors currently being looked at in trials might also have a role in follicular lymphoma very soon.

Lisa Hatfield:

And this patient is asking about the significance of bispecific antibody treatment. And you touched on that a little bit. It looks like she’s also asking if there are specific genetic or molecular markers that can predict a patient’s response. And if I try to translate that, maybe she might be asking about targeted therapy.

Dr. Sameh Gaballa:

Yeah, so bispecific antibodies and CAR T-cell therapy, they target something called CD, either CD19 or CD20, and that’s almost universally expressed on B cells. So most of your follicular lymphoma patients are going to be expressing CD19 or CD20. Tazemetostat is the pill that I talked about.  It inhibits an enzyme called EZH2. Some patients have an EZH2 mutation where it seems to work very well. However, tazemetostat also works in patients who don’t have that mutation. So that’s why it’s not very important to check for the mutation.

It seems maybe it works better in patients who do have the mutation, but it does work as well in patients who do not have that mutation. So unlike other malignancies and other cancers, biomarkers are not yet driving a lot of our treatment decisions in follicular lymphoma as of right now.

Lisa Hatfield:

Thank you. Another question. Is it common for follicular lymphoma to transform into a more aggressive type of lymphoma? And how would that change a treatment plan? And maybe how common is it for that to happen?

Dr. Sameh Gaballa:

Yeah. There’s about a 2 to 3 percent chance per year that the slow-growing lymphoma can transform to an aggressive lymphoma. That, if it does transform, I mean we talked about the symptoms and signs, you get sick quickly, rapidly enlarging lymph nodes, loss of weight, loss of appetite, drenching night sweats. No, a transformation, typically we would do a PET scan, see what’s the most active lymph node, try to get a biopsy from that and confirm there is a large cell transformation. Now, that’s a completely different disease, it needs to be treated completely differently, typically with chemoimmunotherapy.

Something like R-CHOP, for example, is one of the most common regimens we use in this scenario. And the goal of treatment here is to try to get rid of the aggressive lymphoma component here so that it does not recur again. I mentioned it’s about a 2 to 3 percent per year, but it depends on how long the patient lives. So if they live many, many, many decades, their lifetime risk is anywhere between 20 to 30 percent max during their lifetime.

Lisa Hatfield:

And As a blood cancer patient myself, this is a great question this patient is asking, “Is there a risk of secondary cancers after receiving treatment for follicular lymphoma?”

Dr. Sameh Gaballa:

So that’s always a concern, and it depends on what treatment they had. So chemotherapy that can potentially damage DNA can lead to second malignancies, including things like acute leukemia. Luckily, that’s not a high risk. That’s a rare side effect from some of those chemotherapies. Some of the pills can do that as well. Something like lenalidomide can sometimes have second malignancies. But we’re talking about rare incidences, and the benefits usually would outweigh the risks. But it’s not with all treatments, meaning some of the other immune therapies that do not involve chemotherapy would not typically be associated with some of those second malignancies. So it just really depends on what exactly the treatment you’re getting.

Lisa Hatfield:

Can you speak to maintenance therapy and monitoring in follicular lymphoma? And what signs of infection should patients and care partners be aware of during treatment?

Dr. Sameh Gaballa:

Yeah, so there have been randomized studies in slow-growing lymphomas that show that if you do, after you get your standard treatment for follicular lymphoma, if you do what we call a maintenance treatment, usually with rituximab, which is an immune therapy, where you do it every two to three months for about two years, we have data showing that that decreases or delays the risk of relapse. However, it doesn’t change the overall survival, meaning that it just has patients in remission longer. When their disease comes back, they just get treated again at that point, and it doesn’t really affect survival.

So it’s one of those shared decision-making with the patients. I usually go over the risks and benefits of maintenance therapy. It’s optional. It’s not a must. During COVID, we pretty much stopped all maintenance treatments, because the risks were outweighing the benefits because maintenance treatment is…will suppress the immune system more, is associated with more infections. And these infections can be anything. I mean, it could be a pneumonia, could be recurrent urinary infections. It could be any type of infection. So there’s always this risk and benefit that we have to discuss with the patient.

Lisa Hatfield:

Well, Dr. Gaballa, thank you so much for being part of this Patient Empowerment Network START HERE program. It’s these conversations that help patients truly empower themselves along their treatment journey. And on behalf of patients like myself and those watching, thank you so much for joining us, Dr. Gaballa.

Dr. Sameh Gaballa:

No, thank you, Lisa. I really appreciate it. Thank you.

Lisa Hatfield:

I’m Lisa Hatfield. Thank you for joining this Patient Empowerment Network program.


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Is It Aging or My CLL?

Is It Aging or My CLL? from Patient Empowerment Network on Vimeo.

How can chronic lymphocytic leukemia (CLL) patients tell the difference between treatment side effects and normal aging issues? Expert Dr. Ryan Jacobs explains his perspective when fatigue is the only issue for the patient versus patients having multiple symptoms.

Dr. Ryan Jacobs is a hematologist/oncologist specializing in Chronic Lymphocytic Leukemia from Levine Cancer Institute. Learn more about Dr. Jacobs.

Download Resource Guide   |  Descargar Guía en Español

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Transcript:

Lisa Hatfield:

A patient had asked, and I love this question because I often wonder myself when I get up in the morning, my bones are creaking and popping, “How do you know the difference between,” this patient’s talking about fatigue. How does a patient discern, “Well, this is fatigue from my cancer or my treatment,” versus just normal aging? Whether it’s fatigue or bruising or any side effect.

Dr. Ryan Jacobs:

Yeah. Fatigue is a really…I had an attending physician when I was in my training that said, “Treating fatigue makes me fatigued.” But it’s hard. If it’s really the only problem the CLL patient is having, it can be…all those other problems I had mentioned earlier, the low red cells, the low platelets, the painful nodes, the night sweats, I with close to 100 percent certainty know I can fix those with treatment.

Fatigue, I’m not as confident when that’s the only issue that a patient’s having. I try to differentiate between fatigue from other causes and old age, and specifically to CLL. They try to put it as a metric and say, if you’re having to spend half the day or more just lying around and you’re not able to do your normal activities of daily living, like that’s a severe level of fatigue and treatment should be considered. I’m looking for somewhat of a precipitous decline, not necessarily just kind of the gradual fatigue that you might more relate to aging.

The problem with treating fatigue is you’ll look, if you look at the possible side effects of all of these medicines I talked about, fatigue will be a potential side effect. So you’re sometimes trading one problem and getting another, or maybe the fatigue does get better, but then the patient has some different side effect that’s even worse than the fatigue. So it’s hard to really help when fatigue’s the only issue. But certainly, I have helped some patients with fatigue. We don’t have a test that we can do to know for sure is the fatigue coming from the cancer, or is it coming from something else.


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What MPN Patient Type Is a Good Candidate for Telemedicine Visits?

What MPN Patient Type Is a Good Candidate for Telemedicine Visits? from Patient Empowerment Network on Vimeo.

What myeloproliferative neoplasm (MPN) patient type makes a good candidate for telemedicine visits? MPN expert Dr. Jamile Shammo shares her perspective of patient situations that work well for telemedicine and those who can benefit from in-person visits as part of ongoing care.

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Transcript:

Lisa Hatfield:

As more institutions start to have in-person visits instead of only telemedicine visits, you might be wondering if you should keep doing telemedicine visits or move back to seeing your physician in-person. Some people might want to continue doing telemedicine for a number of reasons, including convenience/no travel involved and  limiting your exposure to colds/infection from other patients. There are certain MPN patients that could be seen with telemedicine visits or fewer in-person visits. Listen as Dr. Jamile Shammo explains.

Dr. Jamile Shammo:

So, when I think of the patient that might benefit most from seeing the physician via televisit, for example, it would be someone who perhaps has a stable disease, someone who I may want to monitor perhaps every three to six months, someone who may have stable counts, and we’re just talking to about their symptoms and monitoring those types of things every so often. And perhaps I look at the labs, and you can discuss their symptoms and whether or not they have splenomegaly and issues like that. 

Lisa Hatfield:

As Dr. Shammo notes, if your MPN is considered stable and you typically only see your doctor every three to six months, it might be worth continuing telemedicine visits instead of going back to in-person visits. 

Dr. Jamile Shammo:

Someone who may already be on a stable dose of medication and we don’t have to do any dose adjustments and even if we have to do those adjustments, perhaps we could do labs a little more frequently, so that would be all right too.

Lisa Hatfield:

If you are on a stable dose of your medication and don’t need any modifications or just have minor adjustments, you could consider staying with telemedicine visits. 

But what patients should consider doing more in-person visits, now that COVID-19 precautions are lighter? Dr. Shammo goes on to explain THAT patient could be…

Dr. Jamile Shammo:

Someone in whom I would like to initiate in treatment, someone in whom the disease may be progressing a little too quickly, someone who I may want to do an exam and assess their spleen, I suppose you could send them to an ultrasound facility and obtain an MRI or a CT, or an ultrasound of the imaging study that is. But there’s nothing like an actual exam of the patient. You are thinking about the disease progression, so those sorts of patients in which the disease is actually changing its pace, you may want to take a look at it, the full smear look and examine the skin for certain TKI and signs and symptoms of low platelets and that sort of thing. Look in the mouth for ulcers and things of that nature. 

Lisa Hatfield:

As always, please discuss with your health care team before deciding to switch to only telemedicine visits or going back to in-person visits. They know your history and can help decide what is best for you and your care at this particular time. 


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How Can You Thrive With an MPN? Advice for Navigating Care.

How Can You Thrive With an MPN? Advice for Navigating Care. from Patient Empowerment Network on Vimeo.

How can you thrive with an MPN? In this animated explainer video, an MPN specialist and myelofibrosis patient discuss how to make informed decisions about your care and live a full life with an MPN.

 

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How Can Patients Navigate Care and Thrive with an MPN?


Transcript:

Brian: 

Hi, I’m Brian. Nice to meet you! Many years ago, I was diagnosed with a condition called myelofibrosis. At first, it was a scary to learn that I had cancer, but once I found the right treatment option for me, I’ve been living a full life.  

Meet, Dr. Liu – my doctor. 

Dr. Liu: 

Hi! I’m Dr. Liu, and I’m a hematologist specializing in the care of people with myeloproliferative neoplasms or MPNs.   

MPNs are a group of blood cancers that are characterized by the bone marrow overproducing a certain type of cell. The three types of MPNs are essential thrombocythemia, or ET,  polycythemia vera or PV, and myelofibrosis, or MF. 

As Brian mentioned, with the right treatment, it is possible to live a full life and to thrive with an MPN. 

Brian: 

It’s so true. Navigating my care has been much easier because I partner with my healthcare team – participating in decisions makes me feel like an important member of the team. 

Dr. Liu: 

That’s right, Brian. When considering treatment, it’s important to weigh all of your options.  

While your healthcare team is the expert when it comes to the clinical side of your disease, you as the patient, are the expert on how treatment will impact YOU and your lifestyle.  

Brian: 

And as someone who knows my needs well, my wife is another key member of my team.  She comes with me to appointments and takes notes during visits, and when it is time to make decisions about my care, we both feel well-informed about the options. 

So, Dr. Liu – what factors should be considered when choosing an MPN treatment?  

Dr. Liu: 

Well, it’s important to note that everyone’s MPN is different so what may work for one person, may not work for another. In general, we consider certain factors,1 such as: 

  • The type of MPN, whether it is ET, PV, or MF. 
  • The patient’s age and overall health. 
  • Test results, including blood work or any genetic testing that has taken place. 
  • The symptom burden, which basically means how much the disease symptoms are interfering with a patient’s quality of life. 
  • Any pre-existing health issues. 
  • Finally, and most importantly, the patient’s preference.  

Brian: 

And I like to make informed decisions. So, when considering therapy, I also did some research on my own, and then discussed the information with my healthcare team. It helped my wife and me understand what we’d learned, and confirmed our decision. 

Dr. Liu, what sort of questions should patients ask their doctor when considering a treatment plan? 

Dr. Liu: 

Great question. When choosing therapy, patients should ask: 

  • How is the treatment administered, and how often will I need treatment? 
  • What are the potential side effects of the treatment? 
  • How will the effectiveness of the treatment be monitored? 
  • And, what are options if this treatment doesn’t work for me? 

Brian: 

That’s great advice. Once you’ve begun treatment, it’s important to continue to share how you are feeling with your healthcare team – be sure to mention any side effects or symptoms you may be having with your team. 

Dr. Liu: 

That’s right, Brian. If you speak up about what’s bothering you, we can usually find a way to manage the issue. 

It’s also important point to tell your doctor if you’ve missed a dose of your medication. Many of the newer MPN therapies are self-administered, and it’s important to let us know so we can adjust the plan if necessary. 

So, what steps should you take to thrive in your life with an MPN? 

Brian: 

  • First, understand and participate in treatment decisions. Be sure to share your personal preferences. 
  • Then, communicate regularly with your healthcare team – don’t wait to share information only when you have an appointment.  
  • And, utilize your whole team – nurses, nurse practitioners, and others, are all there to help you. 
  • Use your patient portal. You can view lab work and test results, or even use the messaging feature to communicate with your team. 
  • Bring a friend or loved one to appointments and always write down any questions or concerns in advance.  

Dr. Liu: 

And, most importantly, remember you are at the center of your care. Advocate for yourself! 

To learn more, visit powerfulpatients.org/MPN to access a library of tools. Thanks for joining us! 

Signs It Is Time to Treat Your CLL

Signs It Is Time to Treat Your CLL from Patient Empowerment Network on Vimeo.

When is it time to treat your chronic lymphocytic leukemia (CLL)? Dr. Catherine Coombs reviews the criteria doctors consider when deciding whether it is time to begin therapy. 

Dr. Catherine Coombs is an Assistant Professor of Medicine in the Division of Hematology at The UNC Lineberger Comprehensive Cancer Center. Learn more about Dr. Coombs here.

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Transcript:

Katherine:

When is it time to treat? What factors do you look at? 

Dr. Coombs:

There’s pretty well-established guidelines for when treatment is indicated. The international workshop for CLL has these published guidelines, so it’s something you could Google. Off the top of my head, the main reasons that I do treatment, which are included in these guidelines, are one, if the patient has low blood counts due to the CLL, so that could be anemia or low platelets. Two, if they have bulky lymph nodes. They actually define bulky as 10 centimeters. So, that’s pretty big.  

Or, if the lymph nodes are being symptomatic in some way, they’re bothering the patient, they don’t have to be that big. Three, if the patient has bulky spleen enlargement or if it’s causing symptoms. The spleen is next to the stomach. So, say some patients may not be able to eat a full meal, that’s another reason we could do treatment.   

Another reason is if the CLL is causing constitutional symptoms. Sometimes these are black and white. One is unintentional weight loss of 10 percent or more of the body weight. The one that’s not always black and white is fatigue. Patients can have fatigue from the CLL, but I’ve found often fatigue can be due to other causes. So, that’s something I consider an important job of mine is to make sure we don’t jump into CLL treatment if say, there’s some other cause for the tiredness, such as, say the thyroid’s off, or there’s a huge amount of stress due to some other factor outside of the CLL.  

Then, some other constitutional symptoms are CLL can cause fever or drenching night sweats. Those two it’s important to make sure that there’s not a concurrent infection because infections can also cause those symptoms. The last indication is patients with CLL can develop autoimmune cytopenias. That’s when the immune system attacks some component of the blood cells. Most commonly that’s an autoimmune anemia or autoimmune thrombocytopenia. That’s the term for low platelets.  

Usually, we can treat that with steroids or occasionally CD-20 by itself like rituximab to calm down the immune system. However, if those immune-based therapies fail the patient, then we could consider treating the CLL to help fix that problem.  

What Is the Prognosis of CLL?

What is the Prognosis of CLL? from Patient Empowerment Network on Vimeo.

Chronic lymphocytic leukemia (CLL) can progress in two different ways. Watch to learn about the prognosis, monitoring, and treatment for each CLL type.

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Transcript:

Chronic lymphocytic leukemia (CLL) patients generally have a better outlook compared to other cancer types – with a higher 5-year survival rate of about 83 percent. There are two types of CLL – one being a slower-growing type and the other a faster-growing type. 

The slower-growing type features higher lymphocytes with slightly low platelets, neutrophils, and red cells. While the faster-growing type produces too many CLL cells in the blood that prevent proper function of red cells and platelets. With the two different types of CLL, patients may have very different patient journeys depending on their disease 

While some CLL patients experience very gradual disease progression and are actively monitored during a watch-and-wait phase, other patients may experience a more expedited CLL progression and will need more frequent treatment. 

Dr. Kerry Rogers:                 

“So, for many people, CLL is a very manageable disease. Like I said, some people have had CLL longer than I’ve been a doctor and have needed no treatment for it. However, there are people with CLL that go on to have a lot of difficulty from it, including not doing well with more than therapy or needing really new, advanced therapies, like something called CAR T-cell therapy.

So, for any individual person, you can never say how it’s going to turn out for them, but we do use our experience taking care of lots of people with CLL to make an educated guess as to if this person’s going to be someone that’s going to expect to need a lot of treatment in their lifetime, or maybe no treatment in their lifetime.”

CLL research continues to advance, and clinical trials bring more refined treatments for patients to improve both CLL symptoms and treatment side effects over time. Ask your CLL specialist if you have questions about research advances and check reliable sources like the Patient Empowerment Network, The Leukemia & Lymphoma Society (LLS), and the American Society of Hematology (ASH) and American Society of Clinical Oncology (ASCO) annual conferences.

MPN Treatment Tools and Advancements

MPN Treatment Tools and Advancements from Patient Empowerment Network on Vimeo.

Dr. Kristen Pettit from Rogel Cancer Center shares MPN treatment updates and recent approvals for patient care.

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Transcript:

Dr. Kristen Pettit:

So research in MPNs has really been moving at an extremely fast pace over the last few years, so just over the past few years, we’ve seen advances in nearly every aspect of MPN care, from diagnosis to risk stratification, to MPN treatment, to support of care. We’ve even seen two new drugs, approved for MPN treatment over the past year, the first was ropeginterferon alfa-2b or Besremi approved for polycythemia vera in December 2021. And the most recent was pacritinib (Vonjo) for patients with myelofibrosis with low platelets approved in February of 2022. So there have been lots of exciting improvements very recently, and I think very, many more to come over the next few years.

But there’s still a long ways to go, some unmet needs in the field still include challenges in treating patients with low blood counts, either anemia or thrombocytopenia, low platelets, both of those are still challenging clinical situations. Also, the situation when JAK inhibitors either don’t work well enough for a patient or stop working overtime, that’s a situation that’s very challenging as well. Fortunately, we have a lot of clinical trials and new investigations going on in both of those areas, and patients with low blood counts and patients who have had inadequate or loss of response to JAK inhibitors. So, stay tuned over the next year or two, I think we’ll see major changes in both of those periods.

Myeloproliferative Neoplasms Defined: What Are ET, PV, and MF?

Myeloproliferative Neoplasms Defined: What Are ET, PV, and MF? from Patient Empowerment Network on Vimeo.

What are essential thrombocythemia (ET) , polycythemia vera (PV), and myelofibrosis (MF) exactly? Dr. Naveen Pemmaraju explains how each of these blood disorders manifests along with the symptoms observed in these MPN patients.

Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju, here.

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Transcript:

Katherine Banwell:    

Can you help us understand the differences between ET, PV, and MF?

Dr. Pemmaraju:         

Yeah, this is very important because we toss these words around as if there’s some big definition that was given, and oftentimes, that never happens. So, let’s pause to do that. So, this goes back to the 1950s when William Damashek, who really postulated the modern MPDs at that time as they were known – myeloproliferative disorders – really thought that there were four diseases that were similar at some level and then presented differently. So, that’s polycythemia vera, essential thrombocytosis, myelofibrosis, and CML, chronic myeloid leukemia.

Then, as the modern era comes in, CML is divided off because of the Philadelphia chromosome, BCR-ABL, which is present in 100 percent of those patients.

So, now we know CML is its own thing. And now we have the big three, sort of non-Philadelphia chromosome MPNs, as they’re now known, because neoplasm – cancer – instead of disorder. Within the subtype, and this is important, the subtypes that you mentioned are the most common.

So, polycythemia vera – poly meaning many, cythemia, cells, vera is Latin for true.

This is the designation for the patient who has a higher than expected blood red cell mass or hematocrit. And it actually, interestingly, Katherine, most patients with P vera have an increase in all three of their blood lines, so the red cells, hemoglobin, hematocrit, platelets, and white count. Those patients with PV are especially at risk for both bleeding and clotting, transformation to myelofibrosis, and even transformation to acute leukemia in maybe 5 to 7 percent of patients.

So, the usual treatment there, Katherine, is to bring off the blood mass. That’s the phlebotomy.

And then in the patient who is above the age of 60 or has a prior blood clot, to give some form of chemotherapy, hydroxyurea (Hydrea), or interferon, for example.

Now, the second grouping is ET, essential thrombocytosis. Again, this word vera or essential, meaning not reactive, not benign, not from a regular cause like a surgery or a trauma or an inflammation. So, it means a cancerous cause, an autonomous cause, something that’s coming on its own.

Thrombocythemia or thrombocytosis, meaning too many platelets. So, usually, patients with ET have too many platelets as their predominant manifestation. But again, as with P vera, patients can get into problems with that. Very, very high platelets, usually a million-and-a-half or higher, can actually lead to bleeding. Not necessarily clotting, but extra bleeding. And then patients with any platelet levels, because the platelet level doesn’t exactly correlate, can have either bleeding or clotting. So, that’s usually the predominant factor. And again, the underlying problem with these MPNs is that they can transform to the other ones – PV, MF, even acute leukemia.

And then, finally, myelofibrosis, which we could spend the whole hour on just by itself, is the more advanced state out of these.

So, it can either arise out of the PV or ET or stand alone. And really here, this is an advanced bone marrow failure state with bone marrow scarring or fibrosis. And now, usually, most patients, their blood counts, rather than high are now low because the bone marrow is unable to produce enough cells. And then, therefore, the sequela of the disease – anemia, thrombocytopenia. So, low blood, low platelets.

Then you need transfusions. The liver and the spleen get larger because they remember how to make blood cells. People can have a wasting away appearance. And then here, more than the PV or ET, this is more of an acute disease for many where if you have intermediate to high stage, these patients can transform more readily to leukemia and have a decreased overall survival.

How to Make an Informed MPN Treatment Decision

How to Make an Informed MPN Treatment Decision from Patient Empowerment Network on Vimeo.

When faced with several options, how can you decide on the best therapy for your essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF)? In this explainer video, Katrina and her doctor walk through important considerations when choosing treatment and provide advice for partnering with your healthcare team.

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Transcript:

Katrina:

Hi, I’m Katrina. Nice to meet you!

Several years ago, I started having headaches and felt very tired. After a trip to the doctor and undergoing bloodwork, I was diagnosed with polycythemia vera, or PV, which is a rare blood cancer that causes my body to produce too many blood cells. It was overwhelming at the time to learn that I had a blood cancer, but my hematologist, Dr. Liu, told me more about the condition and how it’s managed.

Here’s Dr. Liu–she can explain it further.

Dr. Liu:

Hi! I’m Dr. Liu, and I’m a hematologist specializing in the care and treatment of people with myeloproliferative neoplasms or MPNs. MPNs are a group of blood cancers that are characterized by the bone marrow overproducing a certain type of cell. Katrina was diagnosed with PV, which is one of the three MPNs. The three types of MPNs are:

Essential thrombocythemia, or ET, which means that the body is producing too many platelets. The second is polycythemia vera or PV. PV is characterized by the overproduction of red blood cells, and, in some cases, elevated white blood cells and platelets. And the third is myelofibrosis or MF, which causes scarring in the bone marrow that disrupts the normal production of blood cells.

When a patient is diagnosed with any of these conditions, there is a chance they could progress from one condition to the next.

Those that have been diagnosed with ET, PV or MF, should have regular visits with their hematologist to monitor their condition and find the most appropriate treatment to manage their MPN.

Katrina:

After I was diagnosed, I met with Dr. Liu and she walked me through the goals of treatment for PV.

Dr. Liu:

Right! First, we talked about the clinical goals of treatment for PV, which are to reduce the risk of a blood clot and ease or eliminate any symptoms.

And, it’s important to note that because each of the MPNs is different, they are treated differently – be sure to discuss the specific goals of YOUR MPN with your doctor.

Katrina and I reviewed the effectiveness of each treatment option, including how treatment would be administered, and took all of her test results into consideration to make sure we found the best, most personalized treatment option for her PV. Then, we went over what our next steps would be if the treatment plan needed to be adjusted.

Katrina:

Next, we talked about another key treatment goal: symptom management. Dr. Liu let me know that I should make her aware of any symptoms that I may be having, even if I don’t think it’s related to my PV.

Dr. Liu:

Exactly, Katrina. A significant change in symptoms can indicate that it may be time to switch treatments or that the disease might be changing. Those symptoms may include enlarged spleen, fever, itching, fatigue and anemia, among others. This is why it’s always important to not only have blood counts checked regularly, but it’s essential to tell your doctor or nurse about any symptoms you may be having, even if you don’t think it’s related to your MPN.

And, last but not least, we discussed the most important treatment goal: Katrina’s goals. Katrina let me know that she’s very social and enjoys playing golf and tennis with her friends – we wanted to make sure she could continue doing the activities she loves.

Katrina:

Dr. Liu reviewed each of the treatment approaches with me, including potential side effects for every therapy and how it could impact my lifestyle. We discussed the pros and cons of each option, together.

Dr. Liu:

Exactly! When deciding on therapy, you and your doctor may also consider:

Your age and overall health, any presence or history of other medical problems, and the financial impact of a treatment plan.

Katrina:

In addition to asking questions, my daughter, Sarah, took notes during our appointments, since it was often hard for me to absorb everything at once.

We also made sure to talk about the appointment on our way home, while the information was fresh on our minds. And we did our part by researching PV and bringing a list of questions to each appointment.

Sarah found an office visit planner on the Patient Empowerment Network website that helped me organize my health info and questions.

Dr. Liu:

As you can see, Katrina and her daughter were actively engaged in each care decision. It’s vital that patients feel empowered to speak up. If you can, bring a friend or loved one along to your appointment.

And, if you are able, it’s a good idea to seek a second opinion or a consultation with an MPN specialist to help you feel confident in your care decisions.

Katrina:

Dr. Liu let me know that she would monitor my condition through regular physical exams, blood work and frequent communication. She made Sarah and I feel included in the decision-making process, as if it were a collaboration.

Dr. Liu:

That’s right. This is a partnership. So, what steps can you take to be more engaged in your MPN care?

  • Bring a friend or loved one to your appointments.
  • Understand and articulate the goals of your treatment plan.
  • Learn about your options and weigh the pros and cons of each approach.
  • Consider a second opinion or a consult with a specialist.

Katrina:

That’s great advice, Dr. Liu. To learn more, visit powerfulpatients.org/MPN to access a library of tools.

Thanks for joining us!

How Does CLL Progress? Understanding the Stages of CLL

How Does CLL Progress? Understanding the Stages of CLL from Patient Empowerment Network on Vimeo.

What are the specific stages of chronic lymphocytic leukemia (CLL), and how does CLL progress? Dr. Matthew Davids details the stages of CLL and indications for when it’s time to treat the condition.

Dr. Matthew Davids is Director of Clinical Research in the Division of Lymphoma at Dana-Farber Cancer Institute. Learn more about Dr. Davids here.

See More from Engage CLL


Related Resources:

 

An Overview of CLL Treatment Types

Transcript:

Katherine:

Okay. So, how does CLL progress? When do you know when it’s time to treat?

Dr. Davids:

The stages of CLL involve the progression of the disease. When we first meet patients, often they only have cells circulating in the blood, and that’s called stage 0 disease. It’s one of the few cancers where there’s actually a Stage 0 before even Stage I, and the reason for that is that many patients can go for years on Stage 0 disease. But as the burden of the CLL cells begin to accumulate in the body they can start to collect in their lymph nodes, and the lymph nodes can start to swell up whether it’s in the neck or the armpits or elsewhere. That’s stage I disease.

They can accumulate in the spleen, which is an organ in the abdomen. It’s kind of a big filter for your bloodstream, and as the filter traps more of these lymphocytes the spleen can slowly enlarge over time. That’s stage II disease.

And then finally, the CLL cells can get into the bone marrow, which is like the factory for making your blood cells. And if the factory floor gets all gummed up with CLL cells it can’t make the normal red cells, that’s called anemia. Or it can’t make the normal platelet cells, that’s called thrombocytopenia. And when we start to see those more advanced stages III and IV of CLL, that usually does require treatment. And what the treatment does is it clears out the factory floor and it allows for the normal machinery to make the normal blood cells again. So, that’s one of the more common reasons why treatment is needed is due to anemia and low platelets. Second reason can be if the lymph nodes or spleen get so bulky that they’re uncomfortable or threatening organs internally. We want to treat before that becomes a real threat.

And then, the third thing that usually happens as the disease progresses, patients can develop some symptoms, what we call constitutional symptoms. These can be things like unintentional weight loss, drenching night sweats that are happening on a consistent basis, and those sorts of things. So, if that’s happening at the same time as these other factors are progressing, those would be reasons to treat.

And notice that one thing I did not say is the white blood cell count itself.

That’s a common misconception. Some people think that as the white blood cell count goes higher – and people use all different thresholds, 100, 200 – that by crossing that threshold you need to start treatment. And in fact, that’s not the case. We have many patients whose white blood cell count can get very high but then it can kind of level off and plateau for a period of several years, and as long as they don’t meet those other treatment indications, they don’t need to be treated just based on the white count alone.