What are key challenges in myeloproliferative neoplasm (MPN) care, and how do symptoms evolve over time? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses constitutional MPN symptoms, strategies to manage fatigue, neurovascular symptoms, and symptom management.
[ACT]IVATION TIP
“…we have a variety of symptoms that can be associated with kind of myeloproliferative neoplasms as a whole, as well as each one of these distinct disease entities. And the therapy for each of these differs based on the particular symptom.”
Dr. Kuykendall. I’d like you to speak to some of the key challenges in managing MPN symptoms. So based on the available treatment options, what are the most challenging symptoms associated with polycythemia vera, PV, or myelofibrosis and essential thrombocythemia, ET? And what are we learning about how these symptoms evolve as the disease progresses?
Dr. Andrew Kuykendall:
When we think about myelofibrosis, that’s probably the disease we associate with the most clear, distinct symptoms. And patients often have what we call “constitutional symptoms.” And this could be fevers, chills, night sweats, bone pain, weight loss. These are classic symptoms of a disease that is really causing a lot of inflammation, right? And driving a lot of these inflammatory pathways, and these types of symptoms are quite well-addressed with JAK inhibitors, these disease specific anti-inflammatories, of which we now have four that are approved for myelofibrosis in different capacities. But there are more symptoms beyond those. I think when we think about polycythemia vera, we get less constitutional symptoms, although that certainly can be seen in a subset of patients.
But we see more itching is probably the classic polycythemia vera symptom. This itching that is quite challenging, doesn’t necessarily respond to antihistamines, and can be something that’s exacerbated by like taking hot showers or being in hot water. There’s a fancy name for it called aquagenic pruritus. And patients may not even be aware this is related to their disease. I’ve met many patients who’ve come in, who’ve been diagnosed with PV who complain of this challenge with showering or being in hot water, who really never put two and two together.
And sometimes the itching isn’t even described as itching. It feels like fire ants all over their body. And you have patients that are really avoiding right, showering. And so they’re doing it maybe once a week or once every two weeks. And so again, this is a symptom that responds quite well to ruxolitinib (Jakafi), which is approved in the second line here.
Beyond that, I think the biggest symptom across myeloproliferative neoplasms is fatigue. And I don’t have great magic tips for fatigue other than to say what we found out is probably non-pharmacologic interventions are better than pharmacologic interventions for fatigue. Things like just making sure you have good sleep hygiene, getting good sleep, healthy diet, exercise, yoga, mindfulness, resting, these probably are more successful in treating fatigue than any specific drug or agent that we have. And I think that speaks to really a failure on our part to develop better therapies. But certainly it’s something that we’re all very well aware of. And so it’s something we monitor in any of our clinical trials when we are developing agents is how does fatigue change over time? And lastly, I’d say for ET I think you can start to see some symptoms that are more kind of neurovascular.
So things like headaches, migraines, ringing in the ears, or tinnitus, or tinnitus. These can be unique to ET and may predate or preempt the actual diagnosis. So a lot of our young patients that are diagnosed with ET may come to attention of physicians because they’ve been dealing with migraines or headaches or fatigue plus migraines and headaches for a long time. Then lo and behold, blood work shows that they have a very high platelet count.
And so over time, I think when we look at these symptoms, certainly there can be waxing and waning of some symptoms, response to therapies, lack of response to therapies. But if the symptoms really do change rapidly, which we don’t see that often, oftentimes this can accompany a change in the disease, right? And that’s the time to go in and see if something’s changed. Sometimes reevaluate the disease status. And so my [ACT]IVATION tip for this is that we have a variety of symptoms that can be associated with kind of myeloproliferative neoplasms as a whole, as well as each one of these distinct disease entities. And the therapy for each of these differs based on the particular symptom.
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How are emerging polycythemia vera (PV) treatments addressing quality of life? Expert Dr. Andrew Kuykendall from Moffitt Cancer Center discusses disease control versus quality of life issues for PV patients and shares updates about rusfertide and hepcidin mimetic clinical trials.
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“…there’s a lot of things that factor into a suboptimal quality of life for patients with PV. And we need to think about all of those as we try to chip away and make patients’ quality of life as good as possible.”
Dr. Kuykendall, since current treatments may help control polycythemia vera but don’t significantly improve symptoms, what steps are being taken to develop new therapies that not only control PV, but also improve patients’ overall quality of life?
Dr. Andrew Kuykendall:
Yeah, so right now the therapies we have for polycythemia vera essentially the main goal is to reduce as I tell patients, to reduce the things that could kill you, right? To reduce the risk of thrombotic events, cardiovascular events, strokes, blood clots, heart attacks, things like that. As we know that that is a huge issue for these patients that are at increased risk for cardiovascular events.
So we do that with a variety of different strategies, but I think increasingly what we’re realizing is we need to make a primary focus of treatment being on improving and maximizing quality of life. And this is something that really we should be thinking about across all malignancies. But specifically polycythemia vera (PV), where this is a disease that if we are able to avoid some of these cardiovascular events, patients have a very good quantity of life expected with their disease.
So they may live with their disease for 15, 20, 30 years. So if that’s the case, we really need to be thinking about how we can do that as best as possible, right? We’re talking about a third of your life living with this disease. And so we need to do that in a quality way. So agents like ruxolitinib (Jakafi) that have been approved for polycythemia vera in the second-line setting.
Ruxolitinib is known that it is improving some of the disease-related symptoms that come with PV, fevers, chills, itching, night sweats, bone pain really does help with those things. But I think that we can move beyond that. And we’re developing agents like rusfertide. Rusfertide is the hepcidin mimetic that is aiming to reduce the amount of phlebotomies patients need. And for me, that’s important in a variety of ways.
One if you don’t need to get phlebotomies all the time, you’re not tied to the healthcare system, right? Nearly as much. And that could be a huge dissatisfactor. And so at the same time, getting a phlebotomy is not that fun either. It requires going in and sitting there getting blood drawn, you may get lightheaded, fatigued that comes with that.
So eliminating that aspect of negative quality of life. At the same time, we’re starting to see with rusfertide whispers that it may help with some of these symptoms that may be related to iron deficiency. Things like brain fog, concentration issues, fatigue. And so if we can help a little bit with that aspect of things too, man, we could start to kind of, you know, chip away at some of the quality of life issues that are ongoing.
And then down the road, I think some of these JAK2-specific inhibitors may have the continued ability to modify the underlying disease. And certainly that’s a huge goal, right? If we can actually start to get true responses really get at the core of the disease to get the disease to go away. And I think that ultimately that will hopefully result in better quality of life as well. So, at the same time, I think my [ACT]IVATION tip for this question is that there’s a lot of things that factor into a suboptimal quality of life for patients with PV. And we need to think about all of those as we try to chip away and make patients’ quality of life as good as possible.
Lisa Hatfield:
Okay. Thank you. And is there any hope of any of these newer therapies being of limited duration, or are all of them continuous therapy? Because I know as a patient myself, that quality of life is impacted by knowing that I’ll be on therapy forever, some kind of therapy forever. Any hope for that?
Dr. Andrew Kuykendall:
Yeah, so certainly with some agents there is hope for that. So some of the agents I referenced, ruxolitinib, rusfertide, these are agents that probably are, are going to be continued therapy. We always call it indefinite, right? As long as we think that the benefits outweigh the risks, we continue that. If we stop those, typically the reasons we’re using them, those come back quite quickly.
But I would say it’s not necessarily the case with agents like interferon. So interferon is an agent that’s less associated with symptomatic improvement, although we do see it in a subset of patients, it’s more associated with the ability to potentially modify the underlying disease. And so what we’ve seen with interferon is that we can measure patients’ JAK2 allele burdens, the number of cells that have the JAK2 mutation that drives the disease.
And in patients that are on interferon for 2, 3, 4 years, we see the number of cells that have the JAK2 mutation go down over time quite consistently. And even in the case that in some patients it goes less than 10 percent or to a level, we really can’t pick it up with our standard testing. And my experience with that is we can actually stop interferon in some of these patients for a pretty extended period of time.
So we have patients where we stopped for one or two years with blood counts that remain quite well-controlled, patients feeling well. In time, we might have to restart it as things start to to pop up, but I think that we are starting to get to these kind of at least treatment reductions, dose reductions where we can spread things out, but also kind of brief treatment interruptions where we get this kind of treatment-free period that certainly can be attractive to some patients.
Lisa Hatfield:
Okay. Thank you. And I’ll just do a quick shoutout to physicians like yourself who deal primarily with MPNs or work a lot with MPNs, that if a patient is watching this and doesn’t have somebody who really specializes in MPNs, everything you’re talking about, clinical trials, it might be helpful even to just get a consult or what I call an expert or second opinion on how to manage your MPNs. So anyway, thought I’d throw that out there. Thank you.
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Myeloproliferative neoplasm (MPN) expert Dr. Abdulraheem Yacoub discusses common challenges in managing MPN symptoms, goals for improving patient quality of life, and proactive patient advice for optimal care.
[ACT]IVATION TIP
“…you should be aware of your own health and your own health challenges and your symptoms, and you should bring to your doctor what is it that you’re bothered by. Some symptoms are a lot more challenging than others, like fatigue, fatigue, and bone pain. These are symptoms that are very resistant to many of our interventions. But that doesn’t mean we shouldn’t keep trying, and we should have an open communication between the patient and their doctors regarding methods to improve that, whether it’s drugs or non-pharmacological interventions or others that we can try for these patients.”
Dr. Yacoub, I’d like you to speak to some of the key challenges in managing symptoms based on the available treatment options. What are the most challenging symptoms associated with the different classic MPNs, PV, MF, and ET?
Dr. Abdulraheem Yacoub:
You know as we address patients with blood cancers ET, PV, and myelofibrosis, there are multiple priorities in these patients. One, we want patients to be safe and protected from the disease by lowering their blood counts to the right goal. For myelofibrosis, we want to achieve disease control by JAK inhibitors and reduction in spleen and consider curative therapy with bone transplantation. So the goals of care are multifaceted and multi-layered in these patients, but we always forget about the quality of life. So patients with ET, PV, and MF live with a high burden of constitutional symptoms that are non-relenting and they impact patients’ quality of life. They impact their psychological health and their physical health. They impact their personal lives and their professional careers. And we try as much as possible to mitigate that impact on patients’ lives and quality of life with the tools that we have. Our tools are imperfect.
Every time I go over all the things we can do, we can use hydroxyurea (Hydrea), interferon, JAK inhibitors, and then that’s it. And then we stop. And there’s really, we don’t have as many tools as we want. Of course, the field is getting better, we are getting better tools to help our patients, but we should always keep patients’ quality of life at the center point of healthcare. In addition to getting the objective metrics controlled, the counts in the right range, and the spleen the right size, we also need to make sure that what we’re doing to patients is also adding quality to their lives.
And my [ACT]IVATION tip for patients is that you should be aware of your own health and your own health challenges and your symptoms, and you should bring to your doctor what is it that you’re bothered by. Some symptoms are a lot more challenging than others, like fatigue, fatigue, and bone pain. These are symptoms that are very resistant to many of our interventions. But that doesn’t mean we shouldn’t keep trying, and we should have an open communication between the patient and their doctors regarding methods to improve that, whether it’s drugs or non-pharmacological interventions or others that we can try for these patients.
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How can myelofibrosis patients and providers collaborate in helping rural doctors? Expert Dr. Michael Grunwald from Levine Cancer Institute discusses common myelofibrosis symptoms and proactive patient advice for educating themselves and their providers about symptoms and care.
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“…patients with newly diagnosed myelofibrosis and longstanding myelofibrosis can help educate primary care physicians about the disease. Often, primary care physicians want to be able to partner with specialists in the care of complex conditions.”
Dr. Grunwald, given how rare this disease is, how can primary care physicians in rural areas be better equipped to recognize and refer patients with suspected myelofibrosis? And what topics, in terms of questions, should patients be asking their primary care providers in rural areas?
Dr. Michael Grunwald:
I think the answer lies in education. It is difficult for primary care physicians to keep up with all organ systems, all diseases. I think it’s important for us as hematologists, for the hematology field, to let primary care providers know the signs of myelofibrosis and also the fact that treatment has become more nuanced and complex in recent years with multiple new therapies available. It also helps if patients educate themselves and educate their providers. So some of the signs, some of the initial signs of myelofibrosis can include splenomegaly, unexplained symptoms such as itching, night sweats, and/or bone pain, sometimes fatigue.
And if patients experience those symptoms, they can seek out primary care help to have their blood tested. Oftentimes, myelofibrosis patients will have abnormalities that are detectable on the peripheral blood with a CBC, a complete blood count with differential. Patients who know that they have myelofibrosis can help teach their primary care providers about their journey in MF care, thereby increasing knowledge and letting providers know how far myelofibrosis care has come. So I think hematologists and patients can work together to try to teach primary care providers about this disease.
My [ACT]IVATION tip for this question is patients with newly diagnosed myelofibrosis and long-standing myelofibrosis can help educate primary care physicians about the disease. Often, primary care physicians want to be able to partner with specialists in the care of complex conditions.
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What renal medullary carcinoma (RMC) symptoms do high-risk patients need to be aware of? Respected expert Dr. Nizar Tannir shares common symptoms that can signal RMC and advice for patients to help ensure they receive urgent care when needed.
Dr. Nizar Tannir is a Professor in the Department of Genitourinary Medical Oncology, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center.
[ACT]IVATION TIP
“…if an individual who has sickle cell trait sees blood in the urine or they have flank pain, that those are warning signs, they need to seek medical attention, they need to contact their physician or go to a local emergency room or healthcare facility, and be checked. They can start with having an ultrasound or a CAT scan to really evaluate the kidneys, to look at the kidneys.”
For those who may be considered high-risk, what are the symptoms of renal medullary carcinoma?
Dr. Tannir:
RMC, is the most aggressive type of kidney cancer so as any kidney cancer, the symptoms relate to the tumor in the kidney, so there will be blood in the urine, that’s one symptom another symptom is pain, flank pain or abdominal pain, belly pain, those are the symptoms related to the, these are local symptoms related the finding of the tumor in the kidney, but if there is a spread of the cancer and RMC, the reason it is one of the most aggressive cancers any person can get, and the most aggressive kidney cancer type is because of its propensity to spread to organs, and, of course, if there is a spread of the cancer or RMC to organs, there will be symptoms related to the spread of the cancer to these organs, for example, if they cancer spread to the lungs, the patient or the subject, we have cough or shortness of breath, or chest pain, if it spreads to bone they have bone pain, they may have weight loss and fever, and these are called constitutional symptoms.
So fatigue, so these are symptoms related to advanced cancer as it is with any advanced cancer, but specific local symptoms related to RMC would be flank pain and or blood in the urine. These should be warning signs. So my activation tip is if an individual who has sickle cell trait sees blood in the urine or they have flank pain, that those are warning signs, they need to seek medical attention, they need to contact their physician or go to a local emergency room or healthcare facility, and be checked. They can start with having an ultrasound or a CAT scan to really evaluate the kidneys, to look at the kidneys. So this is an important activation tip for individuals who see, who experience or encounter any of those symptoms I just mentioned.
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How can the burden of myeloproliferative neoplasm (MPN) symptoms be lessened through the use of mobile health apps? Blood cancer patient Lisa Hatfield shares common MPN symptoms that patients experience and explains wellness strategies and mobile app study results that decreased the symptom burden for patients.
As an MPN patient, you might experience symptoms like fatigue, night sweats, difficulty sleeping, abdominal discomfort, bone pain and others. However, early data using integrative approaches for the treatment of MPNs are promising, including aerobic activity, yoga, meditation, and strength training, to reduce the symptom burden and improve inflammation. With the evolution of smartphone technology, mobile apps have been increasingly popular to document wellness strategies. With this in mind, the University of Arizona Andrew Weil Center for Integrative Medicine developed and successfully piloted a global wellness mobile app, My Wellness Coach (MWC), to guide MPN patients on self-management strategies for their symptom burden.
The app had patients set at least two wellness goals with clear action steps within these seven areas: nutrition, movement, sleep, resilience, environment, relationships and spirituality to work on over the course of 12 weeks. Within the app, there were links to curated resources and tips. Participants were sent 24- to 72-hour interval reminders before and after each action step and a goal deadline to encourage action throughout the intervention. At the end of the study, improvements were observed in inactivity, impaired concentration, dizziness, numbness, sexual dysfunction, night sweats, bone pain, and quality of life.
If you’d like to implement something similar to what the participants did, try the following:
Reflect on why you want to change your symptom burden so you feel motivated
Determine which of these categories: nutrition, movement, sleep, resilience, environment, relationships, and spirituality would you like to set goals in
Create two goals from those categories and make them SMART- specific, measurable, attainable, relevant, and time-bound.
Utilize resources available to you through support groups or online tools
Set reminders on your phone or calendar for each step you need to take to complete your SMART goals
Mobile-based apps are another example of how MPN patients can use telemedicine in their day-to-day life and improve care.
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How can myeloproliferative neoplasm (MPN) patients benefit from integrative health? Experts Dr. Krisstina Gowin, Dr. AnaMaria Lopez, and patient Lisa Hatfield discuss common symptoms of MPN patients, integrative health techniques, and benefits of including integrative medicine.
So I have multiple myeloma, and, of course, that comes with side effects from the different therapies and symptoms of their own. We have a great integrative health center at our cancer center here locally where I live, and I’ve used it for acupuncture for some of my symptom management. I’ve also watched you on different platforms, through webinars and patient support groups where you describe different integrative health techniques and that type of thing. So I’m wondering…two questions. The first part is, what symptoms do MPN patients face the most? And then how can they use integrative health to do that, particularly as it relates to telemedicine? Are there telemedicine options for integrative health? I suppose things like acupuncture, maybe not, but other types of, of integrative health, and can they get a consult for integrative health? Can they even go as far as getting a consult? So if you can answer those questions, the symptoms they face, how to use integrative health, and if they can get a consult for integrative health, that would be great. We’d appreciate that.
Dr. Krisstina Gowin:
Yeah. Well, Lisa, I want to take a moment just to validate your journey that you’re going through and to congratulate you for your self-advocacy to go look for those integrative therapies to support yourself. And for MPN patients, I will say that it’s a really unique group, and so all cancer patients experience symptoms. But in myeloproliferative neoplasms, it’s really kind of this heterogeneous what we call a symptom burden. And so most patients will experience fatigue about 80 percent of MPN patients. But then beyond that, there’s really a whole slew of different sequelae that can be associated with the disease, which you may or may not think about when you’re thinking about MPNs, such as psychosocial issues, sleep issues, sexual issues.
And then we have kind of the classical issues that happen with MPNs, such as dizziness, but we talked about the fatigue, bone pain, itching, abdominal discomfort from an enlarged spleen and early satiety, or feeling full quickly. It’s really a huge symptom complex, if you will. And we now have validated measurement tools to better understand those. It’s the MPN symptom assessment form, which has really, I think, revolutionized how we look at MPN. It’s no longer just treating the blood counts. We’re treating the patient as a whole, and even within our NCCN guidelines, kind of how we as oncologists go through the algorithms of how to change therapy and how we look at patients. We now have symptoms in there. So even if blood counts are controlled, we may change therapies or even do a bone marrow based on symptoms alone. So symptoms are a huge thing in MPN. So getting to your second question for integrative health.
So I think that MPN…the patients in the community are really early adopters for digital engagement, which is fantastic. Everyone’s very engaged, and I’ve had the opportunity to work on meditation apps, yoga apps, a wellness-based app here from the University of Arizona, and patients just really accrue fast. Everyone’s so excited. And most of these, though, were very small kind of pilot trials, looking at feasibility, can’t we really do these things? But most of them as well are showing some impacts on depression, anxiety, sleep, and total symptom burden. So I do think that these modalities through digital platforms certainly can make a difference on the symptoms. And we’ve seen that with meditation. We’ve seen it with yoga, and we’ve seen it with a seven domain wellness app. And is it the digital engagement? I don’t think so.
I think it’s likely the integrative therapies that they’re receiving through that platform, right? We know meditation works, we know yoga works, perhaps not so well in MPNs. We need to build that evidence base, but other solid cancers, we know those interventions really work. But it’s wonderful to get that kind of early data, say it not only works, but it also works when you’re doing it at home, when you’re doing it on a digital platform. And so I would encourage all patients listening to this to, yes, look at what’s around you, what are the resources, what are the clinical trials? Looking at these different digital modalities for integrative medicine, but also to go get an integrative consultation.
And as Dr. Lopez already had mentioned, she does all of her integrative medicine via telemedicine now, which is fantastic. And so you, it’s really, it’s that, you know, your fingertips. You now have access to wonderful oncologists like Dr. Lopez to guide you in this journey. And the journey is not only allopathic Western medicine, but it’s treating you as a person, you as a whole symptom complex. And that’s really what integrative medicine aims to support you through.
Lisa Hatfield:
And you mentioned that Dr. Lopez also does her integrative health via telemedicine. So I’m going to ask, Dr. Lopez, can you speak to that a little bit more? How do you do that with patients? Do they just contact you and set up an appointment for an integrative health consult or appointment? And do you conduct some of that yourself, or do you send them to particular resources in the community?
Dr. AnaMaria Lopez:
Sure. So, yes, patients can make an integrative oncology appointment directly. I really like to do the consults through tele simply because I can…as I was mentioning, it’s like a virtual house call to really get a sense of the patient. Often a partner, significant other, caregiver might be present as well, and as we know there’s the survivor and there’s the co-survivor. So including both can be very helpful to some people, and I think the initial intake…again, as Dr. Gowin was saying, it depends so much on what the person wants to do. So the first opportunity for coming together is simply, “Where are you? What are your goals? What’s important to you? And of the panoply of options, which might be the easiest or the one that you are most interested in?”
And so depending on what it is, we might work together, we might also bring in others if the person is really interested in making lifestyle changes, let’s say related to nutrition. The person might work closely with a nutritionist for some period of time and then come back, and we’d come together and reassess. You mentioned the acupuncture, and you can’t do acupuncture at a distance, but you can certainly teach people about the points and consider acupressure for certain points. So there are so many ways to engage and interact, but yes, I think like a lot of medicine, it’s a team-based approach.
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What happens in early stages of multiple myeloma? Watch as early multiple myeloma is explained as expert Dr. Rafael Fonseca details what occurs in the body, and patient Lisa Hatfield shares the symptoms that she experienced early in her myeloma journey.
These cells live predominantly inside the bones in the space we call the bone marrow. They can do a number of things that actually lead to the symptoms and to the clinical presentation. As they grow in the bone marrow, they take some of that real estate. A person may experience fatigue and that is because they have anemia. The myeloma cells are also very characteristic because they can erode into the structure of bones, so destruction of bone is another feature that we see in patients with myeloma. That can be either seen on X-rays or sometimes people will present with symptoms related to bone pain or discomfort with movement or weight bearing. Those are signs that we look for.
Lisa Hatfield:
For me, early on with myeloma, I really had none of the classic symptoms. All of my blood work was coming back normal. I would see my regular primary care physician every two years. My lab work was coming back normal. Nothing really stood out. I wasn’t anemic. My kidney function was okay. What did stand out over the course of two years was I was experiencing progressively worsening pain in my hip. It felt like kind of a pinched kink pain in my hip to the point where it progressed to the point where I could barely walk was when I finally talked to my primary care doctor. And requested very strongly to have a scan done, and that’s when I was diagnosed with myeloma.
So the primary reason I went in was for the pain to begin with, and my doctor did look at the pain. He tried to assess it several times over the course of two years. But it wasn’t until I had the MRI that showed a large plasmacytoma on my spine when I realized that something was wrong. A couple other signs that I did have looking back now that I complained about to my doctor and I thought were rather curious, I shrunk a little bit. I shrunk in height. My daughters were laughing, and they’re like “Mom, we’re just growing.” But I did shrink in height by about 2-1/2 inches from the compression fractures in my spine and the plasmacytoma that had eaten away at my spine. And then another thing that a lot of people don’t talk about is sometimes people will have foamy urine. We don’t like to talk about body functions.
But it’s important to know that if you experience that, there are proteins that they can find that are called Bence Jones proteins that are a sign of multiple myeloma. So if you notice anything unique like that – foamy urine, extreme fatigue, anemia in your blood tests, it’s definitely worth asking your doctor about. And also relentless, persistent pain in your hips, in your back, in your ribs, any of those areas, it’s worth talking to your doctor about just to assess those thoroughly to make sure there’s not something more significant going on.
If myeloma goes undiagnosed and untreated, the cancer cells can make a patient experience:
Lowered immune function due to white cells being crowded out, resulting in frequent infections
High levels of protein in the urine and blood, which may cause kidney damage
Build-up of cancer cells in the bones, which can cause bone weakening, bone pain, and bone fractures
https://powerfulpatients.org/wp-content/uploads/What-Are-the-Beginning-Stages-of-Multiple-Myeloma-MM.png600600Kara Rayburnhttps://powerfulpatients.org/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-03-02 10:39:552023-05-04 09:58:18What Are the Beginning Stages of Multiple Myeloma (MM)?
Dr. Abdullah Khan, a myeloma specialist, discusses the types of tests that myeloma patients should undergo before choosing therapy, at diagnosis, and if they relapse.
Dr. Abdullah Khan is a hematologist specializing in multiple myeloma and plasma cell disorders at the Ohio State University Comprehensive Cancer Center – The James. Dr. Khan is also an assistant professor in the Division of Hematology at The Ohio State University. Learn more about Dr. Khan.
What testing should take place before choosing a myeloma treatment?
Dr. Khan:
I thought I could answer this question in an indirect way first.
Katherine:
Okay.
Dr. Khan:
I just wanted to let the audience know that anyone, including those that are not in the medical field, can create an account with the nccn.org. That’s the National Comprehensive Cancer Network’s website.
And from there they can download the myeloma guidelines, which are available to all myeloma providers as well. And in the guidelines, there are sections for workup, treatment, how to follow patients with myeloma, and many other things.
So, going back to the question, the first patient encounter will likely include a thorough history and physical exam. Initial lab work includes blood counts, the serum chemistries so we know about the liver and kidney function, multiple myeloma markers. And patients about get very familiar with monoclonal protein, the serum immunoglobulins, and the serum-free light chains.
These are used as the surrogates for responses when you’re undergoing treatment for the myeloma. We will also at the first visit probably also do a 24-hour urine collection, and that’s looking for the abnormal protein in the urine.
There’s imaging. In the past, we used to do x-rays head to toe. That’s sometimes called the myeloma survey or the skeletal survey. But the new recommendations are actually looking for something a bit more sensitive.
So, at our practice, what we do is a PET scan.
So, that includes functional information as well the images themselves. And some institutions may do a PET scan head to toe using low-dose radiation. The final test we will do in patients with newly diagnosed myeloma is a bone marrow biopsy and an aspirate.
So, the biopsy’s looking at the bone itself and the architecture. And the aspirate, you take the liquid part of the bone marrow, and you can ascertain a lot of information including the burden of myeloma when the patient’s newly diagnosed.
Katherine:
What do you mean by “burden”?
Dr. Khan:
You can quantify the number of cancerous plasma cells in the bone marrow. So, some of the information says you have a healthy amount of good bone marrow cells, 50 percent, 60 percent, for example, but of that 50 percent, 60 percent, maybe 80 percent is taken over by myeloma. So, you will get burden of myeloma information from there.
Katherine:
What additional testing should take place following a relapse?
Dr. Khan:
I’ll start that response by first talking about the types of relapses, and there are two broad categories. If we see the myeloma coming back as just the monoclonal protein going back up from its lowest, or maybe the serum-free light chain going up – and there are very specific criteria for what defiance a relapse. But if it’s just a number, we call it a biochemical relapse.
On the other side, there’s a clinical relapse. And at that point, there might be new end organ damage. We’ve heard of the acronym CRAB when we’re describing myeloma. That stands for hypercalcemia, renal or kidney insufficiency, anemia, and bone disease. So, these are end organ damage directly from the multiple myeloma.
So, typically, we’ll try to change the management at biochemical relapse, because a new organ injury may contribute to the patient’s frailty, or it might even limit the treatment options. The testing out of relapse is pretty similar to the first diagnosis. We’ll repeat the history and the physical example, the labs, imaging. And more often than not, I’ll also recommend a bone marrow biopsy to see is that myeloma changing genetically, and does it help me kind of determine new treatment options.
https://powerfulpatients.org/wp-content/uploads/What-Tests-Are-Essential-Before-Choosing-a-Myeloma-Treatment-Approach.png600600Kara Rayburnhttps://powerfulpatients.org/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2022-10-31 14:28:532022-10-31 14:39:22What Tests Are Essential Before Choosing a Myeloma Treatment Approach?
Multiple myeloma patient Danielle was a very active person – and even went on vacation – right before receiving her diagnosis. Her myeloma journey unfolded with her myeloma symptoms, diagnosis, treatment, and participation in a life-altering clinical trial. “I decided to do the study trial because I also wanted to help individuals. If it wasn’t going to help me, then my data that they collect from the study trial will definitely help the scientists, researchers, the doctors. It would help them try to find a cure.”
My myeloma story began in 2011. I was experiencing pain in my hip and my back area, and it was the pain that would come and go. I was also very lethargic in 2011 and couldn’t understand why I was so extremely tired, so I thought the pain in my hip and back area was due to sciatic nerve, and I just didn’t do anything about it, ignored the pain. My husband and I went on our first trip without our sons in October of 2011, and two days before the trip, I developed this really bad nasty pain in my hip and leg area, which actually altered my walk, but I had no idea what the heck was going on, and so I was so frustrated that I… As soon as we got home, I went to see an orthopedic doctor because at that time I was working out like five times a week, so I thought maybe I pulled something, a pinched nerve or something. So I went to see him, he took X-rays, I believe it was an MRI, couldn’t be sure, but when I went back to get my test results, he sat me down and said, “Mrs. Spann, there’s a mass here in your fibula, and I’m going to recommend you to an orthopedic oncologist.” So, that was the very beginning of my diagnosis, initial diagnosis. Of course, I was in denial because I’m like, I knew what an oncologist was, but he must not be talking to the right person, but I went ahead and I met with the orthopedic oncologist. He ran a bunch of tests and mentioned to me that I had myeloma, which is concentrated in one area, which was my fibula, and then he recommended that I have my fibula removed on my right leg. Two days before surgery was scheduled, I received a phone call from his office, saying, “Mrs. Stann, you have lytic lesions all throughout your skeletal structure, and we’re recommending that you go see a bone marrow transplant oncologist.” So now it’s becoming real. The diagnosis is what it was, and I just wanted to know how I could basically fight this. I’m the type of person where you tell me one thing and let’s try to find a solution, so I met with the bone marrow transplant specialist, the oncologist, and then we formulated a plan, and that plan was for me to go on my first study trial. And so that was my introduction into my having multiple myeloma.
I made the decision to participate in a trial, because I trusted my doctor. He had the expertise to understand where my myeloma was, the counts, how aggressive it was, and he recommended that I go on the study trial. He also told me that if the study trial was not going to work for me, or if it wasn’t helping me, that he was going to take me off the study trial. So, I was on the study trial from like January to March…to the end of March, and he sat me down and said that it was not working, my numbers weren’t really moving, and that he was taking me off the study trial. And he took me off the study trial, there were some other treatments that were involved, and then I had two stem cell transplants. After the transplant in 2012, I went ahead and started another treatment regimen, and I was on that for several years, which worked well. My numbers were coming down, but then unfortunately they started going back up, so he mentioned that I should go on another study trial. I weighed the odds, and I knew that he would not lead me down the wrong path. So, I went ahead and I participated in the study trial that I’m still on today, and I’ve been on it for about three, four years.
I decided to do the study trial because I also wanted to help individuals. If it wasn’t going to help me, then my data that they collect from the study trial will definitely help the scientists, researchers, the doctors. It would help them try to find a cure. And so that’s what I wanted to help in some form or fashion, and when I first was diagnosed going to the Winship Cancer Center twice a week, there was a quote that was posted in the cancer center, and that quote was by Dr. Martin Luther King Jr., the quote read, “Life’s most important and persistent question is, ‘What have we done to help others?’” And I would go into the center and I’m like, “Yeah, what have I done to help others?” And me participating in the study trial, I felt like I’m helping others indirectly, and it wasn’t always just about myself, it was, “Okay, yes, the study trial gives the data, and it’s helping me, but it’s also helping that next person as well.”
So, I always look at my life as before diagnosis and after, and my after does not look like my before, I can’t do the same things, I can’t do the same things that I used to do. And one of those things is going to the mall and being in there like 10 hours, that’s so remedial, but it just goes to show like I cannot exert myself the same type of energy that I could before diagnosis. And again, that’s my new normal. I stay positive with everything in life, things happen, but you just have to do what you can to make it better, no matter what it is.
I am happy and proud and so grateful and thankful to mention that as of January 2021, my myeloma is 0% detectable, which means there’s no presence of multiple myeloma in my blood, in my urine, nor in my bone marrow. And so I’m still on a study trial, and I have two different chemo meds that I have to take, and I just act accordingly if I know that one of the chemo meds that I have to take twice a week gives me an upset stomach. I just accordingly in finding different ways to push through it. It is what it is, and my motto when I was having my bone marrow transplants was, “This too shall pass.” And so no matter what I’m going through in life, no matter how down I get. This moment will pass. And so tomorrow, you’ll look back on today and say, “You know what, I did it, I made it.” And you’ll do that for the next day, until you realize that you’re just constantly defeating that previous day, and you’re moving forward.
So, I’ve heard the terminology of a clinical trial, never really paid attention to it because I never had to…I had an idea what the clinical trial was. But once it really came home to me, I realized that, in my words, the clinical trial is collecting the data necessary, they’re going to give you the trial medication, because they’re looking to get this, this medicine approved to put on the market. These medications would not get approved by the FDA, acetaminophen (Tylenol) at one point had to go have a study trial and then get approved by the FDA and then can be distributed to the masses. And so it’s the same with these other drugs. We need individuals to participate positively, knowing that if this is not helping me right now, it will help someone in five years, in two years, in 20 years. The advice that I would give is to trust your doctor, your doctor would not recommend a study trial if he felt that there was a medication that’s already on the market that would help you better. If the study trial you’re on is keeping you with your family, and at the same time is…the scientist, the researchers they’re gathering all this data, it could come to be an actual medication in three, five, seven years. And so just think of it as something that you’re helping society…and your fellow…and your fellow man.
Breast cancer expert Dr. Julie Gralow explains how you can advocate for the best metastatic breast cancer care, through speaking up, utilizing care team members and taking key steps to achieving better care.
Dr. Julie Gralow is the Jill Bennett Endowed Professor of Breast Medical Oncology at the University of Washington, Fred Hutchinson Cancer Research Center, and the Seattle Cancer Care Alliance. More about this expert here.
For patients who may be hesitant to speak out for themselves and advocate for their own care and treatment, what advice do you have?
Dr. Gralow:
You have a whole team who’s behind you, and I’m the MD on the team, but I’ve got a nurse practitioner, and a nurse, and a scheduler, and a social worker, and a nutritionist, and a physical therapy team, and financial counselors. I’ve got a whole team who works with me. And so, a patient might be hesitant to speak up during the actual appointment with their physician. It’s a short amount of time. I would recommend come into it with written-down questions because things go fast. You don’t get a lot of time with your doctor.
Things go fast, but don’t come in with 25 questions, either. Pick your top few that you want to get taken care of this visit because if you come in with 25 or 30, you’re going to lose the answers to most of them. Maybe bring somebody with you who’s an advocate and a listener for you who could be taking notes, so you can process and you don’t have to write it down, or ask if you can record it. It’s really important if you’re newly diagnosed or maybe there’s a progression and you’re going on a new treatment. That’s okay too.
But, I would also say you have a whole team behind you, so sometimes, if you don’t have time or if you’re hesitant to speak up in your doctor’s visit, you can ask the nurse, or maybe you can ask the social worker for help, even. See if there’s support groups around.
Interestingly, we’ve got a peer-to-peer network where patients can request to talk to somebody else who’s matched to them by some tumor features, and their stage, and things like that. Maybe finding somebody else who’s gone through something similar, and somebody independent to talk to instead of relying on your family.
It can also be really helpful to talk to a therapist or a psychologist about your fears, and sometimes, you want to be strong for your family, strong for your children and all, but you need a safe space with somebody that you can just express your fears and your anger if that’s what’s going on, or your depression or anxiety to while you’re trying to hold a strong face for others in your family. So, I would encourage patients to look at who is the whole team and talk to the other members of the team as well, and sometimes, they can help advocate.
Also, find somebody who might be able to come to your appointments with you, somebody who will help you advocate or remind you – “Didn’t you want to ask this question?” – or be another set of ears that you can process it with afterwards.
Katherine:
Dr. Gralow, we’ve covered a lot of useful information today for patients. Thank you so much for joining us.
Dr. Gralow:
Thank you, Katherine.
Katherine:
And, thank you to all of our partners. To learn more about breast cancer and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell.
https://powerfulpatients.org/wp-content/uploads/How-Can-You-Advocate-for-the-Best-Breast-Cancer-Care_-1-1.png600600Kara Rayburnhttps://powerfulpatients.org/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2020-11-04 09:33:162024-09-05 15:02:06How Can You Advocate for the Best Breast Cancer Care?
Genetic tests can help guide metastatic breast cancer care. Dr. Julie Gralow discusses essential genetic tests for metastatic breast cancer, and how results impact treatment decisions.
Dr. Julie Gralow is the Jill Bennett Endowed Professor of Breast Medical Oncology at the University of Washington, Fred Hutchinson Cancer Research Center, and the Seattle Cancer Care Alliance. More about this expert here.
For a patient to get diagnosed, what are the essential tests?
Dr. Gralow:
So, we’re talking about metastatic breast cancer here, and in the U.S., maybe up to 10% or slightly less of breast cancer is technically Stage 4 or metastatic at diagnosis. That means at the time we first found it in the breast, it had already spread beyond. So, an important thing that we’ll do with a newly diagnosed breast cancer is especially if there are a lot of lymph nodes are involved or the patient has symptoms that might say there’s something in the bone, liver, or lung is staging.
So, we’ll use scans – maybe a CAT scan, bone scan, or PET scan – and we will look at whether the disease has gone beyond the breast and the lymph nodes, and if so, where. So, maybe 8-10% of breast cancer diagnosed in the U.S. already has some evidence that it has spread beyond the breast, but the most common way that metastatic breast cancer happens is that a patient was diagnosed possibly years and years ago, treated in the early-stage setting, and now it comes back, and that is the most common presentation for metastatic breast cancer, and sometimes that can be due to symptoms.
As I said, if it comes back in the bone, maybe that’s bone pain. If it’s in the lung, it’s a cough. There are symptoms. Sometimes, it’s because we’ve done a blood test or something and we find some changes there.
And so, when a breast cancer has recurred, it’s really important to document that it’s really breast cancer coming back, first of all, and so, if we can, we generally want a biopsy, and we want to stick a needle in it if it’s safe to do, and look and verify that it looks like breast cancer, and also, it’s really important that we repeat all those receptors that we talked about from the beginning because it can change.
So, a cancer up front 10 years ago could have been positive for estrogen receptor, but the only cells that survived – mutated, changed – were estrogen receptor negative, so what comes back could be different. So, it’s really critical to get that biopsy, repeat the estrogen/progesterone receptor and HER2, and also, in an ideal world, now that it’s 2020 and we’re moving more toward genomics, to do a full genomic profile and look for other changes and mutations that could drive our therapeutic options.
So, staging, knowing where the cancer is, getting a good baseline by understanding where it is and how big it is so that we can follow it and hopefully see that it’s responding to treatment, and then, repeating all of the biology components so that we know what the best options are for treatment are really critical.
Katherine:
Right. How can patients advocate for a precise breast cancer diagnosis, and why is that important?
Dr. Gralow:
Well, all those things I just mentioned are key. Knowing exactly where it is so that we can monitor it – for example, if the cancer has come back in the bones, we would add what we call a bone modifying agent, a drug like zoledronic acid or denosumab – Zometa or Xgeva – which can suppress bone destruction from the cancer, but if it’s not in the bone, we wouldn’t add that.
And, we want to have a good look everywhere so that we can see if it’s responding because sometimes, the tumor can respond differently in one area than another. Also, I think it’s really important to know what your treatment options are by doing that biopsy, getting a full panel, and looking at potentially hundreds of genes that could be mutated, deleted, or amplified so that we know what our treatment options are.
And, we’re not going to use all the treatment options up front, so it’s helpful for knowing that if this treatment doesn’t work or is too toxic, what are the second-line or third-line options? So, we make sure that there’s what we call good staging up front so we know where the cancer is, and then we make sure that we’ve looked at it as best we can in 2020 with all the genomics.
That would give us the best chance of being tailored – individualized – to the tumor. Sometimes, if we can’t biopsy it, like with a needle that would go into a liver spot, then increasingly, we’re looking at what we call liquid biopsies, and that can be drawing the blood and seeing if we can find parts of the tumor, whether it be the DNA or the RNA that’s floating around in the blood, and sometimes we can get that information out of the blood as well.
https://powerfulpatients.org/wp-content/uploads/What-Are-Essential-Genetic-Tests-for-Metastatic-Breast-Cancer-Patients_-1.png600600Kara Rayburnhttps://powerfulpatients.org/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2020-10-20 14:14:592022-11-08 14:07:59What Are Essential Genetic Tests for Metastatic Breast Cancer Patients?
Dr. Daniel Pollyea, an AML specialist, dispels common myths around the causes and symptoms of AML and shares advice so that you can identify credible resources for information. Download the Program Guide here.
Dr. Daniel A. Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center.
I’m Ross Reynolds. Today we’re gonna be debunking some common misconceptions about the causes and symptoms of AML.
And joining me is Dr. Daniel Pollyea. Dr. Pollyea, could you introduce yourself?
Dr. Pollyea:
Yeah. Hi. Good morning, everyone. I’m Dan Pollyea. I’m an Associate Professor of Medicine here at the University of Colorado, where I am the Clinical Director of Leukemia Service.
Ross:
I wanna emphasize to you that this program is not a substitute for medical advice, so be sure to consult your healthcare team when it comes to solid information about it. But you will get some background that I think you’re gonna find useful. And you might have some questions as we go along.
Dr. Pollyea, let’s start out with the basics. What are the causes of AML?
Dr. Pollyea:
Yeah. So, Acute Myeloid Leukemia, it’s a disease, a cancer of the bone marrow.
And it’s the result of an accumulation of mutation and chromosomal abnormalities that affect the DNA of a precursor cell in the bone marrow, otherwise known as a stem cell.
And those abnormalities accumulate until that cell can no longer properly mature, and it also can’t properly die. And so, a cell like that just makes copy after copy after copy of a cell until it crowds out the whole bone marrow with these sorta useless, immature cells.
And the end result of that is the failure of the bone marrow, which causes all of the problems associated with this disease. So, biologically, that’s sort of what happens to make this disease occur.
Ross:
What are some of the myths that you hear from patients that come in and they say, “Oh, this must’ve caused my AML,” but you have to tell them that’s not so?
Dr. Pollyea:
Right. So, I mean, this is one of the most frustrating issues for patients and their families after diagnosis. I mean, it’s a rare disease, only about 30,000 cases a year in the United States. And so, trying to associate a rare disease with external or environmental factors is difficult to impossible. So, although there are a variety of exposures that probably contribute to this disease, we have very little understanding of what those exposures typically are or how that all works.
So, there’s a few things that we know pretty well; large doses of radiation, either associated with like industrial accidents like the Chernobyl disaster, or some of the radiation therapies that patients receive for other types of cancer. Other types of chemotherapy that are used to cure other cancers can contribute to this disease in later years.
We know that there are certain precursor conditions that can evolve to AML, so a person with myelodysplastic syndrome, for instance, has a fairly high chance of someday evolving to develop Acute Myeloid Leukemia. But beyond these sort of a few associations, there isn’t a whole lot that’s known or proven.
Ross:
Now there is radiation associated with X-rays, and some people think that X-rays can cause AML. Is that true?
Dr. Pollyea:
Yeah.
So, I mean, I think a priori no because millions of people get X-rays every day, and only 30,000 people a year get AML. So, clearly it’s not a simple association between getting an X-ray and developing AML. But I think that there is an unknown interaction between environmental exposures and a person’s individual genetic makeup that makes a person more or less susceptible to developing something like AML with respect to exposure to the environment or X-rays and things.
So, while you cannot say that getting an X-ray will lead to AML, certainly there are some people who are more sensitive to the damage that’s done by something like an X-ray. And so, the best course of action is to be cautious and judicious about your exposure to these things, but not to not get these things when they are medically necessary.
So, that’s the challenging balance.
Ross:
Here’s something else we’ve heard, that weed killers can be a risk factor for AML. Is that true?
Dr. Pollyea:
I mean, I think there’s a lot coming out now about weed killers and their association with other types of cancers. Again, I go back to the limitation we have in that in only 30,000 people a year in the United States get AML. Millions of people are exposed to weed killers.
We’re statistically never going to be able to make a clear association. I think that there are certainly some risks for some people. Whether you’re that person who’s more susceptible to developing leukemia or any other cancer because of exposure to a weed killer is impossible to know.
So, like all of these things, I think the advice we have is you have to live your life. You have to do your best to sort of avoid things that you can avoid that you think would be… Or that may cause problems. But not to let those things prevent you from living a normal life.
I know that’s not a satisfying answer, but at the moment that’s the best answer we have.
Ross:
Is formaldehyde exposure another risk factor for AML?
Dr. Pollyea:
Yeah. We think that it is, and kind of along the lines of benzene. But, again, we think that those studies that have shown those types of association show it in very high amounts, amounts that most people in this country would not be exposed to. But I do think, or we do think that there is something to that, to formaldehyde somehow contributing to this.
Ross:
What’s the difference between a risk factor for AML and a cause of AML?
Dr. Pollyea:
Yeah. So, I think risk factors by definition are things that may contribute to AML. And a risk factor for AML by that definition could be walking down the street and having some exposure to radiation from the sun. A cause of AML is something that is a much more solid sort of well-understood factor.
Like I said before, having myelodysplastic syndrome, there is a high chance that that can evolve to Acute Myeloid Leukemia. And if that happens then the MDS, the myelodysplastic syndrome, could be considered or would be considered the cause of your AML. So, very, very different in terms of the amount of evidence that goes into making those determinations
Ross:
Is there a genetic component to this? Can this run in a family?
Dr. Pollyea:
Yeah. So, this is a disease of the genome.
So, I mean, in a lot of respects it is a genetic disease. But the question is very different when you ask is this an inherited genetic disease? Is this disease due to a gene that I inherited from a parent or could pass along to a child?
For many, many years, the answer from the medical community was, “No.” This was not considered to be a disease that clustered in families or that could be inherited. We now know that that’s not necessarily the case. There are some very rare cases where this does seem to travel in families or cluster in families. And we’re now beginning to understand who those people are and what those genes are.
But the vast majority of people with this disease did not inherit a gene to contribute to it and cannot pass this along to a child. This is a random, spontaneous event that occurred within one person’s own body and is not traveling within family. So, we’re learning more and more about this, but really, the vast majority of this is not an inherited genetic condition.
Ross:
You’ve mentioned gene mutations. What mutates a gene? What causes that to happen that could lead down the line to AML?
Dr. Pollyea:
Yeah. Yeah. That’s a great question. Most of the time we do not know the answer to that. These gene mutations occur spontaneously, randomly, and we don’t understand why they happen when they do happen.
And I know that’s, again, not a satisfying answer. It’s very frustrating, particularly patients come in, and, “I’ve lived a healthy lifestyle. I’ve done everything right. I exercise. I eat right. How could this have happened?”
These are things that for the most part are out of the control of a person. These aren’t impacted by your diet or your activity levels, what you eat or don’t eat, what you do or don’t do. That’s a real frustration. In the end, in almost all cases we don’t know or understand why these gene mutations or these, I call them mistakes in the body, occur when they occur. We don’t understand them.
And, Dr. Pollyea, someone asked if benzene can be a risk factor for AML.
Dr. Pollyea:
Yeah. So, benzene is one of the sort of rare environmental exposure associations that we do have clear associations with AML.
But the level of benzene that a person would need to be exposed to is really something that hasn’t been seen in this country in a very long time.
We’d be talking about like an industrial accident type exposure in almost all cases, so being exposed to a cleaning solution or some other fairly minor exposure to benzene, we don’t think is enough, in most cases, to prompt this disease. But benzene in very high doses, like an industrial accident, yes, that is something that we understand can certainly contribute or cause AML.
Ross:
Autoimmune diseases, such as arthritis, can they increase the risk of AML?
Dr. Pollyea:
Oh, boy. That is a really interesting one. So, there are papers in the literature that do support those associations. And I know in my own practice I certainly see that trend. So, I do think that there is something there. There is a proven association between autoimmune conditions and myelodysplastic syndrome, which I said before can be a clear precursor condition to AML. So, certainly, that is an association that is a possibility.
It can be a little difficult to tease out whether it’s those diseases that are associated with ultimately developing AML, or the treatments that people get for some of those autoimmune diseases. Those treatments can modulate the immune system in certain ways that may, in fact, contribute or drive the disease. So, that’s a difficult thing to tease out.
But in general terms, yes, I think there are some associations. Now not by a long shot everyone with an autoimmune disease gets AML. It’s a teeny, tiny fraction. But I think there is an association there.
Ross:
How easy is it to diagnose AML?
Dr. Pollyea:
Well, I mean, I think there’s very clear diagnostic criteria for AML. But I guess that doesn’t really answer the question. And we certainly have patients who come to us after many months of frustration without a clear diagnosis.
So, those scenarios can play out. Many times AML’s a very dramatic presentation, so people get very, very sick very, very quickly with extraordinarily high white blood cell counts and suppression of all the other blood counts that come from the bone marrow like red blood cells and platelets.
In those cases it’s pretty clear that there is a type of acute leukemia going on. There can be some difficulty distinguishing Acute Myeloid from Acute Lymphoblastic Leukemia; those are sort of like cousins, but very different and treated differently. So, it kinda runs the gamut. I mean, it can be pretty clear, but it’s sometimes missed, so yeah.
Ross:
This is a great lead-in to my next question, which is about the symptoms of AML. What should be the warning signs that this might be something you need to get looked at?
Dr. Pollyea:
Right. So, at presentation, the main symptoms are reflective of the fact that the bone marrow, the organ that makes all the cells of the blood, has failed.
So, that can cause severe anemia. Signs of anemia: a white sort of appearance, feeling dizzy or lightheaded when standing, short of breath, weak, tired, fatigue. Those are all pretty clear presenting symptoms for AML. Because the bone marrow also is responsible for making platelets that clot the blood, some people will present with a bleeding complication, or a very subtle rash made up of these particular red dots. We call that a petechial rash. And that rash can come on when the platelet count gets very low.
Sometimes a person will present with an infection or infections that don’t go away or don’t clear because of decrease in white blood cells, the infection-fighting cells of the bone marrow. Those are made in the bone marrow and can fail in the setting of this disease. So, those are the most common symptoms at presentation, symptoms that are reflective of bone marrow failure.
Ross:
You mentioned that sometimes the presentation could be very dramatic, and it sounds like the symptoms are very severe, very quickly. Is that always the case? Is that often the case?
Dr. Pollyea:
That is the case in, I would say, a minority of times. That’s usually the case. It’s more often seen in younger patients with AML. Typically, older patients with AML have a more smoldering course and a much less dramatic presentation, although this sort of very dramatic and dangerous presentation can happen in older patients, but it’s probably something like a third of the time that those very dramatic and medical emergency presentations occur.
Ross:
How important is early diagnosis?
Dr. Pollyea:
Well, I mean, it’s crucial. I mean, in particular in those cases where it’s a very dramatic and proliferative diagnosis, or presentation. A quick diagnosis and recognition of this condition is very important because the sooner a person starts effective treatment the better the ultimate outcome is.
I would say in general terms that applies to all AML patients, but certainly there’s some degrees of variation. So, there’s some AML patients that when I hear about their case on the phone from a referring doctor, it’s appropriate to see them next week in the clinic.
So, it’s not always a medical emergency, but we would never, even in those next-week-in-the-clinic patients, this isn’t something that can wait for weeks or certainly months. This is something that needs to be addressed fairly quickly.
Ross:
What are the best ways to manage those symptoms?
Dr. Pollyea:
Right. So, I mean, at presentation, all those symptoms, the best way to manage those are to start treatment as quickly as possible. So, impacting the underlying cause of this disease is the most important and critical factor to getting a person feeling better because all of these problems stem from the disease in the bone marrow, and so everything else that you do to sort of help a person’s symptoms are Band-Aids when you’re not talking about getting to the root cause.
So, that’s at presentation. Now once we start treatment, there are many potential side effects to any number of treatments. And it all is dependent on what treatment you’re getting and other things about you that will make this a significant problem in some cases. And in that setting, we do have ways that we can aggressively manage a person’s side effects.
Ross:
Can you manage all of the symptoms? Or can people still be experiencing symptoms even after they’re in treatment?
Dr. Pollyea:
Absolutely. So, a person with this disease, depending on how long they’ve had it and some of the features, may not be feeling back to their baseline self for potentially weeks or months after treatment starts in the best-case scenario. So, that can be very frustrating, but a person needs to sort of be able to continue to have a good outlook and stay positive.
Because we are able in many cases to make a big impact on this disease and return a person to their pre-disease quality of life.
Ross:
What are some of the myths that you hear, Dr. Pollyea, about the treatment? Some things that people come in to you saying they think that it helps, but there’s no science to back that up?
Dr. Pollyea:
So, myths about treatment, so many people have a lot of preconceived notions about the intensity of a therapy that they’re going to be asked to withstand. And although sometimes we do treat this disease very intensively, that’s not always the case, and now we have some very effective lower-intensity regimens that can be used in a variety of different scenarios.
There are a lot of people who have a lot of preconceived notions about a stem-cell transplant or a bone-marrow transplant and whether or not they would be eligible for this based on maybe what they’ve heard from friends or family, or what they’ve seen in the internet.
And those are often incorrect. And so, keeping an open mind about treatment options, and discussing those in detail with your doctor are really, really important.
Ross:
You mentioned sometimes it presents in young people, sometimes in older people. What’s sort of typical?
Dr. Pollyea:
This is a disease of predominantly older patients, so the median age of presentation is 68. So, that means that over half of the patients are over 68 years old at diagnosis. So, while this does happen, can happen in younger patients, that’s really an unusual situation. This disease is, like I said, it is predominantly a disease of older patients.
Ross:
There are some patients who I understand think that supplements can deal with the symptoms of AML. Is that accurate?
Dr. Pollyea:
You know, I mean, I think the supplement question is always a challenge. A lot of these supplements, or most of these supplements have never been tested with the rigor of treatments that we’re accustomed to in the medical establishment.
That being said, I won’t deny that some of the supplements can help patients based on what patients’ experiences are and what they tell me. I think what’s really important is just be very open and honest with your doctor about the supplements that you’re taking or want to take to ensure that there are no sort of unanticipated interactions with treatments.
Because I think most doctors are very open to having their patients care for themselves in the ways that they’ve become accustomed to, and they know their bodies very well, and we’re very open to that. But there are sometimes that a drug or a supplement might have a bad interaction with the treatment.
And so, a good example in my practice is antioxidants. So, there’s a lot of literature, a lot of interest in antioxidants as cancer-prevention treatment.
And a lot of that is not well-established, but still I don’t see much harm. But when it comes time to treating a cancer, that’s a very different situation. When we give a patient treatment to try to kill the cancer cells, many times we’re trying to provoke oxidation. That’s part of how these drugs and these treatments work.
So, if you’re taking those treatments, but also at the same time taking antioxidants, there’s the potential you could sort of be cutting your therapy off at the knees, fighting it with one hand behind your back. So, for the period of time when my patients are getting an active treatment, I ask that they don’t take it antioxidant.
And they can resume that in the future in the hopes of preventing another cancer. But the time to prevent with an antioxidant isn’t appropriate when you’re dealing with an active cancer. So, that’s just one example.
Ross:
Fatigue could be a symptom of AML, but there are a lot of causes of fatigue.
How do you differentiate between something that really could be AML and something that isn’t?
Dr. Pollyea:
Yeah. That’s a challenge because I think these are, as I said, older patients. And older patients have a lot of other medical problems. And older people get fatigued, just that’s unfortunately part of the normal aging process. So, we would usually make an assumption that a person’s fatigue and diagnosis is due to the leukemia, the anemia as a result of the leukemia.
But as we successfully treat a patient if they are responding based on their numbers and other objective criteria, but the fatigue is not improving then I think that’s where we would start to look at other contributing factors, and there can be many, so having an open mind at that point is important.
But at the beginning, this is such a monster of a disease, it’s so overwhelming, I think the focus is usually on assumption that the fatigue is due to the disease or to a treatment associated with this disease.
Ross:
This question: is loss of appetite a symptom of AML?
Dr. Pollyea:
Yeah. I definitely see that, hear that, so sometimes people come in and they say that. Sometimes it may not be a loss of appetite, but an extreme weight loss, so a lot of different types of cancer, including AML, can cause that, just basically unintentional weight loss.
A person’s not trying to lose weight. They’re eating what they think is their normal amount and they’re losing tremendous amounts of weight. So, those are both potential presenting symptoms with AML. And loss of appetite, unfortunately, can be associated with some of the treatments for this disease. And taste changes, things not tasting good, can all contribute to that as well, so those are all challenges that our patients face.
Ross:
How important is to get a second opinion? I mean, are all doctors like you pretty much on the same page when it comes to symptoms and treatment?
Dr. Pollyea:
So, this is a challenge. So, the answer to the second question first is unfortunately, no. A lot of this hasn’t quite been standardized. And some doctors, oncologists, cancer doctors, they’ll predominantly be treating the things that are common: colon cancer, breast cancer, prostate cancer. And they will probably only have a few cases of acute leukemia a year.
And so, their approach to this is going to be different than somebody who spends all day seeing patients with AML and thinking about AML.
So, a second opinion is a very nice thing to be able to do. The problem with this disease is that most times it doesn’t afford that opportunity. So, with other conditions you have some time to go out, read about it, talk to some different doctors, get a good plan together.
With AML, often that’s not a possibility. A person is so urgently sick that you have to sorta deal with the resources where you are. The best recommendation I have there, if you do find yourself in a situation where there’s not a lot of expertise is to ask your doctor to just call somebody in the region or email somebody in the region who may have that expertise.
And most doctors all over the country have that sort of resource or partner that they will go to and talk the case through with them, and maybe a transfer to one of those high-volume centers is appropriate.
And maybe that’s not a possibility or appropriate, but maybe you would benefit from just talking… Maybe your doctor would benefit from talking this through. But in cases where it’s not such a dramatic presentation, then yeah, for sure, I think a second opinion can be appropriate. But this isn’t something that can be sort of drawn out for long period of time.
Ross:
You know, when you find out something like this, your tendency might be to jump on the web and start searching for AML. How do you vet those sources that you look at? How do you figure out that their – what would be a sign that they’re bogus sources?
Dr. Pollyea:
Yeah. I mean, I think this field is so rapidly changing and the treatment that we have, that I would, for the most part, assume that what you’re finding on the web is not relevant and is not an up-to-date resource. So, the resources that I listed, the NCCN, UpToDate, the Leukemia & Lymphoma Society, I should mention.
A very important resource that has up-to-date information, and they have even phone numbers for patients and their families to call to get connected with the proper people in a particular city, so that is a really important resource. But I’d be really, really cautious about what you find on the internet because things are changing so fast in this field. There’s a lot of outdated and misinformation on the internet.
Ross:
Well, then there’s outright scams. One of the things you mentioned before we went on is be cautious if someone’s asking you to put money upfront, or if it’s a nonmedical facility. What are some things that people should watch out for?
Dr. Pollyea:
Yeah. So, one of the things that is so important in our area is clinical trials and participating in clinical trials. Patients who opt to do this and receive experimental therapies can sometimes get the treatment of the future, get a drug that’s not currently available through the FDA, but may have a lot of promise.
And this is the way that we fight this disease. We’ve recently had an onslaught of approvals for AML and that’s because the patients being willing to participate in sanctioned clinical trials. So, participating in a sanctioned clinical trial is crucial, and it’s always a recommendation of all leukemia doctors.
When you participate in a conventional clinical trial, you’re asked to sign a consent form that explains what you’re doing and why. There is a confirmation that this has been vetted by an institution’s regulatory board that is prioritizing the safety and well-being of you, the patient. This has been approved by the FDA as a clinical trial. Nobody would ever ask you to pay money. That’s not ethical to participate in a clinical trial. Insurance covers whatever standard of care. And the clinical trial covers anything that isn’t.
So, if you find yourself in a situation where you’re not being asked to sign a consent form, where a clinical trial has not been reviewed by a regulatory board, where your doctor is not a leukemia specialist, where the FDA has not sanctioned the treatment, all of those are alarm signs.
Because there are people out there that are preying on patients in a desperate situation, a very difficult time in their life, and giving them sort of false hope and leading them down paths that are not legitimate.
One easy thing to do to sorta check to see if a clinical trial is legitimate is to go onto clinicaltrials.gov.
This is a resource set up by our national healthcare system that now feeds in every legitimate clinical trial from all over the world, needs to be registered on clinicaltrials.gov. So, if you can’t find your clinical trial on clinicaltrials.gov, I would have a lot skepticism and caution about that.
Ross:
Like what advice do you have for people when they’re first diagnosed? What are the first things they should try to do?
Dr. Pollyea:
Yeah. I mean, that reaction is totally normal and natural. I mean, many times these people are perfectly healthy or have been perfectly healthy, and this news is a complete shock.
And so, it is normal and appropriate to have some period of grieving for the healthy life that you are losing. But I would also, while giving yourself that time to grieve, first, draw on your support system, your family, your friends. Allow them to help you. Accept that assistance that they have. And to be optimistic because we are getting so much better at treating this disease.
I had mentioned before, there has been an onslaught of approvals for drugs in this area the likes of which hasn’t been seen in decades. We have new tools and weapons in our arsenal that we couldn’t have dreamed of even a few years ago.
We in our community are very excited and hopeful about the future and we hope that that will translate ultimately to patients, but being depressed or being down, being scared, all of that is normal.
All of that is expected. Anyone would feel like that. Allowing yourself to have those feelings and emotions is important, as long as it doesn’t get in the way of doing what you need to do to fight this disease.
Ross:
It sounds like you’re hopeful about new treatments for the disease. How about a cure? What’s the science? What’s the medical science say about that? Are we getting any closer to that?
Dr. Pollyea:
We are getting closer to curing this in more cases. So, like I mentioned before, as bad as this is, we can already cure some subsets of patients. There’s one type of Acute Myeloid Leukemia called Acute Promyelocytic Leukemia, APL. It’s an uncommon form of AML, less than 10 percent.
But we can cure close to 99 percent of people with APL. And APL, 15 years ago, was universally the worst form of acute leukemia to get. So, that dramatic 180 that we’ve seen in APL, we are hoping to translate into other forms of AML.
Some other forms of AML have cure rates as high as 50 percent, 60 percent, 70 percent in the right setting. Sometimes we can cure patients with a stem cell transplant fairly reliably. So, we are very, very hopeful about our ability to continue to make progress and cure more and more and more of these patients. That’s the future that we see.
Ross:
Dr. Pollyea, thank you so much. And thank you so much for ending on such a positive note. We really appreciate it. And thank you for joining us for this program today.
To learn more about AML and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Ross Reynolds. Thanks for joining us.
https://powerfulpatients.org/wp-content/uploads/Pollyea-1.png600600Kara Rayburnhttps://powerfulpatients.org/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-06-05 15:38:122024-09-05 14:51:22Fact or Fiction? AML Causes & Symptoms
