MPN Specialized Care and Technology: Digital Health and Symptom Management
MPN Specialized Care and Technology: Digital Health and Symptom Management from Patient Empowerment Network on Vimeo.
Nearly 80% of patients living with a myeloproliferative neoplasm (MPN) are affected by fatigue. Can digital health alleviate symptom burden in MPN care? What exactly is mobile app intervention, and how can it help me? Dr. Krisstina Gowin and Dr. AnaMaria Lopez discuss technological interventions in MPN symptom management, telemedicine limitations and the importance of connecting with an MPN specialist.
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Transcript:
Lisa Hatfield:
Welcome to this Patient Empowerment Network Program, I’m your host Lisa Hatfield. In this unique program, we explore cancer care and technology, specifically the importance of specialized care in myeloproliferative neoplasms, MPNs for short, and the role of digital health in symptom management. Today, I’m joined by two incredible experts, Dr. Krisstina Gowin is a hematologist, oncologist treating MPNs. I’m gonna shorten that, simplify it, treating MPNs. Dr. Gowin is Assistant Professor of Medicine at the University of Arizona. And Dr. Gowin it’s such a pleasure to connect with you.
Dr. Krisstina Gowin:
Oh, it’s such an honor to be on today and connect with you all. Thank you so much for having me.
Lisa Hatfield:
Also joining us is respected oncologist, Dr AnaMaria Lopez, Professor of Medical Oncology at Sidney Kimmel Cancer Centre, Thomas Jefferson University. Dr Lopez is a telemedicine pioneer as the founding medical director of the Arizona Telemedicine Program. Welcome, Dr Lopez, thank you for being here.
Dr. AnaMaria Lopez:
Thank you so much. So looking forward to the discussion.
Lisa Hatfield: Thank you. So, Dr Lopez, I’d like to start with you to talk about cancer care and technology and how far we’ve come with technology and cancer care. We’ve made a fair number of strides in cancer care as they relate to digital health. Can you speak to that a bit?
Dr. AnaMaria Lopez:
Sure. I always say that the COVID pandemic allowed us to advance telehealth in a couple of months what probably would have taken 70 years. So we went from maybe 10% of the visits being done through tele to 90% plus. So a huge, huge change, and a lot of lessons learned, both in the, how do we do it clinically as well as how do we integrate this? How do we integrate this into clinical trials and to move beyond? We really thought telemedicine and really thinking telehealth because there are so many technologies, so there could be monitors, there could be… A lot of us were doing work in patient-recorded outcomes, how do you integrate that? How do you make it easy for patients to use this? And maybe it’s not simply the patient, but it’s also the patient and the caregiver who can help with this reporting. What are really the implementation efforts that need to be done? I could go on and on because there are so many lessons learned and it really shook things up. So people are thinking of this as a new technological revolution. So technology plays a big role in care and certainly a very big role in cancer care.
Lisa Hatfield:
And just out of curiosity, Dr Lopez, do you have a lot of your patients who still continue to see you via digital health or telehealth, who prefer that?
Dr. Krisstina Gowin:
Yes. Yeah. For example, in some of the psychiatry literature, which I think is a little bit unexpected ’cause you think psychiatry is such an intimate interaction. Well a lot of patients actually feel safer when it’s digital, when it’s through tele. So yeah, I do. And we were talking earlier about doing integrative medicine, and almost all of my integrative medicine patients, we do at a distance. I really think of it as it’s a way to bring back the house call.
Lisa Hatfield:
Yes. Well, thank you for that overview, Dr Lopez. So the pandemic has resulted in significant changes to many aspects of daily living for many of us, but for patients living with cancer like myself, there are different realities that we’ve had to deal with. Do we go in for our monthly blood draws, or do we wait a couple of months? So question for Dr Gowin, can you give us an overview of the impact that Covid-19 has had on MPNs or MPN care?
Dr. Krisstina Gowin:
Absolutely. Well, there was a really wonderful study that was done, really led out of Mayo, by Jeanne Palmer and Ruben Mesa, and it was an international study, and it looked at 1500 MPN patients. And they asked questions like, how many of you are actually having telemedicine? And this was in 2020, kind of at the beginning. And over half of them had already been engaging in telemedicine. And about a quarter of them felt that their care actually was delayed a little bit and that there were actually consequences to that delay, so that really speaks to an international kind of change in the paradigm of how we’re delivering care for MPN patients. The other thing is the lockdowns, the lockdowns that were occurring for us here in the US and really internationally. And what they did is, they asked patients their MPN symptom burden, and those that were on lockdown, not surprisingly I think to all of us, had a significantly higher symptom burden.
So I think that really speaks to that A, yes, there was a very large impact of COVID on the development of telemedicine and the need for telemedicine. But it also underscores the need for symptom management that we now have a group of patients that are having a higher symptom burden, probably likely secondary to more sedentary behavior, more anxiety, more depression, but a higher symptom burden because of COVID. And so we really need not only more therapeutics and perhaps non-pharmacologic interventions to support their symptom burden, but it needs to be delivered on a digital platform.
Lisa Hatfield:
Thank you for that, Dr. Gowin. So you brought up a really good point, and this is a great segue to talk about integrative health. 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 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 wanna 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% 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:
Thank you for that. Dr. Gowin. So you talked about an app that patients can use. Is this app accessible to any patient or is it just within a trial or a study that you’ve done?
Dr. Krisstina Gowin:
No, it is widely accessible. It is free, even better. [laughter], it’s called my Wellness Coach. And that’s…
Lisa Hatfield:
I’m writing that down. Okay. [laughter]
Dr. Krisstina Gowin:
Yeah. Yeah. It’s really wonderful. Many domains of wellness. It’s based on motivational interviewing and smart goals. It gives you little reminders of, hey, and you set your own goals, which is wonderful.
Lisa Hatfield:
And what types of interventions then does that contain? Does it have things like you said, meditation and does it have a yoga program for patients? Or what types of interventions?
Dr. Krisstina Gowin:
Not quite yet. I think that that’s what we aspire to is really this multidimensional intervention. It’s not really an intervention. It’s looking at your life. It’s saying, “what are you eating? What are your nutrition goals? How are you moving? What are your exercise goals?”
How is your resiliency? How is your spiritual health? How are your relationships? And so it’s asking all of these domains of what our wellness is and helps to identify where perhaps you would like to devote more time and energy to, and also gives some resources and education around each of those domains and why they’re so important. But you set your own goals and then…
Lisa Hatfield:
That’s great.
Dr. Krisstina Gowin:
You’re accountable for your goals.
Lisa Hatfield:
Great. Well, thank you so much for that information. And you mentioned that Dr. Lopez also does her integrative health via telemedicine. So I’m gonna 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’s so many ways to engage and interact, but yes, I think like a lot of medicine, it’s a team-based approach.
Lisa Hatfield:
Great, thank you. Dr. Lopez. I do have to say with acupuncture, one of the side effects of a lot of the cancer medications is neuropathy. And a lot of patients like myself try every option, every pharmacological option, whether it’s gabapentin or something else, and they don’t work an acupuncture for me anyway, personally. I had a significant reduction in the painful neuropathy… Not so much in the tingling, but a significant reduction that, so I am very strong advocate for integrative health. So thank you for explaining that a bit more and as long as we have you on Dr. Lopez, can you also speak more broadly to innovative telehealth tools that are making an impact on symptom management and overall cancer care?
Dr. AnaMaria Lopez:
Sure. So one of the things that we know, is that for example, people have appointments every three weeks, or they have appointments once a month with the oncologist, and a lot can happen in that time. So setting up systems that are assisted by technology, so that patients can report their symptoms in real time can be very helpful. And some of this may require… It may not be a common way where the person may be familiar going to a computer or going to their phone to kind of say, “This is how I’m feeling.” So that may require some engagement education, but often regardless of age, regardless of background, people find that really easy and find that so helpful to be able to say, “Oh, was it two weeks ago that I had that?” As opposed to just saying, “Hey, I just had this,” and then it can happen anytime day or night that the patient can report. And that way there’s… It’s just so helpful to have an intervention in real time.
The other part that’s good is that often some of these systems can kind of track. So we can look at it together and say, “You know what? Your fatigue tends to be a couple of weeks after therapy, so how can we either prepare for that?” Or just to have the reassurance that, “Yes you have that depth, but it gets better and you get through it.” So being able to look retrospectively and identify that can be helpful and I think also just the ease for people to be able to connect with multiple specialists, sometimes to have multidisciplinary visits where not only does the patient meet with everyone, but the patient can see that we are all meeting and interacting together. So all of those are incredible tools, one of my favorites though, one of my favorites is patients who are in the hospital and patients who are in the hospital a long time, on some occasions. So and even if a person’s not there a long time, it can feel like a long time, so to use the technology, not just to connect the patient, the healthcare team, but to use the technology to connect the patient with his or her family. And I think especially… I mean, a lot of people have smartphones, but it’s using your minutes, sometimes the internet may not be so strong. So to use the technology that would be used for the clinical piece to have that available in the inpatient setting so that patients can feel connected.
Lisa Hatfield:
Yeah, that’s a really great thought that you brought up, too. I know when the pandemic was in full swing, but patients were starting to go back into the office to see their provider. For me, I was not allowed to take my husband in with me, so I went in alone. I was far enough along in my journey. I didn’t necessarily need a care partner with me, but some patients do, maybe a newly diagnosed patient. So that is a really great point. Say, a patient has to come in by himself or herself, is that a technology they can use? Are you willing to let them use their phone to maybe FaceTime during that call or we had to use the actual physical landline because my phone did not connect, the signal wasn’t strong enough. But do you allow that during your appointments to have patients contact somebody?
Dr. AnaMaria Lopez:
Absolutely.
Lisa Hatfield:
Okay. That’s great. Yeah.
Dr. AnaMaria Lopez:
And also there’s pandemic, but there’s also… People live everywhere. So you could say their sun could be in California and I’m in Philadelphia and this way it’s okay, we’ll just beam them in.
Lisa Hatfield:
Yes. Well, thank you for that information. And some patients might be a little more reluctant to use telehealth or telemedicine. How can patients and their care partners feel more confident in voicing their concerns or communicating with their healthcare teams regarding any telemedicine options that are out there?
Dr. AnaMaria Lopez:
So you mentioned that I had been the founding medical director of the Arizona Telemedicine Program, and it was such a wonderful experience because skepticism and I really respected that. It was brand new and we had our system in the library. And the library, it was down in the basement, so it was very metaphorical. I would meet the new clinician at the entrance of the library. We would walk down the stairs together and often, the conversation was, “Okay, I’m doing this for you. I’m doing it one time. We’ll see how it goes.” And I was always so reassuring that if for some reason,’cause ultimately the clinician needs to feel comfortable, yes, this works, or no, it doesn’t. And if you have any doubt and you feel that you need to see the patient in person, you just need to say that, I need to see the patient in person. And inevitably as we’re walking up the stairs, “Oh, I know you called me because I was on call. Just call me anytime. Don’t call the on-call person”. This was great. I loved it.
So inevitably, people really like it and it’s good. You see the patient in their own environment, you can interact. You often get insights that you may not have gotten otherwise just because of where you are and how comfortable they feel in their own space. So I think, for me it’s the proofs in the pudding. Give people the opportunity, have the right supports and technology in place and often it’s a very positive experience.
Lisa Hatfield:
Great. Thank you Dr. Lopez. So Dr. Gowin, a couple of questions for you. Is technology playing a role in accelerating progress in MPN care?
Dr. Krisstina Gowin:
Oh, absolutely. And I think some of the ways that it really accelerates progress is pulling us together. So what we need to recognize is that myeloproliferative neoplasms truly is a rare disease and we just celebrated Rare Disease day. But there’s a lot of challenge in treating patients and progressing the field forward in rare diseases because you can’t do the big clinical trials. It’s hard to come together ’cause everything’s siloed and there’s just a couple patients here, a couple patients there in each practice. But with digital health and clinical trials that are offered on a digital platform, it pulls the nation together and even the world together. And we’ve seen that. I’ve done a international survey-based analysis and I had 858 MPN patients from 52 countries participate in that survey. And so that just shows how it pulls the world together. And for the web app that we just discussed, we had 93 patients say they were interested within three weeks, and within actually a week, we identified them all and then took three weeks to actually accrue them to the trial. So it really speaks to A, how MPN patients are digitally engaged and excited about these kinds of platforms. And then B, how effective it really can be to pull the groups together.
So yes, I think it’s… And that’s really how we’re gonna get progress is through these kind of interventions with a rare disease. And I hope it’s okay if we jump back to something you said, Dr. Lopez, which is, I think telemedicine is so so important to bring everyone together. And in particular, I see that on the transplant ward. And so in myelofibrosis, that’s the only curative therapy. And so many myelofibrosis patients actually go through allogeneic stem cell transplantation. And my goodness, that is a socially isolating experience. Patients are in the hospital, not uncommonly for at least 30 days and then have to be near their transplant center for three months, which often is away from home. So to pull in their support system, both through the acuity of the transplant themselves and then the couple of months after is so crucial to a successful transplantation. And I think through FaceTime and also the MPN support groups, which is very robust, the patient advocacy and the way the MPN network sticks together on a digital platform, I think is really unique and offers unique support.
Lisa Hatfield:
Thank you. And then what role does technology play in the disease symptom management, and in particular, in clinical trials too. What role does technology play with clinical trials?
Dr. Krisstina Gowin:
Well, I think it helps us through different, clinical trial accrual patterns, we can see who’s eligible where, so it helps us identify patients. It helps us to, understand the different kind of precision based medicine approaches so we can start to pool the data, say for, particular mutations… ASXL1 mutations. And so it helps us in the precision medicine aspect of clinical trials and now we’re looking at symptom management and how do we really integrate that. So large survivorship platforms like Carevive, if you’ve ever heard of Carevive, is now integrating our validated symptom assessment form into the Carevive platform. So now we can really collect that data and use that to mine it for potential kind of retrospective analysis. So it’s helpful for clinical trials as well as for our clinicians and clinics to really identify changes in symptom burden.
And just as Dr. Lopez was mentioning, that we can track these over time and it can flag and say, “Oh, your symptoms are changing, they’re increasing over time,” and maybe we need to be thinking about that. And so Carevive is really kind of a electronic medical record driven it’s really a healthcare driven platform, but now there’s patient ones too. And I just learned about this two weeks ago, I was at an MPN conference in Phoenix and learned about MPN Genie. And so MPN Genie apparently is tracking… Patients are putting their symptoms in and that’s shooting that information to the electronic medical record to their doctors. And so I think that’s fantastic, ’cause, we now get that information real time and we can change our clinical management, maybe bring that patient in sooner, maybe do a bone marrow earlier. We never would’ve identified that if it weren’t for those kind of digital engagements, so I think it’s a really exciting time. And I think we’re gonna see more and more of these new platforms and ways for different EMRs and smartphones to be communicating back and forth between patients and providers.
Lisa Hatfield:
Great. That MPN Genie is fascinating to me that we can have that real-time communication going back and forth as a patient, I would love that. So going back to clinical trials, I’m curious if you think that, technology has progressed enough, so in the past… Say I’m an MPN patient or a myeloma patient, I see a clinical trial or hear about one, I have to be onsite for that clinical trial for monitoring maybe for six months to a year. Do you think that technology has progressed enough that clinical trials might allow a patient to be at home, maybe in a more remote area and monitored remotely? Whereas in the past, that same clinical trial required them to be at the facility? Do you think that we’ve progressed to that point in some clinical trials?
Dr. Krisstina Gowin:
You bet. Yeah. I think COVID out of necessity has forced us to do that. And I have in my own clinical trials, even with pharmacologic clinical trials conducted telemedicine visits that were approved by the sponsor. So the paradigm is shifting, and particularly when it is oral therapeutics, I think that’s really accessible when they’re, IV subcutaneous, I think that has different challenges. Obviously you can’t do that as remote, but when they’re oral therapeutics are non-pharmacologic intervention, such as our integrative interventions, I think it really lands well to a more remote experience.
Lisa Hatfield:
Okay. And then would that require communication between the local oncologist and maybe someone like yourself, the investigator on that clinical trial to know what is going on with that particular patient? I assume that that communication would be ongoing?
Dr. Krisstina Gowin:
Absolutely. Always.
Lisa Hatfield:
Okay. Yeah. All right, great. Well, thank you for that information. So, Dr. Lopez, kind of a similar question for you. What are some examples of how technology is influencing cancer care right now?
Dr. AnaMaria Lopez:
Yeah, let me just add on the clinical trial question.
Lisa Hatfield:
Oh, Yes.
Dr. AnaMaria Lopez:
That there’s also the opportunity. Again, there were so many things that we thought, “Oh no, we just can’t be done.” But because of the necessity, necessity is the mother of invention, we do remote consent, so that was a big deal in the past. We can also do a tele visit ahead of the appointment, and screen for the cancer clinical trials, people travel large distances for studies and instead of traveling four or five hours, and then to be told, “Oh, actually you don’t meet the criteria.” To be able to do all of that at a distance, to get the records, to get the images, to review all that needs to be reviewed. And then to say yes, and not only yes, but we can also do your consent at a distance in some situations.
And then when you come, there’s actually the more substantive, perhaps even the treatment. There’s also a large, movement around hospital at home and that these patients that are eligible for that would be able… With digital support, be able to get hospital level care in some cases at home. So some of that may involve infusion, some of that… Again, but that visual connectivity and in the past you really had to kind of conceptualize it and it was kind of space aging to talk about it. But we now, we’ve all done FaceTime, so I think we all really can understand what it entails, so tremendous shifts and, we wanna try to keep that momentum going for our patients. So, I do think that, there’s so many ways that technology has impacted cancer care, even when we talk about the electronic record and patients accessing the electronic record and patients having the opportunity to go into a portal and to see their labs, to see their reports…
To be able to track their changes. All of that is really, really powerful. You know, patients with… The most common I think is patients with diabetes who track their blood sugar sometimes to the minute and they can say, “Oh, I ate that and now I see the impact.” So the opportunity for monitoring, the opportunity for also bringing in experts. So let’s say there’s a patient with a rare disease and the expert is elsewhere, there might be the opportunity to bring people together. We do tumor boards. That’s just part of what we do in cancer care. And also as many… There are health systems now so that it’s not one hospital, it’s multiple hospitals together where we can bring all of those folks together, bring in local expertise, regional expertise, national expertise, all for the patient’s benefit.
So there are so many ways that technology even something as simple as the note. Now this is something we experimented with and it’s still in experimentation phase, but there were these Google classes where you could interact with the patient and as I’m talking, the Google glass would record kind of the conversation and would come up with some sort of a structure for the note. So for what that encounter had been like. So there are lots of ways of how do you capture natural language in real time to really help the workflow, the documentation process. So I think there’s aspects to help the patient, to help the families, to help the clinical teams and to help everybody work together.
Lisa Hatfield:
Great. Thank you. And you talked about the patient portal and I’m one of those patients at fault of seeing a lab result before my doctor saw it and calling him or sending a message via MyChart saying, “Hey, this is going up. What’s wrong with this?” So I’m sure you don’t have to mention any names. I’m sure you’ve seen the challenges of, digital health too, are having that patient portable or portal accessible to patients. So anyway, just wanted to throw that out there that I’m sure that brings challenges to you. Also few little challenges here and there.
Dr. AnaMaria Lopez:
But at the same time, that’s so good, right? It’s so good that patients are engaged. It’s so good that you’re engaged. And I think as long as, we’re communicating that yes, you may see this before me, so you may have questions and then, we just get together and answer the questions.
Lisa Hatfield:
And thank you for saying that Dr. Lopez, because a lot of us patients who do that occasionally feel a little bit guilty for sending a note right away to our doctors. “We know you’re busy. We know you’ve already, you’ll look at those labs. If you’re concerned, you’ll call us or let us know”. And sometimes we jump the gun a little bit. So thank you for reassuring us that that’s okay, that that’s okay to do that. So we appreciate that. So Dr. Gowin, do you have anything to add on, how MPN care or just cancer care in general could change with different technologies? We didn’t touch a lot on things like artificial intelligence and that type of thing, and we can speak to that or, any other type of technology that you’re familiar with.
Dr. Krisstina Gowin:
Well, I think the artificial intelligence aspect is really going to change the paradigm again on how we’re designing, studies. And I think one of the biggest challenges that we have in myeloma and as well as myeloproliferative neoplasms, is to think about how do we optimally sequence our therapies to achieve best survival, right? And I think this is a wonderful problem to have. We have now not only one JAK inhibitor on the market, but several and more in the pipeline and several other therapeutic targets. And so now the question is which therapy and when do we employ it? So things like artificial intelligence will help us to answer that question with machine learning decision tree analysis, all of that is going to be answered through those kind of platforms. And so I think that is going to be a shift we will see in the next five years is many different machine-based learning algorithms to better understand those problems we cannot have tackled traditionally otherwise.
Sensors though is another one, right? And so a big thing in MPNs is not only addressing the blood counts and reducing risk of thrombosis, and to address symptom burden, but it’s really addressing lifestyle because it’s things like cardiovascular disease, stroke that really we’re worried about as some of the sequelae of having the disease and what we’re trying to prevent with therapeutics. And so even going back to this NCCN guidelines, it’s addressing cardiovascular risk factors as part of our core treatment goals. And so how do we really do that? And it’s really through lifestyle medicine and that’s where the sensors come in. And so now we have, these Fitbits and smartphones that connect to our Apple watches and we have Garmins and all these wonderful devices that are prompting us to move more, prompting us to be cognizant of our heart rate and stress response prompting us to meditate. And so I can envision those evolving over time and connecting to the EMR and being very seamlessly interwoven into our clinical trials. And we’re already doing that. In fact, we’re talking about doing one very soon in MPN patients. And so I think the sensors are gonna be another big way that we’re going to be integrating, into our clinical trials and symptom management tools.
Lisa Hatfield:
That’s fascinating. Thank you for that. And Dr. Lopez, do you have anything to add about other technologies and how they may affect cancer care in the future?
Dr. AnaMaria Lopez:
Sure. When Dr. Gowin mentioned the sensors, it just reminded me, we’re building this new building, patient care building and oncology will be there. And I did a tour recently, and we’re used to going to the doctor, you stop in, they get your blood pressure, they get your weight, et cetera. Here, you’ll walk in directly to your exam room and you check in at a kiosk, so you just kinda check in [chuckle] with a little robot kiosk, and then it’ll tell you where you’re going. You’ll go to Room 3, let’s say, and Room 3 will say, “Welcome, Lisa.” [chuckle] And so you know that you’re in the right place. And you’ll walk in, there’s your gown, you’ll sit in the exam chair, and the exam chair automatically is gonna take your vital signs. So it just seems, really these built-in aspects to the technology. And one of the things, again, what I just love about this work is that it’s a very interdisciplinary, multidisciplinary. And one of the projects that we were working on, which it ties into this, when I was in Arizona with the telemedicine program is we worked with the College of Architecture and with this concept of smart buildings.
So it’s kinda like that. Why should you do these different sensors that detect, but that it could also detect. You might walk into the room and you might be really nervous as you might be really cold, and it would detect that and it would warm the room for you. Or you might be coming in and be having hot flashes and it would just cool the room for you. So the technology has so much potential to really improve the patient experience.
Lisa Hatfield:
And that’s amazing to me. I think that would be incredible to walk into a building to have that experience, as long as it doesn’t take away the compassion and care I get from my providers. I am so fortunate to have extraordinary providers, so I don’t think it will ever take over that aspect of it, I think that is a fear people have, especially with artificial intelligence and that type of thing, I think it can only go so far. Can’t provide the humanness that’s required for patient care, so yeah.
Dr. AnaMaria Lopez:
Yeah. These are tools.
Lisa Hatfield:
Yes, that it. Great, well, thank you very much. Dr. Gowin, can you provide or share some examples of how telemedicine is influencing personalized medicine and MPN care, and how can MPN patients best advocate for themselves to get the latest in MPN care?
Dr. Krisstina Gowin:
Well, I think it’s going back to some of the conversations we’ve already had, is that now with telemedicine, you can really access academic centers no matter where you are. And so rural areas now can go to academic centers, very accessible without travel, and so what that lends to is more access to precision-based clinical trials, and very often now we’re doing next generation sequencing panels for patients with MPN. We’re looking at what are the genetic features of the disease and we may be accruing trials based on those genetic features. And so that kind of conversation really only happens at academic centers, and so I think it’s really allowing those that live far away, a few hours away, to really have those personalized and precision-based conversations. And then tying in again the aspect of integrative medicine. And then what is integrative medicine all about is personalizing your treatment plan, asking what are your goals, what is your lifestyle, what is your culture, and how do we really get you on a plan that makes sense for you, that is local for you and sustainable for you to really achieve your optimum wellness?
And so if I were counseling patients listening to this, I would say, start with the in-state academic centers and say, “What are the telemedicine services there? Is there an integrative medicine department there”? And then get a quarterback within that department and say, “Okay, this is the plan”, and then that quarterback can say, “Well, now let’s look local. What do you have? What are your resources there? Let me do some homework with you and hook you up with really evidence-based high quality providers to achieve your personalized needs in your local community”. And I think that’s how we’re really going to get all of our patients in a precision and personalized approach no matter where they live, and that’s again, the beauty of telemedicine and digital health.
Lisa Hatfield:
Great, thank you, Dr. Gowin. And I know we’ve spoken, both you and Dr. Lopez have spoken to all of the rewards of telehealth. Are there any risks or drawbacks that you see to telehealth or telemedicine for digital health?
Dr. AnaMaria Lopez:
The most important thing is to remember that the technology is a tool, and if the person feels that there’s a limitation, so for example, if the patient is seen and they say their heart is racing or skipping beats or something, now, there are ways, there are electronic stethoscope, so you can really do a full exam except for palpation through telemedicine. But not everybody has that even in a clinic, but certainly in our own home, we don’t have that technology. So if a patient is expressing a concern for which the clinician really feels that needs a closer evaluation, then that’s the right next step, so we’re not… The technology is a tool to help us care for people, and if it’s not all available right there, then we need to see the patient in person. So I think that’s the risk is just sometimes people may feel limited like, “Oh, well, I’m not really sure.” It’s okay, I’m not really sure I need to see you, or you need to go here or go there for the care.
And the other, which is a really big threat, is that part of the reason we did 70 years work in a couple months is because it was reimbursed and we’re reaching the end of the pandemic, the federal… And with that, the payers may go backwards. We all know that if that happens, we will go backwards in telemedicine. [chuckle] There will just be decreased, decrease use. And it may lead to people then going back to traveling four hours, waiting, only to be told, “Oh, you know what? There’s not this. This clinical trial doesn’t work for you.” So we don’t want to lose ground. And part of not losing ground is that we really need to continue to have advocacy around reimbursement.
Lisa Hatfield:
Thank you, Dr. Lopez. And I feel compelled, just to follow up with one more question regarding that, because I’m very passionate about this. With some of these rules and guidelines coming to an end, I know in my particular state that I will no longer be able to access my specialist. I see a myeloma specialist. We do not have any here locally. I can access a specialist via telemedicine. I will not have that opportunity. So as all of us know, there are disparities and there are financial disparities in cancer patients. There are racial disparities in cancer patients, there are socioeconomic disparities. Telemedicine has been a tremendous… Has had a tremendous impact on the care and the outcomes and the quality of life of so many patients. So as a patient and as an advocate, do you have any recommendations? Do I go to my doctor and say, “Okay, how can I move forward and still talk to my specialist, who’s out of state? Do I go to my state legislature? Do I talk to my insurance company? How can we get this to continue?” Because this has had such a significant impact on the quality of life and on the outcomes for patients, who otherwise, would not have been able to access that care.
Dr. AnaMaria Lopez:
Yeah, I mean, I think all of the above. Partnering with other advocates, the American Telemedicine Association has a map that kind of says where are all the shifting sands regarding the different rules and legislative changes. But I think it’s led us to a place, where we are all advocates and where physicians, nurses, patients, pharmacists, everybody in the same way that we do team-based care, that we do team-based advocacy and it’s all for our patients.
Lisa Hatfield:
Great. Thank you for that. Dr. Gowin, any last words that you may have about accessing specialists or telemedicine options?
Dr. Krisstina Gowin:
Well, I think we covered the basics, but I just wanna end with just how empowering the access to digital health interventions really is. And so I don’t think there is a one size fits all approach to every patient. So what I would encourage patients to do is just to really think, “How do I compliment my care? What am I missing? How do I achieve my best wellness? And how do I get those resources in my home to make them more convenient for me?” And to start doing some research and self-advocacy to really get those resources ’cause they are out there and in almost… In every domain, there is now a digital version that is accessible to you now.
Lisa Hatfield:
Thank you for that. So it is time to wrap up our program. I could ask you many more questions. From a patient perspective, it has been so refreshing to take a minute to understand how far we’ve come and to have a look at the exciting innovations ahead. As always, we appreciate all the new tools being added to the toolbox, and I am eternally grateful for Dr. Gowin and Dr. Lopez and all providers who are willing to come on these webinars and answer questions from patients. It is so empowering to us, and we’re so appreciative of your time and your energy and your expertise. So thank you so much for being here today. And just a reminder to all patients, to always consult with your medical team about what is right for you. Thank you again so much to Dr. Lopez and Dr. Gowin for joining us for this Patient Empowerment Network program. I’m Lisa Hatfield. Thank you.
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Managing Life With an MPN | What You Need to Know
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Transcript:
Katherine Banwell:
Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today’s webinar is a continuation of our Thrive series. And we’re going to discuss how to manage life with an MPN. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you.
Let’s meet our guest today. Joining me is Dr. Raajit Rampal. Dr. Rampal, welcome. Would you please introduce yourself.
Dr. Raajit Rampal:
Hi. Thank you so much for having me. I’m Raajit Rampal from Memorial Sloan Kettering Cancer Center where I focus on myeloproliferative neoplasms.
Katherine Banwell:
Thank you so much for being with us today.
Dr. Raajit Rampal:
My pleasure.
Katherine Banwell:
As we do with each of the webinars in our Thrive series, let’s start with this question. In your experience, what do you think it means to thrive with an MPN?
Dr. Raajit Rampal:
It’s a great question, right. I think taking a step back, when we think about our patients with MPNs, one of the questions I always have for patients are what are your goals. And inevitably and invariably, people want two things. They want to live longer and they want to live better. And so, I think that thinking about thriving with an MPN to me is about how do we minimize the impact of an MPN in someone’s life. And that means a couple of things. One that means how do we deal with symptoms or things that are causing medical problems.
But two, how do we deal with the anxiety of a diagnosis? In many cases in my experience, that can be just as detrimental to somebody’s well-being as the actual physical symptoms of the disease.
Katherine Banwell:
When it comes to choosing therapy for polycythemia vera essential thrombocythemia, or myelofibrosis, it’s important to work with your healthcare team to identify what is going to work best for you. So, to begin, would you define shared decision making and why is this critical to properly managing life with an MPN?
Dr. Raajit Rampal:
Yeah. Shared decision-making, to me, is really about the physician or whoever is on the healthcare team providing the patient all of the information needed to make a good decision. That means what are we trying to do? What is the medication or invention going to accomplish? What are the side effects because there are always side effects.
And what do we think that’s going to do or how is that going to impact the patient’s life? Where things get nuanced is that patients come to us because we have expertise. There are two extremes. One extreme is that the physician says this is the medication you should take. End of discussion. The other extreme though is also not helpful, which is to say to a patient here are five choices. Here are the side effects. You pick one. Our job is to lay out those side effects and the benefits but then, also help guide a decision.
Katherine Banwell:
What are treatment goals and how are they determined?
Dr. Raajit Rampal:
It depends on the disease to a large extent. Now, when we’re dealing with ET and PV, the primary goal of our interventions is to reduce the risk of a clotting event or bleeding event. And that usually involves controlling the blood counts in some cases, not in all patients with ET.
Sometimes aspirin is all we do. Myelofibrosis is a little bit more complicated because it depends on what the problem is. Not all myelofibrosis patients have the same challenges. Some have anemia that needs treatment. Some have a big spleen. Some have symptoms and some have nothing and they just need observation. So, it’s a bigger list with MF patients. But I think the first part of the discussion always is defining what the goal needs to be.
Katherine Banwell:
What factors are considered when choosing therapy for ET, PV, and MF?
Dr. Raajit Rampal:
I think a couple of things. One is what medication we think is going to benefit the patient best. That has to take into account the individual, their willingness to take certain medications, for example, pills versus interferon injection. Some people have an aversion to self-injection, which we have to take that into account. What are the other medical conditions that the patient is dealing with?
And the reality is, in some cases, it’s cost because these medications, depending on a patient’s insurance, can have quite a different spread in terms of cost. Unfortunately, that is something we have to take into account.
Katherine Banwell:
Let’s talk about what sort of tests should be done following an MPN diagnosis. Can you tell me about those?
Dr. Raajit Rampal:
Yeah. Fundamental to the MPN itself, the things that we really want to know is, in most cases, a bone marrow examination is needed because that will tell us really what the disease is that we’re dealing with. It will tell us about the genetics. I strongly believe we have to be comprehensive in our genetic assessments because that does prognosticate and sometimes gives us an opportunity in terms of treatment. Chromosomal analysis. These are the basic bread and butter hematology tests we want to do from the bone marrow to really understand what the patient’s disease is.
Beyond that, I think that particularly in patients with PV and ET, it’s important that we partner with their primary care physicians to make sure that they’ve had, for example, testing for diabetes, a recent lipid profile, any cardiovascular tests, particularly measurements of blood pressure because these things are all important in terms of an ET or PV patient’s risk of having a blood clot. So, there are, again, things that are within hematology realm but then, there are other general health things that become really important in somebody who is diagnosed with PV or ET.
Katherine Banwell:
How often should lab tests of blood work be done?
Dr. Raajit Rampal:
It really depends on the patient. For some patients with PV, for example, they need to have their blood checked every three weeks because they’re having frequent phlebotomies. Whereas some patients with ET could probably go forward to six months between blood tests.
So, it depends on the individual.
Katherine Banwell:
How can results of biomarker testing affect treatment choices for patients with MPNs?
Dr. Raajit Rampal:
question. The genetics are becoming increasingly important in our treatment decisions. So, let’s take a simple example, which is patients with ET. Calreticulin and JAK2 and MPL are the three most common mutations that we see. But they have very different invocation. So, somebody could have a calreticulin-mutated ET and based on them having that calreticulin mutation and no other factors like no history of clotting, that patient may never need to go on a medication aside from aspirin. And even early on, it’s debatable whether or not some of these patients really need aspirin at all.
Whereas somebody who had a JAK-2 mutant ET, our guidelines and data suggests that that person, once they reach a certain age, should probably be on medication. So, that’s kind of perhaps one of our more clearcut examples of a genetic biomarker telling us how to approach treatment.
And then, it gets more nuanced from that and more exciting and interesting in the sense that there are mutations, for example, that occur in myelofibrosis and in patients whose disease is progressing towards leukemia, such as IDH mutations. And these are things that are now targetable with FDA-approved drugs.
And there are now clinical trials combining JAK inhibitors and IDH inhibitors for patients who have more advanced disease who have these IDH mutations. So, you go from on one end, these genomic markers being of prognostic significance and now, on the other hand, we’re getting to a point where, in some cases, they might tell us how to best treat a patient.
Katherine Banwell:
Dr. Rampal, should all patients diagnosed with MPN’s undergo molecular testing?
Dr. Raajit Rampal:
I strongly believe that. I think that we’ve learned so much that these tests have prognostic value.
And in some cases, it may suggest a slightly different diagnosis. I definitely think that should be the case.
Katherine Banwell:
What should patients be asking once they have the results?
Dr. Raajit Rampal:
What does it mean? That’s the most basic and fundamental question. It’s one thing to get a list of mutations. But the real bread and butter question is what does this mean to the disease and my prognosis and my treatment? Those are the key questions.
Katherine Banwell:
So, what are the types of treatments available for MPNs? And let’s start with myelofibrosis or MF.
Dr. Raajit Rampal:
If we had had this discussion five years ago, it would be pretty simple, and it would take a minute or two. And that’s completely changing and that’s amazing, and it’s good for all of our patients.
Right now, for patients with MF, it depends on what the issue is. If the issue is symptoms or spleen, JAK inhibitors are our first line of therapy. Three approved JAK inhibitors are currently available, two on the first side ruxolitinib (Jakafi) and fedratinib (Inrebic). And pacritinib (Vonjo) can be used for patients with really low platelet counts.
There is a fourth JAK inhibitor that we expect to be, hopefully, approved in June of this year, momelotinib. So, the landscape is about to complete broaden in terms of just JAK inhibitors.
But beyond the JAK inhibitors themselves, there are a number of late stage clinical trials that are combining JAK inhibitors with agents that work through a different mechanism that don’t work through inhibition of the JAK pathway. So far, these drugs have all shown promise in early phase trials. Now, the definitive Phase III trials are being done. We have to wait and see what the data tells us. But if these are positive trials, this could completely alter the landscape of MPN.
Katherine Banwell:
There’s also transplants available, right?
Dr. Raajit Rampal:
Correct. Transplants for more advanced patients, which comes with some major risks. And so, that has to be thought of very carefully in terms of the risks and benefit. But it is a potentially curative strategy.
Katherine Banwell:
Let’s turn to polycythemia vera or PV. What types of treatments are available?
Dr. Raajit Rampal:
It’s really quite a range. So, there are things like phlebotomy and aspirin, which has been the mainstay of therapy for many years. There are drugs like hydroxyurea (Hydrea), interferons, JAK inhibitors. So, ruxolitinib is approved in certain settings for treating polycythemia vera. So, the landscape is broad. There are a lot of questions going on right now with polycythemia vera with regards to how it should best be treated. Is the mainstay of phlebotomy and aspirin really what we should be doing or should we be giving patients treatment earlier on.
And there is some data to suggest that. There is this drug called ropeginterferon (Besremi) that’s FDA-approved for polycythemia, which was compared in the study to phlebotomy and aspirin.
And at least the data suggests that there may be better control of the disease and less progression possibly, and it’s a small number of patients, by treating patients earlier. Whereas we would have just given phlebotomy and aspirin. So, it’s something to consider. There are drugs in clinical trials as well that look promising one of which is called rusfertide, which actually works by changing the way iron is used by the body.
Iron is a key component to hemoglobin and it is, of course, a key component to polycythemia in the sense that we phlebotomize patients to make them iron deficient and that’s how we control the disease. But this is a pharmacological way to do that. So, that drug is now in Phase III trials. So, that may also alter the landscape of treatment of PV in the near future.
Katherine Banwell:
Finally, how is essential thrombocythemia treated?
Dr. Raajit Rampal:
So, in some cases, with absolutely nothing as we had talked about a moment ago. There is some thought that in really, really low-risk patients. Maybe you don’t need to do anything except observe them. Whereas most patients are on an aspirin. And beyond that, we have drugs like interferon, pegylated interferon, and hydroxyurea and anagrelide, all of which can be utilized. It’s not entirely clear if there is one distinct first line treatment that is the best but these drugs are all active. JAK inhibitors have been studied in this setting. And to date, the data hasn’t led to their approval but, certainly, people have studied it.
Katherine Banwell:
Dr. Rampal, how can you tell if a treatment is effective? Are there signs that you look for?
Dr. Raajit Rampal:
Well, I think it’s a couple of things.
One, are we meeting the treatment goals in terms of are we controlling blood counts with ET or PV? That’s one of the first principles in management. And with regards to MF, the same thing. Are patients’ symptoms being controlled? Is the spleen being adequately controlled? And then, there’s the symptom burden because just because the blood counts are being controlled, patients may still have symptoms, in which case, they are not being adequately treated. And then, we have to do our best to try to find a treatment strategy that does control their blood counts but also does control their symptoms.
So, there is the blood count perspective but there is the symptom perspective as well.
Katherine Banwell:
How do you know when it’s time to change treatments?
Dr. Raajit Rampal:
Well, I think really two things. One is if we aren’t meeting our goals like we just talked about. But the other aspect of that is if we are incurring toxicities that are just not tolerable to the patient and that’s a reason to change therapy always.
Katherine Banwell:
Many patients, of course, worry about disease progression. Are there key predictors or tests for progression that patients should know about?
Dr. Raajit Rampal:
This is a key area of investigation currently. I think one of the things that patients say to us so often when we meet them is what’s going to happen to me. And right now, we don’t have great prediction tools. We can say on a population level well, there is X percent of chance of progression at 15 years. That’s useful if you’re talking about a population. That’s not really useful if you’re talking to an individual. Because if I say to somebody there’s a 20 percent chance of your disease progressing to leukemia, it doesn’t really make a difference. That’s a meaningless statement because if you’re in the 20 percent who progress, it’s not a relevant statistic anymore.
It’s sort of a binary thing. We’ve got to do better at developing this. This is something that the MPN Research Foundation is really heavily invested in in trying to identify predictive biomarkers.
If we can do that, then perhaps what we can do is say to a patient this is really what we think your actual risk is. And then, the next step is asking the question if we intervene early, can we prevent that progression from occurring. So, that’s where I think we need to go. We aren’t there yet.
Katherine Banwell:
What signs or symptoms do you look for that may indicate that the disease is progressing?
Dr. Raajit Rampal:
The blood counts are often the canary in the coal mine regardless of the disease. They can tell us if ET or PV is progressing into MF or whether MF is progressing to more of a leukemic phase. Changes in symptoms sometimes can be a harbinger of disease progression. So, Patient 2, for example, is doing really well and now, he’s having drenching sweats and losing weight. So, those types of symptoms are a sign that physical findings is the size of the spleen if it’s increasing.
All of those things together give us a hint about progression.
Katherine Banwell:
Well, is there any way to prevent progression?
Dr. Raajit Rampal:
That is the million dollar question. Again, that’s where we ultimately need to be. We want to be able to intervene to a point where patients don’t get that sick. It would be amazing if we’d come to the point where we can intervene early and nobody progresses to late stage MF. Nobody gets leukemia. And I think that’s a worthy goal. That’s not something that we should think is too lofty of a goal. That should be our ultimate goal here. And a number of groups are investigating this exact question. It’s complicated and it’s going to take time. But I think that’s a worthwhile investment.
Katherine Banwell:
Let’s talk about MPN symptoms and treatment side effects. Here’s a question we received from a viewer before the program. How common is peripheral neuropathy in primary myelofibrosis?
And what is the best treatment for it?
Dr. Raajit Rampal:
Well, by itself, it’s not a very common symptom of MF by itself. Can it be a symptom? Sure. But there are also a number of things that can cause peripheral neuropathy. So, I’m not sure there’s a best treatment.
But what needs to be done is a thorough investigation. There can be a number of causes. It could be nerve injury. It could be a deficiency in vitamins like B12. There are a lot of things that could cause it. So, that type of a symptom needs to be thought of in a broad way in terms of diagnosis.
Katherine Banwell:
Jeff sent in this question. How could I manage the itching? Are there new treatments or strategies to live with itching?
Dr. Raajit Rampal:
Very common thing. And it’s an interesting thing explaining to when we teach our trainees about this symptom, we have to impress on them the fact that itching is not the itching that everybody else experiences.
This is a very profoundly different symptom. It’s debilitating for so many people. I have patients who go to the Emergency Room for that. That’s how terrible it could be. There are a lot of things that could be tried. JAK inhibitors, in my experience, work very well for itching but not in everybody. We use sometimes antihistamines that can work well. Sometimes, antidepressants can work well, not because they’re treating depression but because of other properties that they have. And sometimes, UV light therapy can be useful tool here, too. A lot of patients swear by it.
Katherine Banwell:
Another common side effect is fatigue. Do you have any advice for managing this symptom?
Dr. Raajit Rampal:
Fatigue is the most common symptom across MPNs. And it is also one of the most difficult things to treat. Part of the issue is trying to figure out what does fatigue mean to the patient.
When someone says they’re tired, does that mean they’re sleeping all of the time? Does that mean they don’t have get up and go? The first step is always understanding what does fatigue mean to the patient? And then, the second is trying to dissect that. In some cases, it’s related to anemia, in some cases, it’s not related to anemia and it’s just the disease itself.
And in some cases, you have to think outside of the box about general medical issues like thyroid dysfunction that could be at play here. So, there isn’t one best fit.
But the first test is always to dig deep. When someone says they have fatigue to dig deeper and try to figure out what is that really.
Katherine Banwell:
What other common symptoms do you hear about from patients? And what can be done about those?
Dr. Raajit Rampal:
There are a lot of different things. It’s a spectrum. So, I think that itching and fatigue are very common. Feeling full early is, that’s a big thing, particularly in myelofibrosis patients.
Bone pain, that’s another big one, particularly in myelofibrosis. There is not one therapy that is best for all. I think the JAK inhibitors, certainly, benefit many of these symptoms. But they don’t benefit everybody and not to the extent that makes it tolerable for everybody. So, often times, we struggle with this and try a lot of different things. But, again, I think one of the things to always remember is we don’t always want to say that this must be because of the MPN. Sometimes, symptom is arising because of another medical condition that’s going on concurrently.
Katherine Banwell:
That’s good advice. Thank you. Let’s answer a few more audience questions we received. This one is from Calvin, “If your hematologist says you’re stable and responding well to Hydrea, should you still seek out a second opinion?”
Dr. Raajit Rampal:
It’s never wrong to seek out a second opinion. I strongly believe that, especially when you’re dealing with a disease that’s rare like this.
And even seeking out a second opinion, even if you’re under the care of an expert in the field is never a wrong thing. I think that no one person knows everything. And sometimes, people’s experience and perspective is different. So, I don’t think that’s a bad thing ever.
Katherine Banwell:
As a follow-up to Calvin’s question, is it sufficient to just look at what the blood tests reveal? Or does having bone marrow biopsy dictate what treatment you should follow?
Dr. Raajit Rampal:
I think the bone marrow is important, particularly at initial diagnosis or when there is a change. The blood counts are the canary in the coal mine. So, they tell us is there something else going on that we’re not thinking about. And that’s when the bone marrow becomes important. So, I definitely think bone marrow is important at certain points in the disease.
Katherine Banwell:
Sandra has this question, “Are there new treatments for polycythemia vera being researched beyond interferon?”
Dr. Raajit Rampal:
Yeah. So, we talked about rusfertide as an example of this. And there are, certainly, other drugs that have been evaluated in this space. So, there is a lot of work going on for this disease, which is really encouraging.
Katherine Banwell:
Carolyn sent in this question, “Is there a possibility of bone marrow fibrosis reversal in myelofibrosis without a stem cell transplant?”
Dr. Raajit Rampal:
The answer is yes. So, even with JAK inhibitors, we see that about a third of patients will have a reduction in bone marrow fibrosis. And this is a key question being investigated with some of the newer therapies that are being introduced into the treatment of myelofibrosis. And, certainly, we’ve seen data to date that suggests that the fibrosis can be reduced if not potentially eliminated in some cases.
Katherine Banwell:
Dr. Rampal, should all patients diagnosed with MPNs undergo molecular testing?
Dr. Raajit Rampal:
I strongly believe that. I think that we’ve learned so much that these tests are prognostic value.
And in some cases, it may suggest a slightly different diagnosis. I definitely think that should be the case.
Katherine Banwell:
What should patients be asking once they have the results?
Dr. Raajit Rampal:
What does it mean? That’s the most basic and fundamental question. It’s one thing to get a list of mutations. But the real bread and butter question is what does this mean to the disease and my prognosis and my treatment? Those are the key questions.
Katherine Banwell:
Andrew wants to know does Jakafi cause other mutations to develop?
Dr. Raajit Rampal:
That’s a really good question. Right now, we don’t think the answer is necessarily yes. We have seen that in some patients where the disease has progressed on Jakafi, mutations have emerged.
But the problem is that genetic testing has limits of detection. In other words, the mutation appears, it may not have just appeared or been caused by the drug but that it may have been below our limits of detection and actually grew while the patient was on therapy, which does not mean that the drug caused the mutation but that it was allowed to emerge during treatment with the specific drug. So, that is an area of investigation.
Katherine Banwell:
Well, thank you, Dr. Rampal. And please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them answered on future webinars.
You mentioned earlier clinical trials. And I’d like to dig a little bit deeper. Where do these fit into the treatment plan?
Dr. Raajit Rampal:
I think they should always be considered. None of the therapies that we have do we consider curative. And in many cases, standard therapy is fine given a patient’s clinical situation. In a case where standard therapy is not working or where we think that a patient’s prognosis is particularly challenging, or if they have mutations that may confer resistance to current therapies.
I think in those scenarios, a trial should always be considered.
Katherine Banwell:
So, if a patient is interested in possibly participating in a clinical trial, what kinds of questions should they be asking their healthcare team?
Dr. Raajit Rampal:
All of these trials are different. I think the first thing is to discuss what’s the risk, what’s the benefit of any given trial or drug. What stage and development is it? What’s the evidence to support it? And what can I expect from it?
Katherine Banwell:
What about cost?
Dr. Raajit Rampal:
So, trials, in general, have two components. One is what we call standard of care meaning that things we would do normally for in the course of a patient’s treatment would be billed to a patient’s insurance as if they weren’t on a trial.
Almost all trials, the study drug or any tests that are being done specifically with regards to the study drug are all covered by whoever is sponsoring the trial.
Katherine Banwell:
How do patients find out about where the clinical trials are taking place?
Dr. Raajit Rampal:
Usually, their physician should either, if they’re in a specialized center, they’ll have access there. But if they’re interested in trials and they’re being seen, for example, by a physician in the community who doesn’t necessarily specialize, asking for a referral to a major center where that MPN expertise is not an unreasonable approach to that. There is also clinicaltrials.gov where patients can go look for ongoing trials for their particular diagnosis.
Katherine Banwell:
So, if patients want to learn more about MPNs, what sort of resources would you recommend?
Dr. Raajit Rampal:
The thing I always say to patients is the internet is a very dangerous place for a variety of reasons. We have to, I think, do a good job of communicating to patients what are the resources. And the ones that I always point patients to are, for example, the MPN Advocacy International, the MPN Research Foundation, The Leukemia & Lymphoma Society, and the American Cancer Society. Those are sources of information that are vetted by physicians.
Some of that information is specifically for patients. Those, to me, are good sources for patients to read.
Katherine Banwell:
Dr. Rampal, as we close out our conversation, I wanted to get your thoughts on where we stand with progress and MPN care. Are there advances in research and treatment that make you hopeful?
Dr. Raajit Rampal:
Without a doubt. I think I’ve seen more progress in the last three years than I’ve seen in the last 10 years. And we have so many new drugs coming forward, new questions that we’re trying to answer, tough questions as you alluded to. The question about prognosis but also intervening early to prevent progression of disease. These are things that are difficult questions that we are trying to dig into now. So, I think we should be optimistic. We are seeing so many excellent developments. We’ll have to see how far they’re going to take us. I don’t think we know the answer to that. But this is an exciting time.
Katherine Banwell:
Dr. Rampal, thank you so much for joining us.
Dr. Raajit Rampal:
My pleasure.
Katherine Banwell:
And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us today.
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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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!
How Are ET, PV and Myelofibrosis Monitored?
How Are ET, PV and Myelofibrosis Monitored? from Patient Empowerment Network on Vimeo.
MPN specialist and researcher Dr. Joseph Scandura reviews tools that are used to monitor patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF), including routine blood work and symptom management
Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.
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Transcript:
Katherine Banwell:
I would imagine monitoring patients is different for each of the MPNs. So, how are patients typically monitored over time, and let’s start with essential thrombocythemia?
Dr. Scandura:
Yeah. I think – again, it’s similar. You know, what’s near-term, what’s long-term? And so, in all of these diseases, thrombosis risk is a near-term risk. That’s something that I am monitoring in certain ways to help mitigate that risk. In ET and PV, I approach them similarly. Blood counts are certainly – these are diseases of the blood forming system. Certainly, monitoring blood counts I find helpful. But the reality of it is, in ET, there is not a clear linkage between blood counts and risks.
And so, I like to keep the platelet count near normal if I can. But I also recognize that it may not be worth suppressing all of the blood counts to achieve that landmark, because it’s not clear that that’s really reducing the risk any more than just having somebody on a medication that helps control the blood counts. In polycythemia vera, different blood counts are very important. The red blood cells are kind of like part of the clotting risk. We know from clinical trials that keeping the red blood cell parameters within certain ranges reduces the risk of clotting. And so, what I monitor in polycythemia vera is the hematocrit. In women, I like to keep it below 42. In men, I like to keep it below 45.
But I don’t just – I’m not a slave to the hematocrit. I am keeping an eye on the other blood counts and the other red blood cell parameters. So, for instance, what’s the size of the red blood cells? That tells me a little bit about what’s going on in the blood formation for that patient. And what’s the number of red blood cells? So, sometimes people can have very small red cells, because they’re a little iron-deficient and have a huge surplus of the number of red blood cells. And that tells me a little bit about how their blood forming system is responding to therapy.
Iron deficiency in polycythemia vera is very prominent. I personally believe it’s a very major driver of symptoms in patients who are receiving phlebotomy as part of their care. And it’s something that I monitor and really counsel patients on. My goal is to make phlebotomy independent, but it can take a while.
Everybody starts out iron-deficient, and then we take iron out of their body through blood with the phlebotomy. And that makes them more iron-deficient.
Katherine:
Right.
Dr. Scandura:
I monitor symptoms from patients, and sometimes that can tell me that their disease needs to be – their treatment needs to be tweaked a little bit, even something as simple as aspirin. People can sometimes have burning in the skin or itching that is sometimes responsive to changing the aspirin dose or how it’s given, once a day versus twice a day.
And that simple thing can be a big change for a patient who’s kind of, literally, climbing out of their skin or wishing they could and to try and find something that is helping.
I had a patient the other day. He had COVID. I said, “Oh, you should probably get this medication.” Do you have your primary care physician? Who’s taking care of you?” And he goes, “Well, to be honest with you, you’re my guy.” And so, it’s true. I see this patient a lot. And so, sometimes they forget. If I’m not paying attention to their blood pressure, the risks or treatment of diabetes, cholesterol, lipids, their screening programs for mammogram or colonoscopy, health maintenance issues, I do keep an eye on that in patients, because I do think it’s a part of the MPNs.
I think that there are excess risks for patients for some of these factors. Certainly, if you think of it as three strikes, they get a strike for having an MPN. I don’t want them to have any other strikes. So, diabetes, hypertension, those are strikes that I can potentially, at least, treat or refer them to somebody to help comanage with me. And so, that’s kind of my general approach.
Katherine:
What about patients who have myelofibrosis? Are they monitored more closely?
Dr. Scandura:
Yeah, I think it depends a little bit on the patient. Patients with early myelofibrosis often don’t have any symptoms or near-term risks much different than those from ET or PV. As the disease can progress, then some of these patients have more profound problems with symptoms, which I may be trying to find a solution to make them feel better. And also, blood counts can become more of an issue.
Transfusions in some patients who are very high white blood cell count, the spleen is often quite enlarged. Although, in my experience, most patients aren’t really bothered by the size of their spleen as the physicians are. But it is something where I think, on average, they’re monitored a little bit more closely to quite a bit more closely depending on the patient.
Katherine:
What happens if someone suddenly has a change in blood counts? What do you do?
Dr. Scandura:
Yeah. I mean, repeat it. That’s the first thing. Also, check what’s going on. It’s not uncommon in patients with MPNs that I’ll see them and the counts are a little bit out of whack, the white count is much higher than it’s been, and questioning them. “Oh, yeah. I had X, Y, or Z last week or the week before.” It used to be a upper respiratory tract infection, or they had a minor surgical procedure.
And sometimes the responses to these things can be accentuated in patients with MPNs. And so, if that’s what of this story, I certainly would repeat it and let things calm down a little. And that’s often all it is. I’m much more of a monitor of the trends. So, one-time measure doesn’t generally excite me. It might make me want to have a follow-up a little more – in a shorter period of time. Of course, it depends on what the change is. But, for most of the changes that we observe, they’re relatively minor. And I will monitor them over time.
If I see a trend where something is progressively increasing or decreasing over time, then I start thinking about what else is going on. And that’s always in the context of what’s going on with the patient. How are they feeling? What’s their physical exam like? What are the other laboratory values like?
Katherine:
When is a bone marrow biopsy necessary?
Dr. Scandura:
I would say a bone marrow biopsy is absolutely necessary at the time of diagnosis. I personally do not routinely monitor by bone marrow biopsy unless it’s part of a clinical trial.
But I do perform a bone marrow or want to look at the bone marrow morphology if there is one of these changes or at least a trend that I want a little bit more information about. And so, if – or if it’s been a very long time since somebody has had a bone marrow. If it’s been five or ten years, then sometimes I may recommend we look just so we can collect a little bit more up-to-date information.
But I don’t routinely do a bone marrow, but I will do it if there are laboratories that are kind of trending in the wrong direction, there’s symptoms, there’s physical findings that I’m just not sure about. And I think it would help me be more sure as to what’s going on and be able to discuss that with the patient. Sometimes, just to say, “Hey. Look, we were worried about this, but the bone marrow looks really good.”
Thriving With an MPN | Tips for Managing Worry and Anxiety
Thriving With an MPN | Tips for Managing Worry and Anxiety from Patient Empowerment Network on Vimeo.
Dr. Joseph Scandura explains the role of shared decision-making when deciding on an MPN treatment, and why it’s so important for patients to take an active role in their care.
Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.
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Transcript:
Katherine Banwell:
Can you talk about shared decision-making? Why is it so important for patients to work closely with their healthcare team on choosing a therapy?
Dr. Scandura:
Because these are therapies that last for a long time. And, hopefully, the patients and the relationship last for a long time. And so, I think that everybody has to be comfortable with the decision about a therapy. And my personal goal is to try to make sure that everybody understands the rationale for a therapy, the potential ups and downs with the therapy, which every drug has, every approach has, and what I’m kind of watching and monitoring. I’m a very – I think that communication relieves a lot of anxiety. I think that the unknown is far scarier than the known, even if it’s not perfect. And so, I think shared decision-making has a role in relieving some of the scariness of unknown.
If we’re discussing to come to a decision, that means that my job is to give you the knowledge that I have so that you can tell me the knowledge about you and what you’re feeling and what you want back. And that back and forth is what helps me do a better job of taking care of the patient and helps the patient understand what’s going on and relieve some of the stress of the unknown. So, I think it’s a very synergistic approach. I don’t think I could practice medicine in another way.
Katherine:
Managing the worry associated with a diagnosis or concerns even about progression can lead to a lot of anxiety and fear amongst patients. Why is it important for them to share what they’re feeling with their healthcare team?
Dr. Scandura:
I would say this. If our goals are to have people – I mean, this is what I say to patients – I want you to think about this disease when you’re here. And, then, when you’re not here, my goal is to have you not thinking about this disease because you’re feeling okay and you’re comfortable and confident in what’s going on.
So, I want to make it a clinic visit disease. That’s not always possible. But, for many patients, it is. I don’t want somebody to become – to start thinking like a sick person when they’re not. I don’t want the diagnosis to be the disease, right? I want the person if they’re feeling well, to recognize that. Live your life; move on with things. But, at the same time, these kinds of diagnoses are scary.
Katherine:
Yeah.
Dr. Scandura:
And so, it is normal with a new diagnosis or a change in the diagnosis to go through a period of time where you have to adjust. And so, that’s normal, and you have to work your way through it. Some people want to work that all out internally, and that’s good to a certain extent as long as they have good supports at home. But I often want to know how they’re doing, how they’re working through that so I can get a gauge of how it’s affecting their life and the duration where this adjustment is going on.
So, somebody who’s still adjusting to a new diagnosis two years after the diagnosis, and they’re otherwise clinically well, that’s getting into the range where it’s not normal. You might need additional help. You might need counseling. And, in some patients, that might include some medications for a short period of time. The goal is to have the disease affecting you only in so far as it’s affecting you, not the idea of the disease.
So, that’s a – again, it’s a conversation. There are lots of resources. People, being individuals, deal with things in their own way, and I just try to help understand with them how it’s affecting their life. And, if it seems to be more than I would expect, I’ll tell them that.
And then we can discuss that. It doesn’t mean we have to do something today, but I will tell them, “I think this is maybe a little bit more. Why are you so worried? I think you’re doing great.”
Katherine:
Yeah. Yeah. Can a social worker or somebody else on the healthcare team help with these emotional needs that patients have?
Dr. Scandura:
Absolutely. We have great social workers. I tap into them all the time. We also have a group of psychiatrists who are really interested in kind of psychiatry that’s related to oncology and the diagnoses and how it impacts care. I mean, this is New York City, so everybody has a therapist. But a lot of patients have preexisting connections to healthcare providers or support systems. I think, for some patients, groups are helpful.
MPNs and Pregnancy: Why Close Monitoring Is Important
MPNs and Pregnancy: Why Close Monitoring Is Important from Patient Empowerment Network on Vimeo.
What complications can arise from an MPN during pregnancy? Dr. Joseph Scandura, from Weill Cornell Medicine, explains how pregnant women are monitored during pregnancy and in the postpartum period.
Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.
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How Should You Participate in MPN Care and Treatment Decisions? |
Transcript:
Katherine Banwell:
“What complications can arise from an MPN during pregnancy?”
Dr. Scandura:
Well, look, pregnancy – here you have two things, one of them common and complicated and the other one uncommon and complicated. So, common is pregnancy, but every pregnancy is different. And there’s a lot of changes going on in the body, and there’s certain risks that can go along with that as well. So, clotting risks sometimes can be increased in pregnancy. And then you have an MPN, where you have a clotting risk on top of that. The pregnancy really changes what kinds of medications we can think about using. And so, there are certain medications that we use comfortably in patients that would be an absolutely forbidden medication in a pregnant woman.
And so, it depends a little bit on what’s going on with the patient. But, if they have a history of clotting, then certainly, we would think about wanting to control the blood counts. It depends a little bit on what the disease is how we would do that. Interferons are commonly used in pregnancy, and they are safe in pregnancy and can improve the outcomes in some patients with pregnancy.
But short of that, in patients, for instance, who are very thrombotic risk, sometimes we have to sort of balance the risk of having a clot and something that can interfere with the pregnancy and the risk of bleeding. So, it’s not uncommon that people are on blood thinners during pregnancy at some point, but it really depends on the individual patient. What we do here is we keep very close contact with the patients.
And all of our patients are seen by the high-risk OB/GYN. So, it’s not the general obstetrics people who are monitoring the patient, so they’re much more closely monitored for complications of pregnancy. And we are seeing them more frequently during pregnancy to help, from the MPN side, to try to optimize and minimize the risks of clot. And that doesn’t end as soon as the baby’s out. If breastfeeding, their clotting risk is not normalized after pregnancy, as soon as the baby comes out. And so, you know, there’s an adjustment for several months afterwards where we’re still kind of thinking about this person a little bit differently than we would if they were not or had not been recently pregnant.
How to Access Financial Support for MPN Patients
How to Access Financial Support for MPN Patients from Patient Empowerment Network on Vimeo.
Is there financial help for patients living with ET, PV, and myelofibrosis? MPN specialist Dr. Joseph Scandura shares advice and resources to ease the financial burden of care and treatment.
Dr. Joseph Scandura is Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura, here.
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How Should You Participate in MPN Care and Treatment Decisions |
Transcript:
Katherine:
We’d be remiss if we didn’t bring up financial concerns, treatment and regular appointments can really become quite expensive. Understanding that everyone’s situation is different, of course, where can patients turn if they need resources for financial support?
Dr. Scandura:
Yeah. It depends on what the issue is. So, one of the biggest areas that I found this can interfere with care is when we have copays that are really not reasonable and not affordable. And so, how do we fix that? How do we get access to an agent that might be beneficial for a patient but that – you know, and the insurance has approved it, but they’ve approved it with such a high copay that it’s just not an option anymore.
And so, there are foundations. The PAN Foundation, we often will reach out to for copay assistance. And, actually, many companies have copay assistance programs for their individual drugs. And so, we have some of our nurses who are quite good at navigating these different agencies, and some of them are kind of drug-specific.
And because we see a lot of patients with MPNs and the number of drugs is not that great, we’re pretty tapped into what are the options for copay assistance that might be helpful. And it often works. It doesn’t always work. I had a patient I saw pretty routinely, and I kind of like my certain group of labs that kind of make me feel like I have a good sense of what’s going on. But he was getting killed with the lab costs. And he mentioned this to me, and then I have to do what I tell my – I have three teenage daughters, right? And, when they were littler – smaller, younger, we spent a lot of time distinguishing needs from wants, right?
So, this was one of those instances. What laboratory do I need to make sure that this patient is safe? What do I want because it makes me feel like I have a better idea of what’s going on? And maybe I can back off on those wants if I’m seeing the patient pretty frequently, which I happen to be at that time. And so, some of that is a conversation.
And it depends on the specifics of the insurance and a little bit of back and forth and knowing how to kind of minimize that financial burden when that’s starting to compromise care.
What Are Indicators of MPN Progression?
What Are Indicators of MPN Progression? from Patient Empowerment Network on Vimeo.
What are signs that essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF) may be progressing? Dr. Jeanne Palmer, of the Mayo Clinic in Arizona, reviews factors that may indicate progression and discusses how blood counts are used in disease monitoring.
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.
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How Should You Participate in MPN Care and Treatment Decisions? |
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Understanding Treatment Options for ET, PV, and Myelofibrosis |
Transcript:
Katherine Banwell:
Okay. Katie had this question. “What are the signs of progression from PV to MF or AML, both clinically and in blood tests, and when do you need a new bone marrow biopsy to check for this happening?”
Dr. Jeanne Palmer:
So, in terms of progression, there are several things that we see happen.
I think most importantly is, let’s say you have PV, and you’ve always been on medication, and it’s been hard to control. And all of a sudden, you don’t need medication to control it anymore, or the same thing for essential thrombocythemia. You have been taking medication, and all of a sudden your platelets go down, and you don’t need to take drugs anymore. A lot of times people are like, “Oh, that means I’m fixed and I’m well,” not necessarily, you really need to make sure to talk to your healthcare provider and potentially get a bone marrow biopsy.
Now, the other thing – sometimes the blood counts will actually drop too low, so you’ll have somebody who has PV, who has always been too high and then all of the sudden they come in, and their hemoglobin is very low, and they’re anemic, and that’s another situation where you do that. So, anytime the blood counts start to drop is concerning.
Now, it’s a continuum, so the blood counts may drop as you’re at the point of transitioning but it doesn’t – it’s not like if your blood count is dropping you say, “Oh my God, I have myelofibrosis, I need a bone marrow transplant tomorrow.” That’s not necessarily the case. This is generally a transition type process.
Also when the spleen starts to get enlarged. Now, the spleen can be enlarged even in the setting of just ET or just PV, so spleen enlargement does not necessarily mean you’re transforming, but it can be one of the things that we would see that would indicate that.
Katherine Banwell:
Okay.
Dr. Jeanne Palmer:
And then finally white blood cell count increasing can often be a sign of that. Now, in terms of progression to AML, that is generally something we’ll see in the blood. AML or acute myeloid leukemia, is indicated by the presence of blasts at greater than 20 percent. Now, many patients with myelofibrosis, in particular, but even PV and ET, may have blasts in their peripheral blood. Blasts are normal. If I did a marrow on every healthy person out there, they are going to have some blasts, because these are the first part of the development of white blood cells. So, they’re like baby white blood cells. But what the problem is, is when they start to grow too much.
And so in the setting of myelofibrosis and even sometimes with these other diseases, the blasts will be in the peripheral blood primarily because the bone marrow is damaged and doesn’t hold them in very well. It becomes AML when it gets greater than 20 percent, so that blasts of greater than 20 percent in the peripheral blood or in the bone marrow but a lot of times we find it in the peripheral blood is where we indicate this has progressed to AML.
Katherine Banwell:
Yeah.
Dr. Jeanne Palmer:
Blasts of greater than 10 percent are also something that we really want to pay attention to, because that would suggest that the disease is starting to become more aggressive. Now, blasts vary, so for example, I’ve had patients go up to 11 and then drop back down to 3 or 4, and then they say around 3 or 4 or 5. So, you always want to make sure to double-check because one blast count at 11 percent, whereas it’s very important to address, may not necessarily reflect that you need to change in treatment at that time. Again, these blood tests, I always tell people, do not freak out over one blood test.
Make sure you get at least a couple of them to really confirm what you are looking at.
How to Treat PV-Related Itching
How to Treat PV-Related Itching from Patient Empowerment Network on Vimeo.
Dr. Jeanne Palmer, from the Mayo Clinic in Arizona, explains pruritis, which is the itching related to polycythemia vera (PV), and discusses strategies for managing this MPN symptom.
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.
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Understanding Treatment Options for ET, PV, and Myelofibrosis |
Transcript:
Katherine Banwell:
We received some audience questions prior to the program today. This one is from Jacqueline, “What can I do to minimize pruritus or itching due to PV? A typical histamine blocker like Claritin or Zyrtec has done nothing whatsoever.”
Dr. Jeanne Palmer:
Yeah. Unfortunately, the itching of this is not as much mediated by an allergic type reaction or histamine. It’s a lot related to that microvasculature, those tiny little blood vessels. Things like avoiding hot showers, as we talked about, taking cooler showers or not even taking showers, just like cleaning yourself with a washcloth can be helpful. There are certain medications that we can use sometimes that help.
Now, first of all, Jakafi is extremely effective for itching. Of course, it does have side effects. It’s not always approved for your disease, so for example, it’s not approved for essential thrombocythemia. But JAK inhibitors can be helpful in that setting. There are also medications like gabapentin, which is a medicine that we use to treat peripheral neuropathy and that can actually be helpful because actually the itching, a lot of it is related to nerves not functioning right, so gabapentin can be helpful.
And a really old-school medicine that I sometimes use, especially if the itching is most prevalent at night, is a drug called doxepin, and that’s been around for a very long time, but it can be extremely sedating and has to be used with caution, especially in patients who are older.
How Can Patients Navigate Care and Thrive With an MPN?
How Can Patients Navigate Care and Thrive With an MPN? from Patient Empowerment Network on Vimeo.
What does it mean to thrive with an MPN? Dr. Jeanne Palmer, an MPN specialist from the Mayo Clinic, shares advice on navigating MPN care and stresses the importance of communicating openly with your healthcare team.
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.
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Transcript:
Katherine Banwell:
What does it mean to you to thrive with an MPN?
Dr. Jeanne Palmer:
I think living with an MPN can be very difficult. I think there is a number of things. First of all, there’s always the worry of what’s going to happen in the future. Many of these MPNs can start as fairly, for lack of a better term, as benign issues and can convert to something much more serious. So, I think living with that sort of timebomb in the back it can be extremely stressful. So, figuring out how to live with the fact that there is some degree of uncertainty.
I think the other thing is making sure to understand your disease. These are very rare disorders and even if you go to a hematologist-oncologist specialist, a lot of times they don’t have all the information because they don’t see a lot of them every year. So, it’s really important to make sure that above and beyond that you understand what’s going on in your body so that when new things happen, new symptoms happen, you’re able to really address them as opposed to sort of living with something that may make you feel poorly that’s not being addressed.
So, again, I think the biggest piece of this is seeing how do you live with uncertainty and how do you make sure you understand your disease well enough that you know what’s going on in your own body.
Katherine Banwell:
Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions. Why do you think it’s important for patients to speak up when it comes to symptoms and side effects?
Dr. Jeanne Palmer:
Well, there is a lot of things. This is a disease, again, that we can direct our therapy many times towards symptoms, and so when we think about how do I direct my therapy, so how do I treat somebody, symptoms are an incredibly important part of it. And there is nothing worse than having a patient come and see me who I see every six months, because they’ve been pretty stable and they’re like, “Oh, for three months I’ve been feeling awful.” And you’re like, well, “Why didn’t you let me know, we could do something about this?”
So, if there is something that doesn’t feel right, it’s very, very important to talk to your healthcare provider. I would much rather be bothered and handle something earlier on than miss something and really have a lot more catch-up to do afterwards.
The other thing is symptoms may indicate a blood clotting event. We know that patients will have a higher risk of blood clotting. These are extremely important to identify early on because if they go unchecked, they can cause more damage.
Katherine Banwell:
Dr. Palmer, was we close out this conversation I wanted to get your thoughts on where we stand with progress in helping people live longer and truly thrive with MPN. What would you like to leave the audience with?
Dr. Jeanne Palmer:
So, I think that the first thing is make sure you understand your disease. Don’t hesitate to ask for a second opinion. It’s always good to make sure you talk to someone who can really explain so you feel like when you go home you understand what’s going on in your body. Make sure you understand what symptoms to look for, what things to be aware of, because a lot of times people come in and they have no idea that, oh, these symptoms are actually related to their disease.
The other thing to make sure is that you’re very honest with your provider on how you’re feeling. A lot of times people come in and they say, “Oh, how are you feeling?” “I feel fine,” but then they start to ask very specific questions and they’re like, “Oh yeah, I’m really tired, my fatigue is an 8 out of 10,” or something.
So, make sure you’re really honest with your provider. When they ask you how they’re doing, this is not a social visit, this is a visit where they need to know your symptoms, so you don’t need to say I’m fine like you normally would if you were walking down the street.
The next thing is to always make sure to know where there’s clinical trials because we are making enormous great leaps and bounds in this field. It’s a really exciting time for myeloproliferative diseases, and there’s a number of new drugs that are being tested and coming out. So, it’s always important, if the opportunity is available and you can do it, clinical trials are a great way to get treatment.
Plus, you are giving back, because these are things that help us learn whether something works or not. So, you’re not as much a guinea pig, you never get a sugar pill. It’s one of those things will you will always get the treatment you need and then they may add something to it or you may be in the situation where there is no treatment, so they try something.
But clinical trials, I have to emphasize, are a great way to get therapy and really are how we know everything that we know about treatment for these diseases.
Katherine Banwell:
Yeah. It sounds like there’s a lot of progress and hope in the field.
Dr. Jeanne Palmer:
Oh, absolutely.
What Are Common MPN Symptoms?
What Are Common MPN Symptoms? from Patient Empowerment Network on Vimeo.
Dr. Jeanne Palmer, an MPN specialist, reviews the most common symptoms associated with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF).
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.
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Thriving with an MPN | What You Should Know About Care and Treatment |
Transcript:
Katherine Banwell:
Would you walk us through the common symptoms of each of the MPNs? Let’s start with essential thrombocythemia.
Dr. Jeanne Palmer:
Right. So, there are a number of shared symptoms throughout all the diseases and when we start to figure out how to categorize them, they call into several different categories. The first one is inflammation-related symptoms. We know that the inherent pathway that’s dysregulated or that causes these diseases to happen can also result in significant inflammation in a person, that can result in things like fevers, night sweats, weight loss, and overall feeling really fatigued and poorly, which is something that it seems to be much more prevalent in patients with MPNs, all sorts of them, actually.
The next set of symptoms is related to microvasculature, so all the little blood vessels. And sometimes we think, oh, maybe that’s because there’s too many red blood cells or platelets and the blood become viscous. It’s probably more related to the actual dysregulation of that JAK2 pathway, which is inherent to all the myeloproliferative diseases and as a result, the little blood vessels can clamp down and that can give people headaches, visual changes, numbness and tingling in the hands and feet, and even can cause sort of a painful rash called erythromelalgia in the body.
So, these are things that can happen that are probably less appreciated side effects of the disease. And finally, there’s spleen-related symptoms. The spleen is in the left upper quadrant of the abdomen and it’s an organ that generally is about 12 centimeters in length, 10 to 12, but in patients with myeloproliferative diseases it can be enlarged. And as a result of an enlarged spleen people can have feeling like they get fuller early. So, if you’re eating a meal, all of the sudden you can only eat half of that meal versus the whole meal.
Discomfort or pain in the left upper quadrant. Sometimes it’s much more noticeable when you like bend over to tie your shoes. And then sometimes people can actually, when the spleen gets really big, the blood flow can be impaired towards the end of it which can cause some of the spleen tissue to die, and that can be painful. So, these are things that if somebody does start to notice that they’re having fullness in the left upper quadrant, pain, stuff like that, that that may be related to spleen symptoms.
Katherine Banwell:
What about PV or polycythemia vera, what are the symptoms?
Dr. Jeanne Palmer:
So, all of these sorts of relate to all of the myeloproliferative diseases. So, one other one that I didn’t mention, and this is actually more in PV than others, is itching. Itching can be absolutely unbearable when somebody has PV. It’s particularly noticeable after taking a shower. So, a lot of times I’ve met patients who are like I haven’t been able to take a shower in years, because it causes such a high degree of itching.
Katherine Banwell:
Why a shower? Is it different from having a bath?
Dr. Jeanne Palmer:
Water on the body that can cause the problem. So, if people take hot showers, it’s even worse. Although I think that people sort of react to it differently. Usually what patients end up doing is more like sponge bath type of things, rather than actually being exposed to the water.
Taking colder showers or cooler showers can sometimes help mitigate that. But the itching, and even in the absence of a shower, people can have pretty severe itching, and that can also be one of the major side effects.
Thriving With an MPN | Tips and Support for Navigating Care
Thriving With an MPN | Tips and Support for Navigating Care from Patient Empowerment Network on Vimeo.
Dr. Joseph Scandura, an MPN specialist, discusses the management and monitoring of essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF), and shares resources and support for managing day-to-day life with an MPN.
Dr. Joseph Scandura is Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura, here.
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Transcript:
Katherine:
Hello, and welcome. I’m Katherine Banwell, your host for today’s webinar. This program is part of our Thrive series. And, today, we’re going to discuss navigating life with an MPN. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice.
Please, refer to your healthcare team about what might be best for you. Well, let’s meet our guest today. Joining me is Dr. Joseph Scandura. Welcome, Dr. Scandura, would you please introduce yourself?
Dr. Scandura:
Hi. I’m Joe Scandura. I am Associate Professor of Medicine at Weill Cornell in New York City. I am a Physician Scientist. I actually run a lab studying MPNs and hematopoietic stem cells. And I am Scientific Director of the Silver MPN Center at Cornell.
Katherine:
Thank you so much for taking time out of your schedule to join us today. We start all of the webinars in our Thrive series with the same question. In your experience, what do you think it means to thrive with an MPN?
Dr. Scandura:
As a goal, I think it’s very simple, symptom-free and normal life expectancy. Thriving with an MPN is living your life as though you didn’t have an MPN.
Katherine:
And one part of thriving with an MPN is finding a treatment approach that manages your disease, the symptoms of your MPN, and that fits with your lifestyle. So, what are the factors that are considered when choosing treatment for patients with ET, PV, and MF?
Dr. Scandura:
Certainly, the goals of the therapy. So, is the therapy one that I would be looking to maybe delay progression or for long-term potential benefits, or is it something I need now to control short-term risks such as blood clots? The goals of the patient because some therapies may be more suitable to the goals of one patient than another.
And the other – you know, there’s clinical features that may kinda push towards one approach versus another. Certainly, in a 20-year-old patient, I’m thinking about fertility. I’m thinking about a normal life expectancy. In a 90-year-old patient, I have a different set of concerns, multiple medications – what am I going to do that might be affecting their other comorbid conditions?
Katherine:
Right. Right.
Dr. Scandura:
I think about what are my near-term and long-term goals. So, obviously, age becomes a factor there. If I’m 95 years old, no matter what I do that person is not going to live 20 years. If that person’s 20 years old and they’re not living 30, 40, 50, 60 years, that’s a real shame. That’s a huge loss of life. So, that helps kinda point me in one direction or another.
And, then, there’s different types of therapy. There are injectable agents. There are pills. There are drugs that have been used for a long time but don’t really have an FDA approval. There are drugs that are approved for certain indications.
And, as physicians, we can sometimes stretch that based upon clinical judgment. So, I think a lot of that goes into the discussion I have with patients about therapy.
And that’s always – you know, I present to them what the options are, what I think the benefits might be, what the potential toxicities are, and then we discuss.
Katherine:
Right. I would imagine monitoring patients is different for each of the MPNs. So, how are patients typically monitored over time, and let’s start with essential thrombocythemia?
Dr. Scandura:
Yeah. I think – again, it’s similar. You know, what’s near-term, what’s long-term? And so, in all of these diseases, thrombosis risk is a near-term risk. That’s something that I am monitoring in certain ways to help mitigate that risk. In ET and PV, I approach them similarly. Blood counts are certainly – these are diseases of the blood forming system. Certainly, monitoring blood counts I find helpful. But the reality of it is, in ET, there is not a clear linkage between blood counts and risks.
And so, I like to keep the platelet count near normal if I can. But I also recognize that it may not be worth suppressing all of the blood counts to achieve that landmark, because it’s not clear that that’s really reducing the risk any more than just having somebody on a medication that helps control the blood counts. In polycythemia vera, different blood counts are very important. The red blood cells are kind of like part of the clotting risk. We know from clinical trials that keeping the red blood cell parameters within certain ranges reduces the risk of clotting. And so, what I monitor in polycythemia vera is the hematocrit. In women, I like to keep it below 42. In men, I like to keep it below 45.
But I don’t just – I’m not a slave to the hematocrit. I am keeping an eye on the other blood counts and the other red blood cell parameters. So, for instance, what’s the size of the red blood cells? That tells me a little bit about what’s going on in the blood formation for that patient. And what’s the number of red blood cells? So, sometimes people can have very small red cells, because they’re a little iron-deficient and have a huge surplus of the number of red blood cells. And that tells me a little bit about how their blood forming system is responding to therapy.
Iron deficiency in polycythemia vera is very prominent. I personally believe it’s a very major driver of symptoms in patients who are receiving phlebotomy as part of their care. And it’s something that I monitor and really counsel patients on. My goal is to make phlebotomy independent, but it can take a while.
Everybody starts out iron-deficient, and then we take iron out of their body through blood with the phlebotomy. And that makes them more iron-deficient.
Katherine:
Right.
Dr. Scandura:
I monitor symptoms from patients, and sometimes that can tell me that their disease needs to be – their treatment needs to be tweaked a little bit, even something as simple as aspirin. People can sometimes have burning in the skin or itching that is sometimes responsive to changing the aspirin dose or how it’s given, once a day versus twice a day.
And that simple thing can be a big change for a patient who’s kind of, literally, climbing out of their skin or wishing they could and to try and find something that is helping.
I had a patient the other day. He had COVID. I said, “Oh, you should probably get this medication.” Do you have your primary care physician? Who’s taking care of you?” And he goes, “Well, to be honest with you, you’re my guy.” And so, it’s true. I see this patient a lot. And so, sometimes they forget. If I’m not paying attention to their blood pressure, the risks or treatment of diabetes, cholesterol, lipids, their screening programs for mammogram or colonoscopy, health maintenance issues, I do keep an eye on that in patients, because I do think it’s a part of the MPNs.
I think that there are excess risks for patients for some of these factors. Certainly, if you think of it as three strikes, they get a strike for having an MPN. I don’t want them to have any other strikes. So, diabetes, hypertension, those are strikes that I can potentially, at least, treat or refer them to somebody to help comanage with me. And so, that’s kind of my general approach.
Katherine:
What about patients who have myelofibrosis? Are they monitored more closely?
Dr. Scandura:
Yeah, I think it depends a little bit on the patient. Patients with early myelofibrosis often don’t have any symptoms or near-term risks much different than those from ET or PV. As the disease can progress, then some of these patients have more profound problems with symptoms, which I may be trying to find a solution to make them feel better. And also, blood counts can become more of an issue.
Transfusions in some patients who are very high white blood cell count, the spleen is often quite enlarged. Although, in my experience, most patients aren’t really bothered by the size of their spleen as the physicians are. But it is something where I think, on average, they’re monitored a little bit more closely to quite a bit more closely depending on the patient.
Katherine:
Yeah. You mentioned blood counts. And we know that lab results can fluctuate a bit. What happens if someone suddenly has a change in blood counts? What do you do?
Dr. Scandura:
Yeah. I mean, repeat it. That’s the first thing. Also, check what’s going on. It’s not uncommon in patients with MPNs that I’ll see them and the counts are a little bit out of whack, the white count is much higher than it’s been, and questioning them. “Oh, yeah. I had X, Y, or Z last week or the week before.” It used to be a upper respiratory tract infection, or they had a minor surgical procedure.
And sometimes the responses to these things can be accentuated in patients with MPNs. And so, if that’s what of this story, I certainly would repeat it and let things calm down a little. And that’s often all it is. I’m much more of a monitor of the trends. So, one-time measure doesn’t generally excite me. It might make me want to have a follow-up a little more – in a shorter period of time. Of course, it depends on what the change is. But, for most of the changes that we observe, they’re relatively minor. And I will monitor them over time.
If I see a trend where something is progressively increasing or decreasing over time, then I start thinking about what else is going on. And that’s always in the context of what’s going on with the patient. How are they feeling? What’s their physical exam like? What are the other laboratory values like?
Katherine:
When is a bone marrow biopsy necessary?
Dr. Scandura:
I would say a bone marrow biopsy is absolutely necessary at the time of diagnosis. I personally do not routinely monitor by bone marrow biopsy unless it’s part of a clinical trial.
But I do perform a bone marrow or want to look at the bone marrow morphology if there is one of these changes or at least a trend that I want a little bit more information about. And so, if – or if it’s been a very long time since somebody has had a bone marrow. If it’s been five or ten years, then sometimes I may recommend we look just so we can collect a little bit more up-to-date information.
But I don’t routinely do a bone marrow, but I will do it if there are laboratories that are kind of trending in the wrong direction, there’s symptoms, there’s physical findings that I’m just not sure about. And I think it would help me be more sure as to what’s going on and be able to discuss that with the patient. Sometimes, just to say, “Hey. Look, we were worried about this, but the bone marrow looks really good.”
Katherine:
Yeah. Can you talk about shared decision-making? Why is it so important for patients to work closely with their healthcare team on choosing a therapy?
Dr. Scandura:
Because these are therapies that last for a long time. And, hopefully, the patients and the relationship last for a long time. And so, I think that everybody has to be comfortable with the decision about a therapy. And my personal goal is to try to make sure that everybody understands the rationale for a therapy, the potential ups and downs with the therapy, which every drug has, every approach has, and what I’m kind of watching and monitoring. I’m a very – I think that communication relieves a lot of anxiety. I think that the unknown is far scarier than the known, even if it’s not perfect. And so, I think shared decision-making has a role in relieving some of the scariness of unknown.
If we’re discussing to come to a decision, that means that my job is to give you the knowledge that I have so that you can tell me the knowledge about you and what you’re feeling and what you want back. And that back and forth is what helps me do a better job of taking care of the patient and helps the patient understand what’s going on and relieve some of the stress of the unknown. So, I think it’s a very synergistic approach. I don’t think I could practice medicine in another way.
Katherine:
Dr. Scandura, much of our MPN community is highly engaged in their care. What are some educational resources you would recommend for people who are seeking more information about their condition?
Dr. Scandura:
I think that there’s some basic information available from a variety of – for instance, the National Cancer Institute has some basic information. Leukemia & Lymphoma Society has some basic information.
The MPN Research Foundation has some basic information. And then there are some information websites that are run by corporations, which are – I think they try to be even-handed in some of the discussion and has some good information there, too. I think the – none of these is a perfect source of information. I don’t think there is one source that you can go to answer every question that you could ask.
My MPN Center has a website with a bunch of QAs, and we just every now and then add a new one. And it’s just a really long list. So, these are questions our patients frequently ask us, and we sort of put answers there to help guide. But individual details are often more important than sort of generalizations. I find patient – go ahead.
Katherine:
Oh. I was just going to ask, what about the forums, patient forums that are available? Is that something you would recommend?
Dr. Scandura:
What I kind of I find my patients do is they’ll go out and look for information, because patients with MPNs, thankfully, tend to live a long time. And they are often curious about their disease and want to do better and figure out how they can do better. And so, a lot of them will go to whatever sources are available. But, generally, they come back. So, we circle back; we regroup. And sometimes, it’s la-la land, a little bit crazy things, and sometimes it’s really interesting.
I learn a lot, you know, what’s going on in terms of what are patients really reporting, because sometimes in a clinic visit people kind of don’t say everything, or they forget to say something or maybe just my experience. I don’t see every patient in the world, right? So, if it’s something that’s relatively rare, then I may not have seen it with a new drug or something like that.
So, I can learn from that experience as well. So, I think it’s kind of like people go out. They can be like little honeybees and collect all the information from all the flowers out there. And then they come back, and we regroup in the nest. And we discuss and decide what makes sense, what’s relevant to them, and what might help with our decision-making.
Katherine:
Yeah. Managing the worry associated with a diagnosis or concerns even about progression can lead to a lot of anxiety and fear amongst patients. Why is it important for them to share what they’re feeling with their healthcare team?
Dr. Scandura:
I would say this. If our goals are to have people – I mean, this is what I say to patients – I want you to think about this disease when you’re here. And, then, when you’re not here, my goal is to have you not thinking about this disease because you’re feeling okay and you’re comfortable and confident in what’s going on.
So, I want to make it a clinic visit disease. That’s not always possible. But, for many patients, it is. I don’t want somebody to become – to start thinking like a sick person when they’re not. I don’t want the diagnosis to be the disease, right? I want the person if they’re feeling well, to recognize that. Live your life; move on with things. But, at the same time, these kinds of diagnoses are scary.
Katherine:
Yeah.
Dr. Scandura:
And so, it is normal with a new diagnosis or a change in the diagnosis to go through a period of time where you have to adjust. And so, that’s normal, and you have to work your way through it. Some people want to work that all out internally, and that’s good to a certain extent as long as they have good supports at home. But I often want to know how they’re doing, how they’re working through that so I can get a gauge of how it’s affecting their life and the duration where this adjustment is going on.
So, somebody who’s still adjusting to a new diagnosis two years after the diagnosis, and they’re otherwise clinically well, that’s getting into the range where it’s not normal. You might need additional help. You might need counseling. And, in some patients, that might include some medications for a short period of time. The goal is to have the disease affecting you only in so far as it’s affecting you, not the idea of the disease.
Dr. Scandura:
So, that’s a – again, it’s a conversation. There are lots of resources. People, being individuals, deal with things in their own way, and I just try to help understand with them how it’s affecting their life. And, if it seems to be more than I would expect, I’ll tell them that.
And then we can discuss that. It doesn’t mean we have to do something today, but I will tell them, “I think this is maybe a little bit more. Why are you so worried? I think you’re doing great.”
Katherine:
Yeah. Yeah. Can a social worker or somebody else on the healthcare team help with these emotional needs that patients have?
Dr. Scandura:
Absolutely. We have great social workers. I tap into them all the time. We also have a group of psychiatrists who are really interested in kind of psychiatry that’s related to oncology and the diagnoses and how it impacts care. I mean, this is New York City, so everybody has a therapist. But a lot of patients have preexisting connections to healthcare providers or support systems. I think, for some patients, groups are helpful.
Katherine:
We’d be remiss if we didn’t bring up financial concerns, treatment and regular appointments can really become quite expensive. Understanding that everyone’s situation is different, of course, where can patients turn if they need resources for financial support?
Dr. Scandura:
Yeah. It depends on what the issue is. So, one of the biggest areas that I found this can interfere with care is when we have copays that are really not reasonable and not affordable. And so, how do we fix that? How do we get access to an agent that might be beneficial for a patient but that – you know, and the insurance has approved it, but they’ve approved it with such a high copay that it’s just not an option anymore.
And so, there are foundations. The PAN Foundation, we often will reach out to for copay assistance. And, actually, many companies have copay assistance programs for their individual drugs. And so, we have some of our nurses who are quite good at navigating these different agencies, and some of them are kind of drug-specific.
And because we see a lot of patients with MPNs and the number of drugs is not that great, we’re pretty tapped into what are the options for copay assistance that might be helpful. And it often works. It doesn’t always work. I had a patient I saw pretty routinely, and I kind of like my certain group of labs that kind of make me feel like I have a good sense of what’s going on. But he was getting killed with the lab costs. And he mentioned this to me, and then I have to do what I tell my – I have three teenage daughters, right? And, when they were littler – smaller, younger, we spent a lot of time distinguishing needs from wants, right?
So, this was one of those instances. What laboratory do I need to make sure that this patient is safe? What do I want because it makes me feel like I have a better idea of what’s going on? And maybe I can back off on those wants if I’m seeing the patient pretty frequently, which I happen to be at that time. And so, some of that is a conversation.
And it depends on the specifics of the insurance and a little bit of back and forth and knowing how to kind of minimize that financial burden when that’s starting to compromise care.
Katherine:
Yeah. Let’s answer a few audience questions that we received in advance of the webinar. This one is from Sophie, “What complications can arise from an MPN during pregnancy?”
Dr. Scandura:
Well, look, pregnancy – here you have two things, one of them common and complicated and the other one uncommon and complicated. So, common is pregnancy, but every pregnancy is different. And there’s a lot of changes going on in the body, and there’s certain risks that can go along with that as well. So, clotting risks sometimes can be increased in pregnancy. And then you have an MPN, where you have a clotting risk on top of that. The pregnancy really changes what kinds of medications we can think about using. And so, there are certain medications that we use comfortably in patients that would be an absolutely forbidden medication in a pregnant woman.
And so, it depends a little bit on what’s going on with the patient. But, if they have a history of clotting, then certainly, we would think about wanting to control the blood counts. It depends a little bit on what the disease is how we would do that. Interferons are commonly used in pregnancy, and they are safe in pregnancy and can improve the outcomes in some patients with pregnancy.
But short of that, in patients, for instance, who are very thrombotic risk, sometimes we have to sort of balance the risk of having a clot and something that can interfere with the pregnancy and the risk of bleeding. So, it’s not uncommon that people are on blood thinners during pregnancy at some point, but it really depends on the individual patient. What we do here is we keep very close contact with the patients.
And all of our patients are seen by the high-risk OB/GYN. So, it’s not the general obstetrics people who are monitoring the patient, so they’re much more closely monitored for complications of pregnancy. And we are seeing them more frequently during pregnancy to help, from the MPN side, to try to optimize and minimize the risks of clot. And that doesn’t end as soon as the baby’s out. If breastfeeding, their clotting risk is not normalized after pregnancy, as soon as the baby comes out. And so, you know, there’s an adjustment for several months afterwards where we’re still kind of thinking about this person a little bit differently than we would if they were not or had not been recently pregnant.
Katherine:
Yeah. We have another question. This one from Jennifer. She wonders, is there research being done on MPN progression to understand how it happens or even prevent or slow progression?
Dr. Scandura:
Yeah. There’s a lot. I think there is a – from both the sort of basic laboratory using animal models to try to understand what are the kind of systems that are involved in how these diseases change. What genes are involved? How do they talk to each other? You know, these are not cells that live in a vacuum, right? They live in a special microenvironment. What are the signals that crosstalk between the MPN cells, the MPN stem cells, and their microenvironment?
And so, there’s a lot of research on that and the basic side of things. In humans, there’s a lot that has been done over the years in terms of trying to understand what are some of the genetic features of progression. And I think we’re beginning to get a little bit of a better understand of what are the non-genetic things that are associated with progression.
I was part of an effort from the MPN Research Foundation and still am. They have what they call the Progression Network, where they tried to put together a number of investigators from really across the world to share ideas about the nature of progression and how we might look at studying this and understanding ways to prevent progression.
I think we do have some drugs now that show some promise in terms of being able to prevent progression. I think interferons have shown this in polycythemia vera in terms of a promise for improved long-term outcomes and delayed risk progression. I think that the gold standard randomized trials are maturing and are sort of bearing out some of the same findings that have been observed retrospectively, so sort of kind of looking back in time.
But the difficulty is that it can take a long time for patients to progress. And you say, “Oh, that’s great.” And that is great. But, from a research – from a statistical side, it means things are really slow. If you have to wait 15 years to assess whether or not people progressed less in one treatment versus another, it’s really slow going. And so, we have to do a compromise of what’s – you know, what do animal studies say? What does retrospective analysis, when we might have people who started treatment 30 years ago, and now we’re just seeing how did it all work out? It’s not a perfect study, because biases can creep in, but it’s what we have now. And so, there’s a lot. And I think, increasingly, progression is being recognized as a goal of therapy, to prevent progression.
Personally, it is one of major goals, because I think we do a pretty good job at preventing clots with available treatments. But I don’t think we do a very good job at preventing progression, mostly, because we don’t exactly understand what’s driving that. And so, I think until we develop that deeper understanding and really invest the time and effort in terms of learning which approaches can help prevent progression, we’re going to continue to have these questions.
Katherine:
Yeah. Well, thank you for those answers, Dr. Scandura. And please continue to send in your questions to question@powerfulpatients.org, and we’ll work to get them answered on future programs. As we close out this conversation, Dr. Scandura, I would like to get your thoughts on where we stand with progress with MPN care. Are there advances in treatment research that you’re hopeful about?
Dr. Scandura:
Yeah. I think it’s a very exciting time, actually. I think that over the past 5 to 10 years the amount of new drugs that have been developed and tested in patients has grown exponentially. The number of companies that are targeting MPNs for their drug development has expanded dramatically. The number of clinical trials, good quality clinical trials has increased dramatically. And I think the success that’s coming out of that is we start seeing drugs now that are looking to be very, very effective. I don’t want to name individual drugs.
But I know we have a number of clinical trials where we’re seeing things with these agents that we haven’t seen with our traditional therapies, meaning changes in the bone marrow that we haven’t seen before or a normalization of symptoms or blood counts in an area that has been challenging in the past. And so, we now have drugs and a number of drugs going for approval, a number of newly-approved drugs, even interferon, which is a drug that’s been around forever. Well, not forever. But, I mean, I guess forever, yeah, because it’s a natural product.
So, as long as there have been humans, there have been interferons, even before humans. But now we have it. As a pharmaceutical, they’ve been around for decades. And we now have the first – even though we’ve been using it for decades, we have the first approved, FDA-approved interferon for polycythemia vera, which is I think a huge change.
A company invested the money in getting FDA approval for an agent, and that means they have to – the bar’s higher, and they have to prove something that just using it off-label hasn’t. So, I think it’s a tremendously exciting time. I expect it’s going to continue. We’re going to continue to have improvements in care. There’s going to be combinations of drugs. I think that we’re going to see real advances over the next 5 to 10 years.
Katherine:
Well, Dr. Scandura, thank you so much for taking the time to join us today.
Dr. Scandura:
It was a pleasure. It was nice meeting with you.
Katherine:
And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us today.
Thriving With MPNs: Your Role in Managing Your Treatment and Care
Thriving With MPNs: Your Role in Managing Your Treatment and Care from Patient Empowerment Network on Vimeo.
How can patients thrive with a myeloproliferative neoplasm (MPN)? Dr. Jeanne Palmer discusses treatment approaches, strategies for managing disease symptoms and treatment side effects, and advice on how patients can be proactive in their care.
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology. Learn more about Dr. Palmer, here.
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Transcript:
Katherine Banwell:
Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to talk about how to live and thrive with an MPN. We’re going to discuss MPN treatment goals and how you can play an active role in your care. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you.
Well, joining us today is Dr. Jeanne Palmer. Dr. Palmer, welcome. Would you please introduce yourself?
Dr. Jeanne Palmer:
Thank you so much. I am so happy to be here and to help participate in this. My name is Jeanne Palmer. I am a hematologist at Mayo Clinic in Arizona. I specialize in MPNs as well as bone marrow transplant, and I am thrilled to be here.
Katherine Banwell:
Thank you for taking the time out of your busy schedule to join us today, Dr. Palmer. We start all of the webinars in our Thrive series with the same question and that is, what does it mean to you to thrive with an MPN?
Dr. Jeanne Palmer:
I think living with an MPN can be very difficult. I think there is a number of things. First of all, there’s always the worry of what’s going to happen in the future. Many of these MPNs can start as fairly, for lack of a better term, as benign issues and can convert to something much more serious. So, I think living with that sort of timebomb in the back it can be extremely stressful. So, figuring out how to live with the fact that there is some degree of uncertainty.
I think the other thing is making sure to understand your disease. These are very rare disorders and even if you go to a hematologist-oncologist specialist, a lot of times they don’t have all the information because they don’t see a lot of them every year. So, it’s really important to make sure that above and beyond that you understand what’s going on in your body so that when new things happen, new symptoms happen, you’re able to really address them as opposed to sort of living with something that may make you feel poorly that’s not being addressed.
So, again, I think the biggest piece of this is seeing how do you live with uncertainty, and how do you make sure you understand your disease well enough that you know what’s going on in your own body.
Katherine Banwell:
Yeah. That’s helpful to understand, especially as we move through today’s program, and we’re going to cover the three classic MPNs, polycythemia vera, essential thrombocythemia and myelofibrosis. One part of thriving with an MPN is managing the symptoms of the disease. Would you walk us through the common symptoms of each of the MPNs? Let’s start with essential thrombocythemia.
Dr. Jeanne Palmer:
Right. So, there are a number of shared symptoms throughout all the diseases and when we start to figure out how to categorize them, they call into several different categories. The first one is inflammation-related symptoms. We know that the inherent pathway that’s dysregulated or that causes these diseases to happen can also result in significant inflammation in a person, that can result in things like fevers, night sweats, weight loss, and overall feeling really fatigued and poorly, which is something that it seems to be much more prevalent in patients with MPNs, all sorts of them, actually.
The next set of symptoms is related to microvasculature, so all the little blood vessels. And sometimes we think, oh, maybe that’s because there’s too many red blood cells or platelets and the blood become viscous. It’s probably more related to the actual dysregulation of that JAK2 pathway, which is inherent to all the myeloproliferative diseases and as a result, the little blood vessels can clamp down and that can give people headaches, visual changes, numbness and tingling in the hands and feet, and even can cause sort of a painful rash called erythromelalgia in the body.
So, these are things that can happen that are probably less appreciated side effects of the disease. And finally, there’s spleen-related symptoms. The spleen is in the left upper quadrant of the abdomen and it’s an organ that generally is about 12 centimeters in length, 10 to 12, but in patients with myeloproliferative diseases it can be enlarged. And as a result of an enlarged spleen people can have feeling like they get fuller early. So, if you’re eating a meal, all of the sudden you can only eat half of that meal versus the whole meal.
Discomfort or pain in the left upper quadrant. Sometimes it’s much more noticeable when you like bend over to tie your shoes. And then sometimes people can actually, when the spleen gets really big, the blood flow can be impaired towards the end of it which can cause some of the spleen tissue to die, and that can be painful. So, these are things that if somebody does start to notice that they’re having fullness in the left upper quadrant, pain, stuff like that, that that may be related to spleen symptoms.
Katherine Banwell:
What about PV or polycythemia vera, what are the symptoms?
Dr. Jeanne Palmer:
So, all of these sorts of relate to all of the myeloproliferative diseases. So, one other one that I didn’t mention, and this is actually more in PV than others, is itching. Itching can be absolutely unbearable when somebody has PV. It’s particularly noticeable after taking a shower. So, a lot of times I’ve met patients who are like I haven’t been able to take a shower in years, because it causes such a high degree of itching.
Katherine Banwell:
Why a shower? Is it different from having a bath?
Dr. Jeanne Palmer:
Water on the body that can cause the problem. So, if people take hot showers, it’s even worse. Although I think that people sort of react to it differently. Usually what patients end up doing is more like sponge bath type of things, rather than actually being exposed to the water.
Taking colder showers or cooler showers can sometimes help mitigate that. But the itching, and even in the absence of a shower, people can have pretty severe itching, and that can also be one of the major side effects.
Katherine Banwell:
Much of the time the chosen treatment for MPNs manages the symptoms of the condition. I’d like to review the different types and classes of treatment for the three MPNs. So, let’s start with essential thrombocythemia again. When is it time to treat, and what are the options available?
Dr. Jeanne Palmer:
Right. So, with essential thrombocythemia, that’s the disease that sometimes we don’t need to treat.
So, we basically have a risk stratification system and this risk is based on age, history of a blood clot, the presence or absence of a JAK2 mutation. So, for example, if somebody is 28, does not have a JAK2 mutation, which is again one of those driver mutations, and never had a blood clot, they actually don’t necessarily need to do anything and just be monitored.
Somebody who is less than 60 and has a JAK2 mutation or who is greater than 60 and does not have a JAK2 mutation, in that setting, a lot of times you can use aspirin. Now, it gets a little bit gray in terms of that over 60 without the JAK2 mutation with regards to whether at that point you really should start taking some medicine to lower the platelets.
Now, if somebody has a JAK2 mutation, is greater than 60 or has had a blood clot, hands down they need to take medicine to lower the platelets, in addition to aspirin or whatever blood thinner they may need. So, for example, if you have a blood clot in a vein, a lot of times you need to take a blood thinner and that will be a lifelong thing. And again, we do these risk stratifications because we know there is a certain risk of clotting associated with the risk of essential thrombocythemia.
So, for example, somebody who is less than 60 and does not have a JAK2 mutation, never had a clot, their risk of clotting is probably very close to that of the normal population. Whereas if you’re higher risk and have a JAK2 mutation and greater than 60 or have had a history of a clot, the risk of clot is probably about 4 percent per year. So, this is something that can vary quite widely, and even though that 4 percent per year on the short-term doesn’t sound like a lot, if you take it additive over years, that’s why we generally try to be aggressive about lowering the platelets.
In lowering the platelets, the goal is to get less than 400 and doing that can be done through several different medications. The most commonly used medications is a drug called hydroxyurea, which has been around for a number of years, and a drug called anagrelide which is probably a little less commonly used, because it has some more GI side effects and headaches associated with it.
In some cases, especially in younger patients with this disease, we can consider using interferon, which is an injection of a cytokine, which are one of the chemicals that regulates the immune system within the body. But this interferon can actually help lower the platelets and there is a question of whether it may affect the biology of the disease as well.
Katherine Banwell:
Let’s turn to polycythemia vera or PV, what are the different options available for treating it?
Dr. Jeanne Palmer:
So, for polycythemia vera, everyone needs to be on aspirin.
And additionally, everyone needs to make sure to keep their blood count low, to manage their hematocrit, which is one of the measures of red blood cells. So, in men it’s generally recommended to keep below 45 and in women it’s recommended to keep below 42 percent. Now, the studied number was 45 percent and that was a study that was done, I don’t know, it was probably about 10 plus years ago, that actually showed that by keeping the blood hematocrit less than 45 percent you reduce the risk of having negative events like cardiovascular events and heart attacks. Because women tend to run with a lower blood count than men, it’s been extrapolated that 42 percent should be the number used for women.
Now, this can be done by phlebotomy, which essentially is bloodletting. It’s kind of like donating blood except for that the blood unfortunately can’t be donated to anybody, it has to be discarded. But the phlebotomy is one way to do that, and the reason that works is because it makes somebody iron deficient. So, whereas if this is normal, if you’re iron deficient you become anemic. If your baseline hematocrit is here, making you iron deficient brings you back to normal. So, even though we always associate iron deficiency with anemia, iron deficiency in the setting of polycythemia vera is actually kind of a treatment of sorts.
Now, once somebody gets above 60 and 60 seems to be sort of the magic age in these diseases, once somebody gets above 60, it is recommended that cytoreductive therapy is used, which means therapy or treatment that will bring down the red count. And again, for this one, hydroxyurea is an option as well as interferon. And there is recently an approval, actually FDA approval for a newer interferon called ropeginterferon or Besremi, which can help just bring down the red blood cells but it is the first interferon that’s actually been FDA approved for this indication.
Katherine Banwell:
Are JAK inhibitors used as well?
Dr. Jeanne Palmer:
They are. So, if somebody doesn’t respond well to hydroxyurea, the approval for ruxolitinib is actually for patients who have failed hydroxyurea. Although it’s something that we often consider especially in people who have a lot of symptoms. So, the itching, one of the things that can really help itching actually is Jakafi. If people have night sweats, they have weight loss, spleen related symptoms, those are the patients that will benefit from Jakafi. Additionally, if they are on hydroxyurea and can’t seem to get control of their blood count, Jakafi is a good option to help control the blood counts as well.
Interferon is a very nice option because there’s great data that shows that you may actually be able to lower the percentage of JAK2 burden.
So, we’d look at something called an allele burden, which is the percentage of cells that are involved – have the JAK2 mutation. Now, we don’t know whether lowering this percentage necessarily translates to long-term better survival, but I think there is enough data out there, and there is a good biologic underpinning for saying that this actually can help. But yes, Jakafi is another thing.
And the really exciting thing is that there is a newer agent called rusfertide, which is a hepcidin mimetic, which is basically taking a protein in your body that helps metabolize iron and by making it externally and giving it to somebody that it can actually help bring down the hematocrit without having some of the other side effects we know with some of the other medications. That is currently in Phase III studies, so hopefully in the next couple of years we’ll see approval for that.
Katherine Banwell:
Oh, that’s great news. And finally, how is myelofibrosis treated?
Dr. Jeanne Palmer:
So, myelofibrosis is a little bit of a different animal. When you have something like essential thrombocythemia or PV, a lot of this is managing symptoms, preventing blood clots, but if you do appropriate treatment and management of these diseases you could probably live close to a normal life expectancy.
So, I never typically pin a survival on it. With myelofibrosis, it’s a little bit different because there is a survival. Instead of saying you can live close to normal life expectancy, it backs up to saying how many years do I think you can live with this disease. Now, of course, we are horrible at predicting how many years anyone can live, so we have to take that all with a grain of salt. But we can at least sort of risk stratify people.
And the first thing that’s really important is to figure out whether somebody is a transplant candidate or not and if, based on age, disease risk features, stuff like that, or whether we think they ever will be a transplant candidate. So, that kind of helps us sort of think about what your path moving forward is.
Now, the current FDA-approved treatment for myelofibrosis, there are three JAK inhibitors approved, which is like Jakafi, which was the first approved one but there is also Inrebic or fedratinib and Vonjo or pacritinib and these have all been approved over the years.
The role of JAK inhibitors and treatment of myelofibrosis is symptoms-based. So, for example, a lot of patients with myelofibrosis will have weight loss, night sweats, big spleens, really feeling fatigued and poorly and in this setting, the JAK inhibitor can be very helpful. And you don’t have to have a JAK2 mutation, a lot of times people say, well, I don’t have the JAK2 mutation so how can a JAK inhibitor help. So, the JAK inhibitor works on this pathway, which is called the JAK/STAT pathway, irrespective of mutation.
So, if you are having symptoms and you have myelofibrosis, JAK mutation, excuse me, the JAK2 mutation does not predict who is going to have a response. And people who, regardless of which mutation you have, may actually benefit from it.
So, the JAK inhibitors, though, are extremely effective at reducing symptom burden as well as reducing the spleen size. And we know that if a spleen is big and we can make it shrink that, that probably is a surrogate marker for living longer, and I think it’s because inflammation does a lot of wear and tear on the body. So if you can reduce the inflammation and the spleen shrinks, which generally go hand in hand, then you might help somebody live longer. It is not changing the biology of the disease, though, however, it doesn’t change the pathway and that this disease is kind of projecting ahead in terms of creating – it changes, as it goes along, may acquire new mutations or something like that which makes the disease become more serious.
Right now, the approved therapies for it are JAK inhibitors and the Jakafi, ruxolitinib was the first one approved. Inrebic was approved several years back, or fedratinib.
And then the most recent one that was approved is Vonjo or pacritinib and that’s a drug that is a JAK inhibitor that is actually very good for people with low platelets. The reason I bring that up is because if we think of what’s the biggest limiter of JAK inhibitors, JAK inhibitors bring down red blood cells, and they bring down platelets. So, when somebody has low platelets it’s very hard to use a JAK inhibitor, because we’re not really able to increase the dose well enough to get that inflammatory reduction because of the fact that the blood counts will drop too low.
So, now drugs like Vonjo exist which, due to several other mechanisms associated with the drug are actually much more tolerated in somebody with low platelets. So, if you have low platelets, you can actually take the Vonjo, hopefully get the same degree of JAK inhibition to help the spleen shrink, help the symptoms get better without necessarily making the platelets substantially worse. A lot of times they do drop, it doesn’t help bring up the platelets, but it does help people tolerate more JAK inhibition, which ultimately will help with symptoms.
Dr. Jeanne Palmer:
So, one thing I also wanted to add about myelofibrosis treatment is sometimes people present, they don’t have a lot of symptoms, they don’t have a lot of spleen related problems but they have anemia or low blood counts and these can be incredibly hard to treat.
Even with symptoms and low red blood cell count or anemia or low platelets, it can be challenging to treat because many of these medications lower that. To treat the anemia there are several things that we can do. One of the first ones is using erythropoietin, and so there are many agents, they go by the names of like Procrit or darbepoetin alfa, that actually stimulate red blood cell growth by – like we give a recombinant hormone that helps red blood cells grow. This is normally something produced by the kidney.
So, one thing that’s important before going on one of these injections is to make sure that the kidney is not already producing enough. So, for example, if the kidney said, oh geez, I really need more red cells and is making lots of this hormone, erythropoietin, giving more of it is not going to help the system. But in people who don’t have a really high level it can be very beneficial.
The other thing that can help with anemia, specifically, is a drug called danazol (Danocrine).
It’s been around for a very long time. There are multiple presumed mechanisms of action, but one of them is that it is kind of a testosterone derivative. So, this is a medicine that can often help increase red blood cells in probably about 40 percent of people, and it’s a pill that you take twice a day.
Another option, sometimes we use thalidomide or lenalidomide (Revlimid). These are medications that have been used quite frequently in the setting of multiple myeloma and even a little bit in myelodysplastic syndrome, so some other blood disorders.
But in the setting of myelofibrosis, they can be helpful with anemia and sometimes are combined with prednisone or a corticosteroid. And then finally, in terms of drugs that are being tested and hopefully will be approved at some point in the future. There is a drug called momelotinib, which is another JAK inhibitor that actually has some mechanisms that may also help improve hemoglobin.
So, this is something I’m really looking forward to and we anticipate may be approved by the end of the year. And finally, there is another drug called luspatercept (Reblozyl). Luspatercept may work in the setting where your kidneys are already producing enough erythropoietin. So, the luspatercept is an injection that you receive once every three weeks.
It is currently FDA-approved for the treatment of myelodysplastic syndrome but this is something that has been shown to have some efficacy in myelofibrosis as well. So, this could be another therapeutic option for patients with myelofibrosis.
It is also important, especially for people who have polycythemia vera myelofibrosis to make sure that your iron has been checked and B-12 has been checked, because just because you have a bone marrow disorder doesn’t necessarily mean you don’t have a nutrition deficit that may be able to help improve your hemoglobin somewhat. But these are important things to talk to your doctor. I do not recommend just starting to take iron or B-12, however, if you’re anemic because in many cases you are not deficient and taking too much iron can actually be damaged.
Katherine Banwell:
Yeah, that’s great advice.
When would you consider a stem cell transplant?
Dr. Jeanne Palmer:
So, the stem cell transplant is based on disease risk. There is a number of ways we assess disease risk. The first two ones that were published a number of years back were the DIPSS score, which is Dynamic International Prognostic System Score, or the DIPSS Plus, which basically is the DIPSS and then you add to it a few other clinical features. This symptom score is based largely on things that we can see without even a bone marrow biopsy, so things like symptoms, age, number of white blood cells, whether somebody has anemia. And then the number of something called blasts, which is very immature white blood cells. The DIPSS Plus takes into account low platelets, need for transfusions, and chromosome abnormalities, which is the only test among that that needs to be from a bone marrow biopsy.
Now, these were created prior to Jakafi being commercially available. So, we have to take a little bit of a grain of salt with those because of the fact that Jakafi probably has changed how long people can live with this disease.
Now, more recently they’ve tried to account for these other molecular changes. So, when we take the genetic landscape of these diseases, we have the known driver mutations, so the JAK2 mutation which I have talked about, also calreticulin and MPL.
These three mutations all affect that one pathway, the JAK/STAT pathway, so they all affect the pathway that drives the disease and they are known to be kind of mutually exclusive and definitely contribute to the formation of the disease.
Some of these other mutations are called somatic mutations. They could be checked by things next generation sequencing or genetic analysis. There’s a number of different names that people use for this testing, but we look for mutations that are present and these mutations, number one, can sometimes tell us risk. So, there’s certain mutations that are high risk. Other times it can actually give us other opportunities for therapy, especially of the disease progresses. But these mutations are important to know for risk stratification. For example, if somebody has DIPSS score that is maybe not super high risk, but then they have one of these mutations, we know that that probably makes their disease a little bit more aggressive.
And that’s when we think about transplant, is when we know that the disease probably has an average life – when somebody gets to the point in their disease where we estimate their life expectancy is around five years, recognizing that we’re not very good at this. That is the type of point when we start to think about transplant. But the timing of transplant is something that’s extremely difficult and a very personalized decision. It’s something that it’s really important to understand the disease risks, how we assess them and the caveats of these disease risk assessments as we move forward planning and timing of transplant and that’s something that is, again, a very, very important discussion to have at length with your physician.
And I always recommend, there is quite a few of us out there who actually specialize in transplant for myelofibrosis and having discussions with somebody who really understands the biology of the myelofibrosis and important because it’s very different than a lot of the other diseases that are transplanted.
Katherine Banwell:
Yeah. Well, speaking of that, patients can sometimes feel like they’re bothering their healthcare team with their comments and questions. Why do you think it’s important for patients to speak up when it comes to symptoms and side effects?
Dr. Jeanne Palmer:
Well, there is a lot of things. This is a disease, again, that we can direct our therapy many times towards symptoms, and so when we think about how do I direct my therapy, so how do I treat somebody, symptoms are an incredibly important part of it. And there is nothing worse than having a patient come and see me who I see every six months, because they’ve been pretty stable and they’re like, “Oh, for three months I’ve been feeling awful.” And you’re like, well, “Why didn’t you let me know, we could do something about this?”
So, if there is something that doesn’t feel right, it’s very, very important to talk to your healthcare provider. I would much rather be bothered and handle something earlier on than miss something and really have a lot more catch-up to do afterwards.
The other thing is symptoms may indicate a blood clotting event. We know that patients will have a higher risk of blood clotting. These are extremely important to identify early on because if they go unchecked, they can cause more damage.
Katherine Banwell:
With many of the treatments available as pills now, patients have a role in self-administering their treatment regimen. What happens if a patient forgets to take a medication? Does it impact its effectiveness?
Dr. Jeanne Palmer:
Generally no. I think the ones that would are certain blood thinners you really don’t want to miss and you don’t want to miss the doses on it. With drugs like Jakafi, if you miss one dose you probably won’t notice it, but if you miss multiple doses you can actually get very sick from that. So, some of these medications are really important to be consistent on.
Now, I know this could be a challenge. I mean I don’t take very many medications and I sometimes have a hard time keeping track of what I take, so I know that this can be a difficult thing to do. So, one thing is if you really find you’re struggling with it, setting an alarm on your phone or your Apple Watch or whatever…
Katherine Banwell:
…device.
Dr. Jeanne Palmer:
Device you have can be a really helpful way of doing it. Also having a pill box. They make pretty amazing pill boxes these days that can account for taking drugs once a day, twice a day, three times a day. I’ve even seen them up to four times a day, although generally the most you’ll probably have to take a medicine for a myeloproliferative disease is twice a day. But those are different ways that can really help make sure you’re consistent about taking your medication.
Katherine Banwell:
And if a patient misses a dose, do they need to call their healthcare team and let them know?
Dr. Jeanne Palmer:
Not just for one missed dose. If like, for example, they’re run out and they say, “Oh, geez, I don’t have any and many of these drugs are specialty pharmacy,” so they need to be mailed, and you know that you’re going to be missing it for a while. Or let’s say you look at your pill bottle and go, “Oh shoot, I only have so many pills left,” it is helpful to call because a lot of times, for example, if somebody is on Jakafi and they know they’re going to run out of their pills four days before they’re going to get their next shipment in, then what I sometimes do is I lower the dose a little bit to make sure they maintain a dose throughout that time.
But this is something you definitely want to do under the advice of a healthcare provider. You don’t want to just all of the sudden go, “Oh, well I’m going to run out so I’m just going to change my dose,” and kind of do that.
Katherine Banwell:
Yeah, yeah. We received some audience questions prior to the program today. This one is from Jacqueline, “What can I do to minimize pruritus or itching due to PV? A typical histamine blocker like Claritin or Zyrtec has done nothing whatsoever.”
Dr. Jeanne Palmer:
Yeah. Unfortunately, the itching of this is not as much mediated by an allergic type reaction or histamine. It’s a lot related to that microvasculature, those tiny little blood vessels. Things like avoiding hot showers, as we talked about, taking cooler showers or not even taking showers, just like cleaning yourself with a washcloth can be helpful. There are certain medications that we can use sometimes that help.
Now, first of all, Jakafi is extremely effective for itching. Of course, it does have side effects. It’s not always approved for your disease, so for example, it’s not approved for essential thrombocythemia. But JAK inhibitors can be helpful in that setting. There are also medications like Gabapentin, which is a medicine that we use to treat peripheral neuropathy and that can actually be helpful because actually the itching, a lot of it is related to nerves not functioning right, so gabapentin can be helpful.
And a really old-school medicine that I sometimes use, especially if the itching is most prevalent at night, is a drug called Doxepin and that’s been around for a very long time, but it can be extremely sedating and has to be used with caution, especially in patients who are older.
Katherine Banwell:
Here is a question from Daniel. “How often should a person with PV have hematology appointments, and how often should you have blood tests?”
Dr. Jeanne Palmer:
Well, that is something that you need to discuss with a provider, because everyone’s a little bit different. If I have somebody who I’m managing on a medication, they’ve been rock solid stable, it may be every few months that I check blood and maybe every six months that I see them.
If I have somebody who have been particularly difficult to control and I’m sort of adjusting medications or they’re having symptoms, then I try to check blood more regularly, like on a monthly basis. But again, this is something that – I have checked blood as frequently as every two weeks, especially in somebody who has an extremely high red blood cell count that I’m trying to lower. I have checked blood as infrequently as every three months. Again, in somebody who is not undergoing treatment, say, for example, who has essential thrombocythemia, sometimes I check blood even less. So, it really is something that can vary from every two weeks to every six months.
Katherine Banwell:
Okay. Katie had this question. “What are the signs of progression from PV to MF or AML, both clinically and in blood tests, and when do you need a new bone marrow biopsy to check for this happening?”
Dr. Jeanne Palmer:
So, in terms of progression, there are several things that we see happen.
I think most importantly is, let’s say you have PV, and you’ve always been on medication, and it’s been hard to control. And all of a sudden, you don’t need medication to control it anymore, or the same thing for essential thrombocythemia. You have been taking medication, and all of a sudden your platelets go down, and you don’t need to take drugs anymore. A lot of times people are like, “Oh, that means I’m fixed and I’m well,” not necessarily, you really need to make sure to talk to your healthcare provider and potentially get a bone marrow biopsy.
Now, the other thing – sometimes the blood counts will actually drop too low, so you’ll have somebody who has PV, who has always been too high and then all of the sudden they come in, and their hemoglobin is very low, and they’re anemic, and that’s another situation where you do that. So, anytime the blood counts start to drop is concerning.
Now, it’s a continuum, so the blood counts may drop as you’re at the point of transitioning but it doesn’t – it’s not like if your blood count is dropping you say, “Oh my God, I have myelofibrosis, I need a bone marrow transplant tomorrow.” That’s not necessarily the case. This is generally a transition type process.
Also when the spleen starts to get enlarged. Now, the spleen can be enlarged even in the setting of just ET or just PV, so spleen enlargement does not necessarily mean you’re transforming, but it can be one of the things that we would see that would indicate that.
Katherine Banwell:
Okay.
Dr. Jeanne Palmer:
And then finally white blood cell count increasing can often be a sign of that. Now, in terms of progression to AML, that is generally something we’ll see in the blood. AML or acute myeloid leukemia, is indicated by the presence of blasts at greater than 20 percent. Now, many patients with myelofibrosis, in particular, but even PV and ET, may have blasts in their peripheral blood. Blasts are normal. If I did a marrow on every healthy person out there, they are going to have some blasts, because these are the first part of the development of white blood cells. So, they’re like baby white blood cells. But what the problem is, is when they start to grow too much.
And so in the setting of myelofibrosis and even sometimes with these other diseases, the blasts will be in the peripheral blood primarily because the bone marrow is damaged and doesn’t hold them in very well. It becomes AML when it gets greater than 20 percent, so that blasts of greater than 20 percent in the peripheral blood or in the bone marrow but a lot of times we find it in the peripheral blood is where we indicate this has progressed to AML.
Katherine Banwell:
Yeah.
Dr. Jeanne Palmer:
Blasts of greater than 10 percent are also something that we really want to pay attention to, because that would suggest that the disease is starting to become more aggressive. Now, blasts vary, so for example, I’ve had patients go up to 11 and then drop back down to 3 or 4, and then they say around 3 or 4 or 5. So, you always want to make sure to double-check because one blast count at 11 percent, whereas it’s very important to address, may not necessarily reflect that you need to change in treatment at that time. Again, these blood tests, I always tell people, do not freak out over one blood test.
Make sure you get at least a couple of them to really confirm what you are looking at.
Katherine Banwell:
Thank you, Dr. Palmer. And to our viewers, please continue to send in your questions to question@powerfulpatients.org and we’ll work to get them answered on future webinars.
Dr. Palmer, was we close out this conversation I wanted to get your thoughts on where we stand with progress in helping people live longer and truly thrive with MPN. What would you like to leave the audience with?
Dr. Jeanne Palmer:
So, I think that the first thing is make sure you understand your disease. Don’t hesitate to ask for a second opinion. It’s always good to make sure you talk to someone who can really explain so you feel like when you go home you understand what’s going on in your body. Make sure you understand what symptoms to look for, what things to be aware of, because a lot of times people come in and they have no idea that, oh, these symptoms are actually related to their disease.
The other thing to make sure is that you’re very honest with your provider on how you’re feeling. A lot of times people come in and they say, “Oh, how are you feeling?” “I feel fine,” but then they start to ask very specific questions and they’re like, “Oh yeah, I’m really tired, my fatigue is an 8 out of 10,” or something.
So, make sure you’re really honest with your provider. When they ask you how they’re doing, this is not a social visit, this is a visit where they need to know your symptoms, so you don’t need to say I’m fine like you normally would if you were walking down the street.
The next thing is to always make sure to know where there’s clinical trials because we are making enormous great leaps and bounds in this field. It’s a really exciting time for myeloproliferative diseases, and there’s a number of new drugs that are being tested and coming out. So, it’s always important, if the opportunity is available and you can do it, clinical trials are a great way to get treatment.
Plus, you are giving back, because these are things that help us learn whether something works or not. So, you’re not as much a guinea pig, you never get a sugar pill. It’s one of those things you will always get the treatment you need and then they may add something to it or you may be in the situation where there is no treatment, so they try something.
But clinical trials, I have to emphasize, are a great way to get therapy and really are how we know everything that we know about treatment for these diseases.
Katherine Banwell:
Yeah. It sounds like there’s a lot of progress and hope in the field.
Dr. Jeanne Palmer:
Oh, absolutely.
Katherine Banwell:
Thank you so much, Dr. Palmer, for joining us today.
Dr. Jeanne Palmer:
You are very welcome, my pleasure, and it’s always fun to do these things, so thank you for having me.
Katherine Banwell:
And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell, thanks for joining us today. z
How Can MPN Patients Become More Proactive in Their Care?
How Can MPN Patients Become More Proactive in Their Care? from Patient Empowerment Network on Vimeo.
How can MPN patients become more empowered and active in their care? Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares advice for patients to gain confidence to become a more active participant for optimal care.
See More from Best MPN Care No Matter Where You Live
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Transcript:
Dr. Nicole Rochester:
So what advice would you give for patients so that they can really take a proactive approach to their healthcare and feel more confident in talking about their concerns and communicating with their healthcare team, you’ve shared with us how important that is. Do you have maybe two or three specific tips or maybe questions that every MPN patient should ask their healthcare provider?
Dr. Claire Harrison:
I think the first thing to say is, in my personal view is you do not have to be under an MPN expert to get the best care. I know some people differ with regard to that, but these are chronic conditions, there are national and international guidelines, clinicians are connected. We all talk about patients over time, as we like to do that, we like to get the best for our patients, so a local center with a clinician who you trust, who you get on with…where you can get there easily. You trust their team, you know their logistics work for you, maybe it’s a nurse who work who you get on with, well, who comes to the appointment with you, that is just as good as being under the best professor in the state, where you might not actually see them when you turn up and go to the unit, so that’s really important, understanding your condition, and if you don’t understand being empowered to ask questions, and if you’re in a position where you can’t ask a question, something’s wrong. So don’t be afraid, take somebody with you, write it down.
Sometimes it can be a mistake to do a troll on the Internet, so I wouldn’t always encourage that because what’s on the Internet is not always accurate, but go to a trusted website as the clinician…where can I go to find out more information? Some patient advocacy groups run buddy systems that can also be very helpful and it can be very empowering to meet another patient with the same or similar condition, so I think those are all helpful tips from my perspective, also don’t expect to get all the answers all the time, it can be really tricky as a clinician, maybe you get a patient who comes with a big long list of questions, and say What is your top question that you really want answers to.
Dr. Nicole Rochester:
Those are awesome, awesome tips. I’m just going to repeat a few of them, just to highlight, you mentioned prioritizing your concerns which is incredibly important, and acknowledging that the clinician doesn’t have unlimited time, and so really focusing on the things that concern you the most, you mentioned bringing a buddy to appointments, which is something I fully endorse, so that there’s someone else that’s taking notes or…it can be your eyes and ears during that appointment, things that you may have missed either because of anxiety or stress, and you mentioned writing things down, taking notes, even as the patient asking questions, which is so incredibly important, and really the way that I feel patients demonstrate their involvement in their disease and being an active member of the team, so I really, really appreciate those tips, Dr. Harrison, I think that you have given us so information, so much information about how to empower MPN patients and their families so that they can really get the best care at the outset.
How Can MPN Patients Stay Up to Date With New Treatments?
How Can MPN Patients Stay Up to Date With New Treatments? from Patient Empowerment Network on Vimeo.
What are some ways for MPN patients to stay updated on new treatments? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares resources for MPN treatment news and the benefits that data sharing brings for all MPN patients.
See More from Best MPN Care No Matter Where You Live
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Transcript:
Dr. Nicole Rochester:
Dr. Harrison, how can patients best keep up with the new treatments and communicate with their doctors in a way that makes sure that they have access to these new therapies?
Dr. Claire Harrison:
Well, I think patient advocacy groups are really important here, and use of social media and the Internet, you’re only a few clicks away from updated data, programs like these, but you also have to trust your team and trust your doctor. We all have to keep up to date. That is a professional requirement, and we also are all networked, so I’d probably get 10 to 15 emails a day from colleague saying, “Hi Claire, can I talk to you about this?” There was an email just the other day about a patient in Washington, actually a very young child, we are all connected.
We all want the best for our patients. But do you remember that you can contribute as a patient to advance this in your field, and I know many patients are really interested in this, if you are asked to submit in a blood sample, giving permission for us to use your data. So if we can touch maybe on the field of real-world data and real-world data collection here would be good, so what on the real world data? What does that mean? And so this is becoming a really important way that’s recognized by the FDA and other approval agencies in the world, so in Europe, we have EMEA, for example, and in the UK, we have NHRA as a way of collecting data on agents, so once they are approved, we collect data with regard to how the patients do.
We’ve traditionally done this, but increasingly, as we use electronic data for our patients, we’re more able to collect real-world data, how does my patient who is with myelofibrosis on ruxolitinib (Jakafi) in my clinic inside East London do? So if we can pull that data, we learn a lot more about how these agents are working in patients outside of clinical trial, so you will be contributing if you allow us to collect that kind of data, we discovered JAK2 mutations, CALR mutations, etcetera from samples collected from patients and data. If you want to be part of a clinical trial, then by all means, ask your healthcare team, many MPN centers have lists of trials, and you can always look at clinicaltrials.gov, but boy you throw up a lot of different options when you search in that…on that website.
Dr. Nicole Rochester:
Thank you. I think it’s important to talk about real-world data because in this day and age, many of us are very protective about our personal health information and we should be, but as you stated, having access to that data is really a key way to advance the science and technology in the treatment of some of these conditions, so I really appreciate you sharing that.
Dr. Claire Harrison:
I think just to point out, and I’m sure the audience is acutely aware of what did we learn about COVID? We learned an awful lot about COVID from real-world data from all you MPN patients who gave samples, who told us about how you did with COVID, that’s how we learned about what happened to patients during COVID with MPN, how they responded to vaccination, etcetera. It’s really powerful. And your data will be anonymized, it won’t be linked back to you, Nicole Rochester, or me, Claire Harrison it will be completely anonymous.
