Tag Archive for: Robert H. Lurie Comprehensive Cancer Center

Understanding Prostate Cancer Staging and Progression

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Understanding Prostate Cancer Staging and Progression from Patient Empowerment Network on Vimeo.

What are the stages of prostate cancer? Expert Dr. Maha Hussain provides an overview of prostate cancer stages and progression – and explains scans that detect disease to aid in optimal care.

Dr. Maha Hussain is the Deputy Director of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about this expert here.

See More From INSIST! Prostate Cancer

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Transcript:

Katherine:

Dr. Hussain, we’re going to spend most of this conversation talking about advanced prostate cancer. But before we move on, would you give us a brief overview of the stages of prostate cancer?

Dr. Hussain:

Absolutely. So, with any cancer, we count sort of like four stages. But I would say in prostate cancer the biggest thing is when the cancer is newly diagnosed, which could be confined to the prostate or locally advanced, meaning the cancer has gotten outside the capsule of the prostate but still within that pelvic region.

There is the group of patients who have pelvic lymph nodes at time of diagnosis. And of course, that is the patients who have systemic disease, which would be technically stage four. Now, the systemic disease implies any abnormality that is found on scans that is beyond the public region. So, that could be lymph nodes in the back of the belly. That could be thoracic lymph nodes. That could be neck nodes. That could be lung lesions, of course, or bone, or liver.

Now, the most common area where the cancer goes to is really – when we talk about metastatic disease – is the bone. And then lymph is another area where the cancer goes to. Prostate cancer that is confined to the prostate is curable in the vast majority of patients. There is a category of men who undergo surgery or radiation, and then their PSA begins to go up afterwards.

And this is what we call biochemical relapse. And this is a situation where we know that, in all likelihood obviously, especially of the patients who have had their prostate out, that the cancer has spread. With the current imagine, a good chunk of times, we do not find anything because we’re able to pick up PSA that goes from undetectable to 0.2 to 0.3, but there’s not enough cancer to show up on the scans. We’re hoping, obviously, the better scans, the PET Axumin scan, the PSMA scans are going to help us to identify sites of metastases.

But this is a group of men where if there is no cancer visible and the only thing we’re dealing with is PSA that’s going up, if they’ve had surgery, then

there’s room for what we call salvage therapy with radiation and hormonal treatment. The case is a bit different if there’s only just the prostate – if radiation was given previously. And of course, we talked about metastatic disease.

Katherine:

Once someone has been diagnosed, what tests are used to help understand the aggressiveness of their disease and their overall prognosis?

Dr. Hussain:

Well, I think there is different basic things, as in, what was the extent of the cancer? How did it look under the microscope? And what is the PSA levels? So, these are the general things. There are different sort of genomic panels that the urologist will use to kind of decipher and other things to kind of help with figuring out aggressiveness and things like that. What I would say is this, is a patient who is diagnosed and has a cancer, and at a minimum has what we consider a Gleason 7 prostate cancer – so, that’s the scoring system that is done with the original Gleason score, or the new patterns where it’s talking about intermediate risk to high risk – to me, this is a cancer that needs to be treated.

And again, that’s all to do with if a person has other comorbidities, they have some other terminal condition that’s a separate story. But talking generically, that would be when we would recommend. And these are the patients that are generally not seen by the medical oncologist. They’re seen by the urologist, and then they can refer them to radiation oncology also for consultation.

Is the COVID Vaccine Safe and Effective for Prostate Cancer Patients?

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Is the COVID Vaccine Safe and Effective for Prostate Cancer Patients? from Patient Empowerment Network on Vimeo.

 What do prostate cancer patients need to know about COVID-19 vaccines? Expert Dr. Maha Hussain discusses COVID-19 vaccine safety and effectiveness — and what she’s seen with COVID-19 vaccination with her patients.

Dr. Maha Hussain is the Deputy Director of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about this expert here.

See More From INSIST! Prostate Cancer

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COVID Vaccines: What Do Prostate Cancer Patients Need to Know?

Transcript:

Katherine:

Is the COVID vaccination safe and effective for prostate cancer patients?

Dr. Hussain:

The answer is yes and yes. So, I have to say, by default, I deal mostly with older men. Age brings in other comorbidities. And certainly, while I see all kinds of shades of gray in terms of the disease extent, going all the way from newly diagnosed all the way to end-stage disease, the bulk of the patients I end up seeing tend to have more systemic disease and have other issues going on. And I have to say, surprisingly, less than a handful of my patients had the infection.

Only one required hospitalization with supportive measure, but not even needed incubation; however, he needed a lot of CPAP and other respiratory support. I’m not aware of any of my patients or my colleague’s patients who deal with prostate cancer that have died from COVID. So, I would say that’s the good news and that we have not seen a big hit in the population that I deal with.

I also know that I would say 99.9 percent of my patients have opted to be vaccinated, and they have tolerated the vaccine just fine. There’s only one case, which I actually even saw just this week, who had been vaccinated but have a very, very severe end-stage disease with significantly compromised bone morrow, who got infected but hospitalized for a few days and is recovering.

And so, I would say just by the pool of patients I see, my answers are yes and yes.

Katherine:

Very good. Thank you.

Dr. Hussain:

And I would encourage all the audience to go get vaccinated. I myself am vaccinated. And I’ve advised all my family members to be vaccinated, just to clarify that too.

Prostate Cancer Treatment and Research News

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Prostate Cancer Treatment and Research News from Patient Empowerment Network on Vimeo.

Prostate cancer experts recently gathered at the American Society of Clinical Oncology (ASCO) annual meeting to share research updates. Expert Dr. Maha Hussain reviews clinical trial findings presented at the meeting, potential treatments for FDA approval, and credible sources for prostate cancer research information.

Dr. Maha Hussain is the Deputy Director of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about this expert here.

See More From INSIST! Prostate Cancer

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Transcript:

Katherine:

I’d like to start by asking about developments in prostate cancer research and treatment. Experts recently gathered at the annual American Society of Clinical Oncology meeting, also known as ASCO, to share their research.

So, what were the highlights from that meeting that you feel patients should know about?

Dr. Hussain:

I think probably perhaps I can focus on two major – what I would consider major highlights, and those were the results from two randomized Phase III clinical trials.

One of the trials is called the VISION trial. And the VISION trial was a Phase III randomized trial evaluating lutetium-PSMA-617 treatment in patients with metastatic castration-resistant prostate cancer. And the delightful thing about this study is that that study was positive. The PSMA story has been really going on for a few years now. And there’s the PSMA for purposes of scans, imaging, to assess the cancer. And the FDA just approved a PSMA PET imaging this year.

I think it was in May when it was approved. And that would help better define if the cancer is spread or not, and it help with the decision regarding treatment. But the second part is treatment purposes, so identifying the cancer location and trying to attack it with a specific sort of targeted attack to the tumor is really important.

And so, the FDA is currently looking at this particular agent. And I am hopeful that we will hear soon from the FDA, hopefully before the end of the year, and maybe – who knows? – maybe by summer, middle summer or end of summer. Because I do think that would be a major benchmark in there. And so, that’s one thing.

The other clinical trial that I thought was interesting from a data perspective – and for disclosure, I am one of the investigators on this study. And this was an intergroup Southwest Oncology, or SWOG, sponsored clinical trial. So, it’s a federal study that Dr. Aggarwal presented. And this was a study that was aiming at maximizing, again, the anti-tumor therapy with the use of a drug which I call is the younger brother of abiraterone.

So, abiraterone is a drug that is FDA-approved and has been around for several years right now for both castration-resistant prostate cancer and certainly hormone-sensitive metastatic disease. And so, TAK 700 (Orteronel) is a younger brother, I call it, of abiraterone (Zytiga). And one of the potential advantageous when this trial was designed was the fact that you don’t need to use prednisone. And the trial was completed. It was a national clinical trial. And what was interesting is that there is certainly what appears to be a potential benefit, but not in terms of the conclusive based on the way the study was designed.

Having said that, what I thought was remarkable is that patients who basically were only on the control arm was LHRH therapy, so this could’ve been like leuprorelin (Lupron), goserelin (Zoladex), or something like that plus bicalutamide, which is what we call combined androgen deprivation. And that was sort of like the strongest control arm we could do at the time when the trial was designed.

Remarkably, the patients who were on that arm had a median survival of basically 70 months. That’s the median. That’s the bell-shaped curve with the number in the middle. Seventy months is probably the longest ever in any other randomized trials in this disease space, in the hormone-sensitive space. So, that tells us is that men are living longer with prostate cancer, even though it’s metastatic disease; and, yes, it’s not necessarily curable, but men are living longer. And it’s a function of all of the better treatments that are supportive care and everything that was going on.

And so, the control arm, as I mentioned, was the 70.2 months. The actual experimental arm was about 81.1 months. And again, I don’t know where things will go from this. Obviously, I’m not the sponsor not the FDA. But the point here is that men are living longer, and so wellness and health become even more so important than we ever did. And as I tell my patients, every day you’ll live longer. The odds of living longer is there because of better treatments coming on.

So, to me – not to take too much time from the interview – to me, these were the two highlights: new, approved – I’m sorry, new treatment that I’m hoping will be FDA-approved and, obviously, the fact that men are living longer.

Katherine:

How can patients keep up to date on the research that’s going on?

Dr. Hussain:

I’m a bit biased, obviously. I’m a member of ASCO.

And what I would recommend to my patients is to look at the cancer.net website. The cancer.net is a website that is an ASCO-generated website specifically for patients and families to review. It is vetted. The committees are not run just by physicians, oncologists, a multidisciplinary team, but also patient representative. So, the lingo and the presentation are lay-friendly, I call it, there.

The other part I would say, the NCI website, and the American Cancer Society, the American Urological Association. I would say there’s a lot of stuff on the media. The difficulty is vetting what is sort of fake, what is not so accurate, or bias versus there. I also think that the NCCN has also some resources for patients.

And one thing I always tell patients: explore, look, but make sure that you talk to your doctor about the meanings of everything because sometimes it can be not – it could be misleading, I should say, or maybe not very clear on what the implications are.

Shared Decision-Making: The Patient’s Role in Treatment Choices

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Shared-Decision Making: The Patient’s Role in Treatment Choices from Patient Empowerment Network on Vimeo.

What is the role of the patient when it comes to treatment choices? Dr. Brady Stein details how he partners with patients in decision-making for their MPN care. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Transcript:

Katherine:                  

What do you feel is the patient’s role in the decision for therapy?

Dr. Stein:                   

I think it’s a really important role. I think historically – and, this is decades past; this era should be well over and behind us – this era of authoritative medicine is over.

You can’t just have a doctor walk in the room and say, “This is your treatment, this is what you should do, I’ll see you later.” It’s shared decision-making, and that can be troubling for some patients. But, the idea of shared decision-making is us explaining options informing the patient and making decisions together. That’s really the paradigm for modern contemporary medicine.

Some patients have a harder time with that. A lot of patients say, “Well, doc, this is too overwhelming for me. I just want you to decide for me.” And, we try not to do that. That’s a more uncomfortable type of visit for me when a patient is very deferential and says, “Whatever you say, I’ll do.” That’s not really what we want to hear. I want to know that you feel really informed, that you have a good understanding because each of these treatments – any treatment, any medication has its pros and cons.

There are no real magic bullets, and each upside has an equal downside, so you have to engage and open a dialogue, and what that means is that patients need to read and learn. That’s hard, but patients need to become proactive in their approach to their own illness, and all the patients who are listening now are doing that, trying to get more education about your relatively rare illness that’s going to give you a much better framework to help make decisions together.

Katherine:                  

Absolutely. If a patient isn’t feeling confident with their treatment plan or their care, do you recommend that they maybe consider a second opinion or seek a specialist?

Dr. Stein:                   

Of course, yeah. These are rare diseases, and patients often – I would say that in my clinic, a lot of the patients direct their own second opinions. Oftentimes, it’s coming from the patient more so than their doctor. I think the patient community is very active, the patients are networking, and they’re finding the right specialist to get to.

I think it should be really a team approach. It’s never – it’s usually not very convenient to go to a university unless you live really close, so you want to have someone close to home who can handle the routine, and then, someone who maybe is a little bit further away who can see you once a year, can help with the big decisions, can be part of the healthcare team. So, we generally recommend that you have someone near, and that maybe you have someone far who focuses only on MPNs as part of your team, and now, it’s a little different. Telemedicine is becoming a pretty ingrained part of medicine. It’s a little easier to have those visits with a physician who’s far away because of telemedicine.

Self-Advocacy: Advice for Being a Pro-Active MPN Patient

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Self-Advocacy: Advice for Being a Pro-Active MPN Patient from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) patients be more pro-active in their care? Dr. Brady Stein shares advice to help patients educate themselves about the disease, while finding the right balance of knowledge to prevent them from feeling overwhelmed. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Transcript:

Katherine:

Let’s talk about patient self-advocacy now, Dr. Stein. Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions.

Why is it important for patients to speak up when it comes to symptoms and side effects?

Dr. Stein:                   

I smile a little bit because patients – I get a lot of patient emails by MyChart. That’s our medical record, and it’s a secure patient email, and a lot of patients will start their message by saying, “I’m sorry to bother you.”

And, I always say, “Why do you think that? It’s my job. Please don’t apologize for reaching out to me.” So, that’s kind of the first thing. Don’t feel like you’re bothering your doctor. There are certain things that we won’t know unless you tell us, and so, I think that’s pretty clear. When we’re in a patient room and there might be a husband and wife together, and whether it’s the husband is the patient or the wife is the patient, we might ask a question, and we might get, “No, everything is fine,” but all doctors kind of sneak over to the partner, and the partner may be saying – they’re making gestures to us. There may be nonverbal forms of communication to tell us there’s something much worse than what the patient is telling you.

So, again, “advocate” meaning you have to tell us what’s going on with you. If you’re worried about something, please don’t be stoic about it. These diseases are treated a lot based on your symptoms, and so, if you don’t tell uls about your symptoms, we won’t know.

And, in terms of advocacy, I think one of the things is that these are pretty rare diseases. In an academic center, no, this is our focus, but if you’re in a community practice where the doctor’s seeing 10-15 different things during the course of a day, it’s basically impossible to keep up with myelofibrosis, especially if you have one patient in your whole practice. I can’t do that for diseases that I see that I have only one patient. The medical literature can be overwhelming.

So, patients can quickly outpace their doctor in terms of their knowledge of these diseases, but I think it’s really important to read, to learn, and to think about the illness because you may find out things through your research that your doctor wouldn’t know are available. You may find a clinical trial, a new strategy, or a new test that they simply haven’t had the time to keep up with or learn about. So, that’s what advocacy is about. Reading is really important, but you have to find a balance. I want my patients reading, but you’ve got to find the right amount because there’s a certain amount of reading where the patients start to get overwhelmed.

All patients kind of get to this point. They take it in – like taking it in like a fire hydrant in the beginning of the disease, and it’s overwhelming, and then they start to find their balance. I think there’s a point where the reading becomes anxiety-provoking rather than ameliorating anxiety, and all patients just generally find their balance.

Is My MPN Treatment Working?

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Is My MPN Treatment Working? from Patient Empowerment Network on Vimeo.

During myeloproliferative neoplasm (MPN) treatment, specific blood tests and diagnostic measurements help to gauge a patient’s treatment response. Dr. Brady Stein details the criteria he assesses in monitoring the efficacy of a therapy, including patient-reported outcomes.  

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Transcript:

Katherine:                  

Once a patient has started treatment, how do you know it’s working?

Dr. Stein:                   

That’s a good question because this is a very unique area. Yes, of course, in some respects, it’s straightforward with ET or PV. If we’re starting a medication to control a blood count in hopes of having lowered the thrombosis risk, you can look objectively at blood counts.

Okay, your hematocrit is at this goal? Yes, therapy’s working. You have not had a blood clot?

Yes, therapy’s working. So, there are some objective things. In myelofibrosis, there are some objective things like measuring the spleen and seeing it reduce. You can feel that with your hands, or you can do an ultrasound. So, there are some objective parameters of success. But, in this area, patient-reported outcomes are really important, and so, a measure of success is really just asking the patient, “Do you feel like your drug is working? Do you feel better?

It’s kind of a simple question, but it’s really important, and it’s what we ask in patients who are on certain therapies. “Do you feel like the net effect of your therapy is still positive? Do you feel like it’s helping?” Seems like a straightforward type of question, but I think the answer is extremely informative. When a patient says, “Yes, definitely, my medication is still helping me,” then I know that I don’t need to change it.

MPN Treatment Choices: Where Do Clinical Trials Fit In?

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MPN Treatment Choices: Where Do Clinical Trials Fit In? from Patient Empowerment Network on Vimeo.

When considering MPN treatment approaches, clinical trials are a viable option for care. Dr. Brady Stein discusses clinical trials and factors to keep in mind when considering participation.

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Promising ET, PV & Myelofibrosis Therapies in Development 

What Are the Treatment Options for Myelofibrosis?

Transcript:

Dr. Stein:                   

Clinical trials are always a treatment – always an option for patients with myeloproliferative neoplasms because while we have some standards, we can definitely improve upon those standards for certain. So, clinical trials are always a therapeutic option. I think the one thing is that it may not – it’s not always the most convenient option, but it could be a really important option if available to you.

So, clinical trials basically offer something new or novel that would not otherwise be available to other patients. So, ruxolitinib (Jakafi) was approved around 2011, but the first clinical trials were in 2007, so that’s the example I give to a patient about the benefit of a clinical trial.The patient can get access to a drug that’s effective perhaps three to four years before it’s commercially available.

That’s really the biggest advantage, is you can get early access to something that could really help you. The downsides are that clinical trials are not usually as convenient as regular care, there are often more visits, and there are a lot of unknowns – unknowns about whether it will work. Some side effects are known and expected; there are others that are unknown. So, it’s a lot to think about, but I think it’s always important to consider, especially if your first-line therapy has not been effective, if it’s losing its touch, it’s a good thing to think about for a second line.

Katherine:                  

Are there emerging approaches for treating MPNs that patients should know about?

Dr. Stein:                   

Yeah, absolutely. I think the first question – I think patients are often worried that they have a really rare disease, and why would anyone do research in this area, and that’s – the research community is extremely engaged, the productivity is pretty impressive, and there’s a lot of clinical trials in the space, and I think what I try to explain is pharmaceutical companies aren’t just targeting the most common diseases.

They have interests in rare diseases, and findings in rare diseases can be extrapolated to other diseases that you might think are unrelated, but they can share features, so when you find something working in one space, it can have broad applicability. So, there’s an abundance of research in myeloproliferative neoplasms which are emerging?

In PV, I think there’s quite a possibility that there’ll be a drug approval in 2021, a novel type of interferon called ropeginterferon

That is a drug that’s approved abroad; it’s approved in Europe, and I believe it’s approved in Taiwan, and the FDA is looking at it now. So, it’s a possibility that there’ll be a future option for patients with polycythemia vera. So, yes, it’s research now, but it could be available, and so, that’s the drug that I’m starting to talk more and more about for patients with PV.

In myelofibrosis, you have two JAK inhibitors that are approved, ruxolitinib and fedratinib, you have two others in clinical testing, momelotinib and pacritinib, and then you have a whole other class of what we call non-JAK2 type of therapies targeting the vast array of pathway abnormalities in myelofibrosis.

So, there are a number of different clinical trial options, especially in myelofibrosis. I think that’s the disease area where there are the most clinical trials.

Understanding High-Risk vs Low-Risk Disease in ET, PV & MF

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Understanding High-Risk vs Low-Risk Disease in PV, ET & MF from Patient Empowerment Network on Vimeo.

When looking at polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), how is risk determined? Dr. Brady Stein explains factors he examines when assessing risk to provide ideal care for each patient. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

Related Resources

MPN Symptom or Treatment Side Effect? Know the Difference

Monitoring MPNs: When is it Time to Switch Therapies?

Promising ET, PV & Myelofibrosis Therapies in Development 

Transcript:

Dr. Stein:                  

For ET and PV, when we talk about high versus low risk, we’re talking about vascular complications, risk of having a blood clot. We’re not really talking about risk of transformation. We don’t have, I think, wonderful, widely used toolkits to predict those things. We know they can happen, but our treatment is still really based on clotting for ET and PV.

And, MF – each couple of years, the tools that are available to assess prognosis become more and more. So, in MF, we’re using the most comprehensive approach – of course, taking into account things like age and demographics, but also, looking at symptoms, looking at the depth and severity of blood count changes, looking at bone marrow features like the degree of scarring, looking at the rise in blast counts, and then, looking at chromosomes and novel genetic markers. So, we’re definitely the most comprehensive in myelofibrosis at assessing prognosis.

What Are Treatment Options for Essential Thrombocythemia (ET) & Polycythemia Vera (PV)?

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What Are Treatment Options for Essential Thrombocythemia (ET) & Polycythemia Vera (PV)? from Patient Empowerment Network on Vimeo.

When considering treatment options for essential thrombocythemia (ET) and polycythemia vera (PV), where do experts begin? Dr. Brady Stein details treatment considerations and how he determines the best approaches for ET and PV patients. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

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Which MPN Treatment is Right for You? Factors to Consider

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MPN Treatment: Why Testing for Mutations Matters

Transcript:

Katherine:                  

Let’s start with essential thrombocythemia, or ET.

Dr. Stein:                   

So, I think the first thing is taking an inventory of symptoms, seeing how symptomatic the patient might be. Again, there are some patients who are asymptomatic or have few symptoms, and they were told of a high platelet count during a routine visit, so some patients can be observed if they have few symptoms, and especially if they fall into a lower vascular risk category.

So, symptom assessment first. Second, looking at vascular risk, and there are four categories of risk in in ET in terms of predicting the likelihood of a future blood clotting event. There’s a very low, low, intermediate and high risk group, and that’s based on a patient’s age, whether they’ve had a blood clot before, and the type of mutation they have. JAK2 mutations increase the risk of clotting.

So, if a patient falls into a higher-risk group – say they’re older than 60 with a JAK2 mutation or they’ve had a prior blood clot – those are patients who are generally treated more aggressively with cytoreduction.

And then, the other thing is aspirin. We often see aspirin given to all patients with ET, but not all patients with ET necessarily need it. The role of aspirin is actually a little less clear in ET. For a very low-risk patient, there’s a potential for more harm than benefit, especially if the patient lacks a JAK2 mutation. So, the evidence base to support aspirin for all ET patients is just not there; it’s evolving.

Katherine:                  

What about polycythemia vera, or PV?

Dr. Stein:                   

So, there are a few standards. It’s different – the aspirin question in PV is generally answered by randomized data from 16 years ago in 2004. It’s been shown that aspirin reduces the risk of clotting in PV patients, so, generally, we give low-dose aspirin to all patients. And, hematocrit control is really important.

At least, a goal of 45 percent is mandated in PV. And then, there are patients who might fall into a higher-risk category – older than 60 or have had a prior blood clot – they need something more. And then, I’d also emphasize that there are lower-risk patients who may not be traditional candidates for cytoreduction, but they could have symptoms that really interfere with quality of life, and symptoms alone can be the trigger to add something more to the phlebotomy and aspirin program.

Katherine:                  

What about things like interferon?

Dr. Stein:                   

So, interferons have been used in MPNs for decades and decades. So, a longstanding history with interferons. The issue has been tolerability.

These days, there’s a class called pegylated interferon that’s longer acting, and I think there’s been a lot more use, at least in the last 10 years, still much more in an academic setting than a community practice.

But, interferons have a pretty established role in MPNs, especially polycythemia vera, for sure in ET, less so in myelofibrosis.

What Factors Guide Treatment Choices for ET, PV & MF?

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What Factors Guide Treatment Choices for ET, PV & MF? from Patient Empowerment Network on Vimeo.

When making a myeloproliferative neoplasm (MPN) treatment decision, several factors come into play. Dr. Brady Stein explains what criteria he considers to determine the optimal approach for a patient’s unique situation and specific MPN.

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.

Related Resources

Monitoring MPNs: When is it Time to Switch Therapies?

An Expert Shares Key Steps to Take Following an MPN Diagnosis

MPN Treatment: Why Testing for Mutations Matters

Transcript:

Katherine:              

Dr. Stein, I understand that therapy is different for each of the MPNs. What do you take into consideration to help guide the treatment choice?

Dr. Stein:                   

So, that’s a good question. It’s going to require maybe a little bit of a longer answer. It’s fairly nuanced. The treatment of ET and PV – right now, the goals are – the treatment is largely based on a patient’s vascular risk. That’s largely what influences the choice of therapy. And so if a patient has a perceived high risk for vascular complication in ET or PV, that’s when we’re going to be a little bit more aggressive – so, more aggressive than watchful waiting, more aggressive than using aspirin alone, more aggressive than using phlebotomy and aspirin alone in polycythemia vera.

So, if the patient has a higher vascular risk, in general, we’re going to need to do something more than what we consider to be the standard, and that’s where we enter into the question of cytoreductive therapy – therapies designed to lower blood counts apart from phlebotomy.

Maybe that’s going to change. I hope it will change. Right now, the therapy for ET and PV is generally reactive. We either predict high risk and react, or if a patient is lower risk, if something changes – God forbid there’s a blood clotting event – then we may react to it.

So, ET and PV treatment are generally more reactive. In myelofibrosis, certainly, there are patients who can have lower risk and minimal systems, and there are some patients who can be observed with watchful waiting for sure, but more patients are symptomatic, more patients are going to need therapy in myelofibrosis, and there’s sort of two big categories of therapy.

One is the risk-adapted, deciding if the patient is eligible and should consider stem cell transplant versus thinking only about medical therapy in a patient that may be transplant-ineligible.

And, the medical therapy is based on the worst symptoms for the patient. Is the symptom that’s the worst the spleen enlargement? Is it excessive fatigue? Is it weight loss, or inflammation, or fevers? If it’s that category of symptoms, we have a set of therapies. If it’s really the anemia that is the most problematic issue, then we follow a paradigm to treat anemia.

Katherine:                  

What about considerations like the patient’s health, age, genetic markers, things like that?

Dr. Stein:                   

So, of course. The comorbid illnesses can influence therapy choices, so if a patient is older and has other medical conditions, they’re not going to be treated as aggressively.

So, in myelofibrosis, if a patient is older, with other medical illnesses, then it may be inappropriate to consider something like stem cell transplant, for sure. So, age and health comorbidities are highly influential. In terms of genetic features, if you’re asking about things like the type of mutation that a patient has, right now, we’re – in terms of vascular risk, for ET, the type of mutation matters for blood clotting risk, so if patients have different mutations, it could be treated differently. In other subtypes, like PV or myelofibrosis, in general, there’s – the mutation can be prognostic, but it may not be – it may not lead to a precise and distinct therapy just yet.

How Do Test Results Inform MPN Prognosis & Treatment?

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How Do Test Results Inform MPN Prognosis & Treatment? from Patient Empowerment Network on Vimeo.

Dr. Brady Stein explains the diagnostic tests and genetic mutations that are assessed to determine prognosis and what MPN treatment may work best.

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. 

Related Resources

Which MPN Treatment is Right for You? Factors to Consider

Resource Guide: Choosing an MPN Treatment: What Option is Best for You?

MPN Treatment: Why Testing for Mutations Matters

Transcript:

Katherine:                  

Once a patient is diagnosed with MPN, what sort of testing should take place?

Dr. Stein:                   

So, the test that’s going to lead to suspicion is going to be a blood count, and that’s probably going to be done in the primary care doctor’s office, so that’s going to be the first suspicious test, and in general, there’s going to be some abnormality. Myeloproliferative diseases are characterized in general by an overproduction of blood cells, so it’s going to be a higher white count, it’s going to be a high hemoglobin or hematocrit, or a high platelet count, or a combination of the three that’s generally going to lead to suspicion.

Some patients may have pretty unremarkable blood counts and may present with a blood clot in an unusual location that could ultimately lead to the hematology referral. Some patients might have pretty unremarkable blood counts, but they might have palpation of their spleen, enlargement of their spleen in a physical examination. So, they’re generally the ways that patients are getting to the hematologist.

Katherine:                  

And, what about bone marrow biopsy?

Dr. Stein:                   

So, a bone marrow biopsy is a diagnostic test, and it’s generally recommended for all patients who have a myeloproliferative neoplasm either confirmed or suspected.

It’s advised in WHO criteria – World Health Organization criteria. PV can be made without a bone marrow biopsy – a diagnosis of PV – because it’s the most unique of the MPN subtypes. It’s the one that presents with a high hemoglobin.

So, that diagnosis can be straightforward at times for a hematologist when the setting is right, when there’s a high hemoglobin – or, high enough hemoglobin, I should say, a JAK2 mutation, which all patients with PV have that, or a subnormal erythropoietin level.

Oftentimes, we can make that diagnosis without a bone marrow, and the bone marrow becomes more prognostic. ET, a bone marrow is necessary for diagnosis, and myelofibrosis, you can’t make a diagnosis without it.

Katherine:                  

What do the results of these tests tell us about prognosis and treatment choices?

Dr. Stein:                   

That’s a good question. The bone marrow can be diagnostic in the setting of ET and myelofibrosis. In the setting of polycythemia vera, it can be more prognostic. In general, when a bone marrow is done, 1). To confirm diagnosis, 2). To assess prognosis, what we’re looking for as prognostic features are generally the degree of fibrosis or scarring.

So, each of the MPNs can have that. Of course, MF is characterized by the most pronounced scarring. It can happen to a more subtle degree in ET and PV. That’s going to be prognostic in the setting of ET or PV. The pathologist will alert us about immature cells called blasts.

We basically never see them at diagnosis in patients with ET or PV. We can see them rise in patients with myelofibrosis at diagnosis or through the course of follow-up. So, that’s prognostic.

All bone marrows generally have a chromosome analysis that’s called cytogenetics, and so, if there’s an abnormality, that can help place the patient’s prognosis into different risk categories.

And then, nowadays, more so in myelofibrosis than any of the others, there are extended panels done. These are called NGS, or next-generation sequencing, kind of looking at mutations in a greater degree of detail.

So, not just what we call main mutations – JAK2, calreticulin, or MPL. These are looking at additional mutations that basically hold prognostic significance.

These are pretty well defined, and I think more important in MF compared to the other subtypes.

Katherine:                  

Would you explain the driver mutations in MPNs? What are they, and how they – or, what they mean for patients?

Dr. Stein:                   

So, there’s three of what we call driver mutations, and the most common is JAK2V617F, the next most common is calreticulin, and the least common or most rare is a mutation of MPL, the thrombopoietin receptor. So, the driver gene mutations are the three that we assess to help with diagnosis, and the prevalence varies. In ET, about 60 percent have JAK2, 25 percent have calreticulin, 5 to 10 percent have MPL.

In PV, 99 percent have some type of JAK2 mutation, and in MF, the situation is a lot like ET – 60 percent JAK2, 25 percent calreticulin, about 5 to 10 percent MPL. So, the driver mutations – we think of those as the genetic abnormalities that really drive the disease. They’re the main ones we can test for in a diagnostic setting.

I refer to them as the – to a patient, what I’m describing is you have a car, and the driver mutation is the one that’s sort of driving the car, and it’s doing it somewhat recklessly. It’s in the front seat, driving. And, along the way, the driver can pick up hitchhikers, which we should never do. I refer to those other mutations that are found by NGS as hitchhiker mutations that sit in the back seat, cause trouble, and really shouldn’t be there. They’re not the driver, they’re not fully responsible for the disease, but they can make it a bit worse.