Skip to Navigation Skip to Search Skip to Content
Search All Centers

How Do You Choose Which MPN Treatment to Use When?

Read Transcript Download/Print Transcript
View next

Published on August 4, 2015

How does one decide therapy after a diagnosis of MPN? How does one begin to weed through the treatments that are considered older, versus those that are newer and might be more potent? At an MPN town meeting in Chicago, Dr. Brady Stein from Northwestern Medicine and Dr. Stephen Oh from Washington University School of Medicine discuss how they approach treatment decisions in patients with MPNs. 



Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Transcript | How Do You Choose Which MPN Treatment to Use When?

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

Susan Leclair:

Dr. Stein, everybody who gets any serious disease wants a magic bullet.  They want to take a pill and make it all go away.  We’ve had huge changes in the medications that are available, and yet we periodically fall back on phlebotomy and hydroxyurea (Hydrea) as standards. 

And then there are other, the newer ones that are far more targeted to this particular disease or this particular stage.  How do you choose?

Dr. Stein:               

That’s a very good question.  First of all, it’s very difficult to choose, because each patient is very, very unique.  This is a very heterogeneous spectrum of illnesses.  And so if you’ve seen one patient with ET or one patient with PV or one patient with myelofibrosis, then you’ve seen one patient with each of those diseases.  Everyone is unique, and everyone has different needs.

So what we have to try to do is tailor to those needs.  So I think I’ll start with polycythemia vera, because there are certain cornerstones of therapy that everyone agrees upon, virtually everyone agrees upon.  So for polycythemia vera, there are things that we know we really have to do no matter who the patient might be.  They’re our cornerstones of management.  And that is phlebotomy—a very historical treatment of course but very relevant for polycythemia vera.  And we know that we have a pretty clear target.  We have to lower the hematic at below 45 percent.  We know that helps patients. 

We know that can prevent from cardiovascular complications so all patients.  The second thing we know in polycythemia vera is that for almost all patients, unless a patient can’t tolerate it, aspirin can help those patients.  So for polycythemia vera, there are some straightforward things that whether you’re older or you’re younger, whether you’re at lower risk or you’re at higher risk, whether you have certain symptoms or not, these are cornerstones. 

So I think it’s straightforward there.  Whether we decide to add something or not for polycythemia vera, depends on what we perceive the risk of the disease to be for you.  And so when we make decisions about getting more aggressive than phlebotomy and aspirin, we are trying to predict whether your patient is more likely or less likely to have a blood clot.  And if you’re more likely, we may be more aggressive and we may look to a medication like hydroxyurea, a medication like interferon.  

We also have to look at symptoms.  And so this is becoming—it’s very clear in myelofibrosis. 

I think it’s becoming more clear in ET and polycythemia vera is that we have to look at the subjective and not just the objective.  We can’t just look at the blood counts and measure the spleen, we’ve got to look at the burden of symptoms that each individual has.  So our patient with polycythemia vera might have intractable itching and that may be something that we have to treat.  They may have symptoms from an enlarged spleen.  That may be something we really have to treat.

One of the symptoms that is almost—it’s very, very common, universal perhaps in myelofibrosis, but very common in ET, is fatigue—a very difficult symptom to treat.  So it’s a great question about how we make decisions about treatment.  I thought I’d focus on polycythemia vera, and, Stephen, if you want to talk about how you might approach myelofibrosis.

Dr. Oh: 

Sure.  So myelofibrosis is a disease that particularly until recently, we simply didn’t have great options.  And as Brady mentioned, while we are beginning to more and more appreciate symptoms in patients with PV and ET, in myelofibrosis there’s no question that many patients have quite extensive symptom burden. 

So the treatment options are—hydroxyurea is certainly an option.  What it’s particularly effective for in the setting of myelofibrosis is for elevated blood counts—so leukocytosis, elevated white blood cell count; thrombocytosis, elevated platelet count, etc.  These patients often have very enlarged spleens, and that can cause symptoms.  Hydroxyurea can shrink the spleen at least to some degree.  It’s not typically that effective for very enlarged spleens. Other options historically, interferon.  So that is a drug that particularly in the last several years really has sort of seen this resurgence, both for myelofibrosis, as well as for the other MPNs. 

And it can be a very effective drug, particularly for those who have more sort of early stage myelofibrosis.  If the patient has more advanced disease, extensive splenomegaly, interferon tends to not be as effective. 

There are practical issues as well in terms of side effects and also cost for some patients.  More recently, we have had the development of targeted inhibitors of JAK2.  So one of those drugs, ruxolitinib or Jakafi has been approved specific for myelofibrosis. 

That was in 2011.  And there are a number of other inhibitors of JAK2 that are in various stages of clinical development. 

And what we see with ruxolitinib, as well as the other drugs in this class that are in clinical trials pretty consistently is that they’re very effective for symptoms and specifically what we call constitutional symptoms—so fevers, night sweats, weight loss, fatigue.  In some cases, patients will come back. And a few months after starting treatment and they’ll say, I’m a new person; it’s been a miracle drug for me.  And that is, of course, very impressive to see, and we’re very happy for that, for the patients.

Also, the spleens that can become massively enlarged, in some cases they shrink quite considerably.  These drugs work, ruxolitinib and some of the other drugs in their class, quite consistently to the point that we expect to see at least some degree of that benefit in virtually every patient.  There are some who just don’t benefit, but most get at least some benefit in that regard.  So the use of these drugs has really become more and more commonplace and particularly for symptom benefit in myelofibrosis, very effective.

Susan Leclair:     


Dr. Stein:               

And I also want to—there are people with ET in the audience, and I want to talk a little bit about ET.  I think ET presents some unique challenges in terms of treatments.  First of all, with ET, we’re really—the treatment has to be as benign as the disease can be for some patients. 

So with ET, one of the things we see a lot in our clinic [is] more trouble from the therapy than the disease.  We have to be mindful of that.  So we have to think very carefully about which patients with ET really need treatment.  So, again, we’re looking at mainly who we can predict may have a blood clot.  And so we’re looking at some things, which are unfortunately not very individualized or not very personalized.  We’re looking at your age, and we’re looking at whether or not you’ve had a blood clot, and we’re making decisions based on those two things, whether or not you need to be aggressively treated.

And aggressively treated is using a medication like hydroxyurea or anagrelide (Agrylin) or interferon.  With aspirin, I think that things have changed a little bit with aspirin.  I think there are some people who universally recommend aspirin for ET.  There are others who use aspirin only if you have a JAK2 mutation.  There are others who only use aspirin if you have symptoms; symptoms that we think are related to small vessel disturbances, migraines, visual symptoms, burning in your hands or your feet, which we call erythromyalgia.

So for ET, we have to look at a number of different things.  One point I’d like to make is that while the focus of ET is often, if not always, the platelet count in some offices, we don’t always have to treat based on the platelet count alone, unless the platelet count is causing trouble.  So I think that’s somewhat of an important point for ET.  So the treatment landscape is as complex in ET about when we make a decision about who to treat or not.  

And what’s changing here is that we’re trying to be a little bit more personalized in our approach, trying to be better about predicting who may or may not have a blood clot and using things like age, using things like whether or not you’ve had a blood clot are important but also looking at your cardiovascular profile.  Do you have high blood pressure, diabetes? Are you smoking?  And then looking at the type of mutation that you may carry, and there [are] three major ones.  So we’re trying to move from the general a little bit more to the specific with ET when we make treatment decisions.  

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

View next