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Watchful Waiting and Decision-Making with MPNs

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Published on November 6, 2020

A Doctor’s Advice on the Watch and Wait Period for MPNs

Whether you have recently been diagnosed with an MPN or you have been living with one for many years, you have likely heard of or been placed on “watch and wait” for your diagnosis. What goes into the decision-making process for watch and wait vs. treatment? What changes and symptoms are should you and your care team be watching for? What are the pros and cons of this watchful waiting period?

Dr. Gabriela Hobbs, clinical director of Leukemia Service at Mass General, is here to answer these questions and more on watch and wait for MPNs as a part of our “Answers Now” series. She is joined by Ruth Fein, MPN Patient Advocate and host.

This program is sponsored by Incyte and is produced in partnership with the MPNRF. This organization has no editorial control. It is produced by Patient Power. Patient Power is solely responsible for program content.


Transcript | Watchful Waiting and Decision-Making with MPNs

Ruth Fein: I'm Ruth Fein. Today we'll be talking about watching, waiting, and decision-making with MPNs, and it should be helpful, whether you are newly diagnosed or whether you've been living with an MPN for a long time, like myself.

 Okay, let’s just dig in. Dr. Hobbs, one of the few things we are sure about MPNs is that nothing is for sure. Right? So, let's put that in the context of a new MPN patient. When do you actually feel comfortable recommending a watch-and-wait approach before beginning an initial MPN treatment?

In What Circumstances is Watch and Wait Recommended Before Treatment?

Dr. Hobbs: When somebody is recently diagnosed with an MPN, the first thing is to understand what that diagnosis is and give it a name. Often times I'll have patients come to clinic and say, “Oh, you know, my doctor has been following me for high platelets,” or something. It's important to know, what is the diagnosis? What is the name? And then to ask the question, what has to happen for me to need therapy? Do you need medications to lower your blood counts to prevent a complication of the disease, or does a patient need to consider medication to improve the symptoms of a disease?

What Do You Specifically Watch for in Watch and Wait?

Ruth Fein: What would you specifically watch for?

Dr. Hobbs: I think that the answer is a little bit different depending on the specific MPN. So, when thinking about treatment for essential thrombocytosis (ET) and polycythemia vera (PV), one of the things that's important to recognize is that treatment is used for the most part to prevent a complication related to having a blood clot or having bleeding. The care team that's taking care of the patient should think about several different things to decide if the patient is at low risk for having the blood clot or at high risk for having a blood clot. So, generally speaking, patients with polycythemia vera that are younger than 60 and patients that have not had a blood clot are considered to be low risk, and those patients need to be, generally speaking, on aspirin and they have to have phlebotomy, or having a unit of blood removed, and that's enough for treatment to prevent having a blood clot.

The decision-making is sort of similar in patients with essential thrombocytosis, but we'll also think about the type of mutation that the patient has, and we'll decide is that patient low risk, very low risk, intermediate risk or higher risk for having a blood clot. So really what we're looking for initially is what is the risk of the patient having a blood clot.

Sometimes patients need treatment for other reasons. There are sometimes patients that have lots of symptoms and they may be considered low risk based on what I just said and may not need a medication to lower their blood counts, but let's say they have bad fatigue, bad itching, or other things like that. Those things can also make a doctor decide that it's time to start a medication to improve symptoms. And so, then the goal of treatment is not necessarily just to prevent a blood clot, but also to improve quality of life and symptoms.

Ruth Fein: You typically consider medical history, symptoms, blood counts, all of those things. And this helps you determine a person's risk for disease complications, right? Like clots and bleeding that you spoke about. And that helps you inform your recommendations about initial therapy, whether watch and wait is a good choice. Do I have that right so far?

Dr. Hobbs: Absolutely.

What are the Pros and Cons of Watch and Wait?

Ruth Fein: What about the pros and cons of waiting? So, once you've decided.

Dr. Hobbs: In many cases, it's pretty obvious that giving a medication to lower the risk of a blood clot is not the right choice because a patient is very low risk. The physician may say, you're low risk for having a blood clot, we're going to watch and wait. And so, the benefit of that is that the patient doesn't necessarily need to be on any medication, or they may just need to be on an aspirin.

The downside, and I think it's important to really recognize the downside - being diagnosed with anything provokes a lot of anxiety. And having a diagnosis like ET or PV or myelofibrosis (MF) can have a lot of anxiety also, can lead to a lot of anxiety. Living with that uncertainty hanging over your head can be difficult. And so that I would say the biggest downside of observation is the anxiety that goes with it.

How Do You Deal with the Anxiety of Watch and Wait?

Ruth Fein: How do you suggest people deal with the anxiety of the whole watch-and-wait approach? Depression, anxiety aside, because that's often a co-symptom of MPNs — they overlap certainly, but in this case that watch and wait is all about uncertainty, help us out there.

Dr. Hobbs: Make sure that you understand why your doctor has recommended watching watchful waiting. I find that for sure information is power. So, if your doctor has told you starting medication will not be helpful now, we'll just subject you to the side effects of medications, et cetera. And that is the best approach. Make sure that you truly understand what that means and that you understand that getting started on treatment may not be the right thing for you.

Try to take control in the aspects of your life where you can take control. Because I find that when you get diagnosed with something new, all of a sudden, there's this huge loss of control, right? And you have this diagnosis and there's so many parts of this diagnosis that you can't alter.

Take control of the information that you have, advocate for yourself, and then maximize every other aspect of your life. Diet and exercise, I think, are so important to helping to manage the anxiety and the symptoms that come with having an MPN. So starting an exercise routine that's appropriate for you, making sure that you're healthy otherwise, and that you're treating or preventing the development of other medical conditions like high blood pressure, like diabetes, like high cholesterol, et cetera, are so important to your overall wellbeing and to also preventing any complications that could occur from the MPN itself. Get information and take control of what you can.

Ruth Fein: Let me go back to that same patient again, that we first spoke about. You were seeing a new patient, let's put them forward five years and okay, they're on hydroxyurea (Hydrea) or phlebotomies, which you mentioned. And for those who are not familiar with phlebotomy, I think of it as the reversal to a blood transfusion - you can explain it differently. But what's the watch and wait like then? Five years down the road on a medication you continue to watch for new symptoms, what are you monitoring them for? And what are you watching for them?

What Happens if Someone Has Been on Watch and Wait for Many Years?

Dr. Hobbs: For patients that are already on treatment, we're watching for a few different things. Are the blood counts well controlled and within range? And so, for patients with polycythemia vera, we want to make sure that the hematocrit is less than 45 for example. For patients with essential thrombocytosis, we want to make sure the platelets are within a relatively normal range. This is a question I get a lot. There's not like an absolute target of what the platelets should be. First thing, and most obvious and easiest to do is just getting a blood count and making sure that it's in range. The second thing that we look at is how's the patient doing with that. Is the medication helping, not just to make the blood counts look pretty, but also to help the patient feel well?

One of my goals of treatment is not just to normalize or improve the blood counts, but it's also to help to make sure that the patient is truly living with that MPN. Are there symptoms that the patient is experiencing, that we are not taking care of, even though the blood counts may be adequate. And so, talking about those symptoms is really important. When we're looking at those blood counts or when we are doing the physical exam, is there something that would suggest that the disease that initially started as essential thrombocytosis or polycythemia vera is turning into myelofibrosis? And although that, what we call transformation, when one disease turns into another disease, is not something that occurs frequently it is something that the care team should be looking for every time that they see you.

And for patients with myelofibrosis, that's true as well. We want to make sure that the myelofibrosis is stable, that the blood counts are stable, that the spleen hasn't grown and that there's no signs that the myelofibrosis is turning into leukemia, which is also a complication that may occur sometimes.

Does Your Care Team Differ When You’re on Watch and Wait vs. Receiving Treatment?

Ruth Fein: Does the makeup of your care team differ if a patient is under "watch and wait" versus they're receiving a treatment?

Dr. Hobbs: Yes and no. For patients that are under watchful wait approach or regular treatment, the care team should be the same. And the care team also kind of varies depending on where you're treated. Where it really does change is if you're participating in a clinical trial. So, patients that are participating in clinical trials usually get another care team helping manage that person. So, your main physician and or nurse practitioner and nurse that you may be familiar with will continue to take care of you. But if you are in a clinical trial, you'll also meet the research staff. So that changes the makeup of the team a little bit.

Ruth Fein: So, Dr. Hobbs, thanks so much for joining us today. It's really been so helpful. If you left us with a one-sentence hopeful message, what would that be?

Dr. Hobbs: Don't lose hope because the last couple of decades have shown us so much about MPNs. I think that every day you should feel hopeful that there are going to be new treatments for MPNs. 

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