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Tools to Help You Learn More About MPN Clinical Trials

Tools to Help You Learn More About MPN Clinical Trials from Patient Empowerment Network on Vimeo.

Research is quickly evolving for essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF), all due to clinical trials. Dr. Laura Michaelis reviews tools for learning about clinical trials and how you can get involved. 

Dr. Laura Michaelis is hematologist specializing in myeloproliferative neoplasms (MPNs) at Froedtert & the Medical College of Wisconsin, where she also serves as Associate Professor of Medicine. Learn more about Dr. Michaelis here.

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Dr. Michaelis:                       

So, there’s lots and lots of research going on in the field of myeloproliferative neoplasms. One of the best ways to stay informed about this is to check on something called clinicaltrials.gov, which is a national website that warehouses clinical trials in the disease that you might have.

So, if you look up essential thrombocythemia or you look up myelofibrosis, it will show you the clinical trials that are going on in your region, in your state, or in the country. So, that’s a nice way to begin to look and see what’s available.

The other way, of course, is to talk to your doctor. And each institution has a list of clinical trials that they have open, based on what a person’s diagnosis is, what their age is, what other, for example, health issues that they might have. And so, again, when you’re talking to your doctor, say – Is there something I should be thinking about, in terms of clinical trials?

There are clinical trials going on in all facets of the disease – not just in treatment, which is what you often think about. Treatment for symptoms or treatment for ways to control the disease. There are new medicines coming down the road, in terms of what to do when somebody is no longer responding to treatment. Or how can we combine treatments together? Those are important trials, and those are things that people should talk with about their physician.

But there’s also clinical trials on symptom management, and those can sometimes be a trial that you would do online yoga or dietary trials. And there’s trials on familial syndromes, meaning – Yeah, I have polycythemia vera, and my aunt had myelofibrosis, and her daughter has ET. Well, that is a family that should be investigated. And sometimes we find that – even nationally, we find that we’re networking with one another and collecting patients who have family histories so we can learn more about that.

So, there’s a variety of different types of clinical trials. Those are things to talk to your doctor about. And on your own, you can search them out on clinicaltrials.gov or through patient advocacy organizations.

What Are the Treatment Options for Myelofibrosis?

What Are the Treatment Options for Myelofibrosis? from Patient Empowerment Network on Vimeo.

When choosing a treatment for myelofibrosis (MF), where do you start? Dr. Laura Michaelis reviews the available options for MF therapy, including a discussion of stem cell transplant. 

Dr. Laura Michaelis is hematologist specializing in myeloproliferative neoplasms (MPNs) at Froedtert & the Medical College of Wisconsin, where she also serves as Associate Professor of Medicine. Learn more about Dr. Michaelis here.

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Dr. Michaelis:                       

So, myelofibrosis is among the most aggressive of the myeloproliferative neoplasms. And yet, it still has a broad swath of risk associated with it. And that means that compared to essential thrombocythemia and polycythemia vera, myelofibrosis – primary myelofibrosis tends to be more aggressive and needs to be treated more aggressively.

There are a group of patients who have what we call low-risk or even intermediate 1 risk myelofibrosis who don’t need treatment – who can be followed, whose blood counts can be checked, who can be regularly seen by their doctor, and who can avoid treatment for some period of time, depending on their risk factors.

And so, if you’re diagnosed with primary myelofibrosis, the first question is do I need treatment? And the second question – if the answer to that is yes, then you have to figure out why you need treatment. There is currently only one intervention that is known to be curative in myelofibrosis, and that’s the use of stem cell transplant.

Stem cell transplant, which is also called bone marrow transplant or allogeneic hematopoietic stem cell transplant, is basically a procedure where a donor is able to have their stem cells collected. And then the recipient, who’s the person with the myelofibrosis, undergoes a series of chemotherapy treatments to basically wipe out the bone marrow that they have. And it’s then replaced, using basically a blood transfusion of the stem cells of someone else, which then grow up into the recipient.

This has been shown to be safe in myelofibrosis – well, relatively safe – if done in the right person, who’s relatively fit, and done at the right stage of disease. It’s not a procedure that everybody can tolerate. People need to be pretty fit. And it should be performed at a place that has done numerous transplants for myelofibrosis since it’s a relatively complicated form of stem cell transplant. That being said, in the right person at the right time, it’s an excellent opportunity and option for these patients.

Now in patients who can’t tolerate transplant or where that’s not the right step to go, we have medications. And those medications can sometimes delay the worsening of symptoms. They can certainly control spleen size, improve people’s quality of life, and often improve survival. And the medications that we’re talking about here are called JAK-STAT inhibitors.

And the first approved, and the one that’s most commonly used, is a medicine called ruxolitinib.

This is an oral pill – a pill that you take twice a day and has excellent data that supports that it shrinks the spleen in people with myelofibrosis, that it improves symptoms. And in people with advanced polycythemia vera, decreases the blood count without leading to iron deficiency and also improves symptoms and spleen size.

There’s another JAK-STAT inhibitor that’s approved. That’s a medicine called fedratinib. And it was recently approved in people who had progressed off of myelofibrosis or even in people – or after ruxolitinib or in people who – instead of taking ruxolitinib.

Now in essential thrombocythemia, polycythemia vera, and other times even myelofibrosis, we have other treatments that are commonly used that can be exceedingly helpful in controlling symptoms and blood counts. Those include, for example, hydroxyurea, pegylated interferon, and sometimes treatments aimed at helping anemia, like steroids or derivatives of thalidomide.

And finally, I don’t want to let this end without saying that clinical trials are often an excellent possibility for patients with these conditions, like myelofibrosis.

So, when you are contemplating starting a treatment, it’s really important to ask your physician whether or not there’s any clinical trials that are right for you. 

MPN Symptom or Treatment Side Effect? Know the Difference

MPN Symptom or Treatment Side Effect? Know the Difference from Patient Empowerment Network on Vimeo.

How do you distinguish MPN symptoms from side effects? Dr. Laura Michaelis explains the difference, and why it’s important to share any changes with your doctor.

Dr. Laura Michaelis is hematologist specializing in myeloproliferative neoplasms (MPNs) at Froedtert & the Medical College of Wisconsin, where she also serves as Associate Professor of Medicine. Learn more about Dr. Michaelis here.

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Dr. Michaelis:             

So, symptoms and side effects are sort of different things. Symptoms are the characteristics of the disease process. And these are things that often can vary in intensity. They maybe accumulate over time. But those are things like, for example, uncontrolled itching, fatigue, night sweats, fevers at night, unintentional weight loss, discomfort in the abdomen, or feeling full shortly after eating. Those are symptoms that often bring patients to the doctor’s attention in the beginning. And those are symptoms that can tell us that the treatments that we’re using aren’t working very well.

Now, side effects is the term that we use for problems that evolve when somebody starts a treatment for a condition. So, for example, if somebody starts the treatment of ruxolitinib for myelofibrosis, it is known that one of the side effects of this treatment is a small but significant lowering in the red blood cell [count].

That is a side effect of the ruxolitinib and should be anticipated. So, before you start the ruxolitinib, your doctor should sit down with you and talk about some of the side effects. And that might be one that gets mentioned.

In addition, we know that there is uncommonly – but uncommonly, people can have, for example, shingles reactivation once they’re taking treatment for myelofibrosis. And that might be something for which you take a prophylactic antiviral treatment.

Hydroxyurea has side effects. Interferon has side effects. And those are things that you should think about before you start them. They shouldn’t be reasons not to start the treatment because most people who take medicines don’t have the side effects. But it is something to keep in mind. And when then occur, report them to your doctor.

So, rarely, there’s conditions that occur, and you’re not sure. Is this a side effect to the treatment? Or does this mean the disease is progressing in some way? That’s one of the reasons it’s important to report all of these conditions to your physician because they need to know.

One of the things that can be helpful is there’s a common tool called the MPN SAF, which is a symptom assessment form.

If, periodically, you and your doctor fill that out during a clinic visit, you can sort of understand are those symptoms that I had with my disease responding to the treatment? Can we really measure that things have gotten better since I started treatment X or treatment Y?

And in addition, when you sit down with your doctor at your regular checkups, it’s not just about going through your blood counts and doing a physical exam. It’s also about telling them what you’ve noticed in the last two to three months since you saw your doctor with regard to the treatments that you’re taking.