Myelofibrosis: How Do Symptoms Impact Treatment?

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Topics include: Treatment and Understanding

What are your symptoms telling you? Oftentimes, the course of treatment for myelofibrosis reflects the symptoms and circumstance of each individual patient. Listen as MPN specialist Dr. David Snyder shares information about signs that indicate your disease may be progressing and explains appropriate therapies for the condition.

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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.

Beth Kart Probert:

Could you talk to us about myelofibrosis and give our viewers a summary of it, and also address the treatments and the goals of the treatments through the different therapies?

Dr. Snyder:      

Sure. So we’re talking about the family of myeloproliferative neoplasms, and we started with ET; P vera,of course, is the other.

The third type is myelofibrosis. What we call primary myelofibrosis is patients who are diagnosed right from the beginning with myelofibrosis. But we know that both P. vera and ET have the potential to transform over time to what we would call secondary myelofibrosis, meaning that they started with one condition, and over time it transformed into myelofibrosis.

We see certain changes when that happens. As the name implies, there’s increased scarring, scar tissue in the bone marrow. That’s what myelofibrosis means. We tend to see decrease in some of the blood counts, particularly the hemoglobin with anemia and often the platelet count. And at the same time, often the white count is normal, or it can be very elevated.

One of the clues for a patient with P vera for example, is say a patient was requiring a certain frequency of phlebotomies to maintain control of hematocrit or a certain dose of hydroxyurea (Hydrea). And after a while the doctor notices gee, we haven’t done phlebotomy in about five, six months, and still the hematocrit hasn’t gone up.

Or we’ve been on a certain dose of Hydrea for a long time, and everything has been stable, but now it looks like the hemoglobin is starting to drop. That’s the kind of clue that maybe it’s beginning to transform towards myelofibrosis, and the bone marrow no longer is overproducing red cells, but instead there’s a decreased production of red cells.

So along with that comes a number of other features. The spleen often enlarges around that same time, and people may become aware of that enlarging organ in their abdomen. It may impact their ability to eat. There are also a variety of systemic symptoms that can occur. Sometimes we see this in patients with P. vera and less so with ET, but most commonly in patients with myelofibrosis.

Those are things like fevers, weight loss, night sweats, fatigue, itching and others. That’s kind of the clinical picture that you see when a patient either starts right off at the beginning or is progressing. So in terms of goals of therapy, there are a few issues. One, just like with the discussion about ET, patients with myelofibrosis are at increased risk for blood clotting and sometimes bleeding, as well. But our main focus is to help prevent blood clots from occurring. And so the same kind of baby aspirin if needed, is used.

In addition, there is a treatment called ruxolitinib or Jakafi is another name, that is FDA approved for treatment of patients with myelofibrosis. I’ll say that’s the only drug currently that is approved by the FDA for myelofibrosis, despite the fact that there have been many clinical trials that we may talk about with other drugs. But ruxolitinib was approved based on two main endpoints. One was a significant reduction in the size of the spleen for patients whose spleen is enlarged, with relief of symptoms from that big spleen. And second was control of some of these systemic symptoms that I mentioned. Those are the two main benefits that people can achieve.

There may be some other benefits such as gain in weight for people who have maybe lost weight. There may be some prolongation in survival. That’s a little bit of a soft call, but some patients maybenefit that way.

So that’s the main treatment that we think about for patients with myelofibrosis.

Beth Kart Probert:       

It sounds like from what you’ve described, and, of course, what Dr. Scott described is the symptom burden is a big driver in how you’re going to treat patients. And the symptoms are also a big indication of if it’s progressing, if it’s doing what it should be doing. 

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.

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Page last updated on October 12, 2017