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The Goal of Myelofibrosis (MF) Treatment

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Published on August 30, 2016

What are the various therapeutic treatments for myelofibrosis (MF)?  Myeloproliferative neoplasm (MPN) expert, Dr. John Mascarenhas of Mount Sinai School of Medicine, offers this concise yet comprehensive overview of MF treatments.  Listen as Dr. Mascarenhas discusses ruxolitinib, interferon, thalidomide, lenalidomide and danazol, as well as stem cell transplant (SCT) and splenectomy. 

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Transcript | The Goal of Myelofibrosis (MF) Treatment

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.

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. 

Andrew Schorr:

Okay. Let’s go on, Dr. Mascarenhas, to myelofibrosis. I'm listening carefully. So we have one approved medicine, right?

Dr. Heaney:    

Right.    

Andrew Schorr:                  

Others in clinical trials that we’ll talk about in a minute. But there’s a whole bunch of stuff, some of it not originally meant for myelofibrosis. So help us understand.

Dr. Heaney:         

So myelofibrosis, as a disease, is more complicated and as a therapeutic design is also a little bit more complicated. One must decide early on what the therapeutic goals are for an individual patient. So again, it’s a very individualized, personalized approach. So for myelofibrosis, ruxolitinib (Jakafi) is the only approved drug. It’s a drug that is very effective in improving the symptom burden and reducing the spleen and improving the quality of life. There is data that would support that in doing so, survival is a benefit to patients who take this drug versus patients who don’t, or take alternative therapies. 

Unfortunately, ruxolitinib’s limitation is it doesn’t really improve the blood counts in a substantial way in terms of anemia and thrombocytopenia; low red blood cell count or low platelet count which can really be a problem for many patients either early on or with disease progression. So other drugs that are listed here like erythropoietin, which we spoke about, or drugs that try to stimulate the bone marrow to make red blood cells, even prednisone which is used for many different types of diseases, can sometimes help and assist in improving a lot of the symptoms, as well as anemia.

Interferon may be a drug that may benefit patients, particularly early on before they have a lot of scarring in their bone marrow, and that’s still an area of active investigation. Thalidomide (Thalomid) and lenalidomide (Revlimid) are two drugs from a group of family called immunomodulatory drugs, and they’ve been employed and investigated to help with anemia and splenomegaly. Danazol (Danol) is a synthetic male androgen. That’s also been used to help with anemia. I should say that collectively, all these drugs have response rates of about 20 to 40 percent in studies in improving anemia.

And then the only really potentially curative route for patients with myelofibrosis, and this applies to a select group, is a bone marrow transplant, a stem cell transplantation which I'm assuming we’ll talk about today.

For some patients that have really refractory spleen pain, we still will resort to splenectomy, so surgical removal of the spleen. Ruxolitinib is sort of a medicinal way of addressing splenomegaly. And then in some centers, splenic irradiation is still used to try to reduce spleen at least temporarily.

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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