Are There Clinical Trial Therapies That High-Risk Myelofibrosis Patients Should Consider Before Transplant?

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Topics include: Treatment

At a recent MPN town meeting, Dr. Srdan Verstovsek and Dr. Naval Daver from MD Anderson Cancer Center and Dr. Rami Komrokji from Moffitt Cancer discussed treatment considerations for myelofibrosis patients prior to stem cell transplant.  Each of the experts shares their perspective on how genetic testing shapes treatment choices, how age affects the decision, and why or why not a particular patient makes a good candidate for transplant. Watch to learn more about why this is an individualized decision for each patient. 

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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.

Esther Schorr:   

Susan asks for myelofibrosis patients who are considered high risk because of having adverse genetic mutations, are any clinical trial drugs appropriate to consider before going to transplant? 

Dr. Verstovsek: 

This is a really up-to-date question, because we are learning about the disease at a rapid pace with this genetic testing, which we did not have until a few years ago.  We are discovering groups of patients that may not do as well as others, even with application of the old-style diagnostic factors based on age or blood cell count or symptoms.  And that is the real dilemma, when we see patients that are, let’s say, younger and in good shape without any symptoms, without a big spleen.  And now you have a genetic testing done, and there are bad genetics there.

Is it time to intervene, based just on genetic complexity of that person?  Or is it just something that just gives us a sign to be more cautious about that patient and see that patient more often, and see at what pace that patient will progress?  Because everybody eventually does progress, it’s just a matter of when.  Is it in a year, or is it 10 years?  So the doctors are a more cautious sort that are in general not jumping on the one value or one measurement, and that is applicable here with our colleagues in transplant section, as well.

We would recognize that that patient might be at the risk of progressing sooner rather than later, but we would watch that patient closely and see exactly what happens.

Dr. Komrokji:     

Right.  So I think the question also is do we know that the transplant, for example, would overcome the bad players?  We are learning from other diseases, for example, myelodysplastic syndrome.

There is a mutation called p53, which p53 in most diseases is usually not a good mutation.  That actually those patients if they go to transplant, they drive less mileage.  So it could be actually the other way, that those patients that have a—that there are like some bad player, that those patients are probably better down the road to treat them with clinical trials.  We don't know the answer for this.  We are always tempted to say let’s do more, let’s do more intensive therapies.  But that may not be the answer for every patient, actually. 

Esther Schorr:   

So there really isn’t one answer to this. It’s more on a case-by-case basis, it sounds like.

Dr. Daver:             

I think another interesting thing is a lot of these adverse mutations, I think it was Dr. Freireich and Dr. Kantarjian mentioned that prognostic factors are only important until you have a very effective therapy.  So we have seen this in many leukemias.  CD-20 was bad in ALL [acute lymphocytic leukemia].  With the advents now of rituximab (Rituxan), or ofatumumab (Arzerra) or brentuximab (Adcetris), it’s actually equal.  With better ones, it may even become favorable.  The same is being seen with FLT3 inhibitors, they were always very poor.  Now they’re getting neutral.

We would see with new generation if it gets even better.  So I think until—once you have better treatments, it won’t matter what we don’t know yet, is what Rami was saying, is that can we reverse it and it we have those treatments, yet.  Then it will become more important.                   

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 January 27, 2016