Transplant vs. Oral Medication for MPNs: How Can You Decide Which Is the Right Fit?

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Topics include: Treatments and Understanding

After genetic tests revealed a group of aggressive mutational markers, myelofibrosis patient Samantha Trahan has decided to take action despite feeling healthy. In an effort to prevent disease progression to acute myeloid leukemia (AML), Samantha is weighing her options to find the best route for treatment. What factors should patients consider when contemplating transplant versus oral therapy? Watch now to hear MPN expert Dr. Srdan Verstovsek, from The University of Texas MD Anderson Cancer Center, and Samantha discuss the prognostic implications of mutational status, and the risks and benefits of myeloproliferative neoplasm (MPN) treatments. 

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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.

Andrew Schorr:

So, there have been pills, like I’ve been on for years, ruxolitinib (Jakafi), and maybe there are others in trials for sure, you’re kinda skipping over all that. Is that because you see, from this genomics, sort of a freight train coming, and you just wanna hit it head on?

Samantha Trahan:       

Well, it’s tough to make that call. I mean, I’m trading, right now, what is a great quality of life, for a rough year, and that’s if everything goes perfectly, right? There’s nothing easy about the transplant process. But most of the drugs that are out there right now are focused on quality of life. They help with the spleen, they help with fatigue, they help with your overall body state, and they have, as a consequence of that, they also have some life-extending qualities to them, as I understand it.

But there’s nothing out there, and nothing that is so close on the horizon to wait for, that I can take as a pill, that will cure me or myelofibrosis, or that will stop the genetic mutation in its track so that I won’t develop AML. And so if, I’ve had this, been in this family of diseases for a very long time, so I’ve seen the transition from no medication to finding the JAK2 Mutation, to the development of ruxolitinib, to the drug they’re on now, and I’ve looked at the clinical trials to see what else I might be taking, and I’ve looked at PubMed to see what other doctors are researching, and I don’t see something that is going to be available fast enough to stop what is coming, and that, for me, is progression.

I only happen to know that because of all these mutations that they found in my blood recently. But you’ve gotta factor that in, and having factored that in, I would rather undergo the transplant now, when I am at my absolute healthiest.

I’m young, I don’t need transfusions, I’m not transfusion dependent, I don’t have any other co-morbidities, right? I’m young enough. I'm only 43, so I'm not diabetic, I don’t have high blood pressure. I don’t have anything else. I’ve had my heart checked out, my lungs checked out, and I’m absolutely perfectly healthy. That’s the time when your body is the strongest, that you’re going to do a transplant, and you know you’re going to have to do it sooner or later, I’m choosing to do it sooner.

Dr. Verstovsek:

If I may, there are so many good points here that you summarized, and just to touch upon one by one, the number one is that there is no medication, as you pointed out properly, that would prevent progression of acute myeloid leukemia. JAK inhibitors or any other therapies are here to control the disease to the best of our ability, for whatever they do, in terms of the spleen symptoms or anemia, but none of them has been shown to prevent progression of myeloid leukemia. So that risk stays with the patient, even though they might feel better and have a smaller spleen.

The second point is, the mutation number is so high, and some of them are known to be of prognostical importance, in other ways, once the baby cells acquire that mode, that they are changing as they multiply, and they already went from one to five, their likelihood is that there will be 10 or 15, that the trend will continue. So it is reasonable to interrupt it with elimination of the disease, and replacing it with healthy bone marrow now, when, point number three, you are in excellent shape. You’re not suffering from the disease. You can sustain the transplant and the change, so going through the transplant for you in a safe way are much better.

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 April 23, 2019