MPN Therapy: Advice and Guidance for Making Treatment Decisions - 2 | Transcript | Myeloproliferative Neoplasms | Patient Power

MPN Therapy: Advice and Guidance for Making Treatment Decisions

So that is a prognostic significance of some of the abnormalities in genes, and some of the abnormalities in chromosomes and ketogenes, and this is what Samantha was talking about. They would explain, perhaps, why things change, but they also provide prognostic significance for the outcome of the patients with myelofibrosis at this time. And so that has to be accounted for when we talk about what are we going to do about myelofibrosis. We can do the pills to control the spleen, or the symptoms or anemia, which are the three main problems with patients in myelofibrosis.

There are others, but these are the three main: Anemia, progressive and symptomatic splenomegaly, in general body—failure of the quality of life, like myelofibrosis symptoms. So, symptoms, spleen, and anemia. Or, you wanna say, “I don’t wanna deal with the disease. I wanna change my scope of therapy to eliminate disease, and that’s the only way is obviously the transplant.” And then, the decision needs to be made together, between the patient and the physician, when is the best timing, what are the expectations, and why would you go into that right now.

And this is where we talk about Samantha’s situation. In her particular case, these genetic abnormalities have a powerful, unfortunate possible influence on the evolution of myelofibrosis is much faster rate. You see, usually for the transplant, we utilize characteristics of the patients. That would be if you are very anemic, you would have a very high red cell count, you have progressive inflammation in the spleen, quality of life, transfusion dependence. These are the prognostic factors to assess the aggressiveness of the disease.

And you would say if your disease has a risk of shortening your life expectancy to less than five years, based on our prognostic factors, then you should go for the transplant. We now account for changes in genetics, on top of the clinical evaluation, and the chromosomes also. So, this complex way of looking at individuals by putting all this together: biology, and the clinical perspective, and then you say, “When is the time?” And I think Samantha made up her mind already that now is the time, although she’s not suffering a lot, and that’s good, from the myelofibrosis, because the biological part is so unfavorable, now I think it’s reasonable to consider transplant.

Andrew Schorr:         

Wow, so Samantha, and Nick, we’re gonna get to you in a second, but this is a big decision point she’s at. 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 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.

Andrew Schorr:         


Nick Napolitano:        

Andrew, can I ask a question?

Andrew Schorr:         

Please, go ahead. This is a discussion.

Nick Napolitano:        

So, we spend our lives trying to be proactive, personal, professional, and with this particular disease, there’s so much that’s unknown, and me personally, I’ve inquired with several different doctors about bone marrow biopsies, and how often we should do that, and to me, if you’re able to see progression in the bone marrow biopsy, why wouldn’t we do one more often? Why wouldn’t we try and be proactive and take a look at that bone marrow more often, to stay ahead of the progression and be as prepared as possible, right?

Samantha talked about that before we came on, about trying to be prepared, and I have gotten different viewpoints on how often we should do a bone marrow biopsy. I’ve received advice that, “I’m not going to treat you any differently if I see progression,” or, “I’m gonna treat you based on your symptoms.” But to me, that doesn’t compute when we’re trying to be as proactive as possible, and if you can stay ahead of it, possibly, in the bone marrow biopsy, why wouldn’t we do one more often?

Dr. Verstovsek:           

Okay, so it’s okay to comment on that? Okay. That is an outstanding question, and I cannot tell you how many times I hear about the proactive stance, not perhaps bone marrow biopsies all the time, but proactive. And the sad situation is that the answer that you provided in your comment, is the one that is proper answer. Let’s say that we do bone marrow biopsies in every PV patient every year, and we will find some patients that have more fibrosis than in the past. In fact, 20 percent of PV patients at the time of diagnosis already have some fibrosis. And we know that they are the hardest progression to post in myelofibrosis.

But, the bone marrow biopsy sample is a little bit different form time to time, either the hemopathology’s a little bit different, and you will have to account for these differences and stay put until the next year and make sure that there is real change. And the question is, if you say yes, myelofibrosis is a little bit higher, but otherwise, everything else is the same. I still need phlebotomies, or still have high platelets and whatnot, related to PV. What am I going to be doing differently? And the sad situation is, nothing, really. We don’t have a tool that will be applicable in the patient with PV or ET that has more fibers in the bone marrow than at the beginning. I don’t have a myelofibrosis medication.

I’m not really able to provide the interference that people usually ask about, as opposed to medication to prevent progression, because it hasn’t been proven really to do that. It’s more or less an individual experience within the MPN community that that is a possibility but never documented, so the society, or if you wanna even say insurance companies, or the doctors are not willing to treat people with the medication that has some toxicity and works only for five years usually in patients, because the tolerance is poor long term. And that is questionable long-term benefit in the prevention of any change, or even reversal of fibrosis. So really, now, and if you do this testing, you would say, “What am I gonna do with these results?” Nothing actionable, and that’s why we don’t really do that. In fact, I would give you the alternative test that I would like to do. Why do the bone marrow biopsies? Why not do the bone marrow testing every six months, on blood? You don’t need to do the bone marrow biopsies to look for fibrosis. You can look at the development of new mutations in a patient’s blood.

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Page last updated on August 23, 2019