Published on March 27, 2019
How is treatment effectiveness monitored in myeloproliferative neoplasm (MPN) patients? How long can patients stay on a single therapy? This MPN Partners program features Patient Power founder Andrew Schorr, leading MPN expert Dr. Srdan Verstovsek, from The University of Texas MD Anderson Cancer Center, and patient advocates Samantha Trahan and Nick Napolitano, and offers a meaningful and informative conversation on factors that influence MPN treatment decisions. The panel discusses identifying high-risk disease characteristics, progression, the role of genetic testing and finding an appropriate course of treatment. The panel also gives expert and patient perspectives on symptom reporting and management, significant lab values and long-term effects of therapy. Tune in to learn more about a collaborative approach to choosing therapy and assessing treatment response.
This program is sponsored by Incyte Corporation.
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Transcript | MPN Therapy: Advice and Guidance for Making Treatment Decisions
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.
Hello, and welcome to Patient Power, I’m Andrew Schorr, and welcome to our Partners program, for those of us living with a myeloproliferative neoplasm, and MPN. I’m in Carlsbad, California, near San Diego, but we have some wonderful guests with us: Two from Houston, and one who’s normally in the New York area, today he’s on the road for his business in San Francisco, and I want you to meet them.
Then we’re going to have a discussion, really among patients, with a wonderful, world-class MPN expert as our advisor, as we talk about, really advice and guidance for treatment decisions, which you may need now, or you may need another one later, or later after that. So, joining us are some regulars on Patient Power. First, let’s go to Houston. Samantha Trahan, who’s an attorney in Houston, and Samantha, welcome back to Patient Power.
Thanks so much, Andrew.
So Samantha, just to review for a second, you were diagnosed at a really early age with ET, and now we’re like, 20 years later, and what seems to have gone on, going through polycythemia vera, is now myelofibrosis, right?
That’s right. I’ve now hit the trifecta. I started out with ET and moved to PV, and now, myelofibrosis.
Okay, all right. Well we’re gonna talk a lot more about that, because you’re at quite a crossroads. Okay, over to San Francisco, where Nick Napolitano, living with PV, is away on business. Nick, so you’ve been on baby aspirin, and that’s been working pretty well, and you’ve worked hard with your family and yourself to kinda get your head straight, and I know that’s been an issue, having a young family, and being pretty young yourself. And now you’re questioning, are some symptoms changing, and maybe will there be other treatment needed for you, right?
Yeah, and you know, it’s changed pretty quickly here over the last couple of months, and so we’re going to have discussions about regression and different treatments and things like that, and so it’s a very relevant topic for me, as I’m starting to experience some changes in my diagnosis.
Okay, well we’re gonna talk. Now we have someone who’s seen literally thousands of patients, and advises doctors around the world, and that’s Dr. Srdan Verstovsek, who is the chief of the MPN section of MD Anderson, and noted researcher. He’s been my doctor along the way, he’s Samantha’s doctor, part of her team. Dr. Verstovsek, welcome back to Patient Power.
Pleasure, thank you very much for having me on this valuable program. Friendly faces, glad that things are evolving for us all, and we’re gonna have a nice discussion about what to recognize in a patient, how to deal with and what to expect. Thank you for the opportunity.
Oh, sure. Okay, so first of all, Samantha, just to go back to you, so you and Nick, Me, too, and our viewers, probably, we do a lot of research, and at some point, though, you have to land on something that you believe will help extend your life, and so you can live well. So, how have you been, first of all, how did the myelofibrosis show up? So you went from PV, what changed?
So, I still feel great. So, it’s not that I had a sudden change in symptoms, but when I see Dr. Verstovsek every three to six months, and I have all of my—I have years' worth of data. And what we saw was a slight trend downwards, in hemoglobin, hematocrit, number of red blood cells. And it was enough of a very slow but, once you put it all together, very obvious trend that Dr. Verstovsek suggested it was time to do another bone marrow biopsy and look to see if anything had changed. We did that in December, and then had the change over to diagnosis when I met him in January.
Okay, now, I understand that a transplant is on the table. Why that?
Well, it’s interesting, even though I feel great, I am a well-informed patient, and while the bone marrow biopsy in itself was not too terrible, they also did a full genetic workup, and whereas I used to just be JAK2 positive, like most other, or almost all PV patients. The mutation panel actually brought back this year, I think, five different mutations. And some of those were on the very ugly side of the spectrum.
You know, so many mutations are not so bad, and some of them really speak to an advanced, or a more aggressive disease state. And so, I have two of those, and after doing a bunch of research, and talking with the stem cell team, they think that now is the time to treat that aggressively, because with these mutation patterns, it’s only really just a matter of time before I would progress to something more aggressive like AML.
Wow, and you have children?
I do. I have one. He’s 17-and-a-half. He’s a senior this year, about to graduate high school.
Okay, and so is there a current search going on for a donor?
There is. Originally, they could not find a perfect, unrelated match, so someone out there on the registry that was perfect 10 for 10, and so we had my son typed, and he is a great haplo match, so we were looking down that avenue. And then just last week, they tell me that they found a 10 for 10 unrelated match, and so I went from having really, no options, surprisingly, because everyone thought that finding a match would be pretty easy, but went from having no options to now having two really good, viable options.
Are you on pins and needles right now?
The thought makes me want to throw up every time I think about it, but you know, what else do you know? You know, you’ve got to deal with what you got.
Let’s skip over to your doctor, Dr. Verstovsek. So, Dr. Verstovsek, just a little bit about this sort of genomic testing now, so not all myelofibrosis is alike, is it? So what you might recommend to me, depending on what you saw in my biopsy, might be different for her. Tell us about the subsets now, in a decision to maybe use the big gun, a transplant.
Yeah, I’m sorry that the evolution of the disease has happened and that we are talking about such an intervention as aggressive as it is now a bone marrow transplant for Samantha. But in her short summary, Samantha has touched upon quite a few important aspects of disease evolution. And just briefly, just first to say, that the change, and this was quite well elaborated at the beginning by someone, that the change from more benign conditions, and in her case, it was polycythemia vera changing to myelofibrosis, takes time. Nothing happens right over night.
So you can see, that’s what we always talk to the patients about. We cannot predict who is gonna change, and the risk of change to myelofibrosis is a small one, but it takes time. So what happens in her case is that she described, anemia developed. Then you look at LDH. LDH is the chemistry test that comes from the dead cells, the chemical. It goes up, and then you may see some of the molecules in the white blood cell types, and then you wonder what’s happening because the production of red blood cells is abnormal. The cells are dying much faster. LDH goes up, and then you see baby cells from the bone morrow and blood, and then you say it must be that fibrosis is developing.
And then you do the bone marrow biopsy to document that change. And with the presence of abundance of fibers, plus these clinical findings, then you say, yes you are transforming, or you have already transformed. It’s not one test. It’s not only bone marrow biopsy that would show the fibers. It’s a combination of bone marrow findings, physical exam, chemistry test, and the blood test, all together needs to be taken into account to talk about progression. Then, once you talk about myelofibrosis, then you say, yes this is, you have to know if this is a more aggressive condition.
It tends to change, and it changed already. The cells tend to change, and you try to explain why the change happened. And we can, and that is becoming standard practice in academic centers throughout the United States. Look beyond just the—in terms of genetics, look beyond the JAK2 mutation, or one of these, what you call driver mutations, that they usually will include the JAK2 mutation, calreticulin mutation, or MPL mutation. These are three mutations that almost everybody has one of the three. But we look for others, and to see whether this is the explanation why the disease has changed.
We actually note, everybody has many other mutations, but in Samantha’s case, there were many others, and so that is plausible explanation why the disease became more aggressive. There might be other reasons in the bone marrow of the patients why things changed. We are focused on genetics because we can do the genetic testing. This can be done on blood, not necessarily on the bone marrow biopsy. We also look at the chromosomes, the ketogenes. That can be done also on blood, but it’s much better done on the bone marrow, because you do need a lot of baby cells. Chromosomes can be broken in a patient, and that can explain why things change.