Dr. Jeanne Palmer, an MPN specialist, discusses when a stem cell transplant is an appropriate treatment option and provides an overview of how risk is assessed in MPN patients.
Dr. Jeanne Palmer is a hematologist specializing in myeloproliferative neoplasms (MPNs) and bone marrow transplant at the Mayo Clinic in Arizona. Dr. Palmer also serves as Director of the Blood and Marrow Transplant Program and is Vice Chair and Section Chief for Hematology.
(upbeat music) – When would you consider a stem cell transplant? – So the stem cell transplant is based on disease risk. There’s a number of ways we assess disease risk. The first two ones that were published a number of years back were the DIPSS score, which is Dynamic International Prognostic System Score or the DIPSS+, which basically is the DIPSS and then you add to it a few other clinical features. This symptom score is based largely on things that we can see without even a bone marrow biopsy.
So things like symptoms, age, number of white blood cells, whether somebody has anemia, and then the number of something called blasts, which are very immature white blood cells. The DIPSS+ takes into account low platelets, need for transfusions, and chromosome abnormalities, which is the only tested among that that needs to be in from a bone marrow biopsy. Now these were created prior to Jakavi being commercially available.
So we have to take a little bit of a grain of salt with those because of the fact that Jakavi probably has changed how long people can live with this disease. Now the more recently, they’ve tried to account for others molecular changes.
So when we take the genetic landscape of these diseases, we have the known driver mutation, So the JAK2 mutation, which I’ve talked about, also Calreticulin and MPL. These three mutations all affect that one pathway, the JAK-STAT pathway. So they all affect the pathway that drives the disease, and they are known to be kind of mutually exclusive and definitely contribute to the formation of the disease. Some of these other mutations are called somatic mutations. They can be checked by next generation sequencing or genetic analysis.
There’s a number of different names that people use for this testing, but we look for mutations that are present. And these mutations, number one, can sometimes tell us risk. So there’s certain mutations that are high risk. Other times they can actually give us other opportunities for therapy, especially if the disease progresses.
But these mutations are important to know for risk stratification.
For example, if somebody has a DIPSS score that is maybe not super high risk, but then they have one of these mutations, we know that that probably makes their disease a little bit more aggressive. And that’s when we think about transplant is when we know that the disease probably has an average life expectancy, you know, when somebody gets to the point in their disease where we estimate their life expectancy is around five years, recognizing that we’re not very good at this. That’s the time point when we start to think about transplant. But the timing of transplant is something that’s extremely difficult and a very personalized decision. It’s something that it’s really important to understand the disease risks, how we assess them, and the caveats of these disease risk assessments as we move forward the planning on timing of transplant.
And that’s something that is, again, a very, very important discussion to have at length with your physician.
And you know, I always recommend, there’s quite a few of us out there who actually specialize in transplant for myelofibrosis. And having discussions with somebody who really understands the biology of the myelofibrosis is important, because it’s very different than a lot of the other diseases that are transplanting. (upbeat music).
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