Published on June 23, 2020
When does transplant fit into an MPN treatment regimen? How do doctors and patients make this important decision?
In this segment, host Andrew Schorr talks to transplant expert Dr. Jeanne Palmer from the Mayo Clinic Arizona about the factors she considers when thinking about a stem cell transplant, such as patient risk score, timing and donor health. They also discuss CAR-T cell therapy and when it could be available for treating MPNs. Watch now to learn from an expert.
Transcript | When Is a Transplant the Best Option for MPN Patients?
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Welcome to Patient Power. I'm Andrew Schorr in California. Joining me from Mayo Clinic in Scottsdale, Arizona is Dr. Jeanne Palmer, who is a specialist in the MPNs, and also, with some patients, does transplant. So Dr. Palmer, any of us living with an MPN, particularly myelofibrosis, wonder, is transplant in our future? It's kind of a big gun. So where does it fit in and how do you decide with a patient when a transplant is the way to go?
So that's a fantastic question and constantly moving target. I think that when we think about transplant, I think, first and foremost, it is a shared decision making process between a physician and a patient. So it's never something where people can't come in and I'm like, "Okay, this is it. Time to go. Hands down." It's always something where you're thinking about, okay, what does this timing look like? I always try to break it down, first into just looking at overall risk scores, the risk scores we've been using for 10 years, the DIPSS risk scoring system, which takes into account symptoms, hemoglobin the white blood cell count, age, and the number of immature cells in the peripheral blood.
But from there, we have to go even further. We also have to go into what other molecular risk factors do we see. So we can do pretty intense molecular sequencing of these diseases and find a lot of mutations. These mutations can often give us insight into how we think the disease is going to behave moving forward, and that's very, very important to know. They're not perfect. Nothing is perfect. There's people who I think though, there should be a pretty long lasting disease and they progress quickly. And other people who think, oh, this looks like it'll move fast, but it doesn't. So they're not perfect, but they definitely give us a starting point.
And then once we kind of establish where along this risk line a person is, we say, "Well, when is the right time in this? What is the right risk for this person to proceed with transplant?" And for that, we take into consideration lifetime events, for example. I've had patients that I've put off transplant so they could see a grandchild be born, they could see their son's wedding, and various things like that. And for the most part, it shouldn't make a huge difference. There are times when it does, so that's definitely a conversation to have with your transplant physician.
The other thing I take into consideration is the donor. If somebody has a good donor or not, that affects the risk of the transplant and ultimately the outcome. I think one of the things that's really important to know is outcomes are a lot better than they used to be. When you look at the old data of when we transplanted patients who waited until the very last moment, until they were very sick, the outcomes weren't as good. I think we're all starting to recognize in the world of MPNs and the world of transplant, that the timing probably is before you feel that sick. It's waiting then just that moment, trying to find when is it before you start to get worse because sometimes when you get worse, it's so worse that unfortunately it's hard to get people back to a point where they'll have a successful transplant.
So it's trying to find that right timing. And that takes time, that takes constant conversation and good communication between patient, transplant physician, hematologist, if they're the same person, all for the better.
Dr. Palmer, in some other blood cancers, not MPNs, they're using what are called CAR T-cell therapy, chimeric antigen receptor T-cell therapy, to fight the cancer. And it's being approved in some areas and it's in trials for others. What about for MPNs? What about CAR T for someone with an MPN?
MPNs are a unique situation. So when we look at where the CAR Ts have been largely successful, so the current FDA-approved products for an acute lymphoblastic leukemia, and diffuse large B-cell lymphoma. Those are both diseases that have one cell that mutates and grows out of control. So you have one clone. This clone looks the same. All the cells will be very similar. They all express an example of diffuse large B-cell lymphoma. They express CD19. So the CAR T is something that specifically targets cells that express CD19. So if the proteins are on the surface, the cells will recognize it and go and attack that specific cell. That's the beauty of CAR T and what makes them work so well.
In myeloma, you have a similar thing. You can have certain proteins on the cell surface that the CAR T cell can recognize and go after. With myelofibrosis, and we see this with myelodysplastic syndrome and probably with some AMLs, there's multiple areas that are problems. So it's not one cell that mutates, that grows out of control, that you can say, "If I get rid of that one cell, I'm going to get rid of that disease." With MPNs, the whole marrow is affected. So if you were to say, "Okay, I'm going to get rid of the blasts," or the very immature white blood cells that sometimes reflect, if they get too high in percentage, they reflect leukemia. If you got rid of the blast, you're not helping because there's still lots of other cells in there that are going to be impacted.
So unfortunately, for a disease like myelofibrosis, myelodysplastic syndrome is another good example, the disease involves the whole marrow so the only way to get rid of it is to get rid of the whole marrow. And if you do that, you have to replace it with something. And if you don't replace it with something, you're in a lot worse shape than you started out.
Dr. Jeanne Palmer from the Mayo Clinic in Scottsdale, Arizona. Thank you for helping us understand why CAR T might not be right for both MPNs and myelodysplastic syndrome. I'm Andrew Schorr. Remember, knowledge can be the best medicine of all.
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