There are a number of considerations both in the short and long term to consider when making treatment decisions for younger patients with myeloproliferative neoplasms (MPNs).
Managing a younger person with a myeloproliferative neoplasm (MPN) includes a lot of long-term considerations, including their chance of progressing to a more aggressive disease, like acute myeloid leukemia, said Jennifer Vaughn, MD, hematologist-oncologist and assistant professor in the Division of Hematology at The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute.
Transcript
Since the age distribution of who is affected by MPNs can be broad, with patients possibly being diagnosed in their teens and 20s, how might you approach treatment and management differently for a younger vs an older patient?
It's actually kind of paradoxical in the way that I sort of look at it. On one hand, a patient who presents at a young age is theoretically going to be lower risk, in that their risk of an acute thrombotic event tends to be less because they're under, like I mentioned before. there's this cut off of being over age 60 or below age 60, you know that sort of puts you in a low- versus high-risk category. And those patients tend to be overall healthier. They don't have as many cardiovascular risk factors as someone who presents at a later age. In that respect, the initial treatment may be less aggressive, in that we might avoid adding additional cytoreductive therapies or medications. The benefit to that, too, is that if they do start one of those medicines, they'll have to be on those medications longer, because right now, we don't have clear guidance as to a stop/start recommendation.
Once you start one of those treatments, the thought is that you'll likely be on it for long term, depending on various aspects of what happens in life and in the course of their disease. That can have a couple of negative ramifications, which is, one, a lot of these drugs are patented or have some additional financial cost that comes along with them, and a younger person who initiates therapy early on is going to be facing many years of having to cover those medications financially. Two, we don't have a whole lot of data about the long-term effects of being on certain medications for a long period of time. There's always been a concern about the drug hydroxyurea and that it might lead to an increased risk of progression to a more aggressive MPN in the future or leukemia, for example, that data hasn't really borne out in retrospective studies.
However, I think the thought process has been perhaps avoiding those drugs for long-term treatment in younger patients and considering other drugs such as interferons that we think may be safer. There's also an issue with family planning. Patients who are trying to conceive or have children probably shouldn't be on things like hydroxyurea, as those are teratogenic. There a lot of lot of factors that go into it when you're dealing with someone at a young age.
On the other side, though, a patient who is diagnosed in their 20s, 30s, 40s, has many years to live with the disease. And an active area of research now is trying to identify which of those patients may be at higher risk of developing progression to myelofibrosis or leukemia in the future, and whether or not we should be putting those patients on something—if we can find it—the drug that's going to somehow reduce their risk of progressing in the future. While we don't have the perfect medication for that just yet, there are signals, again, that we may be able to reduce the JAK2 [Janus kinase 2] or driver mutation allele burden in some of these patients, which theoretically could have a long-term benefit. Again, we’re waiting for the data to pan out on that.
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