MPN Workshop of the Carolinas 2024 | Important considerations when deciding whether a young patient with MPN should receive cytoreduction

I think in young patients it’s a little trickier, and I certainly think we always want to do no harm, and so I think when we think about cytoreduction, I think really identifying when you think is the right time is sort of the hardest part.

There are definitely cases where I’m sure most people would agree. You know, certainly if a patient presents with a thrombosis, we’ve certainly seen teenagers present that way where they get diagnosed with Budd-Chiari, which is one of the splanchnic thromboses that’s common in MPNs, and that is actually how their MPN gets picked up. So in that setting, I think everybody would agree they need to be on cytoreduction of some kind.

Certainly if somebody presents with a major hemorrhage because their platelets are high, that’s a really important time to think about it.

I think then beyond that, you get into a little bit of a gray area. You know, again, in pediatrics, often a kid will present with a very high platelet count and someone might have a knee jerk reaction to just start them on cytoreduction to lower that count. But they may be asymptomatic and completely well, and that’s a time when maybe we shouldn’t be reducing.

So I think there are times when it’s really clear we should, and then there are times where we shouldn’t just be reducing based on a number in an otherwise healthy person. 

Then I think you have sort of the in-betweens, you know, the concept of what if somebody has acquired von Willebrand’s disease, and they’re at risk of bleeding but they’re a competitive athlete and they really want to continue doing their sport and that may be is how they’re going to get to college or something that’s important for their lifestyle. So that may be a time to think about cytoreduction. Maybe someone has symptoms that aren’t responding to aspirin and they’re really debilitating, that may be a time to think about cytoreduction.

And then sort of the concept of disease modification, you know, in a young patient who’s going to have a disease for many more decades than someone who’s diagnosed when they’re 60 or 70, maybe thinking about starting interferon early if they have no symptoms and no contraindications because of the potential disease modification benefits. There was some really nice data presented at ASH from a European group showing that myelofibrosis-free survival is really improved with interferon. 

So that’s something I talk about with patients and, you know, if it’s something that a family is interested in or a young person is interested in, I think that’s a great thing.

So I think there are clear times when we do need to try to cytoreduce and times where we clearly shouldn’t just react to a number and don’t necessarily need to cytoreduce. But there are a lot of these in-between conversations that I have with young patients and their families that I think that’s where it’s a little more nuanced. And so thinking about it more and having more conversations with patients and understanding their goals as well is really helpful.