EHA 2025 | Real-world data on the treatment of adolescents and young adults with MPNs

Whilst we think about MPNs as diseases of patients who are usually diagnosed in their 60s, it’s definitely true that we do see a good proportion of patients, 20% or so, who present less than the age of 40, and particularly for ET and indeed PV, we do see adolescents and young adults. So in the UK, we would describe that as a person being diagnosed less than age 25. And we’ve been working on this as part of the EHA special working group for MPN for some time now, describing the literature, and then doing a collaborative analysis of outcomes for patients across many different centres in the SWG. And if listeners are interested in MPN, and want to join this group please do look at the EHA website – shameless advert but it’s a very, very friendly collaborative group; doesn’t matter what stage you are at, doesn’t matter what your profession is, you’re really welcome to join. 

So we’ve been gathering data and updating it and the most recent data to come from our group with regard to this we presented at ASH and we published recently in Leukemia which shows that longer-term outcomes, in particular myelofibrosis, was impacted by the use of interferon. So using interferon for these younger patients actually resulted in 100% myelofibrosis-free survival compared to other treatments such as hydroxycarbamide and anagrelide or indeed no treatment. So that supports to some extent, and there are all sorts of caveats around real-world data, but it supports to some extent and further direction in which the field is moving, which is early intervention with a well-tolerated drug that may modify disease. And so interferon generally is well-tolerated. Not every patient can take it, but it could be a useful agent. And this data certainly adds to other data in the field, suggesting that it might be able to modify disease. And how we might measure that, of course, might be going back to allele burden reduction, but we need to do some other studies. These kind of real-world studies, though, are very important in a field such as ours where, you know, events can occur after 15 to 20 years, and so, you know, it’s difficult to fund and do prospective studies in that context, and so we can still get very important insights from real-world data.

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