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MPN Workshop of the Carolinas 2024 | Are thrombotic risk and progression in ET predictable?
Joseph Scandura, MD, PhD, Weill Cornell Medicine, New York, NY, discusses the need to broaden the focus beyond thrombotic risk when managing patients with essential thrombocythemia (ET). Dr Scandura highlights the possibility of predicting composite risk by identifying the specific driver mutation harbored by a patient, as this provides insight into whether they are at greater risk for thrombosis or progression. This interview took place at the 1st Annual MPN Workshop of the Carolinas in Asheville, NC.
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Transcript
So this is an issue in essential thrombocythemia is that this is a disease where everything we know about and everything that we focus on relates to the increased risk of blood clots, thrombosis, in those diseases. And there really has been limited data in terms of long term outcomes. And many of these patients are younger who are expected to live decades, many decades for some, with this disease...
So this is an issue in essential thrombocythemia is that this is a disease where everything we know about and everything that we focus on relates to the increased risk of blood clots, thrombosis, in those diseases. And there really has been limited data in terms of long term outcomes. And many of these patients are younger who are expected to live decades, many decades for some, with this disease. And yet we focus on the thrombotic complications and ignore the progression part of that.
And so what we found, and this is a partnered with a couple other papers that showed very similar things, is that the driver mutation in ET dictates the risks. So patients with the JAK2 mutation have a much higher risk for thrombotic complications. Patients with a CALR mutation or a MPL mutation have much lower thrombotic rates but much higher progression rates. So we almost never see blood clots in CALR-mutated ET, but over the long term, 20% of them progress to myelofibrosis, which is a very more morbid disease. And so we need systems that include those risks and consider not just thrombosis risk, but the total composite risk a patient will experience throughout their lifetime with that disease.
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Disclosures
Incyte, Abbvie, Constellation/Morphosys, SDP Oncology, Calico, Morphic, PharmaEssentia.
