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MPN Workshop of the Carolinas 2025 | The evolving treatment landscape for patients with high-risk ET in the era of novel agents

In this video, Alexander Coltoff, MD, Medical University of South Carolina, Charleston, SC, briefly comments on the evolving treatment landscape for high-risk essential thrombocythemia (ET). Dr Coltoff notes that, while hydroxyurea is effective in managing blood counts and decreasing thrombotic risk, it does not address disease progression or modify the disease. He envisions that novel agents and therapeutic approaches will enable disease modification and play a key role in eliciting deeper responses, ultimately improving patient outcomes. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.

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Transcript

For high-risk ET, which has traditionally just been treated with hydroxyurea, I think that does an okay job with treating the blood counts and maybe mitigating some of the thrombotic risk. But we know that the high-risk ET patients aren’t just high risk for thrombosis or blood clots that are high risk for disease evolution to myelofibrosis, to leukemia, to decreased overall survival...

For high-risk ET, which has traditionally just been treated with hydroxyurea, I think that does an okay job with treating the blood counts and maybe mitigating some of the thrombotic risk. But we know that the high-risk ET patients aren’t just high risk for thrombosis or blood clots that are high risk for disease evolution to myelofibrosis, to leukemia, to decreased overall survival. And so I think studying some of these newer agents, either older but newer, like interferons, which have had a kind of revolution, or the newer targeted agents to try and actually kind of harness newer medicine to target deeper responses and target disease modification is really how I see the field evolving in the next few years. and trying to maybe even take someone who’s high-risk for ET and kind of reduce their risk, not only for blood clots, but for everything that we care about.

 

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Disclosures

Speaking fees: Incyte, Blueprint Medicines, Sobi; Advisory Boards: PharmaEssentia, Blueprint Medicines.