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MPN Workshop of the Carolinas 2025 | Advice for physicians managing patients with MPN-AP/BP
In this video, Anand Patel, MD, University of Chicago Medical Center, Chicago, IL, provides some advice for physicians managing patients with accelerated/blast-phase (AP/BP) myeloproliferative neoplasms (MPNs), highlighting that clinical trial enrolment should be considered due to the lack of effective standard therapies in this patient population. Dr Patel stresses the importance of comprehensive clinical profiling, including molecular testing, to identify potential targetable aberrations, as well as early referral to a center with expertise in AP/BP disease to optimize care and clinical outcomes. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.
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Transcript
The biggest thing I want to stress here is that there is no standard therapy right now for patients with accelerated-/blast-phase. There are several therapies we can try, but none of them are very effective. So this is really a population where the best option for them is oftentimes a clinical trial because our standard therapies are ineffective.
With that in mind, I think it’s also very important to clinically profile patients, not just in terms of peripheral blood counts, performing a bone marrow biopsy, but also making sure you’re doing molecular testing...
The biggest thing I want to stress here is that there is no standard therapy right now for patients with accelerated-/blast-phase. There are several therapies we can try, but none of them are very effective. So this is really a population where the best option for them is oftentimes a clinical trial because our standard therapies are ineffective.
With that in mind, I think it’s also very important to clinically profile patients, not just in terms of peripheral blood counts, performing a bone marrow biopsy, but also making sure you’re doing molecular testing. About 25% of these patients will have an IDH1 or IDH2 mutation, so we do have targeted therapies that are available. Our role of targeted therapies in myeloid malignancies is expanding. Some of these patients may have other targetable aberrations like FLT3, NPM1, or KMT2A rearrangements, where we can leverage other FDA-approved targeted therapies.
Early referral to a center that has expertise in accelerated-/blast-phase disease is important, particularly thinking about if the disease is well-controlled, a patient will need to ultimately receive a transplant if they’re eligible for it. So the earlier on we can be involved in that process, I think that is the best route to hopefully have a durable long-term cure for a patient.
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Disclosures
Honoraria: Abbvie, Astellas, Amgen, Jazz, Sobi, Syndax; Research funding (inst): Incyte, Servier, Pfizer, Sumitomo.
