Emerging agents and regimens for myeloproliferative neoplasms: updates from ASH 2025 Annual Meeting
Tiantian Zhang1,2, Heng Jiang3, Delong Liu4,5
1Department of Internal Medicine, University of Central Florida, Pensacola, FL, 32514, USA.
Abstract:
Current Myeloproliferative neoplasm (MPN) therapies provide meaningful symptom and event-risk control but are limited by infrequent molecular remissions, treatment-limiting cytopenias (particularly anemia), and continued reliance on non-mutation-directed cytoreduction or phlebotomy with attendant morbidity. At ASH 2025 Annual Meeting, multiple investigational strategies aimed to address these limitations, including clone-directed targeting (INCA033989), phlebotomy-sparing physiologic therapy (rusfertide), and epigenetic disease-modifying combinations (pelabresib). We summarized the latest updates on these emerging agents and regimens for MPNs from the 2025 ASH Annual Meeting.
Insights
New myeloproliferative neoplasm (MPN) therapies are emerging to overcome current treatment limits. Investigational agents targeting MPN clones, sparing phlebotomy, and modifying disease offer potential for improved outcomes.
Area of Science:
- Hematology
- Oncology
- Molecular Medicine
Background:
- Current myeloproliferative neoplasm (MPN) treatments offer symptom control but have limitations.
- These include infrequent molecular remissions and treatment-limiting cytopenias, particularly anemia.
- Existing therapies often rely on non-mutation-directed cytoreduction or phlebotomy, causing morbidity.
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