New research in myeloproliferative neoplasms (MPNs) presented at the 2025 American Society of Hematology (ASH) meeting points toward more targeted treatments, better symptom control, and tools to personalize care for people living with these rare blood cancers.
New drugs for symptom control and disease activity
Several studies looked at new medicines or combinations aimed at improving symptoms like fatigue, spleen enlargement, and the need for blood transfusions in myelofibrosis and polycythemia vera. These included updated results with JAK inhibitors (such as ruxolitinib, momelotinib, fedratinib, and pacritinib) and new partner drugs that may help reduce anemia, shrink the spleen, and improve quality of life when standard therapies are not enough.
Targeted and “first-in-class” therapies
Early-phase trials tested highly specific drugs designed to go after key drivers in MPNs, such as mutant JAK2 and mutant calreticulin, as well as new agents like bezuclastinib, nuvisertib, pelabresib, bomedemstat, and others. These studies are still in the safety and dose-finding stages but suggest that more precise, biology-driven treatments may become available in the future for conditions like myelofibrosis, essential thrombocythemia, polycythemia vera, and systemic mastocytosis.
Reducing transfusions and improving anemia
Several abstracts focused on helping people with MPNs who struggle with anemia and frequent blood transfusions. Trials combining momelotinib or fedratinib with other agents, and studies of drugs like elritercept, luspatercept, and zilurgisertib, explored ways to improve hemoglobin levels, reduce transfusion dependence, and see whether those improvements are linked with better survival.
Interferon and longer-term disease control
New data on ropeginterferon alfa-2b in polycythemia vera and essential thrombocythemia examined how this long-acting interferon may help control blood counts and possibly affect the underlying disease over time. These studies included patients across different risk levels and settings, adding to the evidence that interferon-based approaches remain an important option for some people with MPNs.
Tools to personalize monitoring and care
Beyond drugs, researchers also highlighted new ways to track disease and support patients, including a patient-developed “MPN journal” app to record symptoms and share information with care teams. Other work looked at blood markers like the neutrophil-to-lymphocyte ratio, bone marrow fat cells, and genetic changes as potential biomarkers to better predict response, guide treatment choices, and understand how MPNs progress over time.
Reference
67th ASH Annual Meeting. Presentations 80, 81, 83, 482, 484, 485, 486, 909, 910, 1021, 1022, 1024, 2019, 2023, 2025, 2051, 2052, 3794, 3795, 3803, 3809, 3810, 5527, 5569, 5572, 5581, 5589, 5590
