Opinion|Videos|July 7, 2025

The Evolving MPN Landscape: Future Therapeutic Directions and Addressing Unmet Needs in Myelofibrosis and Polycythemia Vera

Panelists discuss how the future treatment landscape for myeloproliferative neoplasms is evolving with many promising therapies in development, including calreticulin antibodies, selective JAK2 inhibitors, and novel agents targeting various pathways.

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In the final segment, Dr. Vachhani asks Dr Rampal about emerging developments in the myeloproliferative neoplasm (MPN) field that might change the treatment landscape. Dr Rampal notes many ongoing developments, including new targeted agents being used as add-on or upfront therapies and calreticulin antibodies. He acknowledges that positive results from these studies will create a more complex treatment landscape but welcomes this challenge as it will expand options for patients.

Dr. Vachhani elaborates on specific promising therapies in development for myelofibrosis, including type 2 selective JAK2 inhibitors, JAK2 V617F selective inhibitors, and drugs with novel mechanisms of action. These are being investigated in both newly diagnosed and second-line settings. He mentions several specific agents: selinexor (XPO1 inhibitor) in the SENTRY study, navtemadlin (MDM2 inhibitor) in the POESIS study, which targets patients with suboptimal ruxolitinib response, BET inhibitors like pelabresib and an inSight BET inhibitor, and numisetib for symptom management.

For polycythemia vera, Dr. Vachhani is particularly excited about the paradigm shift from focusing solely on hematocrit control to more comprehensive disease control, potentially slowing disease progression. Both experts agree that while these developments are promising, time will reveal their full impact on disease outcomes. The segment concludes with mutual appreciation for the discussion, highlighting the rapidly evolving nature of MPN treatment approaches and the increasing sophistication of management strategies.


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