A 60-Year-Old Man with Uncontrolled Polycythemia Vera - Episode 6

Unmet Needs and Future Directions in Polycythemia Vera Management

Description: Before closing out his discussion on polycythemia vera management, Prithviraj Bose, MD, shares excitement for emerging therapeutics in this setting.


Prithviraj Bose, MD: As far as future directions and unmet needs, and potential for drug development in PV [polycythemia vera], the need to modify the risk of myelofibrosis and AML [acute myeloid leukemia] is probably the No. 1 unmet need that we need to investigate new therapies for. As far as new drugs in the field, there’s a drug called rusfertide, which is a hepcidin mimetic. This is a drug with a narrow but specific role, which is to reduce the need for phlebotomy. By mimicking the action of hepcidin, it can make sure that the iron is less available for erythropoiesis and is sequestered in the reticular endothelial system.

There is a limitation in terms of the iron that’s available for erythropoiesis. In this way it’s able to reduce or eliminate the need for phlebotomy and correct iron deficiency. That’s a novel mechanism and a drug that does what it’s designed to do. This is something on the horizon, but its role is well defined. It would be used to reduce the need for phlebotomy, rather than a more holistic approach to the disease in terms of reducing clots, ameliorating symptoms, reducing spleen size, and controlling blood counts, which are achieved by ruxolitinib [Jakafi]. Of course, reducing the need for phlebotomy and controlling the hematocrit remains a central goal in PV, and rusfertide is an exciting drug in that sense.

Transcript edited for clarity.