Treatment of polycythemia vera (PV) is shifting from focusing only on hematocrit control to a broader approach that also targets white blood cells, platelets, and symptoms. Data from large studies like REVEAL study show that higher levels of white blood cells and neutrophils, not just hematocrit, are linked to increased risk of blood clots.
Keeping hematocrit below 45% remains the main treatment goal, but frequent phlebotomy can lead to iron deficiency and symptoms like fatigue. A new drug, rusfertide, is under review by the U.S. Food and Drug Administration and may reduce or eliminate the need for phlebotomy by limiting iron use for red blood cell production. In the Phase 3 VERIFY trial, most patients receiving rusfertide avoided needing phlebotomy compared with standard care.
For low-risk patients, treatments are also evolving. ropeginterferon alfa-2b is now commonly recommended to help stabilize blood counts and reduce treatment burden, offering better tolerability than older interferon therapies.