Rusfertide improves hematocrit control in phase III polycythemia vera trial

The phase III VERIFY trial evaluated rusfertide, a subcutaneous hepcidin mimetic peptide, in patients with polycythemia vera requiring frequent phlebotomy and with poorly controlled hematocrit. During the 32-week Part 1a, 293 patients were randomly assigned, double-blind, to rusfertide (147) or placebo (146), alongside standard care with or without cytoreductive therapy. The primary endpoint was clinical response, defined as absence of phlebotomy eligibility during weeks 20–32.

Clinical response was achieved by 113 patients receiving rusfertide (77%) versus 48 receiving placebo (33%; P < .0001). From baseline to week 32, the mean number of phlebotomies was 0.5 versus 1.8, and 63% versus 14% maintained hematocrit below 45% (both P < .0001). Rusfertide also improved PROMIS fatigue scores and MFSAF symptom scores versus placebo (P = .0252 and P = .0240, respectively); the abstract did not provide the magnitude of these changes.

Injection site reactions, anemia, and fatigue were the most common adverse events; no deaths were reported. These findings support improved hematocrit control and reduced phlebotomy burden in this population. However, the available abstract covers only 32 weeks and does not report effects on thrombotic events or long-term disease outcomes. Adverse-event rates and full methodological details were unavailable.