PACIFIC phase II final results: Rusfertide in PV

Results from the single-arm, open-label, phase II PACIFIC trial (NCT04767802) evaluating rusfertide, a hepcidin mimetic, in 20 patients with polycythemia vera (PV) and elevated baseline hematocrit (>48%) were published in Leukemia Research by Chew et al.

Key data: By Week 8, 85% of patients achieved hematocrit <45%, and 95% achieved ≥5% absolute reduction from baseline. Median time to first hematocrit <45% was 4.9 weeks. No patients received phlebotomy during rusfertide treatment and 85% of patients experienced treatment-emergent adverse events (TEAEs), most of which were Grade 1/2.

Key learning: Rusfertide resulted in rapid and sustained improvement in hematocrit in patients with PV and elevated baseline hematocrit, and was generally well tolerated. Sustained hematocrit control reduces the need for therapeutic phlebotomy and may reduce thrombotic and cardiovascular events over the long term.