Multicenter randomized phase 2 trial of ruxolitinib versus best available therapy for polycythemia vera after hydroxycarbamide failure

Polycythemia vera (PV) is characterized by JAK/STAT activation, thrombotic/hemorrhagic events, systemic symptoms, and transformation to acute leukemia. Hydroxycarbamide (HC) is the most common chemotherapy drug used to treat PV. The multicenter randomized phase 2 MAJIC-PV trial compared ruxolitinib versus best available therapy (BAT) for PV in patients resistant/intolerant to HC. Complete response rate, duration of response, event-free survival, and molecular response were superior with ruxolitinib over BAT. Molecular response was linked to event-free, progression-free, and overall survival.