Leukemia: Myeloproliferative Neoplasm (MPN)

    Actionable Target Abnormality Clinical Experience with Targeted Agent
    Janus kinase 2 (JAK2) Somatic, constitutively activated mutation, V617F Ruxolitinib (JAK2 inhibitor):
    • Phase 3, COMFORT-I trial, for intermediate 2 or high-risk myelofibrosis: Spleen volume reduction of 35% or more at 24 wk: 41.9% (0.7% in placebo); ≥50% improvement in total symptom score at 24 wk: 45.9% (5.3% in placebo) (40)
    • Phase 3, COMFORT-II trial: Spleen volume reduction of 35% or more at 48 wk: 28% (vs. 0% in best available treatment) (41)
    Sotatercept (JAK2 inhibitor):
    • Phase 2, for myelofibrosis with 36% of pts had anemia response (42)
    Other topics in Targets by Organ Site