Peripheral blasts are associated with responses to ruxolitinib and outcomes in patients with chronic‐phase myelofibrosis

BACKGROUND: The presence of peripheral blasts (PB) is a negative prognostic factor in patients with primary and secondary myelofibrosis (MF) and PB ≥4% was associated with a particularly unfavorable prognosis. Ruxolitinib (RUX) is the JAK1/2 inhibitor most used for treatment of MF‐related splenomegaly and symptoms. Its role has not been assessed in correlation with PB. METHODS: In 794 chronic‐phase MF patients treated with RUX, we evaluated the impact of baseline percentage of PB on response (spleen and symptoms responses) and outcome (RUX discontinuation‐free, leukemia‐free, and overall survival). Three subgroups were compared: PB‐0 (no PB, 61.3%), PB‐4 (PB 1%‐4%, 33.5%), and PB‐9 (PB 5%‐9%, 5.2%). RESULTS: At 3 and 6 months, spleen responses were less frequently achieved by PB‐4 (P = .001) and PB‐9 (P = .004) compared to PB‐0 patients. RUX discontinuation‐free, leukemia‐free, and overall survival were also worse for PB‐4 and PB‐9 patients (P = .001, P = .002, and P < .001, respectively). CONCLUSIONS: Personalized approaches beyond RUX monotherapy may be useful in PB‐4 and particularly in PB‐9 patients.