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

Palandri, Francesca;Bartoletti, Daniela;Iurlo, Alessandra;Bonifacio, Massimiliano;Abruzzese, Elisabetta;Caocci, Giovanni;Elli, Elena M;Auteri, Giuseppe;Tiribelli, Mario;Polverelli, Nicola;Miglino, Maurizio;Heidel, Florian H;Tieghi, Alessia;Benevolo, Giulia;Beggiato, Eloise;Fava, Carmen;Cavazzini, Francesco;Pugliese, Novella;Binotto, Gianni;Bosi, Costanza;Martino, Bruno;Crugnola, Monica;Ottaviani, Emanuela;Micucci, Giorgia;Trawinska, Malgorzata M;Cuneo, Antonio;Bocchia, Monica;Krampera, Mauro;Pane, Fabrizio;Lemoli, Roberto M;Cilloni, Daniela;Vianelli, Nicola;Cavo, Michele;Palumbo, Giuseppe A;Breccia, Massimo

2022-01-01

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Abstract

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.