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.
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. - In: CANCER. - ISSN 0008-543X. - 128:13(2022), pp. 2449-2454. [10.1002/cncr.34216]
Peripheral blasts are associated with responses to ruxolitinib and outcomes in patients with chronic-phase myelofibrosis
Breccia, Massimo
2022
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
