A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis

Margherita Maffioli, Barbara Mora, Somedeb Ball, Alessandra Iurlo, Elena Maria Elli, Maria Chiara Finazzi, Nicola Polverelli, Elisa Rumi, Marianna Caramella, Maria Cristina Carraro, Mariella D'Adda, Alfredo Molteni, Cinzia Sissa, Francesca Lunghi, Alessandro Vismara, Marta Ubezio, Anna Guidetti, Sabrina Caberlon, Michela Anghilieri, Rami KomrokjiDaniele Cattaneo, Matteo Giovanni Della Porta, Toni Giorgino, Lorenza Bertu, Marco Brociner, Andrew Kuykendall, Francesco Passamonti

Research output: Contribution to journalArticlepeer-review


Ruxolitinib (RUX) is extensively used in myelofibrosis (MF). Despite its early efficacy, most patients lose response over time and, after discontinuation, have a worse overall survival (OS). Currently, response criteria able to predict OS in RUX-treated patients are lacking, leading to uncertainty regarding the switch to second-line treatments. In this study, we investigated predictors of survival collected after 6 months of RUX in 209 MF patients participating in the real-world ambispective observational RUXOREL-MF study (NCT03959371). Multivariable analysis identified the following risk factors: (1) RUX dose,20 mg twice daily at baseline, months 3 and 6 (hazard ratio [HR], 1.79; 95% confidence interval [CI], 1.07-3.00; P 5.03), (2) palpable spleen length reduction from baseline #30% at months 3 and 6 (HR, 2.26; 95% CI, 1.40-3.65; P 5.0009), (3) red blood cell (RBC) transfusion need at months 3 and/or 6 (HR, 1.66; 95% CI, 0.95-2.88; P 5.07), and (4) RBC transfusion need at all time points (ie, baseline and months 3 and 6; HR, 2.32; 95% CI, 1.19-4.54; P 5.02). Hence, we developed a prognostic model, named Response to Ruxolitinib After 6 Months (RR6), dissecting 3 risk categories: low (median OS, not reached), intermediate (median OS, 61 months; 95% CI, 43-80), and high (median OS, 33 months; 95% CI, 21-50). The RR6 model was validated and confirmed in an external cohort comprised of 40 MF patients. In conclusion, the RR6 prognostic model allows for the early identification of RUX-treated MF patients with impaired survival who might benefit from a prompt treatment shift.

Original languageEnglish
Pages (from-to)1855-1864
Number of pages10
JournalBlood advances
Issue number6
Publication statusPublished - Mar 22 2022

ASJC Scopus subject areas

  • Hematology


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