Baseline factors associated with response to ruxolitinib: An independent study on 408 patients with myelofibrosis

Francesca Palandri, Giuseppe Alberto Palumbo, Massimiliano Bonifacio, Mario Tiribelli, Giulia Benevolo, Bruno Martino, Elisabetta Abruzzese, Mariella D'Adda, Nicola Polverelli, Micaela Bergamaschi, Alessia Tieghi, Francesco Cavazzini, Adalberto Ibatici, Monica Crugnola, Costanza Bosi, Roberto Latagliata, Ambra Di Veroli, Luigi Scaffidi, Federico de Marchi, Elisa CerquiBarbara Anaclerico, Giovanna De Matteis, Marco Spinsanti, Elena Sabattini, Lucia Catani, Franco Aversa, Francesco Di Raimondo, Umberto Vitolo, Roberto Massimo Lemoli, Renato Fanin, Francesco Merli, Domenico Russo, Antonio Cuneo, Maria Letizia Bacchi Reggiani, Michele Cavo, Nicola Vianelli, Massimo Breccia

Research output: Contribution to journalArticlepeer-review


In patients with Myelofibrosis (MF) treated with ruxolitinib (RUX), the response is unpredictable at therapy start. We retrospectively evaluated the impact of clinical/laboratory factors on responses in 408 patients treated with RUX according to prescribing obligations in 18 Italian Hematology Centers. At 6 months, 114 out of 327 (34.9%) evaluable patients achieved a spleen response. By multivariable Cox proportional hazard regression model, pre-treatment factors negatively correlating with spleen response were: high/intermediate-2 IPSS risk (p=0.024), large splenomegaly (p=0.017), transfusion dependency (p=0.022), platelet count < 200x109/l (p=0.028), and a time-interval between MF diagnosis and RUX start > 2 years (p=0.048). Also, patients treated with higher (≥10 mg BID) average RUX doses in the first 12 weeks achieved higher response rates (p=0.019). After adjustment for IPSS risk, patients in spleen response at 6 months showed only a trend for better survival compared to non-responders. At 6 months, symptoms response was achieved by 85.5% of 344 evaluable patients; only a higher (> 20) Total Symptom Score significantly correlated with lower probability of response (p < 0.001). Increased disease severity, a delay in RUX start and titrated doses < 10 mg BID were associated with patients achievinglower response rates. An early treatment and higher RUX doses may achieve better therapeutic results.

Original languageEnglish
Pages (from-to)79073-79086
Number of pages14
Issue number45
Publication statusPublished - 2017


  • Myelofibrosis
  • Predictive factors
  • Response
  • Ruxolitinib
  • Splenomegaly

ASJC Scopus subject areas

  • Oncology


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