Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients: Results from an independent study

Francesca Palandri, Mario Tiribelli, Giulia Benevolo, Alessia Tieghi, Francesco Cavazzini, Massimo Breccia, Micaela Bergamaschi, Nicola Sgherza, Nicola Polverelli, Monica Crugnola, Alessandro Isidori, Gianni Binotto, Florian H. Heidel, Francesco Buccisano, Bruno Martino, Roberto Latagliata, Marco Spinsanti, Lydia Kallenberg, Giuseppe Alberto Palumbo, Elisabetta AbruzzeseLuigi Scaffidi, Antonio Cuneo, Michele Cavo, Nicola Vianelli, Massimiliano Bonifacio

Research output: Contribution to journalArticle

Abstract

Patients with myelofibrosis at intermediate-1 risk according to the International Prognostic Score System are projected to a relatively long survival; nonetheless, they may carry significant splenomegaly and/or systemic constitutional symptoms that hamper quality of life and require treatment. Since registrative COMFORT studies included only patients at intermediate-2/high International Prognostic Score System risk, safety and efficacy data in intermediate-1 patients are limited. We report on 70 intermediate-1 patients treated with ruxolitinib according to standard clinical practice that were evaluated for response using the 2013 IWG-MRT criteria. At 6 months, rates of spleen and symptoms response were 54.7% and 80% in 64 and 65 evaluable patients, respectively. At 3 months, ruxolitinib-induced grade 3 anemia and thrombocytopenia occurred in 40.6% and 2.9% of evaluable patients, respectively. Notably, 11 (15.9%) patients experienced at least one infectious event ≥grade 2. Most (82.6%) patients were still on therapy after a median follow-up of 27 months. These data support the need for standardized guidelines that may guide the decision to initiate ruxolitinib therapy in this risk category, balancing benefit expectations and potential adverse effects.

Original languageEnglish
JournalHematological Oncology
DOIs
Publication statusAccepted/In press - 2017

Keywords

  • Intermediate-1 risk
  • IPSS
  • MF
  • Myelofibrosis
  • Ruxolitinib

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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    Palandri, F., Tiribelli, M., Benevolo, G., Tieghi, A., Cavazzini, F., Breccia, M., Bergamaschi, M., Sgherza, N., Polverelli, N., Crugnola, M., Isidori, A., Binotto, G., Heidel, F. H., Buccisano, F., Martino, B., Latagliata, R., Spinsanti, M., Kallenberg, L., Palumbo, G. A., ... Bonifacio, M. (Accepted/In press). Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients: Results from an independent study. Hematological Oncology. https://doi.org/10.1002/hon.2429