Life after ruxolitinib: Reasons for discontinuation, impact of disease phase, and outcomes in 218 patients with myelofibrosis

F. Palandri, M. Breccia, M. Bonifacio, N. Polverelli, E.M. Elli, G. Benevolo, M. Tiribelli, E. Abruzzese, A. Iurlo, F.H. Heidel, M. Bergamaschi, A. Tieghi, M. Crugnola, F. Cavazzini, G. Binotto, A. Isidori, N. Sgherza, C. Bosi, B. Martino, R. LatagliataG. Auteri, L. Scaffidi, D. Griguolo, M. Trawinska, D. Cattaneo, L. Catani, M. Krampera, R.M. Lemoli, A. Cuneo, G. Semenzato, R. Foà, F. Di Raimondo, D. Bartoletti, M. Cavo, G.A. Palumbo, N. Vianelli

Research output: Contribution to journalArticlepeer-review


Background: After discontinuing ruxolitinib, the outcome of patients with myelofibrosis reportedly has been poor. The authors investigated whether disease characteristics before the receipt of ruxolitinib may predict drug discontinuation in patients with myelofibrosis and whether reasons for drug discontinuation, disease phase at discontinuation, and salvage therapies may influence the outcome. Methods: A centralized electronic clinical database was created in 20 European hematology centers, including clinical and laboratory data for 524 patients who received ruxolitinib for myelofibrosis. Results: At 3 years, 40.8% of patients had stopped ruxolitinib. Baseline predictors of drug discontinuation were: intermediate-2–risk/high-risk category (Dynamic International Prognostic Score System), a platelet count <100 ×10 9 per liter, transfusion dependency, and unfavorable karyotype. At last contact, 268 patients (51.1%) had discontinued therapy, and the median drug exposure was 17.5 months. Fifty patients (18.7%) died while taking ruxolitinib. The reasons for discontinuation in the remaining 218 patients were the lack (22.9%) or loss (11.9%) of a spleen response, ruxolitinib-related adverse events (27.5%), progression to blast phase (23.4%), ruxolitinib-unrelated adverse events (9.2%), and allogeneic transplantation during response (5.1%). The median survival after ruxolitinib was 13.2 months and was significantly better in the 167 patients who discontinued ruxolitinib in chronic phase (27.5 vs 3.9 months for those who discontinued in blast phase; P <.001). No survival differences were observed among patients who discontinued ruxolitinib in chronic phase because of lack of response, loss of response, or ruxolitinib-related adverse events. The use of investigational agents and/or ruxolitinib rechallenge were associated with improved outcome. Conclusions: The survival of patients with myelofibrosis after discontinuation of ruxolitinib is poor, particularly for those who discontinue in blast phase. Salvage therapies can improve outcome, emphasizing the need for novel therapies.

Original languageEnglish
Pages (from-to)1243-1252
Number of pages10
Issue number6
Publication statusPublished - 2020


  • investigational agents
  • myelofibrosis
  • outcome
  • ruxolitinib
  • treatment failure
  • adult
  • aged
  • allogeneic stem cell transplantation
  • anemia
  • Article
  • blast cell
  • blood toxicity
  • cancer growth
  • cancer mortality
  • cancer patient
  • cancer prognosis
  • cancer survival
  • clinical outcome
  • cohort analysis
  • controlled study
  • data base
  • disease course
  • drug withdrawal
  • Dynamic International Prognostic Score System
  • heart failure
  • high risk patient
  • human
  • infection
  • intermediate risk patient
  • karyotype
  • major clinical study
  • metastasis
  • multicenter study
  • neurologic disease
  • observational study
  • platelet count
  • pleura effusion
  • priority journal
  • prognostic assessment
  • retrospective study
  • risk factor
  • salvage therapy
  • spleen
  • thrombocytopenia
  • thrombosis
  • treatment response
  • very elderly


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