Real-life ruxolitinib experience in intermediate-risk myelofibrosis

Abstract

Background In this retrospective cohort of patients with primary, post-polycythemia vera, or post-essential thrombocythemia myelofibrosis, 57 patients with MF who received ruxolitinib for MF-related symptoms or symptomatic splenomegaly were evaluated. Methods The median age of the patients in this cohort was approximately 58 years. Of these, there were 33 patients (57.9%) in INT-1, 23 patients (40.4%) in INT-2, and 1 patient (1.8%) at high risk. Overall, spleen size reduction of at least 35% (spleen response) was achieved in 56.6% and 63.3% of all cohort and INT-1 risk at any time, respectively. Results Symptom response and clinical improvement were observed in 21.7% and 60.7% of patients, respectively. Anemia and thrombocytopenia were prevalent, but manageable. About 73.7% of patients continued treatment during a median follow-up of 22 months. Two-year OS probability was approximately 84.5% (95% CI, 63.1-94.0%) and 62.3% (95% CI, 37.5-79.6%) for the intermediate-1 and -2 risk groups, respectively. Conclusion Real-life experience in a community-based hospital confirms the efficacy and safety profile of ruxolitinib in intermediate-risk myelofibrosis. Treatment discontinuation rates were lower than those in clinical trials.