High-dose ruxolitinib in MF: A real-world cohort study

Results from a single-center, retrospective cohort study, evaluating ruxolitinib in patients with myelofibrosis (MF; N = 24), were published in Annals of Hematology by Mendicino et al. Objectives of this study included characterization of longitudinal treatment exposure, spleen response, hematologic and non-hematologic safety, infection risk, second malignancies, and clinical outcomes.  

Key data: Overall, 45.8%, 25.0%, and 29.2% of patients were classified as receiving high-, intermediate-, and minimal-intensity ruxolitinib doses, respectively. A reduction in spleen length was observed in 81.0% of evaluable patients (n = 21). Median hemoglobin decreased from 13.0 g/dL at baseline to 9.9 g/dL during treatment, while median platelet count decreased from 474.5 × 10⁹/L to 170.0 × 10⁹/L. Second malignancies were observed in three patients and four patients died during follow-up, with no clear association observed between sustained exposure to full-dose or dose-intensified treatment and mortality. 

Key learning: Escalation to sustained, full-dose intensity ruxolitinib appears feasible in select patients with MF, with durable treatment exposure and a manageable safety profile. Further multicenter studies are warranted to confirm findings.