Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial
In brief
In polycythemia vera, ruxolitinib had 2.3% six-month complete responses versus 2.9% with other therapy
In this randomized trial, first-line ruxolitinib did not improve the six-month complete response rate over best available therapy: responses occurred in about 2% to 3% of patients in each group. Ruxolitinib improved hematocrit, spleen enlargement, itching and fatigue more, and patients stayed on treatment longer, but the trial was small and platelet reduction favored other therapy.
- Journal
- Blood cancer journal (Q1)
- Published
- 22 October 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Steffen Koschmieder, Susanne Isfort, Lino L Teichmann, Philippe Schafhausen, Florian H Heidel, Carl Crodel, et al.
- PMID
- 42918761
- DOI
- 10.1038/s41408-026-01644-2
Why clinicians should know about it
- Picked for Oncology and Radiation Oncology (top studies of the week, 25 October 2026): Randomized first-line ruxolitinib trial in polycythemia vera
- Picked for Hematology (top studies of the week, 25 October 2026): Randomized first-line ruxolitinib trial in polycythaemia vera
Abstract
Ruxolitinib (RUX) is approved as second-line treatment for HU-intolerant/-resistant patients with polycythemia vera (PV). Here, we assessed RUX vs. best available therapy (BAT) as first-line treatment in PV in the randomized multicenter RuxoBEAT clinical trial (NCT02577926). Crossover from BAT to RUX was possible after 6 months. Baseline characteristics in the RUX (n = 44) and BAT (n = 34) arms were comparable. The primary endpoint, the clinico-hematologic complete response (CR) rate at month 6, was not met (2.3% vs. 2.9%, p = 1.000). Analyses of secondary and exploratory endpoints showed that RUX was significantly more effective than BAT in improving hematocrit, splenomegaly, and patient-reported pruritus and fatigue, but less effective in reducing platelet counts. Compared to baseline, JAK2V617F variant allele frequency decreased significantly in both RUX and BAT groups at 6 months. The time on treatment (p < 0.001) and duration of treatment (p = 0.022) were significantly superior for RUX vs. BAT during the 24-month study period. There were no new safety signals. These findings demonstrate that RUX did not lead to higher CR rates than BAT and suggest that RUX may induce clinically meaningful additional disease improvements in first-line therapy of PV patients.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.