Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial
19 affiliations
- 1Aachen, Germany
- 2Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany
- 3Hamburg, Germany
- 4Hannover, Germany
- 5Jena, Germany
- 6Minden, Germany
- 7TU Dresden, Germany
- 8Munich, Germany
- 9Magdeburg, Germany
- 10Ulm, Germany
- 11Essen, Germany
- 12Mainz, Germany
- 13Nürnberg, Germany
- 14Freiburg, Germany
- 15Düsseldorf, Germany
- 16Winnenden, Germany
- 17Gemeinschaftspraxis, Aschaffenburg, Germany
- 18Lübeck, Germany
- 19Cologne, Germany
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.
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BibTeX
@article{koschmieder2026firstline,
title = {{Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial}},
author = {Steffen Koschmieder and Susanne Isfort and Lino L. Teichmann and Philippe Schafhausen and Florian H. Heidel and Carl Crodel and Andreas Hochhaus and Martin Griesshammer and Katja Sockel and Philipp J. Jost and Denise Walther and Konstanze Döhner and Joachim R. Göthert and Markus Radsak and Marinela Augustin and Heiko Becker and Aristoteles Giagounidis and Markus Schaich and Martine Klausmann and Nikolas von Bubnoff and Frank Stegelmann and Maike Kortmann and Michelle Zentis and Julia Frank and Anna Hagemeier and Martin Hellmich and Tim H. Brümmendorf},
journal = {Blood cancer journal},
year = {2026},
month = oct,
doi = {10.1038/s41408-026-01644-2},
url = {https://europepmc.org/article/PMC/PMC13691084},
note = {CC BY},
}
RIS
TY - JOUR
TI - Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial
AU - Steffen Koschmieder
AU - Susanne Isfort
AU - Lino L. Teichmann
AU - Philippe Schafhausen
AU - Florian H. Heidel
AU - Carl Crodel
AU - Andreas Hochhaus
AU - Martin Griesshammer
AU - Katja Sockel
AU - Philipp J. Jost
AU - Denise Walther
AU - Konstanze Döhner
AU - Joachim R. Göthert
AU - Markus Radsak
AU - Marinela Augustin
AU - Heiko Becker
AU - Aristoteles Giagounidis
AU - Markus Schaich
AU - Martine Klausmann
AU - Nikolas von Bubnoff
AU - Frank Stegelmann
AU - Maike Kortmann
AU - Michelle Zentis
AU - Julia Frank
AU - Anna Hagemeier
AU - Martin Hellmich
AU - Tim H. Brümmendorf
PY - 2026
DA - 2026/10/22
JO - Blood cancer journal
DO - 10.1038/s41408-026-01644-2
UR - https://europepmc.org/article/PMC/PMC13691084
AB - 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.
SN - 2044-5385
ER -