Journal Article

DKFZ-2026-02620

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial.

Koschmieder, S. ; Isfort, S. ; Teichmann, L. L. ; Schafhausen, P. ; Heidel, F. H. ; Crodel, C. ; Hochhaus, A. ; Griesshammer, M. ; Sockel, K. ; Jost, P. J. ; Walther, D. ; Döhner, K. ; Göthert, J. R. ; Radsak, M.DKFZ* ; Augustin, M. ; Becker, H.DKFZ* ; Giagounidis, A. ; Schaich, M. ; Klausmann, M. ; von Bubnoff, N. ; Stegelmann, F. ; Kortmann, M. ; Zentis, M. ; Frank, J. ; Hagemeier, A. ; Hellmich, M. ; Brümmendorf, T. H. ; Neoplasms, G. S. G. f. M. (Collaboration Author)

2026
Springer Nature [London]

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.

Keyword(s): Humans (MeSH) ; Polycythemia Vera: drug therapy (MeSH) ; Polycythemia Vera: genetics (MeSH) ; Polycythemia Vera: pathology (MeSH) ; Female (MeSH) ; Pyrimidines (MeSH) ; Nitriles (MeSH) ; Male (MeSH) ; Pyrazoles: therapeutic use (MeSH) ; Pyrazoles: adverse effects (MeSH) ; Pyrazoles: administration & dosage (MeSH) ; Janus Kinase 2: genetics (MeSH) ; Aged (MeSH) ; Middle Aged (MeSH) ; Treatment Outcome (MeSH) ; ruxolitinib ; Pyrimidines ; Nitriles ; Pyrazoles ; Janus Kinase 2 ; JAK2 protein, human

Note: #DKTKZFB9#

---

Contributing Institute(s):

1. DKTK Koordinierungsstelle Frankfurt (FM01)
2. DKTK Koordinierungsstelle Freiburg (FR01)

Research Program(s):

1. 899 - ohne Topic (POF4-899) (POF4-899)

Appears in the scientific report 2026

---

Database coverage:
; ; ; Article Processing Charges ; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; DOAJ Seal ; Essential Science Indicators ; Fees ; IF >= 10 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

---