Ruxolitinib versus standard therapy for the treatment of polycythemia vera

Alessandro M. Vannucchi, Jean Jacques Kiladjian, Martin Griesshammer, T. Masszi, Simon Durrant, Francesco Passamonti, Claire N. Harrison, Fabrizio Pane, Pierre Zachee, Ruben Mesa, Shui He, Mark M. Jones, William Garrett, Jingjin Li, Ulrich Pirron, Dany Habr, Srdan Verstovsek

Research output: Contribution to journalArticle

266 Citations (Scopus)

Abstract

Background Ruxolitinib, a Janus kinase (JAK) 1 and 2 inhibitor, was shown to have a clinical benefit in patients with polycythemia vera in a phase 2 study. We conducted a phase 3 open-label study to evaluate the efficacy and safety of ruxolitinib versus standard therapy in patients with polycythemia vera who had an inadequate response to or had unacceptable side effects from hydroxyurea.

Methods We randomly assigned phlebotomy-dependent patients with splenomegaly, in a 1: 1 ratio, to receive ruxolitinib (110 patients) or standard therapy (112 patients). The primary end point was both hematocrit control through week 32 and at least a 35% reduction in spleen volume at week 32, as assessed by means of imaging.

Results The primary end point was achieved in 21% of the patients in the ruxolitinib group versus 1% of those in the standard-therapy group (P

Conclusions In patients who had an inadequate response to or had unacceptable side effects from hydroxyurea, ruxolitinib was superior to standard therapy in controlling the hematocrit, reducing the spleen volume, and improving symptoms associated with polycythemia vera.

Original languageEnglish
Pages (from-to)426-435
Number of pages10
JournalNew England Journal of Medicine
Volume372
Issue number5
DOIs
Publication statusPublished - Jan 1 2015

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Polycythemia Vera
Hydroxyurea
Hematocrit
Therapeutics
Janus Kinase 1
Spleen
Janus Kinase 2
Phlebotomy
Splenomegaly
Group Psychotherapy
INCB018424
Safety

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Vannucchi, A. M., Kiladjian, J. J., Griesshammer, M., Masszi, T., Durrant, S., Passamonti, F., ... Verstovsek, S. (2015). Ruxolitinib versus standard therapy for the treatment of polycythemia vera. New England Journal of Medicine, 372(5), 426-435. https://doi.org/10.1056/NEJMoa1409002

Ruxolitinib versus standard therapy for the treatment of polycythemia vera. / Vannucchi, Alessandro M.; Kiladjian, Jean Jacques; Griesshammer, Martin; Masszi, T.; Durrant, Simon; Passamonti, Francesco; Harrison, Claire N.; Pane, Fabrizio; Zachee, Pierre; Mesa, Ruben; He, Shui; Jones, Mark M.; Garrett, William; Li, Jingjin; Pirron, Ulrich; Habr, Dany; Verstovsek, Srdan.

In: New England Journal of Medicine, Vol. 372, No. 5, 01.01.2015, p. 426-435.

Research output: Contribution to journalArticle

Vannucchi, AM, Kiladjian, JJ, Griesshammer, M, Masszi, T, Durrant, S, Passamonti, F, Harrison, CN, Pane, F, Zachee, P, Mesa, R, He, S, Jones, MM, Garrett, W, Li, J, Pirron, U, Habr, D & Verstovsek, S 2015, 'Ruxolitinib versus standard therapy for the treatment of polycythemia vera', New England Journal of Medicine, vol. 372, no. 5, pp. 426-435. https://doi.org/10.1056/NEJMoa1409002
Vannucchi AM, Kiladjian JJ, Griesshammer M, Masszi T, Durrant S, Passamonti F et al. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. New England Journal of Medicine. 2015 Jan 1;372(5):426-435. https://doi.org/10.1056/NEJMoa1409002
Vannucchi, Alessandro M. ; Kiladjian, Jean Jacques ; Griesshammer, Martin ; Masszi, T. ; Durrant, Simon ; Passamonti, Francesco ; Harrison, Claire N. ; Pane, Fabrizio ; Zachee, Pierre ; Mesa, Ruben ; He, Shui ; Jones, Mark M. ; Garrett, William ; Li, Jingjin ; Pirron, Ulrich ; Habr, Dany ; Verstovsek, Srdan. / Ruxolitinib versus standard therapy for the treatment of polycythemia vera. In: New England Journal of Medicine. 2015 ; Vol. 372, No. 5. pp. 426-435.
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