JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis

Claire Harrison, Jean Jacques Kiladjian, Haifa Kathrin Al-Ali, Heinz Gisslinger, Roger Waltzman, Viktoriya Stalbovskaya, Mari McQuitty, Deborah S. Hunter, Richard Levy, Laurent Knoops, Francisco Cervantes, Alessandro M. Vannucchi, Tiziano Barbui, Giovanni Barosi

Research output: Contribution to journalArticlepeer-review

Abstract

BACKGROUND: Treatment options for myelofibrosis are limited. We evaluated the efficacy and safety of ruxolitinib, a potent and selective Janus kinase (JAK) 1 and 2 inhibitor, as compared with the best available therapy, in patients with myelofibrosis. METHODS: We assigned 219 patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis to receive oral ruxolitinib or the best available therapy. The primary end point and key secondary end point of the study were the percentage of patients with at least a 35% reduction in spleen volume at week 48 and at week 24, respectively, as assessed with the use of magnetic resonance imaging or computed tomography. RESULTS: A total of 28% of the patients in the ruxolitinib group had at least a 35% reduction in spleen volume at week 48, as compared with 0% in the group receiving the best available therapy (P

Original languageEnglish
Pages (from-to)787-798
Number of pages12
JournalNew England Journal of Medicine
Volume366
Issue number9
DOIs
Publication statusPublished - Mar 1 2012

ASJC Scopus subject areas

  • Medicine(all)

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