Ruxolitinib vs best available therapy for et intolerant or resistant to hydroxycarbamide

Claire N. Harrison, Adam J. Mead, Anesh Panchal, Sonia Fox, Christina Yap, Emmanouela Gbandi, Aimee Houlton, Samah Alimam, Joanne Ewing, Marion Wood, Frederick Chen, Jason Coppell, Nicki Panoskaltsis, Steven Knapper, Sahra Ali, Angela Hamblin, Robyn Scherber, Amylou C. Dueck, Nicholas C.P. Cross, Ruben MesaMary Frances McMullin

Research output: Contribution to journalArticlepeer-review

107 Scopus citations


Treatments for high-risk essential thrombocythemia (ET) address thrombocytosis, diseaserelated symptoms, as well as risks of thrombosis, hemorrhage, transformation to myelofibrosis, and leukemia. Patients resistant/intolerant to hydroxycarbamide (HC) have a poor outlook. MAJIC (ISRCTN61925716) is a randomized phase 2 trial of ruxolitinib (JAK1/2 inhibitor) vs best available therapy (BAT) in ET and polycythemia vera patients resistant or intolerant to HC. Here, findings of MAJIC-ET are reported, where the modified intention-to-treat population included 58 and 52 patients randomized to receive ruxolitinib or BAT, respectively.Therewasnoevidenceof improvement incomplete responsewithin1year reported in 27 (46.6%) patients treated with ruxolitinib vs 23 (44.2%) with BAT (P 5.40). At 2 years, rates of thrombosis, hemorrhage, and transformationwere not significantly different; however, some disease-related symptoms improved in patients receiving ruxolitinib relative to BAT.Molecular responses were uncommon; there were 2 completemolecular responses (CMR) and 1 partial molecular response in CALR-positive ruxolitinib-treated patients. Transformation to myelofibrosis occurred in 1 CMR patient, presumably because of the emergence of a different clone, raising questions about the relevance of CMR in ET patients. Grade 3 and 4 anemia occurred in 19% and 0%of ruxolitinib vs 0% (both grades) in the BAT arm, and grade 3 and 4 thrombocytopenia in 5.2% and 1.7% of ruxolitinib vs 0% (both grades) of BAT-treated patients. Rates of discontinuation or treatmentswitching didnot differ between the 2 trial arms. TheMAJIC-ET trial suggests that ruxolitinib is not superior to current second-line treatments for ET.

Original languageEnglish (US)
Pages (from-to)1889-1897
Number of pages9
Issue number17
StatePublished - Oct 26 2017
Externally publishedYes

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology


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