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

Harrison, Claire N., Mead, Adam J., Panchal, Anesh, Fox, Sonia, Yap, Christina, Gbandi, Emmanouela, Houlton, Aimee, Alimam, Samah, Ewing, Joanne, Wood, Marion, Chen, Frederick, Coppell, Jason, Panoskaltsis, Nicki, Knapper, Steven ORCID:, Ali, Sahra, Hamblin, Angela, Scherber, Robyn, Dueck, Amylou C., Cross, Nicholas C. P., Mesa, Ruben and McMullin, Mary Frances 2017. Ruxolitinib vs best available therapy for ET intolerant or resistant to hydroxycarbamide. Blood 130 (17) , pp. 1889-1897. 10.1182/blood-2017-05-785790

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Treatments for high-risk essential thrombocythemia (ET) address thrombocytosis, disease-related 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. There was no evidence of improvement in complete response within 1 year reported in 27 (46.6%) patients treated with ruxolitinib vs 23 (44.2%) with BAT (P = .40). At 2 years, rates of thrombosis, hemorrhage, and transformation were not significantly different; however, some disease-related symptoms improved in patients receiving ruxolitinib relative to BAT. Molecular responses were uncommon; there were 2 complete molecular 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 treatment switching did not differ between the 2 trial arms. The MAJIC-ET trial suggests that ruxolitinib is not superior to current second-line treatments for ET.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Additional Information: Title on accepted manuscript slightly different to title on published paper.
Publisher: American Society of Hematology
ISSN: 0006-4971
Date of First Compliant Deposit: 11 October 2018
Date of Acceptance: 24 July 2017
Last Modified: 11 Nov 2023 22:15

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