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

Harrison, Claire N and Mead, Adam J and Panchal, Anesh and Fox, Sonia and Yap, Christina and Gbandi, Emmanouela and Houlton, Aimee and Alimam, Samah and Ewing, Joanne and Wood, Marion and Chen, Frederick and Coppell, Jason and Panoskaltsis, Nicki and Knapper, Steven and Ali, Sahra and Hamblin, Angela and Scherber, Robyn and Dueck, Amylou C and Cross, Nicholas C P and 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. ISSN 1528-0020.

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Official URL: http://www.bloodjournal.org/content/130/17/1889

Abstract

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. This trial was registered at www.isrctn.com as #ISRCTN61925716.

Item Type: Article
Subjects: WH Haemic and lymphatic systems. Haematology
Divisions: Planned IP Care > Oncology and Clinical Haematology
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Depositing User: Miss Emily Johnson
Date Deposited: 26 Jan 2018 13:54
Last Modified: 26 Jan 2018 13:54
URI: http://www.repository.heartofengland.nhs.uk/id/eprint/1450

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