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Ruxolitinib versus best available therapy for ET intolerant or resistant to hydroxycarbamide in a randomized trial.

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, Ruben and Dueck, Amylou C and Cross, Nicholas C P and Mesa, Ruben and McMullin, Mary Frances (2017) Ruxolitinib versus best available therapy for ET intolerant or resistant to hydroxycarbamide in a randomized trial. Blood. ISSN 1528-0020. This article is available to all HEFT staff and students via ASK HEFT Discovery tool http://tinyurl.com/z795c8c using their HEFT Athens Login.

Full text not available from this repository.
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 II trial of ruxolitinib (JAK1/2 inhibitor) vs Best Available Therapy (BAT) in ET and polycythemia vera (PV) patients resistant or intolerant to HC. Here findings of MAJIC-ET are reported, where the modified intention-to-treat population included 58 & 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 two complete molecular responses (CMR) and one partial molecular response (PMR) in CALR positive ruxolitinib-treated patients. Transformation to myelofibrosis occurred in one CMR patient, presumably due to the emergence of a different clone raising questions about the relevance of CMR in ET patients. Grade 3&4 anemia occurred in 19% & 0% of ruxolitinib vs 0% (both grades) BAT arm, grade 3&4 thrombocytopenia in 5.2% & 1.7% of ruxolitinib vs 0% (both grades) of BAT treated patients. Rates of discontinuation or treatment switching did not differ between the two trial arms. The MAJIC-ET trial suggests that ruxolitinib is not superior to current second-line treatments for ET.

Item Type: Article
Additional Information: This article is available to all HEFT staff and students via ASK HEFT Discovery tool http://tinyurl.com/z795c8c using their HEFT Athens Login.
Subjects: QZ Pathology. Oncology
WH Haemic and lymphatic systems. Haematology
Divisions: Planned IP Care > Oncology and Clinical Haematology
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Depositing User: Mr Philip O'Reilly
Date Deposited: 27 Nov 2017 14:29
Last Modified: 27 Nov 2017 14:29
URI: http://www.repository.heartofengland.nhs.uk/id/eprint/1532

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