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

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Date
2017-10
ICR Author
Yap, Christina
Author
Harrison, CN
Mead, AJ
Panchal, A
Fox, S
Yap, C
Gbandi, E
Houlton, A
Alimam, S
Ewing, J
Wood, M
Chen, F
Coppell, J
Panoskaltsis, N
Knapper, S
Ali, S
Hamblin, A
Scherber, R
Dueck, AC
Cross, NCP
Mesa, R
McMullin, MF
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Type
Journal Article
Metadata
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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.
URI
https://repository.icr.ac.uk/handle/internal/3459
DOI
https://doi.org/10.1182/blood-2017-05-785790
Collections
  • Clinical Studies
Subject
Humans
Disease Progression
Hemorrhage
Hydroxyurea
Pyrazoles
Treatment Outcome
Withholding Treatment
Drug Resistance
Adult
Aged
Aged, 80 and over
Middle Aged
Female
Male
Thrombocythemia, Essential
Research team
Clinical Trials & Statistics Unit
Language
eng
Date accepted
2017-07-24
License start date
2017-10
Citation
Blood, 2017, 130 (17), pp. 1889 - 1897

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