Koschmieder, Steffen ORCID: 0000-0002-1011-8171, Isfort, Susanne, Wolf, Dominik, Heidel, Florian H., Hochhaus, Andreas, Schafhausen, Philippe, Griesshammer, Martin, Wolleschak, Denise, Platzbecker, Uwe, Doehner, Konstanze, Jost, Philipp J., Parmentier, Stefani, Schaich, Markus, von Bubnoff, Nikolas, Stegelmann, Frank, Maurer, Angela, Crysandt, Martina, Gezer, Deniz, Kortmann, Maike, Franklin, Jeremy, Frank, Julia, Hellmich, Martin and Bruemmendorf, Tim H. (2023). Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group. Ann. Hematol., 102 (2). S. 349 - 359. NEW YORK: SPRINGER. ISSN 1432-0584

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Abstract

Patients (pts) with polycythemia vera (PV) suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis. Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. The RuxoBEAT trial (NCT02577926, registered on October 1, 2015, at clinicaltrials.gov) is a multicenter, open-label, two-arm phase-IIb trial with a target population of 380 pts with PV or ET, randomized to receive RUX or best available therapy. This pre-specified futility analysis assesses the early clinical benefit and tolerability of RUX in previously untreated PV pts (6-week cytoreduction was allowed). Twenty-eight patients were randomly assigned to receive RUX. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46 to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend for reduced night sweat scores (1.5 to 0). JAK2V617F allele burden, as part of the scientific research program, also significantly decreased. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Koschmieder, SteffenUNSPECIFIEDorcid.org/0000-0002-1011-8171UNSPECIFIED
Isfort, SusanneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wolf, DominikUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Heidel, Florian H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hochhaus, AndreasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schafhausen, PhilippeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Griesshammer, MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wolleschak, DeniseUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Platzbecker, UweUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Doehner, KonstanzeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jost, Philipp J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Parmentier, StefaniUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schaich, MarkusUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
von Bubnoff, NikolasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stegelmann, FrankUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Maurer, AngelaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Crysandt, MartinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gezer, DenizUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kortmann, MaikeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Franklin, JeremyUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Frank, JuliaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hellmich, MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bruemmendorf, Tim H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-696352
DOI: 10.1007/s00277-022-05080-7
Journal or Publication Title: Ann. Hematol.
Volume: 102
Number: 2
Page Range: S. 349 - 359
Date: 2023
Publisher: SPRINGER
Place of Publication: NEW YORK
ISSN: 1432-0584
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
MYELOPROLIFERATIVE NEOPLASMS; ROPEGINTERFERON ALPHA-2B; STANDARD THERAPY; DOUBLE-BLIND; SYMPTOM; RISK; BURDEN; SWEDENMultiple languages
HematologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/69635

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