Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia.
David J KuterMerlin EfraimJiri MayerMarek TrněnýVickie McDonaldRobert BirdThomas RegenbogenMamta GargZane KaplanNikolay TzvetkovPhilip Young-Ill ChoiArend Jan Gerard JansenMilan KostalRoss BakerJaromir GumulecEun-Ju LeeIlona CunninghamIsaac GoncalvesMargaret WarnerRalph BocciaTerry GernsheimerWaleed GhanimaOlga BandmanRegan BurnsAnn NealeDolca ThomasPuneet AroraBeiyao ZhengNichola CooperPublished in: The New England journal of medicine (2022)
Rilzabrutinib was active and associated with only low-level toxic effects at all dose levels. The dose of 400 mg twice daily was identified as the dose for further testing. Overall, rilzabrutinib showed a rapid and durable clinical activity that improved with length of treatment. (Funded by Sanofi; ClinicalTrials.gov number, NCT03395210; EudraCT number, 2017-004012-19.).
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