In a phase II single-arm trial, patients with high-risk acute myeloid leukaemia (AML) received the FLT3 inhibitor quizartinib: patients >60 years of age with relapsed (within 1 year) and/or refractory AML were assigned to cohort 1, while patients >18 years of age with relapsed and/or refractory AML following salvage chemotherapy or haemopoietic stem cell transplantation were assigned to cohort 2. The majority (74.4%) of patients had detectable FLT3-internal tandem duplications, which are typically associated with an inferior prognosis. Similar response rates of 77% and 74% were observed in cohorts 1 and 2, respectively, with similar frequencies of composite complete remissions (56% and 46%) and complete remission (3% and 4%) also observed. Quizartinib is currently under further investigation in phase III trials.
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Cortes, J. et al. Quizartinib, an FLT3 inhibitor, as monotherapy in patients with relapsed or refractory acute myeloid leukaemia: an open-label, multicentre, single-arm, phase 2 trial. Lancet Oncol. https://doi.org/10.1016/S1470-2045(18)30240-7 (2018)
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Sidaway, P. Quizartinib tested in patients with high-risk AML. Nat Rev Clin Oncol 15, 532 (2018). https://doi.org/10.1038/s41571-018-0061-8
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DOI: https://doi.org/10.1038/s41571-018-0061-8
