Journal
EUROPEAN JOURNAL OF HAEMATOLOGY
Volume 93, Issue 6, Pages 533-536Publisher
WILEY
DOI: 10.1111/ejh.12334
Keywords
Acute myeloid leukemia; FLT3-ITD mutation; NPM1 mutation; sorafenib; all trans retinoid acid; targeted therapy
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Knowledge of the molecular basis of acute myeloid leukaemia has increased considerably in the past few years, and therapies targeting specific molecular defects of this disease are intensively investigated. Patients with both NPM1 and FLT3-ITD mutations encompass 20% of cytogenetically normal AML. The multikinase and FLT3 inhibitor, sorafenib, has shown some efficacy in patients with relapsed FLT3-ITD+ AML. In addition, it is suggested that all-trans retinoic acid (ATRA) used in combination with chemotherapy has shown to improve outcome of patients harbouring NPM1 mutations. We report here the clinical course of three patients with refractory or relapsed FLT3-ITD+/NPM1(+) AML who achieved significant response upon sorafenib and ATRA combination.
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