FLT3-targeted Therapy Restores GATA1 Pathway Function in NPM1/FLT3-ITD Mutated Acute Myeloid Leukaemia

One-third of newly diagnosed adult acute myeloid leukaemia (AML) carry mutations, which frequently occur together with nucleophosmin () mutations and are associated with worse prognosis. inhibitors are widely used in clinics with limitations due to drug resistance. AML cells carrying mutations in both mouse models and patients present low expression of GATA1, a gene involved in haematopoietic changes preceding AML. Here, we show that FLT3 inhibition induces cellular responses and restores the GATA1 pathway and functions in 1/-ITD mutated AML, thus providing a new mechanism of action for this drug.