Abstract: OBJECTIVE To investigate the injuring effect and mechanism of multikinase inhibitor chiauranib on FLT3-ITD positive acute myeloid leukemia(AML). METHODS CCK8 and colony-forming assay were separately utilized to determine cell viability and proliferation of FLT3-ITD positive AML cells exposure with various concentration of chiauranib. FLT3-ITD positive AML cell lines and primary cells treated with increasing dosage of chiauranib in designated timepoints were assessed with Annexin V/PI dual-staining assay. Click-iT^® EdU kit was used to evaluated the distribution of cell cycle. Subcutaneous xenograft model by injection with FLT3-ITD AML cells was adopted to evaluate the in vivo antileukemia efficacy of chiauranib. The underlying mechanism of chiauranib cytotoxicity was explored by Western blotting assay. RESULTS In this study, treatment FLT3-ITD positive AML cell lines with chiauranib resulted in significant reduction of cell viability, suppression of cell colony formation, blockade of cell cycle and promotion of cell apoptosis; primary FLT3-ITD AML cells obtained from 8 AML patients were separately exposed to various chiauranib doses and exhibited great sensitivity to chiauranib cytotoxicity. The anti-AML efficacy of chiauranib was validated in the FLT3-ITD AML xenograft model and had no significant safety profiles. Dephosphorylation of VEGFR2 and its downstream targets MEK and Erk was associated with the antitumor effects of chiauranib on FLT3-ITD positive AML cellular models. CONCLUSION This study demonstrates that chiauranib shows potent antileukemic effects on FLT3-ITD positive AML cells in vitro and in vivo possibly via perturbation of the activity of VEGFR2/MEK/Erk pathway.