DZNep showed potent anti-AML efficacy in vitro and in vivo. (A) AML cell lines were treated with either DMSO control or DZNep at dose of 0.5, 1, and 5μM for 48 hours. Cells were harvested, washed, and stained with annexin V/PI double dye, then subjected to flow cytometric analysis. (B) Mice were treated with PBS control or DZNep 2 mg/kg/d, respectively. Tumor volume curves were constructed with measurements taken by caliper. Average of tumor volume was calculated as the average of 7 mice in each group ± SD. (C) Survival analysis showed that DZNep treatment improved the survival time of mice inoculated with human leukemia (P < .05). Seven mice in each group were used for the construction of the survival curves.