Exposure to atovaquone reduces disease burden and prolongs survival in a xenograft model of AML. (A-B) Female NSG mice were injected by tail vein with 5 × 105 THP-1.ffluc cells. Treatment with atovaquone (AQ; 200 mg/kg; n = 6) or VC (n = 5) by daily oral gavage began on the day of cell injection. Bioluminescent imaging demonstrated decreased disease burden in AQ-treated mice. These mice were not followed for survival. (C-D) Male NSG mice were injected by tail vein with 5 × 105 THP-1.ffluc cells. Treatment with AQ (200 mg/kg per day; n = 7) or VC (n = 7) by daily oral gavage began on the day of cell injection. Bioluminescent imaging demonstrated decreased disease burden at the time points noted (curves were significantly different by analysis of variance P = .0069). (E) Male AQ-treated mice had significantly prolonged survival compared with controls. *P < .05 by Student t test.