Fig. 5

Sorafenib and Regorafenib treatment suppressed AML engraftment in vivo. Magnetically sorted CD34⁺ pooled BM cells and splenocytes from secondary engrafted NSG mice were injected intrahepatically in newborn NSG pups after sublethal irradiation (1 × 10⁵ cells per pup). Successfully engrafted mice with more than 30 human CD45⁺ cells per microliter of blood (between week 12 to 16 post-engraftment) were randomly assigned to either untreated (n = 3), Regorafenib (n = 6; 5 mg/kg body weight; gavage-fed once daily) or Sorafenib (n = 6; 10 mg/kg body weight; gavage-fed once daily) treatment groups and monitored for 1 month. a Longitudinal effect of Regorafenib and Sorafenib treatment on peripheral blood engraftment at week 0, 2, and 4 post-drug treatment. Change in AML engraftment for each group at each time point is expressed as fold change relative to the absolute human CD45⁺ count per microliter of blood at week 0 post-drug treatment. Data are presented as mean fold change ± SEM. Two-tailed Mann Whitney U test; *p < 0.05. b Soft tissue sarcoma and reduced spleen size were observed in Regorafenib- or Sorafenib-treated mice. Representative images of abdomen and spleen was shown; scale bar: 1 cm. Weight of spleen are presented as mean ± SEM. Two-tailed Mann Whitney U test; *p < 0.05. c Comparison of the absolute count of human CD45⁺ cells (c, above) and CD34⁺ cells (c, below) in peripheral blood, spleen, and BM between different treatment groups after 4 weeks post-drug treatment. Data are presented as mean absolute count ± SEM. Two-tailed Mann Whitney U test; *p < 0.05