Figure 2.

Mitapivat-treated transfused β-thalassemia mice show reduced liver iron accumulation and improved iron homeostasis. (A) Left and central panels: iron staining (Perl’s Prussian blue is a semi-quantitative method to assess organ iron accumulation) in liver from wild-type (WT) and Hbb^th3/+ mice treated with either vehicle or transfusion (Tr.) or transfusion plus mitapivat. One representative image from 5 with similar results. Right panel: quantification of iron staining in liver. Data are mean ± standard error of the mean (SEM) (n=5). °P<0.05 compared to WT, *P<0.05 compared with vehicle Hbb^th3/+ mice and ^#P<0.05 compared with vehicle-treated transfused (Tr.) Hbb^th3/+ mice. (B) Liver mRNA expression normalized over liver iron concentration (LIC) as determined using the bathophenanthroline method. Data are presented as means ± SEM (n=3). ^#P<0.05 compared with vehicle-treated transfused Hbb^th3/+ mice. (C) Transferrin saturation in Hbb^th3/+ mice treated with either vehicle or transfusion or transfusion plus mitapivat. Transferrin saturation was calculated as the ratio between serum iron and total iron binding capacity, using the Total Iron Binding Capacity Kit (Randox Laboratories, UK) and 50 mL of serum, according to the manufacturer’s instructions. Data are presented as means ± SEM (n=3). *P<0.05 compared with vehicle Hbb^th3/+ mice and ^#P<0.05 compared with vehicle-treated transfused Hbb^th3/+ mice. RBC: red blood cells.