Effects of Thalidomide on Erythropoiesis and Iron Homeostasis in Transfusion-Dependent β-Thalassemia

Thalidomide is a therapeutic option for patients with β-thalassemia by increasing fetal hemoglobin and thereby reducing the requirement for blood transfusions. However, information on changes in erythropoiesis and iron homeostasis during thalidomide treatment is lacking. This study investigated the effects of thalidomide treatment on hematologic, erythropoietic, and ironstatus parameters in 22 patients with transfusion-dependent β-thalassemia (TDT). Thalidomide significantly improved anemia endpoints, including increases in hemoglobin (p<0.001), red blood cells (p<0.001), and hematocrit (p<0.001), as well as reducing erythropoietin levels (p=0.033) and ameliorating erythropoiesis. Thalidomide treatment significantly reduced serum iron levels (p=0.018) and transferrin saturation (p=0.039) and increased serum transferrin levels (p=0.030). Thalidomide had no observed effect on serum ferritin or hepcidin, but changes in hepcidin (r=0.439, p=0.041) and serum iron (r=-0.536, p=0.010) were significantly correlated with hemoglobin increment. This comprehensive study indicates that thalidomide treatment can ameliorate erythropoiesis and iron homeostasis in patients with TDT, thus supporting the effectiveness of this drug.