- Are New Treatment Options for Beta Thalassemia Ready for Widespread Use?
Saturday, June 1st, 2019
Patients with transfusion-dependent beta thalassemia, the most severe form of the disease, require lifelong management with red blood cell (RBC) transfusions, presenting a substantial cost and burden to patients. The only curative treatment for patients with transfusion-dependent beta thalassemia is allogeneic hematopoietic cell transplantation (alloHCT), which also is associated with a variety of adverse events. [...]