Beta Thalassemia: Monitoring and New Treatment Approaches.

Beta thalassemias are a significant global health problem. Globin chain imbalance leads to a complex physiologic cascade of hemolytic anemia, ineffective erythropoiesis, and iron overload. Management of the broad spectrum of phenotypes requires the careful use of red blood transfusions, supportive care, monitoring, and management of iron overload. In this article, the authors discuss recommendations for monitoring of individuals with thalassemia, as well as ongoing preclinical and clinical trials of therapies targeting different aspects of thalassemia pathophysiology.