Management of thalassaemia

Thalassaemias are the commonest single gene disorders in the world. They result from reduced synthesis of α or β-globin genes and can result in an asymptomatic carrier status, mild anaemia or severe anaemia which may result in intrauterine death or necessitate lifelong transfusion to sustain life. β-thalassaemia major is the most common of the most severe phenotypes. There has been significant improvement in clinical care particularly relating to transfusion safety and management of iron overload. Challenges remain to further improve care, quality of life and dealing with a globally rising incidence and prevalence of thalassaemias. This review gives an overview of the treatment of β-thalassaemia major and the management of its complications.