Luspatercept in patients with non-transfusion dependent β-thalassaemia

Abstract

Ineffective erythropoiesis leads to the development of chronic anaemia and iron overload in patients with non-transfusion dependent β-thalassemia, which is associated with significant morbidity, risk of mortality, and loss of health-related quality of life (HRQoL) especially in patients who have haemoglobin concentrations lower than 10 g/dL.1 There are no approved drugs directed at the management of anaemia in patients with non-transfusion dependent β-thalassemia, leaving supportive care with occasional red blood cell transfusions and iron chelation as the only approved therapeutic options for these patients.