Luspatercept for treating anaemia in patients with α-thalassemia haemoglobin H disease

Luspatercept is being developed to treat anaemia (low red blood cell levels) in adults with alpha (α)-thalassaemia haemoglobin H (HbH) disease. HbH disease is a rare inherited blood disorder caused by faults in three of the four genes that make alpha-globin, a part of haemoglobin. This leads to the formation of abnormal haemoglobin, which damages red blood cells and causes anaemia. People with HbH disease may feel tired, look pale, or develop jaundice (yellowing of the skin and eyes). Some patients need regular blood transfusions, which can cause iron overload and other complications.