Hemoglobinopathies

Blood transfusions have been a common therapy for severe thalassemia, but transfusions do not cure the disease, and frequent transfusions can cause iron overload, an illness caused by excessively high levels of iron. A drug, called an iron chelator, may be given to bind with the excess iron. Iron chelators can produce additional side effects, such as hearing loss and reduced growth.

As with sickle cell disease, gene therapy and bone marrow transplants are very promising therapies for severe thalassemias. While transplants are risky procedures and can cause death, they are more likely to be successful when performed on young and relatively healthy children.