Standard Treatments
Blood Transfusions
Transfusions of red blood cells are the main treatment for people who have moderate or severe thalassemias. This treatment provides healthy red blood cells with normal hemoglobin, although transfusions are typically required every two weeks for those will beta thalassemia major. |
Iron Chelation Therapy
The hemoglobin in red blood cells is an iron-rich protein. Thus, regular blood transfusions can lead to a buildup of iron in the blood. This condition is called iron overload. It damages the liver, heart, and other parts of the body. To prevent this damage, doctors use iron chelation therapy to remove excess iron from the body. |
Vitamin Supplements
Folic acid is a B vitamin that helps build healthy red blood cells and must be taken daily by those with beta thalassemia minor and major in order to counteract anemia. |
Advanced/Future Treatment
Blood and Marrow Stem Cell Transplant
This is the only treatment that can cure beta thalassemia. A blood and marrow transplant replaces the faulty stem cells in bone marrow with healthy ones from a donor. As a result, the stem cells begin to create healthy red blood cells with the proper amount of hemoglobin and oxygen. For low risk patients (i.e. no organ disease, young, etc.) there is about a 90% long term survival rate.
This is the only treatment that can cure beta thalassemia. A blood and marrow transplant replaces the faulty stem cells in bone marrow with healthy ones from a donor. As a result, the stem cells begin to create healthy red blood cells with the proper amount of hemoglobin and oxygen. For low risk patients (i.e. no organ disease, young, etc.) there is about a 90% long term survival rate.
LentiGlobin BB305 In February, 2015, the FDA gave LentiGlobin BB305 "breakthrough therapy" status and allowed it to expedite clinical trials on the drug. It is known as a lentiviral vector (gene delivery vehicle) which inserts a functioning version of the HBB gene into the patient's stem cells ex vivo. The reintroduced cells are then reintroduced into the patient. |
The Bottom Line
BETA THALASSEMIA MINOR: No reduction in life expectancy so long that treatments are taken appropriately.
BETA THALASSEMIA MAJOR: The average life span is 17 years, and most die by their early 30's due to cardiac disease from high iron levels.
BETA THALASSEMIA MAJOR: The average life span is 17 years, and most die by their early 30's due to cardiac disease from high iron levels.