How is thalassemia treated?
Treatments for thalassemia depend on the type and how serious it is. If you are a carrier or have alpha or beta thalassemia trait, you likely have mild or no symptoms and may not need treatment.
If you have a more serious thalassemia type like hemoglobin H disease, beta thalassemia intermedia, or beta thalassemia major you may experience moderate to serious anemia symptoms. You may need treatments such as blood transfusions, medicine, a splenectomy, or a blood and bone marrow transplant.
Blood transfusions are the main way to treat moderate or severe thalassemia. This treatment gives you red blood cells with healthy hemoglobin.
During a blood transfusion, a needle is used to insert an intravenous (IV) line into one of your blood vessels. You receive healthy blood through this line. The procedure usually takes 1 to 4 hours. How often blood transfusions are needed depends on how serious your condition and symptoms are.
- Occasional blood transfusions may be needed for people who have hemoglobin H disease or beta thalassemia intermedia. Specifically, a transfusion may be needed when your body is under stress, such as during an infection, pregnancy, or surgery.
- Regular blood transfusions (every 3 to 4 weeks) may be needed for people who have beta thalassemia major. These transfusions help maintain healthy hemoglobin and red blood cell levels.
Iron chelation therapy
The hemoglobin in red blood cells is an iron-rich . Regular blood transfusions can cause iron buildup, or iron overload, which can lead to potentially life-threatening complications.
To prevent this, doctors use iron chelation therapy in people who receive regular blood transfusions to remove excess iron from the body. Three medicines are used for iron chelation therapy:
- Deferasirox is a pill taken once daily. Side effects can include skin rash, nausea, and diarrhea.
- Deferiprone is a pill that may be used if other treatments do not work. It can lower your white blood cell numbers, which can put you at risk for infections.
- Deferoxamine is a liquid medicine that is given slowly under the skin, usually with a small portable pump used overnight. This therapy takes time and can be mildly painful. Side effects can include problems with vision and hearing.
Talk to your doctor if you’re pregnant or thinking about becoming pregnant. You may need to switch to a different iron chelation therapy medicine.
Learn more about thalassemia and pregnancy.
Blood and bone marrow transplant
A blood or bone marrow transplant, also called a hematopoietic transplant, replaces blood-forming stem cells that aren’t working properly with healthy donor cells. A stem cell transplant is the only treatment that can cure thalassemia. However, only a small number of people who have severe thalassemia are able to find a good donor match and are a good fit for the procedure.
Learn more about the blood and bone marrow transplant procedure.
Even though blood transfusions are the typical treatment, other treatments may be used.
- Medicines called luspatercept (Reblozyl) and hydroxyurea may be prescribed by a healthcare provider to treat thalassemia. Luspatercept can lessen the number of blood transfusions needed for people with moderate to severe anemia as a result of thalassemia. Hydroxyurea is usually used to treat sickle cell disease and can help lower the risk of health problems from thalassemia.
- Splenectomy is surgery to remove the spleen. Your provider may recommend splenectomy to improve your symptoms if you have mild to moderate thalassemia. However, removing the spleen lowers the body’s ability to fight infections.