People who have thalassemia trait generally do not require treatment. Those with mild thalassemia also generally do not require treatment.

Patients with moderate to severe thalassemia may require ongoing therapy. Blood transfusions are the most common form of treatment for severe cases of thalassemia. These transfusions provide the body with healthy red blood cells that carry adequate levels of oxygen. However, blood transfusions provide only temporary therapy and must be repeated for long-term treatment.

Patients with moderate thalassemia may require only occasional transfusions, whereas patients with more severe forms of thalassemia may require regular transfusions. Most patients with significant thalassemia need transfusions that provide an average of 52 pints of blood a year. Transfusions typically are scheduled for every two to three weeks. Patients are also often directed to take folic acid supplements.

Repeated transfusions can cause excessive levels of iron to build up in the body, a potentially fatal condition known as iron overload. Excess iron must be removed to prevent toxic damage to the liver and heart, and abnormal growth rates and delayed sexual development in children.

A treatment known as chelation therapy removes excess iron deposits from the body. It involves injections from a battery-powered infusion pump of an iron-binding drug called desferrioxamine under the skin of the stomach or legs for up to 12 hours a day for five to seven days each week. This drug has been shown to promote normal growth and sexual development and to increase a patient’s lifespan. However, it also can have side effects such as vision damage and hearing loss, and the therapies are often considered inconvenient and painful. Because of this discomfort associated with chelation therapy, many patients discontinue or avoid it. However, this is associated with worsening health problems and early death. Patients are strongly encouraged to continue with their chelation therapy, and physicians are looking for less stressful chelation agents.

Patients who undergo regular transfusions should avoid vitamins or supplements that contain iron because of the dangers of excessive iron levels.

Bone marrow transplants recently have been used to treat especially severe cases of beta thalassemia major. Results have been encouraging, and many patients (typically age 16 or younger) have become disease free. However, bone marrow transplants involve considerable risk, because all the patient’s blood-forming cells must be destroyed and replaced with cells from a donor that perfectly match the patient’s own cells. Typically, these donors are siblings.

Organ damage associated with severe thalassemia may necessitate surgery to remove the affected organ. For example, enlarged spleens may need to be removed, or gallbladders may be removed in patients who have repeated attacks of gallstones.

The blood transfusions associated with thalassemia theoretically put the patient at risk for contracting viruses such as hepatitis or AIDS from contaminated donated blood. However, improved donor screening and testing of blood products have dramatically reduced the risk of such viruses appearing in the blood supply.