Treatment Overview

Treatment for thalassemia depends on the severity of the condition. Thalassemia may be mild, moderate, or severe.

Mild thalassemia is the most common form and does not require treatment.

Moderate thalassemia (thalassemia intermedia and more severe hemoglobin H disease) may be treated with:

• Folic acid (a vitamin that your body needs to produce new red blood cells). People with thalassemia often lack enough folic acid in their diets to keep a good supply of red blood cells.
• Blood transfusions. You may need a blood transfusion when your body is under stress, such as during an infection.

If you have moderate thalassemia, do not take medicines that increase the amount of iron in the body, which can damage organs. These medicines include:

• Iron supplements or multivitamins that contain iron.
• Vitamin C, which can increase the amount of iron that your body absorbs from food.

Severe thalassemia is often treated with:

• Regular blood transfusions. A child with severe thalassemia typically starts to have symptoms when he or she is 6 months old and will need blood transfusions every 4 to 6 weeks. The transfusions often lead to increased iron levels, which also may require treatment.
• Folic acid, which your body needs to produce red blood cells. A person with thalassemia needs folic acid supplements because red blood cells are destroyed faster than normal. As a result, the body often uses up its stores of this vitamin.

Other treatments that doctors consider for severe cases of thalassemia include:

• A splenectomy, which is surgical removal of the spleen.
• In rare cases, a bone marrow transplant. This treatment can cure severe thalassemia, but it must be done before problems related to excess iron stored in the body develop. Phlebotomy, removing blood from the body, is sometimes needed after a successful bone marrow transplant to reduce high iron levels.
• In rare cases, a stem cell transplant.² This procedure involves taking stem cells from the umbilical cord of a newborn. These stem cells are then injected into a child who has severe thalassemia. The donor is most often a brother or sister of the child.

What to Think About

Blood transfusion therapy often creates an excess of iron, which may damage organs if untreated. You may need to use medicines called chelating agents that bind to and remove excess iron from the blood. The chelating agent deferasirox (Exjade) is taken as a pill once a day. The chelating agent deferoxamine mesylate (Desferal) is injected through a tube placed under the skin. A portable pump delivers the medicine in what is usually an all-night procedure. This treatment works best if it is done 5 or 6 nights a week. Both deferoxamine and deferasirox have potentially serious side effects, including hearing loss and kidney, liver, and eye problems.

Researchers are studying how well medicines (hydroxyurea and butyrate) work to get the body to produce fetal hemoglobin in children and adults with thalassemia. Fetal hemoglobin is different than adult hemoglobin. The body does not normally produce it after birth. Fetal hemoglobin may help adult hemoglobin work better in people with thalassemia.⁴