Treatment

Treatment for thalassemia depends on which type you have and how severe it is.

Treatments for mild thalassemia

Signs and symptoms are usually mild with thalassemia minor and little, if any, treatment is needed. Occasionally, you may need a blood transfusion, particularly after surgery, after having a baby or to help manage thalassemia complications.

People with severe beta-thalassemia will need blood transfusions. And because this treatment can cause iron overload, they will also need treatment to remove excess iron. An oral medication called deferasirox (Exjade, Jadenu) can help remove the excess iron.

Treatments for moderate to severe thalassemia

Treatments for moderate to severe thalassemia may include:

  • Frequent blood transfusions. More-severe forms of thalassemia often require frequent blood transfusions, possibly every few weeks. Over time, blood transfusions cause a buildup of iron in your blood, which can damage your heart, liver and other organs. To help your body get rid of the extra iron, you may need to take medications that rid your body of extra iron.
  • Stem cell transplant. Also called a bone marrow transplant, a stem cell transplant may be an option in select cases, including children born with severe thalassemia. It can eliminate the need for lifelong blood transfusions and drugs to control iron overload.

    During this procedure, you receive infusions of stem cells from a compatible donor, usually a sibling.

Nov. 02, 2016
References
  1. Longo DL, et al., eds. Disorders of hemoglobin. In: Harrison's Principles of Internal Medicine. 19th ed. New York, N.Y.: McGraw-Hill Education; 2015. http://accessmedicine.com. Accessed Sept. 15, 2016.
  2. What are thalassemias? National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/health/health-topics/topics/thalassemia/. Accessed Sept. 15, 2016.
  3. Kelly N. Thalassemia. Pediatrics in Review. 2012;33:434.
  4. Thalassemias. Merck Manual Professional Version. http://www.merckmanuals.com/professional/hematology_and_oncology/anemias_caused_by_hemolysis/thalassemias.html. Accessed Sept. 15, 2016.
  5. Yates AM. Prenatal screening and testing for the hemoglobinopathy. http://www.uptodate.com/home. Accessed Sept. 15, 2016.
  6. Benz EJ. Treatment of beta thalassemia. http://www.uptodate.com/home. Accessed Sept. 20, 2016.
  7. Musallam KM, et al. Iron overload in b-thalassemia intermedia: An emerging concern. Current Opinion in Hematology. 2013;20:187.
  8. DeLoughery TG. Microcytic anemia. New England Journal of Medicine. 2014;371:1324.
  9. AskMayoExpert. Thalassemia syndrome. Rochester, Minn.: Mayo Foundation for Medical Education and Research; 2016.
  10. Benz EJ. Clinical manifestations and diagnosis of the thalassemia. http://www.uptodate.com/home. Accessed Sept. 15, 2016.
  11. Schrier SL, et al. Chelation therapy for thalassemia and other iron overload states. http://www.uptodate.com/home. Accessed Sept. 20, 2016.
  12. What is a blood and marrow stem cell transplant? National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/health/health-topics/topics/bmsct/. Accessed Sept. 20, 2016.
  13. Adams RH (expert opinion). Mayo Clinic, Scottsdale/Phoenix, Ariz. Sept. 26, 2016.