Thalassemia

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Treatments and drugs

By Mayo Clinic staff

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.

Treatment 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 used to treat severe thalassemia in select cases. Prior to a stem cell transplant, you receive very high doses of drugs or radiation to destroy your diseased bone marrow. Then you receive infusions of stem cells from a compatible donor. However, because these procedures have serious risks, including death, they're generally reserved for people with the most severe disease who have a well-matched donor available — usually a sibling.
References
  1. What are thalassemias? National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/health/dci/Diseases/Thalassemia/Thalassemia_All.html. Accessed Dec. 30, 2010.
  2. Giardina PJ, et al. Thalessemia syndromes. In: Hoffman R, et al. Hematology: Basic Principles and Practice. 5th ed. Philadelphia, Pa.: Churchill Livingstone Elsevier; 2009. http://www.mdconsult.com/books/about.do?about=true&eid=4-u1.0-B978-0-443-06715-0..X5001-8--TOP&isbn=978-0-443-06715-0&uniqId=230100505-56. Accessed Dec. 30, 2010.
  3. Mueller BU. Prenatal testing for the hemoglobinopathies and thalassemias. http://www.uptodate.com/home/index.html. Accessed Jan. 3, 2011.
DS00905 Feb. 4, 2011

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