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Beta Thalassemia Treatments

Beta thalassemia is an inherited blood disorder that affects the production of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. Beta thalassemia is classified as either minor or major. Beta thalassemia major is also known as Cooley's anemia. According to the Mayo Clinic, beta thalassemia minor normally does not require treatment, except for possibly an occasional blood transfusion. Beta thalassemia major is treated in a variety of ways, depending on the patient's specific situation.

  1. Blood Transfusions

    • Blood transfusions are the most common treatment method for beta thalassemia major. Blood transfusions ensure that beta thalassemia patients have enough hemoglobin in their bloodstreams to carry needed oxygen to the tissues and muscles. Normal red blood cells only live for about 120 days, so regular transfusions are necessary for most patients. According to the National Heart Lung and Blood Institute (NHLBI), patients with beta thalassemia major need regular blood transfusions as often as every 2 to 4 weeks. Blood transfusions are expensive, but there are relatively few risks as blood is heavily screened for infections in the United States.

    Medication

    • Since most beta thalassemia patients require frequent blood transfusions, excessive amounts of iron can build up in the bloodstream. Too much iron can cause severe damage to the body's internal organs, including the heart and liver. Medications called iron chelators help regulate iron levels in the blood and are a common treatment method for patients who receive blood transfusions. Iron chelators are available in pill form or as a liquid medication pumped under the skin. The liquid iron chelators can be somewhat painful, and side effects include loss of vision and hearing. Iron chelators in pill form may cause nausea, vomiting, diarrhea, headache, fatigue and joint pain.

    Supplements

    • It is important for beta thalassemia patients, especially those receiving regular blood transfusions, to avoid iron supplements. Many multi-vitamins contain iron, so patients should carefully check the labels of any supplements or vitamins and talk to their doctors before adding any nutritional supplements. Folic acid helps build healthy red blood cells. Since many people do not get enough folic acid in their diets, beta thalassemia major patients may need a daily supplement to provide an adequate dose of folic acid.

    Blood and Marrow Stem Cell Transplant

    • Stem cells in bone marrow help make blood cells, including red blood cells. A stem cell transplant is the only way to completely cure beta thalassemia. Other treatment methods focus on managing the symptoms only. Bone marrow and stem cell transplants are very risky. If the patient's body rejects the transplant, serious illness or death can occur. Finding a suitable match is vital to minimizing the risk of rejection. Due to the difficulty of finding a match and the serious risks involved, blood and marrow stem cell transplants are usually reserved for patients with severe cases of beta thalassemia who are not responding well to other treatment options.

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