How is thalassemia treated?

Standard treatments for patients with thalassemia major are blood transfusions and iron chelation.

  • Blood transfusion involves injection of red blood cells through a vein to restore normal levels of healthy red blood cells and hemoglobin. Transfusions are repeated every 4 months in patients with moderate or severe thalassemias, and every 2 to 4 weeks in patients with beta thalassemia major. Occasional transfusions may be needed (for instance, during times of infection) for hemoglobin H disease or beta thalassemia intermedia.
  • Iron chelation is removal of excess iron from the body. A danger with blood transfusions is that they can cause iron overload, which may in turn causedamage to other organs. Because of this, patients who receive frequent transfusions also require iron chelation therapy, which can be given in pill form.
  • Nutritional supplements, in the form of folic acid supplements, and monitoring of B12 levels are important, as these nutrients are key components to making healthy blood cells.
  • Bone marrow and stem cell transplant from a compatible related donor is the only treatment that can cure thalassemia. It is the most effective treatment. Compatible means that the donor has the same types of proteins, called human leukocyte antigens (HLA), on the surface of their cells as the person who will be receiving the transplant. Bone marrow transplant from a compatible brother or sister offers the best chance at a cure. Most patients with thalassemia, however, lack a suitable sibling donor. A bone marrow transplant is done in the hospital. Within 1 month, the transplanted bone marrow stem cells will start to make new, healthy blood cells. Given the high risks of a bone marrow transplant, it is not routinely recommended for those with mil or moderate thalassemia.

Last reviewed by a Cleveland Clinic medical professional on 07/14/2018.

References

  • National Heart, Lung, and Blood Institute. What Are Thalassemias? Accessed 3/5/2020.
  • Centers for Disease Control and Prevention. Thalassemia Accessed 3/5/2020.
  • American Academy of Family Physicians/FamilyDoctor.org. Thalassemia Accessed 3/5/2020.

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