Thalassemia Management

Jagdish Kathwate
MD Pediatrics. Assistant Professor, Government Medical College, Aurangabad, India.
First Created: 12/27/2013  Last Updated: 08/01/2015

Patient Education

How Are Thalassemias Treated?

Treatments for thalassemias depend on the type and severity of the disorder. People who are carriers or who have an alpha or beta thalassemia trait have mild or no symptoms. They’ll likely need little or no treatment.

Standard Treatments

Blood Transfusions

Transfusions of red blood cells are the main treatment for people who have moderate or severe thalassemias. This treatment gives healthy red blood cells with normal hemoglobin. During a blood transfusion, a needle is used to insert an intravenous (IV) line into one of your blood vessels. Through this line, patients will receive healthy blood. The procedure usually takes 1 to 4 hours.

Red blood cells live only for about 120 days. So, patients may need repeated transfusions to maintain a healthy supply of red blood cells.

If patients have hemoglobin H disease or beta-thalassemia intermedia, he may need blood transfusions on occasion. For example, he may have transfusions when he has an infection or other illness, or when his anemia is severe enough to cause tiredness.

If the patient has beta-thalassemia major (Cooley's anemia), he will likely need regular blood transfusions (often every 2 to 4 weeks). These transfusions will help you maintain normal hemoglobin and red blood cell levels.

Blood transfusions allow you to feel better, enjoy normal activities, and live into adulthood. This treatment is lifesaving, but it's expensive and carries a risk of transmitting infections and viruses (for example, hepatitis). However, the risk is very low because of careful blood screening.

Iron Chelation Therapy

The hemoglobin in red blood cells is an iron-rich protein. Thus, regular blood transfusions can lead to a buildup of iron in the blood. This condition is called iron overload. It damages the liver, heart, and other parts of the body.

To prevent this damage, doctors use iron chelation therapy to remove excess iron from the body. Two medicines are used for iron chelation therapy.

  • Deferoxamine is a liquid medicine that's given slowly under the skin, usually with a small portable pump used overnight. This therapy takes time and can be mildly painful. Side effects include problems with vision and hearing.
  • Deferasirox is a pill taken once daily. Side effects include headache, nausea (feeling sick to the stomach), vomiting, diarrhea, joint pain, and tiredness.

Folic Acid Supplements

Folic acid is a B vitamin that helps build healthy red blood cells. Your doctor may recommend folic acid supplements in addition to treatment with blood transfusions and/or iron chelation therapy.

Other Treatments

Other treatments for thalassemias have been developed or are being tested, but they're used much less often.

Blood and Marrow Stem Cell Transplant

A blood and marrow stem cell transplant replaces faulty stem cells with healthy ones from another person (a donor). Stem cells are the cells inside bone marrow that make red blood cells and other types of blood cells. A stem cell transplant is the only treatment that can cure thalassemia. But only a small number of people who have severe thalassemias are able to find a good donor match and have a risky procedure.

Possible Future Treatments

Researchers are working to find new treatments for thalassemias. For example, it might be possible someday to insert a normal hemoglobin gene into stem cells in the bone marrow. This will allow people who have thalassemias to make their own healthy red blood cells and hemoglobin. Researchers also are studying ways to trigger a person's ability to make fetal hemoglobin after birth. This type of hemoglobin is found in fetuses and newborns. After birth, the body switches to making adult hemoglobin. Making more fetal hemoglobin might make up for the lack of healthy adult hemoglobin.

Treating Complications

Better treatments now allow people who have moderate and severe thalassemias to live longer. As a result, these people must cope with complications that occur over time. An important part of managing thalassemias is treating complications. Treatment might be needed for heart or liver diseases, infections, osteoporosis, and other health problems.

1. Piomelli S, Loew T. Management of thalassemia major (Cooley's anemia) Hematol Oncol Clin North Am 1991, 5: 557-69.
2. Piomelli S. Cooley's anemia management : 25 years of progress. In :Bucker CD, Gale RP, Lucarelli G. Advances and controversies in thalassemia therapy - bone marrow transplantation and other approaches. NY Alan R Liss 1989, 309: 23-26.
3. Lokeshwar MR, Mangalani M, Rao S, et al. Current trends in thalassemia therapy. Proceedings of international symposium cum workshop on 'anemia in children', 1991, p.21.14
4. Cohen A. Current status of iron chelation therapy with desferrioxamine. Semin Hematol 1990, 27: 86-90.
5. Porter JB, Hyder RC, Huehns ER. update on hydroxy pyridone oral chelating agents. Semin Hematol 1990, 27: 95-100.
6. Olivieri NF, Coren G, Louis PS et al. Studies of oral chelator 1,2 dimethyl-3-hydroxy-pyrid-4-one in thalassemia patient. Semin Hematol 1990, 27: 101-4.
7. Agarwal MB, Vishwanathan C, Ramanathan J. Oral iron chelation with L1. Lancet 1990, i: 601.
8. Lucarelli G, Galimberti M, Polchi P et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990, 322: 417-21.
9. Friedman T. Progress towards human gene therapy. Science 1989, 244: 1275-80.
10. Mehta BC. Thalassemia management symposium - thalassemias. Ind J Blood Transf 1992, 10: 43-62.

Thalassemia Management Thalassemia Management 2015-08-01
Disclaimer: The information given by is provided by medical and paramedical & Health providers voluntarily for display & is meant only for informational purpose. The site does not guarantee the accuracy or authenticity of the information. Use of any information is solely at the user's own risk. The appearance of advertisement or product information in the various section in the website does not constitute an endorsement or approval by Pediatric Oncall of the quality or value of the said product or of claims made by its manufacturer.
0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0