Thalassemia
Alternate Names : Thalassemia Major, Thalassemia Minor, Cooley's Anemia, Mediterranean Anemia
What are the treatments for the disease?
With severe thalassemia, regular blood transfusions are needed. The person will also require folic acid and other nutritional supplements.
An individual who has frequent red blood cell transfusions can develop iron
overload. This can be avoided with chelation therapy. This is a process that removes excess iron from the body. This therapy may need to be started early in childhood.
Some treatments for thalassemia are still being tested in clinical trials. These include:
changing the abnormal hemoglobin genes using medications similar to those
used in cancer chemotherapy
bone marrow transplant and infusions of stem cells, which are starter cells
specific gene therapy targeted at the abnormal chromosomes
These approaches are usually reserved for severe thalassemia.
What are the side effects of the treatments?
Frequent blood transfusions can lead to an iron overload. Excess iron can end up in various body tissues and cause skin discoloration, liver disease, and diabetes. Chelation, which involves daily injections of an iron-binding agent, can help eliminate the excess iron from the body.
There are risks with any blood transfusion. These include an allergic reaction, infection, and
sometimes hepatitis or HIV, the virus that causes AIDS.
Bone marrow transplants can cause the body to attack the new bone marrow. Or the new bone marrow may not function at all.
What happens after treatment for the disease?
A person with severe thalassemia may need frequent blood transfusions for the rest of his or her life.
How is the disease monitored?
Thalassemia is monitored by frequent blood tests. The person will need to be followed closely by the healthcare provider. Any new or worsening symptoms should be reported to the provider.
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