Beta thalassemia is an inherited blood disorder. This means it is passed down through the parent’s genes. It is a form of anemia. Anemia is a low red blood cell or low hemoglobin level. Hemoglobin is part of red blood cells. It carries oxygen to organs, tissues and cells. Beta thalassemia affects the production of hemoglobin.
There are different types of thalassemia. The severity of anemia depends on the type of your child has.
Beta thalassemia is caused by a defect of the gene that controls hemoglobin production. There are three types:
Beta thalassemia is an inherited problem. It is passed from parents to children. Depending on the specific type of thalassemia a parent has, that person's children have different chances that they will also have the disorder or be a carrier for it. It's important for carriers of the thalassemia gene to know how their children may be affected. They should talk with a healthcare provider.
The gene defect that causes beta thalassemia is more common in the following people:
The symptoms of beta thalassemia major occur when an infant is between six and 24 months. They include:
The symptoms of beta thalassemia intermedia happen at a later age and include:
Children with beta thalassemia minor have anemia, but often do not have symptoms.
The healthcare provider will ask about your child’s symptoms and health history. He or she will give your child a physical exam. Your child may also have tests, such as:
Treatment will depend on your child’s symptoms, age and general health. It will also depend on how severe the condition is.
Your child's healthcare provider will likely send you to a hematologist. This is an expert in blood disorders. Treatment for beta thalassemia depends on the type. Your child may have to take daily doses of folic acid, a vitamin needed to make hemoglobin. Treatment may also include:
Complications from frequent transfusions may happen when a child is about 10 or 11 years old. They are from having too much iron. The complications include:
Children with beta thalassemia intermedia may also have complications from iron overload.
The ongoing care of your child should include:
Talk with your provider about genetic counseling.
Each child's symptoms and treatment is different. Make sure you understand your child's symptoms and treatment, including possible side effects. Call your child's provider if you notice any changes or have questions. For example, call the provider if your child has any of the following:
Most patients with beta thalassemia who have received a one-time ZYNTEGLO® treatment have been able to produce sufficient hemoglobin because of the treatment, freeing them from regular blood transfusions.
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