If you or your child has a blood disorder called beta thalassemia, the goal of treatment is to boost the number of healthy red blood cells in the body. Your doctor will figure out the right plan based on the type of disease you have and how severe the symptoms are.
If the condition is mild, you may not need to do anything. For severe beta thalassemia, you or your child may need to get blood transfusions, a treatment called chelation therapy that removes excess iron from your body, or surgery.
Treatment by Type
Beta thalassemia affects your body's ability to make hemoglobin, a protein that helps your red blood cells carry oxygen to all your organs. Too few red blood cells is called anemia. Without enough oxygen, you'll feel tired and weak.
There are three types of beta thalassemia, and each has a different treatment.
Beta thalassemia minor (beta thalassemia trait) is the mildest form. You or your child may not need treatment. If you have mild anemia, folic acid supplements can help. Folic acid is a B vitamin that raises the number of red blood cells that your body makes. In certain situations, adults may need a blood transfusion, like after they have surgery or give birth.
Beta thalassemia intermedia causes more serious anemia. Some people will need blood transfusions to boost their red blood cell count.
Beta thalassemia major is the most severe type. It often starts by age 2 and causes severe anemia and other serious symptoms. Treatment involves regular blood transfusions.
If your child has beta thalassemia major or intermedia, make sure to reach out to family and friends for the emotional backing you need. Social workers and mental health professionals can help if you find yourself stressed or anxious.
Doctors who specialize in blood diseases, called hematologists, treat beta thalassemia. These doctors work at hospitals and beta thalassemia treatment centers. A team of doctors, nurses, physical therapists, and other health care professionals will tailor treatment to you or your child.
If you have beta thalassemia major, you'll need blood transfusions every 2 to 4 weeks to raise the number of your red blood cells. Some people with this type of beta thalassemia need transfusions only at certain times, such as when they have an illness or infection.
During a transfusion, a nurse gives you blood from a donor by placing a small plastic tube into one of your veins. The process takes 1 to 4 hours at your doctor's office or a clinic.
Technicians carefully test the blood used in transfusions to prevent you from getting hepatitis or other diseases. For extra protection, your child will need to get the full three doses of hepatitis B vaccines before they can have a blood transfusion.
The blood cells you get during a transfusion have a lot of iron. After you have many transfusions, iron can build up in your body. Beta thalassemia also makes your body absorb extra iron from food. All that extra iron can damage organs like your heart and liver.
Chelation therapy is a treatment that removes the additional iron from your body. You'll get a pill or shot of a medicine like deferasirox (Exjade, Jadenu), deferiprone (Ferriprox), or deferoxamine (Desferal). These drugs bind to iron in your body, which then leaves your body when you pee or have a bowel movement.
Spleen Removal Surgery
Your spleen filters blood cells. In beta thalassemia, too much iron and too many abnormal red blood cells can collect in that organ and make it swell. An enlarged spleen can cause your hemoglobin levels to drop even lower, plus lower the number of blood cells called platelets that help your blood clot.
The main treatment for an enlarged spleen is surgery to remove the spleen, called a splenectomy. It can improve some symptoms of beta thalassemia, but it has risks.
Your doctor will weigh the decision to remove your spleen carefully. Doctors avoid this surgery in children under age 5 because kids are at higher risk for a very serious blood infection called sepsis.
Your spleen helps your immune system get rid of bacteria and other germs that could make you sick. After surgery, you'll be more likely to catch infections. To lower your chances of getting an illness, you'll get vaccinated before the operation and take antibiotics for at least 2 years afterward.
Stem Cell Transplant
If you or your child doesn't get better with blood transfusions and other treatments, your doctor may suggest a stem cell transplant. "Hematopoietic" stem cells are young cells in your bone marrow -- the spongy center of your bones -- that grow into new blood cells. During a stem cell transplant, you get healthy blood-forming cells from a donor to replace your own damaged cells.
A stem cell transplant can cure beta thalassemia. But because it has risks like infection or rejection of the new stem cells, it's not right for everyone.
The odds of success are higher if the donor's cells closely match yours or your child's cells. A close relative like a brother or sister makes the best match. Your stem cell transplant will also work better if you get it before age 16, or before the liver gets damaged.
Treatments for Complications
Beta thalassemia can cause problems like liver damage, weak bones, and infections. Your doctor will give you medicines and other treatments for these problems if they happen.