How Is Beta Thalassemia Treated?

The goals of treating beta thalassemia include reducing symptoms of anemia and improving quality of life, according to Jonathan Feld, MD, Hematology Specialist at The Mount Sinai Hospital in New York City.

Your exact treatment plan may vary depending on:

• How severe your anemia is 

• How aggressive your beta thalassemia is

• Your age

• Your overall health

• What other conditions you have

• Your preferences

What are the treatment options for beta thalassemia?

Blood transfusions

Blood transfusions help provide more red blood cells to the body. Some people only need blood transfusions occasionally, while others may need frequent blood transfusions.

“Blood transfusions might help shortness of breath and improve energy and fatigue,” says Dr. Feld. “Blood transfusions can help prevent complications related to anemia and beta thalassemia.”

Iron chelation therapy

Getting frequent blood transfusions may sometimes cause you to have excess iron in your body. Iron overload can be harmful to some organs, such as your liver. 

Iron chelation therapy helps lower the amount of iron in the blood and the organs.

Erythroid maturation agents (EMAs)

EMAs are a newer treatment option for beta thalassemia. It helps the stem cells mature correctly into normal red blood cells. This helps reduce anemia and risk of complications. EMAs for beta thalassemia may help lower the number of blood transfusions you need.

Stem cell transplant

A stem cell transplant may be very helpful for treating beta thalassemia, but it’s not an option for everyone. Generally, a transplant may be an option for people who are younger, relatively healthy, and have a donor match available. Stem cell transplants are typically only used for more severe cases of beta thalassemia and they may come with a greater likelihood of complications

There are generally treatment options available that may help beta thalassemia — and more options may be on the way. Dr. Feld mentions gene therapy and stem cell therapy as options that may become more common for treating beta thalassemia in the future.