How Do Erythroid Maturation Agents Treat Beta Thalassemia?

Erythroid maturation agents, or EMAs, are a newer treatment option for beta thalassemia. The first EMA for beta thalassemia received FDA approval in November 2019.

Historically, one primary treatment for beta thalassemia has been administering blood transfusions. Transfusions typically help increase the red blood cell count, which may then improve symptoms of anemia and quality of life.

While blood transfusions can be helpful, some people need them frequently. This can be time consuming, and it may also come with risks. Frequent blood transfusions increase the risk of iron overload, which is when the red blood cells have excess iron, among other issues. Iron overload can damage the organs, including the liver.

How do erythroid maturation agents help treat beta thalassemia?

EMAs work by helping abnormal stem cells mature into healthy red blood cells. This helps improve your blood cell counts and can reduce symptoms of anemia.

This can help reduce the number of blood transfusions someone may need, which may also lessen the risk of transfusion complications.

“EMAs are administered as an injection under the skin that can be given in the [abdomen], the arm, or the leg,” says Jonathan Feld, MD, Hematology Specialist at The Mount Sinai Hospital in New York City.

Not everyone with beta thalassemia is a candidate for EMAs. “Talk to your hematologist to understand if EMAs are right for you,” says Dr. Feld.