What is thalassemia?

There are many types of thalassemia. The thalassemias are a group of inherited blood disorders in which the genes that produce hemoglobin, the protein in red blood cells that carries oxygen from the lungs to all parts of the body, are broken. As a result, the red blood cells do not contain enough hemoglobin, causing anemia that can range from mild to life threatening. Thalassemia can come in different forms depending on the genetic mutations causing it. The transfusion-dependent form, also called thalassemia major or Cooley’s Anemia, requires lifelong follow-up care and regular blood transfusions. Some other forms are more readily managed and may require little or no treatment.

How we care for thalassemia at Dana-Farber/Boston Children’s

At the Dana-Farber/Boston Children’s Thalassemia Program, our experts provide comprehensive care for children and adults with all forms of thalassemia. Treatment for thalassemia depends on the subtype of the disorder, but may include blood transfusions, monitoring, iron chelation therapy, or stem cell transplantation.

Learn more

Find more in-depth information on thalassemia on the Dana-Farber/Boston Children's website, including answers to:

  • What are the different types of thalassemia?
  • What are the symptoms of thalassemia?
  • How is thalassemia in children diagnosed?
  • How is thalassemia in children treated?
  • What is the latest research on thalassemia?
  • What is the long-term outlook for children with thalassemia?

Gene therapy for beta-thalassemia

The gene therapy clinical trial for beta thalassemia consists of a one-time infusion of ST-400 — manufactured from a individual's own blood stem cells, which are removed and then genetically edited in a lab to produce blood with a higher oxygen-carrying capacity. Learn more about this clinical trial and the Gene Therapy Program.