Current Environment:

The Thalassemia Program at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center treats all forms of thalassemia, including thalassemia minor or trait, thalassemia intermedia (also called non-transfusion dependent thalassemia (NTDT),  and transfusion dependent thalassemia (TDT), also known as thalassemia major or Cooley’s anemia.

We have extensive experience with all types of beta thalassemia, including hemoglobin E-beta thalassemia, and all types of alpha thalassemia, including hemoglobin H disease.

We offer a full spectrum of services for the diagnosis, treatment, and ongoing care of even the rarest and hardest-to-treat thalassemias. Because of our long-standing experience, comprehensive subspecialty services, and deep involvement with cutting-edge novel therapies, we have been designated as a center of excellence in thalassemia by the Cooley’s Anemia Foundation.

Zynteglo® gene therapy approved for transfusion-dependent beta thalassemia

In August 2022, the FDA approved betibeglogene autotemcel gene therapy (also known as beti-cel or Zynteglo®) for severe beta thalassemia requiring frequent red blood cell transfusions. We expect to be able to offer Zynteglo® in the near future. Read more.

Blending care and research

Our researchers have been at the forefront of thalassemia research for decades, seeking new methods of diagnosis and better treatments including novel medications and gene therapies. Learn about our research.

Our care team

Children seen by our Thalassemia Program have access to the broadest blood-disorder expertise and pediatric subspecialty care available. We assemble the right team of specialists to address each child’s specific needs.

Our team is led by hematologists with expertise in transfusion medicine and genetics. Because thalassemia is a complex disease, we work closely with specialists in cardiology and endocrinology, and when needed, specialists in gastroenterology, audiology, ophthalmology, nephrology, infectious diseases, and genetic counseling as needed. We also collaborate closely with our Stem Cell Transplant team to offer cellular and genetic therapies. Our thalassemia nurse practitioner works with families to coordinate their child’s care.