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Thalassemia Program | Overview

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 and thalassemia major (Cooley’s anemia), as well as rare forms of the disease such as hemoglobin E-beta thalassemia and hemoglobin H-alpha thalassemia. Because of our innovative approach, we are one of only six programs designated as centers of excellence in thalassemia by the National Cooley’s Anemia Foundation and one of only seven centers sponsored by the federal Centers for Disease Control and Prevention for the prevention of thalassemia complications.

Dana-Farber/Boston Children’s is an integrated pediatric hematology and oncology partnership between Dana-Farber Cancer Institute and Boston Children’s Hospital. We offer, in one specialized program, the full spectrum of services that are needed for diagnosis, treatment and ongoing care of even the rarest and hard-to-treat conditions.

How we diagnose and treat thalassemia

We perform a variety of blood tests to diagnose thalassemia, such as measuring the amount of hemoglobin and iron in the blood. In some cases, genetic tests can identify specific genetic defects that cause thalassemia. Special testing, including chorionic villus sampling and amniocentesis, can be done before birth to determine if a baby has thalassemia and how severe the condition is.

Depending on the type of thalassemia a child has, treatment may include medications and supplements for anemia, or blood transfusions and oral iron chelation therapy to treat iron overload from chronic infusions. We provide advanced assessment with cardiac MRI to help monitor iron overload and prevent complications, and we offer the most effective available chelation treatments, including intravenous deferoxamine (Desferal) and oral deferasirox (Exjade). For patients with hard-to-treat thalassemia, we provide access to deferiprone, an experimental iron chelator currently under study.

For certain cases, stem cell transplantation may be recommended to replace the blood-forming stem cells with the defective hemoglobin gene(s).

Our care team

Thalassemia Program patients have access to the broadest set of blood disorder expertise and pediatric subspecialties available. The breadth of our expertise allows us to assemble a team of specialists to meet the specific needs of each patient.

Our team is led by hematologists with expertise in transfusion medicine and genetics. Because thalassemia is a complex disease, our treatment team also includes specialists in cardiology and endocrinology, and when needed, specialists in gastroenterology, audiology, ophthalmology, infectious diseases, and genetic counseling may also be included. In addition, we have a thalassemia nurse practitioner who works with families to coordinate their child’s care.