The Thalassemia Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center offers expertise in the diagnosis and treatment of thalassemia. Our innovative approach to patient care reduces the risk of complications and helps patients lead healthy, active lives during and after treatment.
Located in Boston, MA, our program is one of only six centers in the United States designated for clinical excellence in thalassemia by the National Cooley’s Anemia Foundation, and one of seven centers of its kind to be sponsored (2012) by the Centers for Disease Control and Prevention for the prevention of thalassemia complications. We treat children with all forms of this condition, including thalassemia minor or trait, thalassemia intermedia, and thalassemia major (Cooley’s anemia), as well as rarer forms such as hemoglobin E beta-thalassemia and hemoglobin H alpha-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).
Dana-Farber/Boston Children's patients have access to the broadest set of blood disorder expertise and pediatric subspecialities 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; 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.