Transfusion-dependent beta-thalassemia is treated with frequent and lifelong blood transfusions which deliver red blood cells to your body to correct the anemia. However, they also cause excess iron to build up in the body, which can damage organs, such as the heart and liver, and cause symptoms, such as abdominal pain, weakness, fatigue and joint pain, because iron accumulation can’t be removed naturally. Patients who receive ongoing blood transfusions also must take medicines to remove the excess iron, called iron chelation therapy.
The only curative treatment option for transfusion-dependent beta-thalassemia is allogeneic hematopoietic stem cell transplant (HSCT), a procedure in which a patient receives blood stem cells from a donor. The donor’s stem cells produce a supply of new blood cells with functioning beta-globin.
The best results for allogeneic HSCT occur with blood stem cells from a donor with a matching tissue type, usually a sibling. However, less than 25 percent of patients who are candidates for transplantation have a sibling with such a match.
Additionally, results of allogeneic HSCT in adults are less than optimal, especially when a sibling matched donor is not available. Children have fewer complications compared to adults when treated with allogeneic HSCT. Additional treatment options are needed.