Understand the void filled by gene therapy in the treatment paradigm for transfusion-dependent β-thalassemia
Evaluate the appropriateness of gene therapy for individual patients with transfusion-dependent β-thalassemia
Case: The parent of a 12-year-old boy of Greek ancestry who has transfusion-dependent β-thalassemia are asking about curative options. He does not have any matched related donors, and a search through the international bone marrow donor registry has not identified a match either. He is transfused every 3 weeks to maintain a pretransfusion hemoglobin level of 9.5 to 10.5 g/dL and is well chelated, with a most recent liver magnetic resonance imaging showing a liver iron concentration of 3.4 mg/g dry weight and a cardiac T2* of 42 ms. The parents are concerned about school absences and their son's desire to participate in competitive sports as he gets older.
Transfusion-dependent β-thalassemia is...
