Burden of Illness of Alpha- and Beta-Thalassemia: A Qualitative Study

Alpha (α)- and beta (β)-thalassemia are inherited red blood cell disorders with a wide spectrum of symptoms, functional manifestations, and disease burden. The standard of care for α- and -β-thalassemia major is regular transfusions and iron chelation therapy. However, symptoms may persist despite treatment. Patients with non-transfusion dependent (NTD) thalassemia are historically considered to have less severe disease than patients who are transfusion-dependent (TD), yet they may experience considerable disease burden negatively affecting their health-related quality of life (HRQoL). Additionally, little is known about the HRQoL of α- thalassemia patients, of whom a majority are NTD.