Ali T. TaherRayan Bou-FakhredinAntonis KattamisVip ViprakasitMaria Domenica CappelliniSiriraj HospitalAmerican University of Beirut Medical CenterUniversità degli Studi di MilanoEthnikó ke Kapodistriakó Panepistímio Athinón2022-08-042022-08-042021-01-01Expert Review of Hematology. Vol.14, No.10 (2021), 897-90917474094174740862-s2.0-85115049539https://repository.li.mahidol.ac.th/handle/20.500.14594/78613Introduction: β-thalassemia is one of the most common inherited monogenic diseases. Many patients are dependent on a lifetime of red blood cell (RBC) transfusions and iron chelation therapy. Although treatments have a significant impact on quality of life (QoL), life expectancy, and long-term health outcomes have improved in recent decades through safer RBC transfusion practices and better iron chelation strategies. Advances in the understanding of the pathology of β-thalassemia have led to the development of new treatment options that have the potential to reduce the RBC transfusion burden in patients with transfusion-dependent (TD) β-thalassemia and improve QoL. Areas covered: This review provides an overview of currently available treatments for patients with TD β-thalassemia, highlighting QoL issues, and providing an update on current clinical experience plus important practical points for two new treatments available for TD β-thalassemia: betibeglogene autotemcel (beti-cel) gene therapy and the erythroid maturation agent luspatercept, an activin ligand trap. Expert opinion: Approved therapies, including curative gene therapies and supportive treatments such as luspatercept, have the potential to reduce RBC transfusion burden, and improve clinical outcomes and QoL in patients with TD β-thalassemia. Cost of treatment is, however, likely to be a significant barrier for payors and patients.Mahidol UniversityMedicineArticleSCOPUS10.1080/17474086.2021.1977116