Emerging Therapies in b-Thalassemia

A new era of novel therapies is emerging with the aim of advancing care and improving outcomes for b-thalassemia patients. For some of these agents, the efficacy and safety has been established, whereas for others more data are awaited. More impor-tantly, reports on the use of these novel therapies in the real world outside clinical trials and subsequent outcomes are awaited. This ultimate test will determine whether these agents can be integrated into the standard care of b-thalassemia and used as monotherapy or in combination with other established/conventional therapeutic approaches.