2021 update on clinical trials in β-thalassemia

The treatment landscape for patients with beta-thalassemia is witnessing a swift evolution, yet several unmet needs continue to persist. Patients with transfusion-dependent beta-thalassemia (TDT) primarily rely on regular transfusion and iron chelation therapy, which can be associated with considerable treatment burden and cost. Patients with non-transfusion-dependent beta-thalassemia (NTDT) are also at risk of significant morbidity due to the underlying anemia and iron overload, but treatment options in this patient subgroup are limited. In this review, we provide updates on clinical trials of novel therapies targeting the underlying pathology in beta-thalassemia, including the alpha/non-alpha-globin chain imbalance, ineffective erythropoiesis, and iron dysregulation.

Automated summary

The treatment landscape for beta-thalassemia patients is rapidly evolving, with unmet needs still existing for both transfusion-dependent and non-transfusion-dependent patients. Novel therapies targeting the underlying pathology in beta-thalassemia are currently in clinical trials, focusing on alpha/non-alpha-globin chain imbalance, ineffective erythropoiesis, and iron dysregulation.