What is your preferred first-line therapy for transfusion-dependent beta-thalassemia?

Transfusions are the backbone of therapy for these individuals. Reducing transfusion burden is advantageous to minimize iron loading, space out transfusions, and improve quality of life. At present, there are 2 agents available - luspatercept and mitapivat. The clinical trial endpoints for both are ...

Transfusion is my preferred first-line therapy (and standard of care) for beta-thalassemia major. For beta-thalassemia intermedia that has evolved into TDT, my preferred first-line treatment is mitapivat over luspatercept, particularly if the patient has extramedullary masses (that have been noted t...