A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia

In adults with transfusion-dependent β-thalassemia (n=336), a larger proportion of patients who received luspatercept plus best supportive care had a reduction in their transfusion burden (of ≥33% and by ≥2 units at weeks 13-24 vs baseline; 21.4% vs. 4.5% with placebo, P<0.001).


New England Journal of Medicine