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In patients with transfusion-dependent β-thalassemia, luspatercept is used to enhance late stages of erythropoiesis and reduce transfusion burden. Patients with non–transfusion-dependent β-thalassemia have less-severe anemia than do those with transfusion-dependent disease, but they have limited treatment options beyond as-needed transfusion, despite compromises in morbidity, mortality, and quality of life.
Now, investigators have conducted a multicenter, randomized, placebo-controlled, phase 2 study to test the efficacy and safety of luspatercept in 145 patients with non–transfusion-dependent β-thalassemia (median age, 40 years; 57% female; median baseline hemoglobin, 8.2 g/dL). The primary endpoint was the proportion of patients with a mea…