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Anemia in patients with beta-thalassemia is due to premature death of erythroblasts, resulting in expansion of the bone marrow, accumulation of iron, and eventual organ failure. Luspatercept promotes erythroid maturation by antagonizing inhibitors of late-stage erythropoiesis.
To examine the safety and effectiveness of luspatercept in adults with beta-thalassemia, investigators conducted an industry-funded, open-label, phase II study in 64 patients; 31 of whom were transfusion-dependent. Luspatercept was given subcutaneously every 21 days in doses ranging from 0.6 to 1.25 mg/kg.
The following were reported:
Hemoglobin increased by ≥1.5 g/dL for ≥2 weeks in 58% of non–transfusion-dependent patients; those with versus without prior splenectomy w…