The New England journal of medicine
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Letter Randomized Controlled Trial
Telacebec (Q203), a New Antituberculosis Agent.
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Randomized Controlled Trial Multicenter Study
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.
Patients with transfusion-dependent β-thalassemia need regular red-cell transfusions. Luspatercept, a recombinant fusion protein that binds to select transforming growth factor β superfamily ligands, may enhance erythroid maturation and reduce the transfusion burden (the total number of red-cell units transfused) in such patients. ⋯ The percentage of patients with transfusion-dependent β-thalassemia who had a reduction in transfusion burden was significantly greater in the luspatercept group than in the placebo group, and few adverse events led to the discontinuation of treatment. (Funded by Celgene and Acceleron Pharma; BELIEVE ClinicalTrials.gov number, NCT02604433; EudraCT number, 2015-003224-31.).