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

Date
2020
Author
Shetty, J. K.
Kattamis, A.
Vlachaki, E.
Origa, R.
Aydinok, Y.
Bejaoui, M.
Ho, P. J.
Chew, L. -P.
Bee, P. -C.
Lim, S. -M.
Lu, M. -Y.
Tantiworawit, A.
Ganeva, P.
Gercheva, L.
Shah, F.
Neufeld, E. J.
Thompson, A.
Laadem, A.
Lal, A.
Zou, J.
Zhang, J.
Miteva, D.
Zinger, T.
Linde, P. G.
Sherman, M. L.
Hermine, O.
Porter, J.
Piga, A.
Cappellini, M. D.
Viprakasit, V.
Taher, A. T.
Georgiev, P.
Kuo, K. H. M.
Coates, T.
Voskaridou, E.
Liew, H. -K.
Pazgal-Kobrowski, I.
Forni, G. L.
Perrotta, S.
Khelif, A.
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Abstract
Background Patients with transfusion-dependent beta-thalassemia need regular red-cell transfusions. Luspatercept, a recombinant fusion protein that binds to select transforming growth factor beta superfamily ligands, may enhance erythroid maturation and reduce the transfusion burden (the total number of red-cell units transfused) in such patients.
URI
http://hdl.handle.net/20.500.12627/174364
https://doi.org/10.1056/nejmoa1910182
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