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

Domenica Cappellini, Maria; Viprakasit, Vip; Taher, Alì; Georgiev, Pencho; H.M. Kuo, Kevin; D. Coates, Thomas; Voskaridou, Ersi; Liew, Hong-Keng; Pazgal-Kobrowski, Idit; Forni, G.L.; Perrotta, Silverio; Khélif, Abderrahim; Lal, Ashutosh; Kattamis, Antonis; Vlachaki, Efthymia; Origa, Raffaella; Aydınok, Yeşim; Béjaoui, Mohamed; Joy Ho, P.; Chew, Lee-Ping; Chong Bee, Ping; Lim, Soo-min; Lu, Meng‐Yao; Tantiworawit, Adisak; Ganeva, Penka; Gercheva, Liana; Shah, Farrukh; J. Neufeld, Ellis; A. Thompson, Alexis; Laadem, Abderrahmane; K. Shetty, Jeevan; Zou, Jun; Zhang, Jennie; Miteva, Dimana; Zinger, Tatiana; G. Linde, Peter; L. Sherman, Matthew; Hermine, Olivier; B. Porter, John; Piga, Antonio

doi:10.1056/nejmoa1910182

Public

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

Published in New England Journal of Medicine • Mar 25, 2020

Authors:

Maria Domenica Cappellini

Vip Viprakasit

Alì Taher

Abstract

BACKGROUND: 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 ...

Subject

Thalassemia

Medicine

Blood transfusion

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