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

Cappellini, M.; Viprakasit, V.; Taher, A.; Georgiev, P.; Kuo, K.; Coates, T.; Voskaridou, E.; Liew, H.; Pazgal-Kobrowski, I.; Forni, G.; Perrotta, S.; Khelif, A.; Lal, A.; Kattamis, A.; Vlachaki, E.; Origa, R.; Aydinok, SERAP; Bejaoui, M.; Ho, P.; Chew, L.; Bee, P.; Lim, S.; Lu, M.; Tantiworawit, A.; Ganeva, P.; Gercheva, L.; Shah, F.; Neufeld, E.; Thompson, A.; Laadem, A.; Shetty, J.; Zou, J.; Zhang, J.; Miteva, D.; Zinger, T.; Linde, P.; Sherman, M.; Hermine, O.; Porter, J.; Piga, ZEYNEP

doi:10.1056/nejmoa1910182

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

Cappellini M. D., Viprakasit V., Taher A. T., Georgiev P., Kuo K. H. M., Coates T., ...More

NEW ENGLAND JOURNAL OF MEDICINE, vol.382, no.13, pp.1219-1231, 2020 (SCI-Expanded, Scopus)

Publication Type: Article / Article
Volume: 382 Issue: 13
Publication Date: 2020
Doi Number: 10.1056/nejmoa1910182
Journal Name: NEW ENGLAND JOURNAL OF MEDICINE
Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, PASCAL, Abstracts in Social Gerontology, AgeLine, BIOSIS, CAB Abstracts, Chemical Abstracts Core, CINAHL, Educational research abstracts (ERA), EMBASE, Food Science & Technology Abstracts, Gender Studies Database, International Pharmaceutical Abstracts, MEDLINE, MLA - Modern Language Association Database, Public Affairs Index, Veterinary Science Database
Page Numbers: pp.1219-1231
Istanbul University Affiliated: Yes

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.