| Autor: |
Domenica Cappellini, M. 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. Lal, A. Kattamis, A. Vlachaki, E. Origa, R. Aydinok, Y. Bejaoui, M. Joy Ho, P. 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. Shetty, J.K. Zou, J. Zhang, J. Miteva, D. Zinger, T. Linde, P.G. Sherman, M.L. Hermine, O. Porter, J. Piga, A. BELIEVE Investigators |
| Jazyk: |
angličtina |
| Rok vydání: |
2020 |
| Popis: |
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 red-cell units transfused) in such patients. METHODS In this randomized, double-blind, phase 3 trial, we assigned, in a 2:1 ratio, adults with transfusion-dependent β-thalassemia to receive best supportive care plus luspatercept (at a dose of 1.00 to 1.25 mg per kilogram of body weight) or placebo for at least 48 weeks. The primary end point was the percentage of patients who had a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval. Other efficacy end points included reductions in the transfusion burden during any 12-week interval and results of iron studies. RESULTS A total of 224 patients were assigned to the luspatercept group and 112 to the placebo group. Luspatercept or placebo was administered for a median of approximately 64 weeks in both groups. The percentage of patients who had a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval was significantly greater in the luspatercept group than in the placebo group (21.4% vs. 4.5%, P |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
|
