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Please use this identifier to cite or link to this item: http://repository.li.mahidol.ac.th/dspace/handle/123456789/54614
Title: A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia
Authors: M. Domenica Cappellini
Vip Viprakasit
Ali T. Taher
Pencho Georgiev
Kevin H.M. Kuo
Thomas Coates
Ersi Voskaridou
Hong Keng Liew
Idit Pazgal-Kobrowski
G. L. Forni
Silverio Perrotta
Abderrahim Khelif
Ashutosh Lal
Antonis Kattamis
Efthymia Vlachaki
Raffaella Origa
Yesim Aydinok
Mohamed Bejaoui
P. Joy Ho
Lee Ping Chew
Ping Chong Bee
Soo Min Lim
Meng Yao Lu
Adisak Tantiworawit
Penka Ganeva
Liana Gercheva
Farrukh Shah
Ellis J. Neufeld
Alexis Thompson
Abderrahmane Laadem
Jeevan K. Shetty
Jun Zou
Jennie Zhang
Dimana Miteva
Tatiana Zinger
Peter G. Linde
Matthew L. Sherman
Olivier Hermine
John Porter
Antonio Piga
l'Institut des Maladies Génétiques Imagine
Azienda Ospedaliera Brotzu - Microcitemico
Centre National de Greffe de Moelle Osseuse
University Hospital St. Marina
American University of Beirut Medical Center
Hopital Farhat Hached Sousse
UCSF Benioff Children‘s Hospital
Università degli Studi di Milano
National and Kapodistrian University of Athens
St.George University Hospital
Hôpital Necker Enfants Malades
Università degli Studi della Campania Luigi Vanvitelli
Laikon General Hospital
UCL
Rabin Medical Center Israel
St. Jude Children's Research Hospital
Celgene Corporation
E.O. Ospedali Galliera
Royal Prince Alfred Hospital
University of Toronto
Keck School of Medicine of USC
University of Malaya Medical Centre
Hospital Sultanah Aminah
Università degli Studi di Torino
Faculty of Medicine, Siriraj Hospital, Mahidol University
Ege University Medical School
National Health Service
National Taiwan University
Hippokration General Hospital
Chiang Mai University
Celgene
Hospital Umum
Ann and Robert H. Lurie Children's Hospital of Chicago
Specialized Hospital for Active Treatment in Oncology
Hospital Sultanah Bahiyah
Acceleron Pharma
Keywords: Medicine
Issue Date: 26-Mar-2020
Citation: New England Journal of Medicine. Vol.382, No.13 (2020), 1219-1231
Abstract: © 2020 Massachusetts Medical Society. 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<0.001). During any 12-week interval, the percentage of patients who had a reduction in transfusion burden of at least 33% was greater in the luspatercept group than in the placebo group (70.5% vs. 29.5%), as was the percentage of those who had a reduction of at least 50% (40.2% vs. 6.3%). The least-squares mean difference between the groups in serum ferritin levels at week 48 was −348 μg per liter (95% confidence interval, −517 to −179) in favor of luspatercept. Adverse events of transient bone pain, arthralgia, dizziness, hypertension, and hyperuricemia were more common with luspatercept than placebo. CONCLUSIONS 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.
URI: http://repository.li.mahidol.ac.th/dspace/handle/123456789/54614
metadata.dc.identifier.url: https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85082380437&origin=inward
ISSN: 15334406
00284793
Appears in Collections:Scopus 2020

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