Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial
| Autor: | Ali T Taher, Maria Domenica Cappellini, Antonis Kattamis, Ersi Voskaridou, Silverio Perrotta, Antonio G Piga, Aldo Filosa, John B Porter, Thomas D Coates, Gian Luca Forni, Alexis A Thompson, Immacolata Tartaglione, Khaled M Musallam, Jay T Backstrom, Oriana Esposito, Ana Carolina Giuseppi, Wen-Ling Kuo, Dimana Miteva, Jennifer Lord-Bessen, Aylin Yucel, Tatiana Zinger, Jeevan K Shetty, Vip Viprakasit, Jassada Buaboonnam, Supachai Ekwattanakit, Archrob Khunhapinant, Efthalia Loka, Maria Moraki, Pagona Flevari, Maria Dimopoulou, Vasiliki Bartzi, Hisham Daadaa, Georges El Hasbani, Suzanne Koussa, Federica Ammendola, Saverio Scianguetta, Marta Puglia, Ilaria Ferrara, Giovanni Ferrero, Carmen Gaglioti, Filomena Longo, Silvia Turrini, Vincenzo Voi, Elena Cassinerio, Anna De, Giovanna Graziadei, Alessia Marcon, Margherita Migone De Amicis, Irene Motta, Patrizia Cinque, Bruno Pannone, Paolo Ricchi, Manuela Balocco, Paola Carrara, Francesco Della Rovere, Martina Lamagna, Valeria Pinto, Sabrina Quintino, Perla Eleftheriou, Maciej Garbowski, Arne de Kreuk, Susan Carson, Christopher Denton, Tom Hofstra, Sayany Veluswamy, John Wood, Sherif Badawy, Rachel Bercovitz, Rukhmi Bhat, Diane Calamaras, Robert Liem, Astrid Mack |
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| Přispěvatelé: | Taher, Ali T, Cappellini, Maria Domenica, Kattamis, Antoni, Voskaridou, Ersi, Perrotta, Silverio, Piga, Antonio G, Filosa, Aldo, Porter, John B, Coates, Thomas D, Forni, Gian Luca, Thompson, Alexis A, Tartaglione, Immacolata, Musallam, Khaled M, Backstrom, Jay T, Esposito, Oriana, Giuseppi, Ana Carolina, Kuo, Wen-Ling, Miteva, Dimana, Lord-Bessen, Jennifer, Yucel, Aylin, Zinger, Tatiana, Shetty, Jeevan K, Viprakasit, Vip |
| Jazyk: | angličtina |
| Rok vydání: | 2022 |
| Předmět: |
Adult
Male Settore MED/09 - Medicina Interna Activin Receptors Type II Hemoglobin E Recombinant Fusion Proteins beta-Thalassemia Hematology Immunoglobulin Fc Fragments Treatment Outcome Double-Blind Method alpha-Globins Quality of Life Unit Cardarelli Hospital Humans Thalassemia Female Settore MED/15 - Malattie del Sangue |
| Popis: | Background In patients with non-transfusion-dependent beta-thalassaemia, haemoglobin concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality, and impaired quality of life. No drugs are specifically approved for anaemia management in patients with non-transfusion-dependent beta-thalassaemia, other than transfusion therapy administered infrequently in accordance with patients' needs. We assessed the efficacy and safety of luspatercept versus placebo in patients with non-transfusion-dependent beta-thalassaemia.Methods We did a phase 2, randomised, double-blind, multicentre, placebo-controlled trial in 12 centres in six countries (Thailand [n=1], Lebanon [n=1], Greece [n=2], Italy [n=5], the UK [n=1], and the USA [n=2]). Eligible patients were aged 18 years or older, had confirmed diagnosis of beta-thalassaemia or haemoglobin E/beta-thalassaemia (concomitant a-globin deletion, mutation, or duplication were allowed), and a baseline haemoglobin concentration of 10.0 g/dL or lower. All patients were non-transfusion-dependent. Patients were randomly assigned (2:1) to luspatercept or placebo using an interactive response technology system and stratified by baseline haemoglobin concentration (>= 8.5 g/dL vs = 3 vs |
| Databáze: | OpenAIRE |
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