학술논문
Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial
Document Type
Article
Author
Lee, Jong Wook; Griffin, Morag; Kim, Jin Seok; Lee Lee, Lily Wong; Piatek, Caroline; Nishimura, Jun-ichi; Carrillo Infante, Cynthia; Jain, Deepak; Liu, Peng; Filippov, Gleb; Sicre de Fontbrune, Flore; Risitano, Antonio; Kulasekararaj, Austin G; Barcellini, Wilma; Barraco, Fiorenza; Beneitez Pastor, David; Capra, Marcelo; Chew, Lee Ping; Chrysavgi, Lalayanni; De Castro, Carlos; de la Tour, Régis Peffault; De Oliveira, Michel Michels; Di Bona, Eros; Forcade, Edouard; Fu, Chieh-Lin; Furha, Cossor; Gaya Valls, Anna; Giannouli, Stavroula; Gonzalez-Fernandez, Ataulfo; Griffin, Morag; Gural, Alexander; Gutierrez, Emilio Ojeda; Hernández-Rodríguez, Inés; Ibrahim, Ibrahim; Iori, Anna Paola; Ishida, Tadao; Jang, Jun Ho; Kim, Jeong-A; Kim, Jin Seok; Kitano, Toshiyuki; Kosugi, Hiroshi; Kreiniz, Natalia; Kulasekararaj, Austin; Lee, Jong Wook; Lee Lee, Lily Wong; Mayer, Jiri; Mitchell, Lindsay; Mori, Yasuo; Nishiwaki, Kaichi; Notaro, Rosario; Nunez, Ramiro; Obara, Naoshi; Oliva, Esther Natalie; Patriquin, Christopher; Pessoa, Viviani; Piatek, Caroline; Piekarska, Agnieszka; Raza, Shahzad; Risitano, Antonio Maria; Rojnuckarin, Ponlapat; Samuel, Desmond; Shammo, Jamile; Tadmor, Tamar; Takami, Akiyoshi; Tamari, Roni; Terriou, Louis; Uchiyama, Hitoji; Vannucchi, Alessandro Maria; Yamaguchi, Hiroki
Source
The Lancet Haematology; December 2023, Vol. 10 Issue: 12 pe955-e965, 11p
Subject
Language
ISSN
23523026
Abstract
Symptoms of anaemia due to clinically significant extravascular haemolysis can affect patients with paroxysmal nocturnal haemoglobinuria (PNH) treated with C5 inhibitors (ravulizumab or eculizumab). The aim of this study was to assess the efficacy and safety of danicopan (ALXN2040), an investigational, first-in-class, oral complement factor D inhibitor, as add-on therapy to ravulizumab or eculizumab in patients with PNH and clinically significant extravascular haemolysis.