학술논문
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Document Type
Article
Author
Coppens, Michiel; Pipe, Steven W; Miesbach, Wolfgang; Astermark, Jan; Recht, Michael; van der Valk, Paul; Ewenstein, Bruce; Pinachyan, Karen; Galante, Nicholas; Le Quellec, Sandra; Monahan, Paul E; Leebeek, Frank W G; Castaman, Giancarlo; Crary, Shelley E; Escobar, Miguel; Gomez, Esteban; Haley, Kristina M; Hermans, Cedric R J R; Kampmann, Peter; Kazmi, Rashid; Key, Nigel S; Klamroth, Robert; Konkle, Barbara A; Kruse-Jarres, Rebecca; Lattimore, Susan; Lemons, Richard; Meijer, Karina; O'Connell, Niamh; Quon, Doris V; Raheja, Priyanka; Symington, Emily; Verhamme, Peter; Visweshwar, Nathan; von Drygalski, Annette; Wang, Michael; Wheeler, Allison P; White, Shanna; Young, Guy
Source
The Lancet Haematology; April 2024, Vol. 11 Issue: 4 pe265-e275, 11p
Subject
Language
ISSN
23523026
Abstract
Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic factor IX in terms of bleeding protection 18 months after gene therapy in a phase 3 trial. We report post-hoc 24-month efficacy and safety data from this trial to evaluate the longer-term effects of etranacogene dezaparvovec in individuals with haemophilia B.