Bella Madan, Margareth C Ozelo, Priyanka Raheja, Emily Symington, Doris V Quon, Andrew D Leavitt, Steven W Pipe, Gillian Lowe, Gili Kenet, Mark T Reding, Jane Mason, Michael Wang, Annette von Drygalski, Robert Klamroth, Susan Shapiro, Hervé Chambost, Amy L Dunn, Johannes Oldenburg, Sheng-Chieh Chou, Flora Peyvandi, Carolyn M Millar, Dane Osmond, Hua Yu, Ebony Dashiell-Aje, Tara M Robinson, Johnny Mahlangu
BACKGROUND: Valoctocogene roxaparvovec transfers a human factor VIII (FVIII) coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection. OBJECTIVE: Present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial. METHODS: GENEr8-1 enrolled 134 adult males with severe hemophilia A who were receiving FVIII prophylaxis. Efficacy endpoints included annualized bleeding rate (ABR), annualized FVIII utilization (AFU), FVIII activity (chromogenic substrate assay; imputed as 1 IU/dL at baseline and 0 IU/dL after discontinuation), and the Haemophilia-Specific Quality of Life Questionnaire for Adults (Haemo-QOL-A)...
April 11, 2024: Journal of Thrombosis and Haemostasis: JTH