Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
National Health Laboratory Service · University of the Witwatersrand · +27 more institutions
Abstract
Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a phase 3 study of the efficacy and safety of valoctocogene roxaparvovec therapy evaluated after 52 weeks in men with severe hemophilia A have been published previously.
vector genomes of valoctocogene roxaparvovec per kilogram of body weight. The primary end point was the change from baseline in the annualized rate of treated bleeding events at week 104 after receipt of the infusion. The pharmacokinetics of valoctocogene roxaparvovec were modeled to estimate the bleeding risk relative to the activity of transgene-derived factor VIII.
Citation impact
- FWCI
- 52.31
- Percentile
- 100%
- References
- 29
Authors
29- JMJohnny MahlanguCorresponding
National Health Laboratory Service, University of the Witwatersrand, Johannesburg Hospital, Indiana University – Purdue University Indianapolis
- RKRadosław Kaczmarek
National Health Laboratory Service, Ludwik Hirszfeld Institute of Immunology and Experimental Therapy, Indiana University School of Medicine, Indiana University – Purdue University Indianapolis
- AVAnnette von Drygalski
National Health Laboratory Service, University of California San Diego, Indiana University – Purdue University Indianapolis
- SSSusan Shapiro
National Health Laboratory Service, National Institute for Health Research, NIHR Biomedical Research Centre at The Royal Marsden and the ICR, Indiana University – Purdue University Indianapolis
- SCSheng‐Chieh Chou
National Health Laboratory Service, National Taiwan University Hospital, Indiana University – Purdue University Indianapolis
Topics & keywords
- Medicine
- Confidence interval
- Clinical endpoint
- Internal medicine
- Factor IX
- Risk factor
- Gastroenterology
- Clinical trial
- Good health and well-being