Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was maintained for 2 years despite declining factor VIII levels.

Saved in:
Bibliographic Details
Published inThe New England journal of medicine Vol. 388; no. 8; pp. 694 - 705
Main Authors Mahlangu, Johnny, Kaczmarek, Radoslaw, von Drygalski, Annette, Shapiro, Susan, Chou, Sheng-Chieh, Ozelo, Margareth C., Kenet, Gili, Peyvandi, Flora, Wang, Michael, Madan, Bella, Key, Nigel S., Laffan, Michael, Dunn, Amy L., Mason, Jane, Quon, Doris V., Symington, Emily, Leavitt, Andrew D., Oldenburg, Johannes, Chambost, Hervé, Reding, Mark T., Jayaram, Kala, Yu, Hua, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B., Henshaw, Joshua, Robinson, Tara M., Wong, Wing Yen, Pipe, Steven W.
Format Journal Article
LanguageEnglish
Published United States Massachusetts Medical Society 23.02.2023
Subjects
Bleeding
Body weight
Coagulation
Coagulation factors
Confidence intervals
Disease prevention
Epidemiology
Factor VIII - therapeutic use
Factor VIII deficiency
Gene transfer
Gene Transfer Techniques
Genetics
Genetics General
Genomes
Half-Life
Hematology
Hemophilia
Hemophilia A - complications
Hemophilia A - drug therapy
Hemorrhage - etiology
Hemorrhage - prevention & control
HIV
Human immunodeficiency virus
Humans
Hypotheses
Life Sciences
Male
Oncology
Pharmacokinetics
Prophylaxis
Recombinant Fusion Proteins - therapeutic use
Safety
Statistical analysis
Online AccessGet full text

Cover

More Information
Summary:Participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was maintained for 2 years despite declining factor VIII levels.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
content type line 14
content type line 23
ISSN:0028-4793
1533-4406
1533-4406
DOI:10.1056/NEJMoa2211075