Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Patients with severe hemophilia A were treated with an adenoviral construct containing coagulation factor VIII cDNA and followed for 1 to 3 years. Median factor VIII activity at 49 to 52 weeks was 24 IU per deciliter, and annualized bleeding rates decreased after treatment. Elevations in alanine ami...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2022-03, Vol.386 (11), p.1013-1025
Hauptverfasser:	Ozelo, Margareth C, Mahlangu, Johnny, Pasi, K. John, Giermasz, Adam, Leavitt, Andrew D, Laffan, Michael, Symington, Emily, Quon, Doris V, Wang, Jiaan-Der, Peerlinck, Kathelijne, Pipe, Steven W, Madan, Bella, Key, Nigel S, Pierce, Glenn F, O’Mahony, Brian, Kaczmarek, Radoslaw, Henshaw, Joshua, Lawal, Adebayo, Jayaram, Kala, Huang, Mei, Yang, Xinqun, Wong, Wing Y, Kim, Benjamin
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Adverse events Alanine Alanine transaminase Alanine Transaminase - blood Anemia Antibodies Anticoagulants Aspartate aminotransferase Bleeding Body weight Coagulation factor VIII Coagulation factors Complementary DNA Dependovirus Disease prevention Employees Enrollments Factor VIII - therapeutic use Factor VIII deficiency Gene Editing Gene therapy Genetic Therapy - methods Genetic Vectors Genetics Genetics General Genomes Hematology Hemophilia Hemophilia A - complications Hemophilia A - therapy Hemorrhage - epidemiology Hemorrhage - etiology Hemorrhage - prevention & control HIV HIV Seronegativity Human immunodeficiency virus Humans Hypotheses Infusions, Intravenous Intention to Treat Analysis Laboratories Male Nausea Oncology Population Prophylaxis Safety Thrombosis
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Beschreibung
Zusammenfassung:	Patients with severe hemophilia A were treated with an adenoviral construct containing coagulation factor VIII cDNA and followed for 1 to 3 years. Median factor VIII activity at 49 to 52 weeks was 24 IU per deciliter, and annualized bleeding rates decreased after treatment. Elevations in alanine aminotransferase were the most common toxic effect and were mainly controlled with glucocorticoids.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2113708