Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
Robert Klamroth, Michael Recht, Nigel S. Key, Wolfgang Miesbach, Steven W. Pipe, Radosław Kaczmarek, Douglass Drelich, Blanca Salazar, Sandra Le Quellec, Paul E. Monahan, Nicholas Galante, Paul van der Valk, Jacqueline Tarrant
Abstract
= 0.0065). For participants with detectable anti-AAV5 NAbs and IgG, there was a high correlation of titers at each visit (median r = 0.96; range: 0.92-0.99). IgM anti-AAV5 antibodies were detectable in only 9% of participants, and seroconversion was infrequent. In conclusion, AAV5 NAb test results were consistent over 6 months, which informs the timing of NAb screening when considering gene therapy for hemophilia B.
Topics & Concepts
AntibodyNatural historyLead (geology)Phase (matter)Natural (archaeology)MedicineVirologyImmunologyHistoryPhysicsInternal medicineBiologyArchaeologyPaleontologyQuantum mechanicsHemophilia Treatment and ResearchPlatelet Disorders and Treatments