The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A

•The inhibitor incidence of N8-GP in previously untreated patients was 29.9% (14.9% high-titer).•In 17 patients, temporarily decreased incremental recovery without inhibitors was observed within the first 5 exposure days to N8-GP. [Display omitted] N8-GP (turoctocog alfa pegol) is a recombinant, gly...

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Published in:Blood advances 2023-02, Vol.7 (4), p.620-629
Main Authors: Male, Christoph, Königs, Christoph, Dey, Sohan, Matsushita, Tadashi, Millner, Anders Holm, Zak, Marek, Young, Guy, Kenet, Gili
Format: Article
Language:English
Subjects:
Child
Factor VIII - therapeutic use
Hemophilia A - drug therapy
Hemorrhage - chemically induced
Hemostatics - therapeutic use
Humans
Recombinant Proteins - therapeutic use
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Summary:•The inhibitor incidence of N8-GP in previously untreated patients was 29.9% (14.9% high-titer).•In 17 patients, temporarily decreased incremental recovery without inhibitors was observed within the first 5 exposure days to N8-GP. [Display omitted] N8-GP (turoctocog alfa pegol) is a recombinant, glycoPEGylated, extended half-life, factor VIII replacement product. Here, we examined the immunogenicity, safety, and efficacy of N8-GP in previously untreated patients (PUPs). pathfinder6 is an ongoing, open-label, phase 3 trial that enrolled PUPs with severe hemophilia A and were aged 5 BU]). Sixty-five patients received N8-GP prophylaxis for an average of 2.17 years with a median annualized bleeding rate (interquartile range) of 1.42 (0.76; 3.13) and a 90.5% hemostatic success rate. Temporarily decreased incremental recovery (IR), defined as ≥2 consecutive measurements of IR
ISSN:2473-9529
2473-9537
DOI:10.1182/bloodadvances.2022007529