| Autor: |
MC Ozelo, J Mahlangu, B Madan, J Mason, F Peyvandi, AV Drygalski, G Yamaguti-Hayakawa, TM Robinson, SW Pipe, TT Gener-Eigh |
| Jazyk: |
angličtina |
| Rok vydání: |
2023 |
| Předmět: |
|
| Zdroj: |
Hematology, Transfusion and Cell Therapy, Vol 45, Iss , Pp S451-S452 (2023) |
| Druh dokumentu: |
article |
| ISSN: |
2531-1379 |
| DOI: |
10.1016/j.htct.2023.09.842 |
| Popis: |
Aims: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) provides endogenous factor VIII (FVIII) production to prevent bleeding in people with severe hemophilia A. This study evaluated bleeding, FVIII activity and safety outcomes 3 years after receiving valoctocogene roxaparvovec. Methods: The open-label, multicenter phase 3 GENEr8-1 trial (NCT03370913) evaluated 6 x 1013 vg/kg valoctocogene roxaparvovec in 134 adult men with severe hemophilia A (FVIII ≤1 IU/dL) without inhibitors. Bleeds and FVIII use were self-reported after regular prophylaxis ended (scheduled for week [W] 4) through data cutoff. Comparisons to baseline on FVIII prophylaxis were performed in a subset of 112 HIV-negative participants enrolling from a non-interventional study (rollover population). FVIII activity per chromogenic assay and quality of life (QOL) per Haemo-QOL-A were assessed in 132 HIV-negative participants (modified intent-to-treat [mITT] population). Safety was assessed in all participants. Results: Median follow-up was 162 weeks (n = 134); 131 participants completed W156. The mean annualized treated bleeding rate in 112 rollover participants over 3 years was 0.8 bleeds/year, mean annualized rate of all bleeds was 1.3 bleeds/year, and mean FVIII utilization was 125 IU/kg/year. During year 3, 73.2% of 110 rollover participants had zero treated bleeds and 61.6% had no bleeds (excluding surgeries/procedures). At W156, mean and median FVIII were 18.8 and 8.4 IU/dL (mITT, N = 132); 11.4%, 56.1%, and 32.6% of mITT participants had FVIII activity ≥40 IU/dL, ≥5 and |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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