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December 9, 2017New England Journal of Medicine694 citationsOpen Access

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

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SRSavita RangarajanLWLiron WalshWLWill Lester

Key Points

  • To assess the therapeutic efficacy and safety of AAV5-hFVIII-SQ gene transfer in individuals with severe hemophilia A.
  • Interventional clinical trial (ClinicalTrials.gov: NCT02576795; EudraCT: 2014-003880-38) evaluating high-dose intravenous infusion of AAV5-hFVIII-SQ.
  • Tracked factor VIII activity levels, hemostatic stabilization, exogenous factor VIII use, and adverse safety events over a 1-year follow-up period.
  • High-dose AAV5-hFVIII-SQ infusion achieved sustained normalization of factor VIII activity over 1 year in 6 of 7 participants.
  • Hemostasis was stabilized with a profound reduction in exogenous factor VIII concentrate use across all 7 participants, with no safety events reported.

Abstract

The infusion of AAV5-hFVIII-SQ was associated with the sustained normalization of factor VIII activity level over a period of 1 year in six of seven participants who received a high dose, with stabilization of hemostasis and a profound reduction in factor VIII use in all seven participants. In this small study, no safety events were noted, but no safety conclusions can be drawn. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov number, NCT02576795 ; EudraCT number, 2014-003880-38 .).

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Cite This Study

Rangarajan et al. (2017) studied this question.

synapsesocial.com/papers/69d9f15084371aa676a3c520https://doi.org/10.1056/nejmoa1708483
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