FVIII activity following FVIII protein infusion or FVIII gene transfer predicts the bleeding risk in hemophilia A rats.

ABSTRACT:

Background

Prophylactic replacement therapy in hemophilia A (HA) patients does not adequately prevent bleeds and arthropathic complications. A more refined understanding of the relationship between coagulation factor VIII (FVIII) levels and bleeding risk during protein prophylaxis, or with gene therapy, is needed to improve patient care.

Objectives

Investigate this relationship in the HA rat, a model exhibiting spontaneous bleeds and development of arthropathy similar to HA patients.

Methods

Human B domain-deleted FVIII was delivered to HA rats via adeno-associated virus (AAV)-mediated gene transfer or multiple intravenous protein injections.

Results and conclusions

After 12 weeks of observation, both approaches significantly reduced bleeds per animal and incre

SUBMITTER: Lovgren KM 

PROVIDER: S-EPMC7786582 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature