Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors.

ABSTRACT:

Background

The formation of neutralizing antibodies (inhibitors) directed against human coagulation factor VIII (hFVIII) is a life-threatening pathogenic response that occurs in 20-30% of severe congenital hemophilia A patients and 0.00015% of the remaining population (i.e. acquired hemophilia A). Interspecies amino acid sequence disparity among FVIII orthologs represents a promising strategy to mask FVIII from existing inhibitors while retaining procoagulant function. Evidence for the effectiveness of this approach exists in clinical data obtained for porcine FVIII (pFVIII) products, which have demonstrated efficacy in the setting of congenital and acquired hemophilia.

Objectives

In the current study, recombinant (r) ovine FVIII (oFVIII) was evaluated for antigenicity and p

SUBMITTER: Zakas PM 

PROVIDER: S-EPMC4295011 | biostudies-literature | 2015 Jan

REPOSITORIES: biostudies-literature