A fully recombinant partial prothrombin complex effectively bypasses fVIII in vitro and in vivo

Thromb Haemost. 2002 Dec;88(6):1003-11.

Abstract

The development of inhibitory antibodies is a serious complication in hemophilic patients, severely compromising therapeutic success. Bleeding episodes in affected patients are controlled by treatment with a plasma-derived prothrombin complex concentrate (PCC), activated PCC (APCC) or recombinant activated factor VII. We hypothesized that a recombinant two-component agent consisting of recombinant prothrombin (rfII) and activated factor X (rfXa) would have substantial fVIII bypassing activity and could be a safe alternative therapeutic option. To test this hypothesis we assembled an agent in vitro solely consisting of rfII and rfXa at a molar ratio of 37,500:1. These factors are believed to be responsible for the activity of APCC preparations. Recombinant fX, used as the source for fXa generation, and rfII were purified from serum-free and protein-free conditioned media of stably transfected CHO and BHK tissue culture cells, respectively. Activation of rfX to rfXa was accomplished by the plant protease ficin, obviating the need for a protease derived from a human or animal source. We found that in vitro the complex reduced the abnormally prolonged activated partial thromboplastin time (APTT) of a high-titer fVIII inhibitor plasma similar to an APCC preparation. Furthermore, addition of increasing amounts of rfII/rfXa to inhibitor plasma resulted in a linear dose-dependent increase in the rate of thrombin generation. In a rabbit fVIII inhibitor model, treatment with rfII/rfXa statistically significantly reduced the intensity of the abnormal cuticle bleeding. In the Wessler test, rfII/rfXa showed no thrombogenicity. These data show that a well-defined, particularly safe and efficacious agent with fVIII bypassing activity can be generated from recombinant fII and fXa.

MeSH terms

  • Animals
  • Blood Coagulation / drug effects
  • CHO Cells
  • Cell Line
  • Cricetinae
  • Factor VIII / antagonists & inhibitors
  • Factor VIII / metabolism*
  • Factor Xa / genetics
  • Factor Xa / isolation & purification
  • Factor Xa / therapeutic use
  • Ficain
  • Humans
  • In Vitro Techniques
  • Partial Thromboplastin Time
  • Prothrombin / genetics
  • Prothrombin / isolation & purification
  • Prothrombin / therapeutic use*
  • Rabbits
  • Recombinant Proteins / genetics
  • Recombinant Proteins / isolation & purification
  • Recombinant Proteins / therapeutic use
  • Safety
  • Thrombin / biosynthesis
  • Thrombosis / etiology
  • Transfection

Substances

  • Recombinant Proteins
  • Prothrombin
  • Factor VIII
  • Thrombin
  • Factor Xa
  • Ficain