FVIII/VWF complex displays a greater pro-haemostatic activity than FVIII preparations devoid of VWF: Study in plasma and cell-based models

Haemophilia. 2020 Jul;26(4):e151-e160. doi: 10.1111/hae.14008. Epub 2020 Apr 23.

Abstract

Introduction: Plasma-derived FVIII/VWF complex was reported to be less sensitive to inhibitors than FVIII preparations devoid of VWF.

Aim: To compare the efficacy of FVIII/VWF complex (Fanhdi) and five different VWF-free FVIII preparations in restoring thrombin generation and activation of thrombin-activatable fibrinolysis inhibitor (TAFI) in haemophilic plasma, with and without inhibitor, and in cell-based models.

Methods: Experiments were performed in haemophilic plasma supplemented with inhibitory IgG or in plasma samples obtained from haemophilia A patients without (n = 11) and with inhibitor (n = 12). Thrombin generation was evaluated by calibrated automated thrombography (CAT) under standard conditions, in the presence of activated protein C (APC) or thrombomodulin (TM), and in cell-based models including endothelial cells, either alone or in combination with platelets or tissue factor-expressing blood mononuclear cells. The kinetics of TAFI activation was determined by a two-stage functional assay in the absence and in the presence of APC.

Results: In haemophilic plasma without inhibitor, Fanhdi enhanced thrombin generation and TAFI activation as well as recombinant (2nd-4th generation) and plasma-derived FVIII preparations devoid of VWF. On the contrary, in plasma with inhibitor, Fanhdi displayed a greater ability to restore thrombin generation and TAFI activation under all tested conditions. Notably, in cell-based models including endothelial cells, Fanhdi proved more efficient than all other preparations in improving thrombin generation even in the absence of inhibitor.

Conclusion: The greater pro-haemostatic activity of FVIII/VWF complex, either in haemophilic plasma with inhibitor or in the presence of endothelial cells, may offer therapeutic advantages.

Keywords: coagulation; endothelial cells; fibrinolysis; inhibitor; thrombin; thrombin-activatable fibrinolysis inhibitor.

Publication types

  • Comparative Study

MeSH terms

  • Carboxypeptidase B2 / drug effects
  • Carboxypeptidase B2 / metabolism
  • Carboxypeptidase B2 / pharmacology
  • Coagulants / pharmacology
  • Coagulants / therapeutic use
  • Combined Modality Therapy
  • Endothelial Cells / drug effects
  • Endothelial Cells / metabolism
  • Factor VIII / pharmacology*
  • Factor VIII / therapeutic use
  • Fibrinolysis / drug effects
  • Hemophilia A / blood
  • Hemophilia A / drug therapy*
  • Hemostasis / drug effects
  • Hemostasis / physiology
  • Humans
  • Immunoglobulin G / metabolism
  • Kinetics
  • Plasma / metabolism
  • Protein C / metabolism
  • Thrombin / drug effects
  • Thrombin / metabolism
  • Thrombomodulin / metabolism
  • Thromboplastin / metabolism
  • Treatment Outcome
  • von Willebrand Factor / pharmacology*
  • von Willebrand Factor / therapeutic use

Substances

  • Coagulants
  • Immunoglobulin G
  • Protein C
  • Thrombomodulin
  • von Willebrand Factor
  • Factor VIII
  • Thromboplastin
  • Carboxypeptidase B2
  • Thrombin