Gly74Ser mutation in protein C causes thrombosis due to a defect in protein S-dependent anticoagulant function

Thromb Haemost. 2017 Jun 28;117(7):1358-1369. doi: 10.1160/TH17-01-0043. Epub 2017 Apr 13.

Abstract

Protein C is a vitamin K-dependent serine protease zymogen in plasma which upon activation by thrombin in complex with thrombomodulin (TM) down-regulates the clotting cascade by a feedback loop inhibition mechanism. Activated protein C (APC) exerts its anticoagulant function through protein S-dependent degradation of factors Va and VIIIa. We recently identified a venous thrombosis patient whose plasma level of protein C antigen is normal, but its anticoagulant activity is only 34 % of the normal level. Genetic analysis revealed that the proband and her younger brother carry a novel heterozygous mutation c.346G>A, p.Gly74Ser (G74S) in PROC. Thrombin generation assay indicated that the TM-dependent anticoagulant activity of the proband's plasma has been significantly impaired. We expressed protein C-G74S in mammalian cells and characterised its properties in established coagulation assays. We demonstrate that the protein C variant can be normally activated by the thrombin-TM complex and the resulting APC mutant also exhibits normal amidolytic and proteolytic activities toward both FVa and FVIIIa. However, it was discovered the protein S-dependent catalytic activity of APC variant toward both procoagulant cofactors has been significantly impaired. Protein S concentration-dependence of FVa degradation revealed that the capacity of APC variant to interact with the cofactor has been markedly impaired. The same results were obtained for inactivation of FVa-Leiden suggesting that the protein S-dependent activity of APC variant toward cleavage of Arg-306 site has been adversely affected. These results provide insight into the mechanism through which G74S substitution in APC causes thrombosis in the proband carrying this mutation.

Keywords: Protein C; factor VIIIa; factor Va; protein S; thrombosis.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Substitution
  • Anticoagulants / blood*
  • Female
  • HEK293 Cells
  • Heterozygote
  • Humans
  • Male
  • Mutant Proteins / blood*
  • Mutant Proteins / chemistry
  • Mutant Proteins / genetics*
  • Mutation, Missense
  • Pedigree
  • Protein C / chemistry
  • Protein C / genetics*
  • Protein C / metabolism*
  • Protein C Deficiency / blood*
  • Protein C Deficiency / genetics*
  • Protein S / metabolism*
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / genetics
  • Recombinant Proteins / metabolism
  • Thrombin / metabolism
  • Thrombomodulin / blood
  • Venous Thrombosis / blood
  • Venous Thrombosis / genetics

Substances

  • Anticoagulants
  • Mutant Proteins
  • Protein C
  • Protein S
  • Recombinant Proteins
  • THBD protein, human
  • Thrombomodulin
  • Thrombin