Department of Pediatrics, Nara Medical University, Kashihara, Japan.
The Course of Thrombosis and Hemostasis Molecular Pathology, Nara Medical University, Kashihara, Japan.
Blood Adv. 2023 Jun 27;7(12):2831-2842. doi: 10.1182/bloodadvances.2022008918.
Factor V (FV) plays pivotal roles in both procoagulant and anticoagulant mechanisms. Genetic mutations, FV-W1920R (FVNara) and FV-A2086D (FVBesançon), in the C1 and C2 domains of FV light chain, respectively, seem to be associated with deep vein thrombosis. However, the detailed mechanism(s) through which these mutations are linked to thrombophilia remains to be fully explored. The aim of this study was to clarify thrombotic mechanism(s) in the presence of these FV abnormalities. Full-length wild-type (WT) and mutated FV were prepared using stable, human cell lines (HEK293T) and the piggyBac transposon system. Susceptibility of FVa-A2086D to activated protein C (APC) was reduced, resulting in significant inhibition of APC-catalyzed inactivation with limited cleavage at Arg306 and delayed cleavage at Arg506. Furthermore, APC cofactor activity of FV-A2086D in APC-catalyzed inactivation of FVIIIa through cleavage at Arg336 was impaired. Surface plasmon resonance-based assays demonstrated that FV-A2086D bound to Glu-Gly-Arg-chloromethylketone active site-blocked APC and protein S (P) with similar affinities to that of FV-WT. However, weakened interaction between FVa-A2086D and phospholipid membranes was evident through the prothrombinase assay. Moreover, addition of FVa-A2086D to plasma failed to inhibit tissue factor (TF)-induced thrombin generation and reduce prothrombin times. This inhibitory effect was independent of PC, PS, and antithrombin. The coagulant and anticoagulant characteristics of FV(a)-W1920R were similar to those of FV(a)-A2086D. FV-A2086D presented defects in the APC mechanisms associated with FVa inactivation and FV cofactor activity, similar to FV-W1920R. Moreover, both FV proteins that were mutated in the light chain impaired inhibition of TF-induced coagulation reactions. These defects were consistent with congenital thrombophilia.
FV(因子 V)在促凝和抗凝机制中都起着关键作用。FV 轻链 C1 和 C2 结构域中的遗传突变 FV-W1920R(FVNara)和 FV-A2086D(FVBesançon)似乎与深静脉血栓形成有关。然而,这些突变与血栓形成之间的详细机制仍有待充分探索。本研究旨在阐明存在这些 FV 异常时的血栓形成机制。使用稳定的人细胞系(HEK293T)和 piggyBac 转座子系统制备全长野生型(WT)和突变型 FV。FV-a-A2086D 对活化蛋白 C(APC)的敏感性降低,导致 APC 催化失活时 Arg306 有限切割和 Arg506 延迟切割,从而显著抑制 APC 催化失活。此外,FV-a-A2086D 在 APC 催化 FVIIIa 失活时通过 Arg336 切割的 APC 辅因子活性受损。基于表面等离子体共振的测定表明,FV-a-A2086D 与 Glu-Gly-Arg-氯甲基酮活性位点封闭的 APC 和蛋白 S(P)的结合亲和力与 FV-WT 相似。然而,通过凝血酶原酶测定可以明显看出 FVa-A2086D 与磷脂膜之间的相互作用减弱。此外,向血浆中添加 FVa-A2086D 不能抑制组织因子(TF)诱导的凝血酶生成并缩短凝血酶原时间。这种抑制作用与 PC、PS 和抗凝血酶无关。FV(a)-W1920R 的凝血和抗凝特性与 FV(a)-A2086D 相似。FV-a-A2086D 在 APC 机制中存在缺陷,与 FVa 失活和 FV 辅因子活性有关,类似于 FV-W1920R。此外,在轻链中发生突变的两种 FV 蛋白均损害了对 TF 诱导的凝血反应的抑制。这些缺陷与先天性血栓形成一致。