Ding Qiulan, Yang Likui, Zhao Xiaoqing, Wu Wenman, Wang Xuefeng, Rezaie Alireza R
Xuefeng Wang, MD, Department of Laboratory Medicine, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, No. 197 Ruijin Second Road, Shanghai, 200025 China, Tel.: +86 21 54667770, Fax: +86 21 64333548, E-mail:
Alireza R. Rezaie, PhD, Cardiovascular Biology Research Program, Oklahoma Medical Research Foundation, 825 N.E. 13th Street, Oklahoma City, OK 73104, USA, Tel.: +1 405 271 4711, Fax: +1 405 271 3137, E-mail:
Thromb Haemost. 2017 Feb 28;117(3):479-490. doi: 10.1160/TH16-10-0750. Epub 2016 Dec 15.
We have characterised the pathogenic basis of dysprothrombinaemia in a patient exhibiting paradoxical bleeding and thrombotic defects during pregnancy and postpartum. Genetic analysis revealed that the proband is homozygous for the prothrombin Arg382His mutation, possessing only ~1 % clotting activity. The proband experienced severe bleeding episodes during her pregnancy, which required treatment with prothrombin complex concentrates, and then pulmonary embolism and deep-vein thrombosis at 28 days postpartum, which required treatment with LMWH and fresh frozen plasma. Analysis of haemostatic parameters revealed that the subject had elevated FDP and DD and decreased fibrinogen levels, indicating the presence of hyperfibrinolysis. Thrombin generation and clotting assays with the proband's plasma in the presence of soluble thrombomodulin and tissue-type plasminogen activator indicated a defect in activation of both protein C and thrombin activatable fibrinolysis inhibitor (TAFI). Unlike normal plasma, no TAFI activation could be detected in the patient's plasma. The expression and characterisation of recombinant prothrombin Arg382His indicated that zymogen activation by prothrombinase was markedly impaired and the activation of protein C and TAFI by thrombin-Arg382His was impaired 600-fold and 2500-fold, respectively. The recombinant thrombin mutant exhibited impaired catalytic activity toward both fibrinogen and PAR1 as determined by clotting and signalling assays. However, the mutant activated factor XI normally in both the absence and presence of polyphosphates. Arg382 is a key residue on (pro)exosite-1 of prothrombin and kinetic analysis of substrate activation suggested that the poor zymogenic activity of the mutant is due to its inability to bind factor Va in the prothrombinase complex.
我们已对一名在妊娠和产后出现矛盾性出血和血栓形成缺陷的患者的凝血异常血症的致病基础进行了表征。基因分析显示,先证者为凝血酶原Arg382His突变的纯合子,仅具有约1%的凝血活性。先证者在怀孕期间经历了严重的出血事件,需要用凝血酶原复合物浓缩物进行治疗,然后在产后28天发生了肺栓塞和深静脉血栓形成,需要用低分子肝素和新鲜冷冻血浆进行治疗。止血参数分析显示,该受试者的纤维蛋白降解产物(FDP)和D-二聚体(DD)升高,纤维蛋白原水平降低,表明存在高纤维蛋白溶解。在可溶性血栓调节蛋白和组织型纤溶酶原激活剂存在的情况下,用先证者的血浆进行凝血酶生成和凝血试验表明,蛋白C和凝血酶激活的纤维蛋白溶解抑制剂(TAFI)的激活均存在缺陷。与正常血浆不同,在患者血浆中未检测到TAFI激活。重组凝血酶原Arg382His的表达和表征表明,凝血酶原酶对酶原的激活明显受损,凝血酶-Arg382His对蛋白C和TAFI的激活分别受损600倍和2500倍。通过凝血和信号试验测定,重组凝血酶突变体对纤维蛋白原和蛋白酶激活受体1(PAR1)的催化活性均受损。然而,该突变体在不存在和存在多磷酸盐的情况下均能正常激活因子XI。Arg382是凝血酶原(原)外位点-1上的一个关键残基,底物激活的动力学分析表明,该突变体酶原活性差是由于其无法在凝血酶原酶复合物中结合因子Va。