Patients with severe congenital protein (P)C deficiency require long-term anticoagulant management. Recombinant PC concentrates for prophylactic use are not available in Japan; prothrombin complex concentrates (PCC), containing factors (F)II, VII, IX, X, and PC (PPSB-HT®), have been used 'off-label' in a few patients. We investigated the combined use of prophylactic PCC and Warfarin (VKA; PT-INR 2.0-2.5) in a severely PC-deficient patient in whom VKA alone did not prevent recurrent purpura. Plasma VKA-dependent factor levels and global PC function (Thrombopath®) were assessed. Plasma activity levels of FII/FVII/FIX/FX post-infusion of PCC (6.3 unit/kg) increased 35/27/27/35 (initial level) to 59/60/38/83 IU/dl, respectively. FVII:C and FIX:C rapidly returned to baseline levels 12-24 h post-infusion, but FII:C and FX:C returned more slowly. PC antigen (< 5%) increased to ~ 15%, followed by return to baseline levels 24 h post-infusion. Global PC function was very low (%PiCi 24%), but improved post-PCC infusion. This potential was slightly detectable even at an undetectable PC level. At day 3, high levels of D-dimer and FDP were observed without thrombotic event, but these improved post-infusion. Although PCC restored VKA-dependent coagulation factors, PC contained in PCC significantly improved global anticoagulation, and was clinically beneficial in this severely deficient patient.

中文翻译：

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在严重C蛋白缺乏的情况下，使用华法林和凝血酶原复合物联合抗凝治疗的止血效果评估。

患有严重先天蛋白（P）C缺乏症的患者需要长期抗凝治疗。在日本没有用于预防的重组PC浓缩物。凝血酶原复合物浓缩物（PCC）包含因子（F）II，VII，IX，X和PC（PPSB-HT®），已在少数患者中使用“标签外”。我们研究了严重PC缺乏的患者中预防性PCC和华法林（VKA; PT-INR 2.0-2.5）的联合使用，其中仅VKA不能预防复发性紫癜。评估血浆VKA依赖性因子水平和整体PC功能（Thrombopath®）。输注PCC后FII / FVII / FIX / FX的血浆活性水平（6.3单位/千克）分别增加35/27/27/35（初始水平）至59/60/38/83 IU / dl。FVII：C和FIX：C在输注后12-24小时迅速恢复到基线水平，但FII：C和FX：C返回的速度更慢。PC抗原（<5％）增加至〜15％，然后在输注后24小时恢复到基线水平。总体PC功能非常低（％PiCi为24％），但PCC后输注得到了改善。即使在无法检测到的PC水平下，也可以轻微检测到该电位。在第3天，观察到高水平的D-二聚体和FDP，没有血栓形成事件，但是输注后这些改善。尽管PCC恢复了依赖VKA的凝血因子，但PCC中包含的PC显着改善了总体抗凝作用，并且对该严重缺乏的患者具有临床益处。观察到高水平的D-二聚体和FDP没有血栓形成事件，但是输注后这些改善了。尽管PCC恢复了依赖VKA的凝血因子，但PCC中包含的PC显着改善了总体抗凝作用，并且对该严重缺乏的患者具有临床益处。观察到高水平的D-二聚体和FDP没有血栓形成事件，但是输注后这些改善了。尽管PCC恢复了依赖VKA的凝血因子，但PCC中包含的PC显着改善了总体抗凝作用，并且对该严重缺乏的患者具有临床益处。