Dreyfus M, Masterson M, David M, Rivard G E, Müller F M, Kreuz W, Beeg T, Minford A, Allgrove J, Cohen J D
Hospital Bicêtre, Paris, France.
Semin Thromb Hemost. 1995;21(4):371-81. doi: 10.1055/s-2007-1000658.
Protein C replacement therapy with a monoclonal antibody purified, virus inactivated protein C concentrate was carried out in nine infants (three male, six female) with severe congenital protein C deficiency and life-threatening purpura fulminans and/or thrombosis associated with disseminated intravascular coagulation (DIC). Eight infants were homozygous for protein C deficiency; one was a compound heterozygote. The treatment period varied from 22 days to three years. The half-life of protein C was found to be as short as two to three hours during activation of the coagulation system, increasing to approximately ten hours after stabilization. During the acute phase, protein C levels of 0.10 to 0.25 IU/mL were associated with elevated markers of coagulation activation indicating DIC, while protein C levels greater than 0.25 were associated with normalization of coagulation markers. No product-related side effects were reported. Episodes of bleeding or purpura recurred in all patients who were switched to oral anticoagulant therapy, necessitating reinstatement of protein C replacement therapy, either as needed to control symptoms, or on a long-term prophylactic schedule, alone or in addition to oral anticoagulation. Home treatment with protein C concentrate allowed a near-normal life-style for patients who otherwise would be hospitalized for long periods of time.
对9名患有严重先天性蛋白C缺乏症且伴有危及生命的暴发性紫癜和/或与弥散性血管内凝血（DIC）相关的血栓形成的婴儿（3名男性，6名女性），采用单克隆抗体纯化、病毒灭活的蛋白C浓缩物进行蛋白C替代治疗。8名婴儿为蛋白C缺乏症纯合子；1名是复合杂合子。治疗期从22天到3年不等。发现凝血系统激活期间蛋白C的半衰期短至2至3小时，稳定后增至约10小时。急性期，蛋白C水平为0.10至0.25 IU/mL与提示DIC的凝血激活标志物升高相关，而蛋白C水平大于0.25与凝血标志物正常化相关。未报告与产品相关的副作用。所有改用口服抗凝治疗患者均再次出现出血或紫癜发作，因此需要恢复蛋白C替代治疗，可根据控制症状的需要，或按长期预防性方案单独或联合口服抗凝进行。使用蛋白C浓缩物在家中治疗使患者能够过上接近正常的生活方式，否则这些患者将需要长期住院。
