Siffel Csaba, Wadhwa Abhinav, Tongbram Vanita, Ogongo Margaret Katana, Sliwka Henrik, Gazda Hanna T, Turecek Peter L
Takeda Development Center Americas, Inc, Lexington, Massachusetts, USA.
College of Allied Health Sciences, Augusta University, Augusta, Georgia, USA.
Res Pract Thromb Haemost. 2024 Aug 5;8(6):102542. doi: 10.1016/j.rpth.2024.102542. eCollection 2024 Aug.
Severe congenital protein C deficiency (SCPCD) is a rare disorder associated with life-threatening purpura fulminans and disseminated intravascular coagulation that typically present within hours after birth. Treatment options for patients with SCPCD include replacement therapy with a plasma-derived protein C concentrate. In this targeted literature review, we summarize information on the use of protein C concentrate as long-term prophylaxis (>1 week of treatment) for patients with SCPCD. In total, 18 publications were included in the review, of which 15 were case studies. Treatment with protein C concentrate (Ceprotin; Baxalta US Inc, a Takeda company; Takeda Manufacturing Austria AG) was reported in 11 publications, and treatment with protein C concentrate (Protexel; LFB Biomedicaments) was reported in 2 publications. One publication reported on both Ceprotin and Protexel. Details of protein C concentrate treatment regimens, including the dose, administration frequency, and route of administration, were reported in 11 publications. Dosing regimens varied across all 11 publications, possibly due to different protein C trough levels among patients or the administration of concomitant medications. Seven of the 11 publications reported on patients who initially received intravenous protein C concentrate and subsequently switched to subcutaneous administration. Treatment outcomes with protein C concentrate were generally favorable, including the prevention of coagulopathy and thrombosis and the healing of cutaneous lesions. Three adverse events in 1 publication were identified as being possibly related to Ceprotin administration. Although published data are limited, this review provides valuable insights into the treatment of patients with SCPCD in clinical practice, including protein C concentrate dosing regimens, administration routes, and associated clinical outcomes.
严重先天性蛋白C缺乏症（SCPCD）是一种罕见疾病，与危及生命的暴发性紫癜和弥散性血管内凝血相关，通常在出生后数小时内出现。SCPCD患者的治疗选择包括使用血浆源性蛋白C浓缩物进行替代治疗。在本次针对性文献综述中，我们总结了关于使用蛋白C浓缩物对SCPCD患者进行长期预防（治疗超过1周）的信息。该综述共纳入18篇出版物，其中15篇为病例研究。11篇出版物报道了使用蛋白C浓缩物（Ceprotin；武田公司旗下的百特美国公司；武田奥地利制造股份公司）进行治疗，2篇出版物报道了使用蛋白C浓缩物（Protexel；LFB生物制药公司）进行治疗。1篇出版物同时报道了Ceprotin和Protexel。11篇出版物报告了蛋白C浓缩物治疗方案的详细信息，包括剂量、给药频率和给药途径。所有11篇出版物中的给药方案各不相同，这可能是由于患者之间蛋白C谷值水平不同或同时使用了其他药物。11篇出版物中有7篇报道了患者最初接受静脉注射蛋白C浓缩物，随后改为皮下给药。蛋白C浓缩物的治疗效果总体良好，包括预防凝血病和血栓形成以及皮肤病变的愈合。1篇出版物中确定了3起不良事件可能与Ceprotin给药有关。尽管已发表的数据有限，但本综述为临床实践中SCPCD患者的治疗提供了有价值的见解，包括蛋白C浓缩物的给药方案、给药途径及相关临床结果。
