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重度纯合子蛋白C缺乏症管理中的挑战:一例报告

Challenges in managing severe homozygous protein c deficiency: a case report.

作者信息

Almatter Eman, Alsharidah Sondus, Bourhama Mona, Bourusly Maha, Obaid Mohamad Adel

机构信息

Department of Hematology, NBK Specialized Children's Hospital, Kuwait, Kuwait.

出版信息

Blood Coagul Fibrinolysis. 2025 Jan 1;36(1):34-36. doi: 10.1097/MBC.0000000000001332. Epub 2024 Nov 4.

Abstract

Protein C deficiency is a rare autosomal recessive disorder associated with a high risk of thromboembolic complications. This case report describes the challenges in managing a 23-year-old woman with severe homozygous protein C type 1 deficiency diagnosed since early infancy. Her medical history included misdiagnosed cellulitis, recurrent thrombosis, and permanent vision loss in one eye. The laboratory workup confirmed a diagnosis of severe protein C deficiency. Management involved a combination of fresh frozen plasma (FFP), protein C concentrate, warfarin, and heparin, with ongoing challenges due to recurrent thrombosis and anaphylaxis to FFP. This case highlights the challenges in the diagnosis and management of severe protein C deficiency. Although current treatment options provide partial control, further research is crucial to develop safer and more effective therapies to improve long-term outcomes for affected patients.

摘要

蛋白C缺乏症是一种罕见的常染色体隐性疾病,与血栓栓塞并发症的高风险相关。本病例报告描述了管理一名自婴儿早期就被诊断为严重纯合子1型蛋白C缺乏症的23岁女性时所面临的挑战。她的病史包括误诊的蜂窝织炎、复发性血栓形成以及一只眼睛的永久性视力丧失。实验室检查确诊为严重蛋白C缺乏症。治疗包括新鲜冰冻血浆(FFP)、蛋白C浓缩物、华法林和肝素的联合应用,但由于复发性血栓形成和对FFP的过敏反应,治疗仍面临持续挑战。本病例突出了严重蛋白C缺乏症诊断和管理中的挑战。尽管目前的治疗选择能提供部分控制,但进一步研究对于开发更安全、更有效的疗法以改善受影响患者的长期预后至关重要。

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