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血管性血友病。

von Willebrand disease.

机构信息

Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands.

出版信息

Nat Rev Dis Primers. 2024 Jul 25;10(1):51. doi: 10.1038/s41572-024-00536-8.

Abstract

von Willebrand disease (VWD) is the most common inherited bleeding disorder. The disorder is characterized by excessive mucocutaneous bleeding. The most common bleeding manifestations of this condition include nosebleeds, bruising, bleeding from minor wounds, menorrhagia or postpartum bleeding in women as well as bleeding after surgery. Other less frequent symptoms include gastrointestinal bleeding, haematomas or haemarthroses. VWD pathophysiology is complex and results from defects in von Willebrand factor (VWF) glycoprotein. Quantitative deficiencies are responsible for type 1 VWD with a partial decrease of VWF and type 3 with the complete absence of VWF. Qualitative abnormalities cause type 2 VWD, being further divided into types 2A, 2B, 2M and 2N. Although common, VWD is at risk of misdiagnosis, overdiagnosis and underdiagnosis owing to several factors, including complex diagnosis, variability of bleeding symptoms, presence of external variables (blood groups and other physiological modifiers such as exercise, thyroid hormones, oestrogens, and ageing), and lack of disease awareness among non-specialist health-care providers. Establishing the correct VWD diagnosis requires an array of specialized phenotypic assays and/or molecular genetic testing of the VWF gene. The management of bleeding includes increasing endogenous VWF levels with desmopressin or infusion of exogenous VWF concentrates (plasma-derived or recombinant). Fibrinolytic inhibitors, topical haemostatic agents and hormonal therapies are used as effective adjunctive measures.

摘要

血管性血友病(VWD)是最常见的遗传性出血性疾病。该疾病的特征是黏膜皮肤过度出血。这种情况下最常见的出血表现包括流鼻血、瘀伤、轻微创伤出血、月经过多或产后出血以及手术后出血。其他不太常见的症状包括胃肠道出血、血肿或关节积血。VWD 的病理生理学较为复杂,是由于血管性血友病因子(VWF)糖蛋白的缺陷所致。数量缺陷导致 1 型 VWD,VWF 部分减少,3 型 VWD 则完全缺乏 VWF。质量异常导致 2 型 VWD,进一步分为 2A、2B、2M 和 2N 型。尽管较为常见,但由于多种因素,VWD 存在误诊、过诊和漏诊的风险,包括复杂的诊断、出血症状的可变性、外部变量(血型和其他生理调节剂,如运动、甲状腺激素、雌激素和衰老)的存在,以及非专科医疗保健提供者对该疾病缺乏认识。正确诊断 VWD 需要一系列专门的表型检测和/或 VWF 基因的分子遗传学检测。出血的治疗包括使用去氨加压素增加内源性 VWF 水平或输注外源性 VWF 浓缩物(血浆衍生或重组)。纤维蛋白溶解抑制剂、局部止血剂和激素治疗可作为有效的辅助措施。

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