von Willebrand 病。

IF 76.9 1区 医学 Q1 MEDICINE, GENERAL & INTERNAL Nature Reviews Disease Primers Pub Date : 2024-07-25 DOI:10.1038/s41572-024-00536-8
Omid Seidizadeh, Jeroen C J Eikenboom, Cécile V Denis, Veronica H Flood, Paula James, Peter J Lenting, Luciano Baronciani, James S O'Donnell, David Lillicrap, Flora Peyvandi
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引用次数: 0

摘要

冯-威廉氏病(VWD)是最常见的遗传性出血性疾病。这种疾病的特点是皮肤黏膜出血过多。这种疾病最常见的出血表现包括鼻出血、瘀伤、小伤口出血、月经过多或妇女产后出血以及手术后出血。其他较少见的症状包括胃肠道出血、血肿或血肿。VWD 的病理生理学非常复杂,是由 von Willebrand 因子(VWF)糖蛋白缺陷引起的。1 型 VWD 和 3 型 VWD 的病因分别是 VWF 的部分减少和 VWF 的完全缺失。定性异常导致 2 型 VWD,又分为 2A、2B、2M 和 2N 型。虽然 VWD 很常见,但由于诊断复杂、出血症状多变、存在外部变量(血型和其他生理改变因素,如运动、甲状腺激素、雌激素和老化)以及非专业医疗保健提供者缺乏疾病意识等因素,VWD 存在误诊、过度诊断和诊断不足的风险。确定 VWD 的正确诊断需要一系列专门的表型检测和/或 VWF 基因的分子遗传检测。治疗出血的方法包括使用去氨加压素或输注外源性 VWF 浓缩物(血浆来源或重组)来提高内源性 VWF 水平。纤溶抑制剂、局部止血剂和激素疗法是有效的辅助措施。
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von Willebrand disease.

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.

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来源期刊
Nature Reviews Disease Primers
Nature Reviews Disease Primers Medicine-General Medicine
CiteScore
76.70
自引率
0.20%
发文量
75
期刊介绍: Nature Reviews Disease Primers, a part of the Nature Reviews journal portfolio, features sections on epidemiology, mechanisms, diagnosis, management, and patient quality of life. The editorial team commissions top researchers — comprising basic scientists and clinical researchers — to write the Primers, which are designed for use by early career researchers, medical students and principal investigators. Each Primer concludes with an Outlook section, highlighting future research directions. Covered medical specialties include Cardiology, Dermatology, Ear, Nose and Throat, Emergency Medicine, Endocrinology, Gastroenterology, Genetic Conditions, Gynaecology and Obstetrics, Hepatology, Haematology, Infectious Diseases, Maxillofacial and Oral Medicine, Nephrology, Neurology, Nutrition, Oncology, Ophthalmology, Orthopaedics, Psychiatry, Respiratory Medicine, Rheumatology, Sleep Medicine, and Urology.
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