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去氨加压素在遗传性血小板功能障碍中的应用。

Desmopressin in inherited disorders of platelet function.

作者信息

Coppola A, Di Minno G

机构信息

Regional Reference Centre for Coagulation Disorders, Department of Clinical and Experimental Medicine, Federico II University of Naples, Naples, Italy.

出版信息

Haemophilia. 2008 Jan;14 Suppl 1:31-9. doi: 10.1111/j.1365-2516.2007.01607.x.

Abstract

Following the first clinical use in haemophilia and von Willebrand disease in 1977, the synthetic analogue of vasopressin 1-deamino-8-D-arginine vasopressin (DDAVP, desmopressin) was successfully employed for the management of a series of bleeding disorders, including congenital and acquired defects of platelet function. In this setting, few haemostatic approaches are available and, in particular for severe bleeding and major invasive procedures, the transfusion of platelet concentrates is the first-choice treatment. Therefore, DDAVP was (and remains) an attractive therapeutic alternative, being well tolerated, cost-saving, administrable at home (by the intranasal or subcutaneous concentrated formulations) and, in particular, enabling the avoidance of blood product exposition and the related risks (allergic reactions, transfusion transmitted infections). Despite three decades of clinical use, cellular mechanisms of haemostatic effects of DDAVP in platelet defects remain poorly known and the excellent results reported in some case series have not been strengthened by rigorous clinical trials, hampered by the rarity and the heterogeneity of these disorders. However, clinical experience more than evidence-based medicine reserved an established place to DDAVP in the management of inherited platelet disorders. This review will focus the available clinical data and the open issues of DDAVP in this setting.

摘要

1977年去氨加压素(DDAVP,即1-去氨基-8-D-精氨酸加压素)首次在血友病和血管性血友病的临床中应用后,这种加压素的合成类似物成功用于一系列出血性疾病的治疗,包括先天性和获得性血小板功能缺陷。在这种情况下,可用的止血方法很少,特别是对于严重出血和大型侵入性手术,输注血小板浓缩物是首选治疗方法。因此,DDAVP一直是一种有吸引力的治疗选择,耐受性良好、成本低、可在家中给药(通过鼻内或皮下浓缩制剂),尤其能够避免接触血液制品及其相关风险(过敏反应、输血传播感染)。尽管已经临床应用了三十年,但DDAVP在血小板缺陷中止血作用的细胞机制仍知之甚少,一些病例系列报告的出色结果尚未得到严格临床试验的证实,这些疾病的罕见性和异质性阻碍了相关研究。然而,临床经验而非循证医学在遗传性血小板疾病的治疗中为DDAVP保留了一席之地。本综述将聚焦于DDAVP在这种情况下的现有临床数据和未解决的问题。

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