1型血管性血友病和轻度甲型血友病患者在15μg封顶剂量下对去氨加压素的反应性。

Desmopressin responsiveness at a capped dose of 15 μg in type 1 von Willebrand disease and mild hemophilia A.

作者信息

Siew Dou-Anne, Mangel Joy, Laudenbach Lori, Schembri Sheila, Minuk Leonard

机构信息

Bleeding Disorders Program, University of Western Ontario, London, Ontario, Canada.

出版信息

Blood Coagul Fibrinolysis. 2014 Dec;25(8):820-3. doi: 10.1097/MBC.0000000000000158.

DOI:10.1097/MBC.0000000000000158

PMID:24911459

Abstract

Desmopressin (DDAVP) is commonly used in the treatment of patients with type 1 von Willebrand disease (VWD) and mild hemophilia A. A patient's responsiveness to DDAVP based on a 0.3 μg/kg dose determines future therapeutic efficacy of the drug. The aim of the study was to determine whether a capped dose of 15 μg subcutaneous DDAVP is able to achieve the same level of DDAVP responsiveness as previously reported. This is a retrospective chart review of patients from 1995 to 2013 in adults and children with type 1 VWD and hemophilia A weighing more than 50 kg. Levels of factor VIII, ristocetin cofactor, and von Willebrand factor antigen were measured before and after 1 h of administration of 15 μg of DDAVP. In patients with type 1 VWD, the complete response rate was 82.5% with a partial response rate of 12.5% and 5% nonresponders. In patients with mild hemophilia A, the complete response rate was 53.8% with a partial response rate of 38.5% and 7.7% nonresponders. These results using a capped 15-μg dose of DDAVP are similar to previously published reports using the 0.3-μg/kg dose.

摘要

去氨加压素（DDAVP）常用于治疗1型血管性血友病（VWD）患者和轻度甲型血友病患者。基于0.3μg/kg剂量的患者对DDAVP的反应性决定了该药物未来的治疗效果。本研究的目的是确定皮下注射15μg的固定剂量DDAVP是否能够达到与先前报道相同水平的DDAVP反应性。这是一项对1995年至2013年体重超过50kg的1型VWD和甲型血友病成人及儿童患者的回顾性病历审查。在给予15μg DDAVP 1小时前后测量因子VIII、瑞斯托霉素辅因子和血管性血友病因子抗原水平。在1型VWD患者中，完全缓解率为82.5%，部分缓解率为12.5%，无反应者为5%。在轻度甲型血友病患者中，完全缓解率为53.8%，部分缓解率为38.5%，无反应者为7.7%。使用15μg固定剂量DDAVP的这些结果与先前发表的使用0.3μg/kg剂量的报告相似。