评估急性川崎病冠状动脉炎症的新型治疗方法：阿托伐他汀的I/IIa期试验。
Evaluating a novel treatment for coronary artery inflammation in acute Kawasaki disease: A Phase I/IIa trial of atorvastatin.
作者信息
Tremoulet Adriana H, Jain Sonia, Burns Jane C
机构信息
Kawasaki Disease Research Center, Department of Pediatrics, University of California San Diego, La Jolla, California, USA; Rady Children's Hospital San Diego, San Diego, California, USA.
Biostatistics Research Center, Department of Family Medicine and Public Health, University of California San Diego, La Jolla, California.
出版信息
Expert Opin Orphan Drugs. 2015;3(9):967-970. doi: 10.1517/21678707.2015.1066246. Epub 2015 Jul 13.
INTRODUCTION
Since the 1980s, the primary treatment of acute Kawasaki disease (KD) has been intravenous immunoglobulin and aspirin. However, 5-10% of children with acute KD will develop coronary artery abnormalities despite treatment within the first ten days after fever onset. There is no approved adjunctive therapy to prevent progression of coronary artery damage in these patients.
AREAS COVERED
The rationale and study design of a Phase I/IIa trial of atorvastatin in children with acute KD and coronary artery inflammation is presented. The studies of host genetics and KD pathogenesis leading up to this trial are reviewed.
EXPERT OPINION
The repurposing of well-studied drugs used in the adult population is a cost-effective and efficient strategy to identify new therapies for pediatric diseases. Exploiting the anti-inflammatory, non-lipid-lowering effects of statins may open up new applications for this class of drugs for the pediatric age group.
引言
自20世纪80年代以来，急性川崎病（KD）的主要治疗方法是静脉注射免疫球蛋白和阿司匹林。然而，尽管在发热起病后的前十天内接受了治疗，但仍有5%-10%的急性KD患儿会出现冠状动脉异常。目前尚无批准的辅助治疗方法来预防这些患者冠状动脉损伤的进展。
涵盖领域
介绍了阿托伐他汀用于急性KD和冠状动脉炎症患儿的I/IIa期试验的基本原理和研究设计。回顾了在此试验之前关于宿主遗传学和KD发病机制的研究。
专家意见
将在成人中广泛研究的药物重新用于儿科疾病是一种经济高效的策略，可用于识别儿科疾病的新疗法。利用他汀类药物的抗炎、非降脂作用可能为这类药物在儿科年龄组中开辟新的应用领域。
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