University of California at San Diego, La Jolla, and Rady Children's Hospital, San Diego, California.
National Reference Center for Autoinflammatory Diseases, Bicêtre Hospital, AP-HP, University of Paris Sud, Paris, France.
Arthritis Rheumatol. 2017 Feb;69(2):268-276. doi: 10.1002/art.39975.
The decision to move forward with three clinical trials of IL-1 blockade for treatment of acute Kawasaki disease is a case study in translational science. These trials were born on the one hand from transcriptome studies of host response during the acute disease coupled with animal model investigations of key immune signaling pathways and, on the other hand, out of clinical desperation to intervene in patients with severe inflammation in the setting of acute Kawasaki disease. The convergence of laboratory science and clinical observations led to the clinical trials described here and serves as a model for how such observations can be translated into new therapies.
推进三项针对川崎病急性发作的白细胞介素-1 阻断治疗临床试验的决定是转化科学的一个案例研究。这些试验一方面源于急性疾病期间宿主反应的转录组研究,并结合关键免疫信号通路的动物模型研究,另一方面源于对急性川崎病严重炎症患者进行干预的临床需求。实验室科学和临床观察的融合促成了这里描述的临床试验,为如何将这些观察转化为新疗法提供了模式。