Translational Stroke Research: Vision and Opportunities.
Translational Stroke Research: Vision and Opportunities.
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DOI：
10.1161/strokeaha.117.017112
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发表时间：
2017-09
期刊：
影响因子：
8.3
通讯作者：
Koroshetz W
中科院分区：
文献类型：
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作者：
Bosetti F;Koenig JI;Ayata C;Back SA;Becker K;Broderick JP;Carmichael ST;Cho S;Cipolla MJ;Corbett D;Corriveau RA;Cramer SC;Ferguson AR;Finklestein SP;Ford BD;Furie KL;Hemmen TM;Iadecola C;Jakeman LB;Janis S;Jauch EC;Johnston KC;Kochanek PM;Kohn H;Lo EH;Lyden PD;Mallard C;McCullough LD;McGavern LM;Meschia JF;Moy CS;Perez-Pinzon MA;Ramadan I;Savitz SI;Schwamm LH;Steinberg GK;Stenzel-Poore MP;Tymianski M;Warach S;Wechsler LR;Zhang JH;Koroshetz W
Bosetti等NINDS研讨会建议2633学术界，工业界，食品和药物管理局，以及其他公共和私人资助机构。在本研讨会的背景下，翻译是指将一种有前途的治疗药物沿着药物开发管道推进所必需的研究。临床前转化研究需要根据其是探索性还是确证性进行不同的设计。探索性方法可能侧重于疾病机制，并使用更简单的啮齿动物模型来获得关于假定治疗靶点的严格信息。然而，假定疗法的后期验证性研究将受益于临床试验中采用的相同设计特征，包括异质性人群、足够的样本量、预测临床结局的终点、与治疗用途相似的给药途径以及研究结果的报告，包括阴性结果。这份特别报告概述了研讨会参与者的讨论和建议，以帮助推进转化性卒中研究，总结在表中。
Bosetti et al NINDS Workshop Recommendations 2633 academia, industry, the Food and Drug Administration, and other public and private funding agencies. In the context of this workshop, translation refers to the research necessary to move a promising therapeutic along the drug development pipeline. Preclinical translational studies need to be designed differently depending on whether they are exploratory or confirmatory. Exploratory approaches may focus on disease mechanisms and use simpler rodent models to gain rigorous information on a putative therapeutic target. However, later-stage confirmatory studies of putative therapies would benefit from the same design features adopted in clinical trials, including heterogeneous populations, adequate sample size, end points predictive of clinical outcomes, and routes of administration similar to therapeutic use, as well as reporting of study results, including negative findings. This special report outlines the discussions and recommendations developed by the workshop participants to help advance translational stroke research, which are summarized in the Table.