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转化医学肾脏病学:什么是转化医学研究及肾脏病转化医学研究概述。

Translational nephrology: what translational research is and a bird's-eye view on translational research in nephrology.

机构信息

IIS-Fundacion Jimenez Diaz, School of Medicine , Universidad Autonoma de Madrid , Madrid , Spain ; Fundacion Renal Iñigo Alvarez de Toledo-IRSIN and REDINREN , Madrid , Spain.

出版信息

Clin Kidney J. 2015 Feb;8(1):14-22. doi: 10.1093/ckj/sfu142. Epub 2015 Jan 28.

Abstract

The ultimate aim of biomedical research is to preserve health and improve patient outcomes. However, by a variety of measures, preservation of kidney health and patient outcomes in kidney disease are suboptimal. Severe acute kidney injury has been treated solely by renal replacement therapy for over 50 years and mortality still hovers at around 50%. Worldwide deaths from chronic kidney disease (CKD) increased by 80% in 20 years--one of the greatest increases among major causes of death. This dramatic data concur with huge advances in the cellular and molecular pathophysiology of kidney disease and its consequences. The gap appears to be the result of sequential roadblocks that impede an adequate flow from basic research to clinical development [translational research type 1 (T1), bench-to-bed and back] and from clinical development to clinical practice and widespread implementation (translational research T2) that supported by healthcare policy-making reaches all levels of society throughout the globe (sometimes called translational research T3). Thus, it is more than 10 years since the introduction of the last new-concept drug for CKD patients, cinacalcet; and 30 years since the introduction of reninangiotensin system (RAS) blockade, the current mainstay to prevent progression of CKD, illustrating the basic science-clinical practice disconnect. Roadblocks from clinical advances to widespread implementation, together with lag time-to-benefit may underlie the 20 years since the description of the antiproteinuric effect of RAS blockade to the observation of decreased age-adjusted incidence of endstage renal disease due to diabetic kidney disease. Only a correct understanding of the roadblocks in translational medicine and a full embracement of a translational research culture will spread the benefits of the biomedical revolution to its ultimate destinatary, the society.

摘要

生物医学研究的最终目标是维护健康和改善患者的预后。然而,通过各种措施,肾脏健康的维护和肾脏病患者的预后并不理想。严重的急性肾损伤已经通过肾脏替代治疗治疗了 50 多年,但死亡率仍徘徊在 50%左右。在 20 年内,全球因慢性肾脏病(CKD)导致的死亡人数增加了 80%,是主要死亡原因中增长最大的疾病之一。这一戏剧性的数据与肾脏病及其后果的细胞和分子病理生理学的巨大进展相符。这种差距似乎是由于一系列的障碍造成的,这些障碍阻碍了从基础研究到临床开发(转化研究类型 1(T1),从实验室到病床和再回来)的充分流动,以及从临床开发到临床实践和广泛实施(转化研究 T2)的充分流动,而这种流动是由医疗保健政策制定支持的,它可以到达全球社会的各个层面(有时称为转化研究 T3)。因此,自最后一种新型 CKD 药物西那卡塞问世以来,已经过去了 10 多年;自引入肾素血管紧张素系统(RAS)阻断剂以来,已经过去了 30 多年,这是预防 CKD 进展的当前主要方法,说明了基础科学与临床实践之间的脱节。从临床进展到广泛实施的障碍,以及获益的滞后时间,可能是自 RAS 阻断剂的蛋白尿效应描述以来的 20 年中,观察到因糖尿病肾病导致的终末期肾病的年龄调整发病率下降的原因。只有正确理解转化医学中的障碍,并充分接受转化研究文化,才能将生物医学革命的惠益传播给其最终目标——社会。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/36d8/4310441/f7977ddb17e4/sfu14201.jpg

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