Background: Precision medicine focuses on the identification of therapeutic strategies that are effective for a group of patients based on similar unifying characteristics. The recent success of precision medicine in non-critical care settings has resulted from the confluence of large clinical and biospecimen repositories, innovative bioinformatics, and novel trial designs. Similar advances for precision medicine in sepsis and in the acute respiratory distress syndrome (ARDS) are possible but will require further investigation and significant investment in infrastructure. Methods: This project was funded by the American Thoracic Society Board of Directors. A multidisciplinary and diverse working group reviewed the available literature, established a conceptual framework, and iteratively developed recommendations for the Precision Medicine Research Agenda for Sepsis and ARDS. Results: The following six priority recommendations were developed by the working group: 1) the creation of large richly phenotyped and harmonized knowledge networks of clinical, imaging, and multianalyte molecular data for sepsis and ARDS; 2) the implementation of novel trial designs, including adaptive designs, and embedding trial procedures in the electronic health record; 3) continued innovation in the data science and engineering methods required to identify heterogeneity of treatment effect; 4) further development of the tools necessary for the real-time application of precision medicine approaches; 5) work to ensure that precision medicine strategies are applicable and available to a broad range of patients varying across differing racial, ethnic, socioeconomic, and demographic groups; and 6) the securement and maintenance of adequate and sustainable funding for precision medicine efforts. Conclusions: Precision medicine approaches that incorporate variability in genomic, biologic, and environmental factors may provide a path forward for better individualizing the delivery of therapies and improving care for patients with sepsis and ARDS.

中文翻译：

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败血症和急性呼吸窘迫综合征精准医学的研究议程：美国胸科学会官方研究声明。


背景：精准医学侧重于根据相似的统一特征确定对一组患者有效的治疗策略。精准医学最近在非重症监护环境中的成功得益于大型临床和生物样本库、创新的生物信息学和新颖的试验设计的融合。脓毒症和急性呼吸窘迫综合征（ARDS）的精准医疗也可能取得类似的进展，但需要进一步的研究和基础设施方面的大量投资。方法：该项目由美国胸科学会董事会资助。一个多学科、多元化的工作组审查了现有文献，建立了概念框架，并迭代制定了脓毒症和急性呼吸窘迫综合征精准医学研究议程的建议。结果：工作组提出了以下六项优先建议：1）创建脓毒症和急性呼吸窘迫综合征的临床、影像和多分析物分子数据的大型丰富表型和统一知识网络； 2）实施新颖的试验设计，包括适应性设计，并将试验程序嵌入电子健康记录中； 3）持续创新识别治疗效果异质性所需的数据科学和工程方法； 4）进一步开发实时应用精准医疗方法所需的工具； 5) 努力确保精准医疗策略适用于不同种族、民族、社会经济和人口群体的广大患者； 6) 为精准医疗工作确保和维持充足且可持续的资金。 结论：结合基因组、生物和环境因素变异性的精准医学方法可能为更好地个性化治疗和改善脓毒症和急性呼吸窘迫综合征患者的护理提供一条前进之路。