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确定成人肺部与重症监护领域的临床研究重点。美国国立心肺血液研究所工作组报告。

Identifying Clinical Research Priorities in Adult Pulmonary and Critical Care. NHLBI Working Group Report.

作者信息

Semler Matthew W, Bernard Gordon R, Aaron Shawn D, Angus Derek C, Biros Michelle H, Brower Roy G, Calfee Carolyn S, Colantuoni Elizabeth A, Ferguson Niall D, Gong Michelle N, Hopkins Ramona O, Hough Catherine L, Iwashyna Theodore J, Levy Bruce D, Martin Thomas R, Matthay Michael A, Mizgerd Joseph P, Moss Marc, Needham Dale M, Self Wesley H, Seymour Christopher W, Stapleton Renee D, Thompson B Taylor, Wunderink Richard G, Aggarwal Neil R, Reineck Lora A

机构信息

Division of Allergy, Pulmonary and Critical Care Medicine and.

Division of Respirology, University of Ottawa, Ottawa, Ontario, Canada.

出版信息

Am J Respir Crit Care Med. 2020 Aug 15;202(4):511-523. doi: 10.1164/rccm.201908-1595WS.

Abstract

Preventing, treating, and promoting recovery from critical illness due to pulmonary disease are foundational goals of the critical care community and the NHLBI. Decades of clinical research in acute respiratory distress syndrome, acute respiratory failure, pneumonia, and sepsis have yielded improvements in supportive care, which have translated into improved patient outcomes. Novel therapeutics have largely failed to translate from promising preclinical findings into improved patient outcomes in late-phase clinical trials. Recent advances in personalized medicine, "big data," causal inference using observational data, novel clinical trial designs, preclinical disease modeling, and understanding of recovery from acute illness promise to transform the methods of pulmonary and critical care clinical research. To assess the current state of, research priorities for, and future directions in adult pulmonary and critical care research, the NHLBI assembled a multidisciplinary working group of investigators. This working group identified recommendations for future research, including ) focusing on understanding the clinical, physiological, and biological underpinnings of heterogeneity in syndromes, diseases, and treatment response with the goal of developing targeted, personalized interventions; ) optimizing preclinical models by incorporating comorbidities, cointerventions, and organ support; ) developing and applying novel clinical trial designs; and ) advancing mechanistic understanding of injury and recovery to develop and test interventions targeted at achieving long-term improvements in the lives of patients and families. Specific areas of research are highlighted as especially promising for making advances in pneumonia, acute hypoxemic respiratory failure, and acute respiratory distress syndrome.

摘要

预防、治疗因肺部疾病导致的危重症并促进康复是重症监护领域和美国国立心肺血液研究所(NHLBI)的基本目标。数十年来,针对急性呼吸窘迫综合征、急性呼吸衰竭、肺炎和脓毒症的临床研究已在支持治疗方面取得进展,这些进展已转化为患者预后的改善。新型疗法在很大程度上未能将有前景的临床前研究结果转化为后期临床试验中患者预后的改善。精准医学、“大数据”、利用观察性数据进行因果推断、新型临床试验设计、临床前疾病建模以及对急性疾病康复的理解等方面的最新进展有望改变肺部和重症监护临床研究的方法。为评估成人肺部和重症监护研究的现状、研究重点及未来方向,NHLBI组建了一个多学科研究人员工作组。该工作组确定了未来研究的建议,包括:)着重理解综合征、疾病及治疗反应异质性的临床、生理和生物学基础,目标是开发有针对性的个性化干预措施;)通过纳入合并症、联合干预措施和器官支持来优化临床前模型;)开发并应用新型临床试验设计;)深化对损伤和康复机制的理解,以开发和测试旨在实现患者及其家庭生活长期改善的干预措施。特别指出,在肺炎、急性低氧性呼吸衰竭和急性呼吸窘迫综合征方面取得进展的特定研究领域尤其具有前景。

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