Division of Anaesthetics, Pain Medicine and Intensive Care and.
Division of Pulmonary, Critical Care, Allergy, and Sleep Medicine, Department of Medicine, University of California, San Francisco, San Francisco, California.
Am J Respir Crit Care Med. 2024 Jul 15;210(2):155-166. doi: 10.1164/rccm.202311-2086SO.
Critical care uses syndromic definitions to describe patient groups for clinical practice and research. There is growing recognition that a "precision medicine" approach is required and that integrated biologic and physiologic data identify reproducible subpopulations that may respond differently to treatment. This article reviews the current state of the field and considers how to successfully transition to a precision medicine approach. To impact clinical care, identification of subpopulations must do more than differentiate prognosis. It must differentiate response to treatment, ideally by defining subgroups with distinct functional or pathobiological mechanisms (endotypes). There are now multiple examples of reproducible subpopulations of sepsis, acute respiratory distress syndrome, and acute kidney or brain injury described using clinical, physiological, and/or biological data. Many of these subpopulations have demonstrated the potential to define differential treatment response, largely in retrospective studies, and that the same treatment-responsive subpopulations may cross multiple clinical syndromes (treatable traits). To bring about a change in clinical practice, a precision medicine approach must be evaluated in prospective clinical studies requiring novel adaptive trial designs. Several such studies are underway, but there are multiple challenges to be tackled. Such subpopulations must be readily identifiable and be applicable to all critically ill populations around the world. Subdividing clinical syndromes into subpopulations will require large patient numbers. Global collaboration of investigators, clinicians, industry, and patients over many years will therefore be required to transition to a precision medicine approach and ultimately realize treatment advances seen in other medical fields.
重症监护使用综合征定义来描述患者群体，以便于临床实践和研究。人们越来越认识到需要采用“精准医学”方法，整合生物和生理数据可识别出可重复出现的亚群，这些亚群可能对治疗有不同的反应。本文综述了该领域的现状，并探讨了如何成功过渡到精准医学方法。为了影响临床护理，亚群的识别不仅要区分预后，还必须区分治疗反应，理想情况下，通过定义具有不同功能或病理生物学机制（表型）的亚组来实现。现在有多个使用临床、生理和/或生物学数据描述的脓毒症、急性呼吸窘迫综合征和急性肾或脑损伤的可重复出现的亚群的例子。其中许多亚群已经证明具有定义不同治疗反应的潜力，主要是在回顾性研究中，而且相同的治疗反应亚群可能跨越多个临床综合征（可治疗特征）。为了改变临床实践，必须在需要新的适应性试验设计的前瞻性临床研究中评估精准医学方法。目前正在进行几项这样的研究，但仍有许多挑战需要解决。这些亚群必须易于识别，并适用于全球所有重症患者群体。将临床综合征细分为亚群需要大量患者。因此，需要经过多年的全球调查人员、临床医生、行业和患者的合作，才能过渡到精准医学方法，并最终实现其他医学领域看到的治疗进展。
