Opportunities for improved clinical trial designs in acute respiratory distress syndrome
Opportunities for improved clinical trial designs in acute respiratory distress syndrome
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DOI：
10.1016/s2213-2600(22)00294-6
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发表时间：
2022-09-01
影响因子：
76.2
通讯作者：
Matthay, Michael A.
中科院分区：
文献类型：
--
作者：
Wick, Katherine D.;Aggarwal, Neil R.;Matthay, Michael A.
急性呼吸窘迫综合征(ARDS)是一种常见的危重病综合征，发病率和死亡率均较高。目前还没有针对ARDS的有效药物治疗方法。目前对ARDS的定义是基于共同的临床特征，但没有涵盖临床危险因素、影像特征、生理学、发病时间和轨迹以及综合征生物学的异质性。在ARDS临床试验社区内，越来越多的人对设计临床试验以减少试验人群的异质性感兴趣。这一努力必须与正在进行的制定行业的、全球的ARDS定义的工作相平衡，这对试验设计具有重要影响。最终，这两个目标应该共存--设计适用于全球不同ARDS人群的试验，同时促进识别有针对性的特征的机会。在个人观点中，我们建议两个主要策略来改进未来的ARDS试验：开发新的方法来针对临床试验人群中的可治疗特征，以及改善ARDS试验的代表性，纳入全球人群。我们强调，这两个战略是相辅相成的。我们还讨论了扩大ARDS定义的提议如何影响临床试验的未来。
The acute respiratory distress syndrome (ARDS) is a common critical illness syndrome with high morbidity and mortality. There are no proven pharmacological therapies for ARDS. The current definition of ARDS is based on shared clinical characteristics but does not capture the heterogeneity in clinical risk factors, imaging characteristics, physiology, timing of onset and trajectory, and biology of the syndrome. There is increasing interest within the ARDS clinical trialist community to design clinical trials that reduce heterogeneity in the trial population. This effort must be balanced with ongoing work to craft an indusive, global definition of ARDS, with important implications for trial design. Ultimately, the two aims-to design trials that are applicable to the diverse global ARDS population while also advancing opportunities to identify targetable traits-should coexist. In this Personal View, we recommend two primary strategies to improve future ARDS trials: the development of new methods to target treatable traits in clinical trial populations, and improvements in the representativeness of ARDS trials, with the inclusion of global populations. We emphasise that these two strategies are complementary. We also discuss how a proposed expansion of the definition of ARDS could affect the future of clinical trials.