Pragmatic trials for critical illness in neonates and children.
Pragmatic trials for critical illness in neonates and children.
复制标题
新生儿和儿童危重疾病的实用试验。
DOI：
10.1016/s2352-4642(22)00345-5
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Schlapbach LJ
中科院分区：
文献类型：
--
作者：
Schlapbach LJ
在英国，每年有超过10万名婴儿和儿童住进新生儿和儿科重症监护病房。感染和败血症仍然是该患者组入院、短期和长期发病率和死亡率的主要原因。1,2然而，在这种情况下，大多数诊断和治疗实践并不是基于高级别证据。这使儿童面临次优决策和无效甚至有害的干预。这种证据缺失对患者、家庭和社会造成的损失尚不清楚。这仅仅是由于进行临床试验的固有挑战（成本、时间和受过适当培训的工作人员的可用性），还是由于特定的重症监护环境（时间敏感的干预措施、安全问题、工作人员和家长的可接受性，以及急性压力下同意的复杂性）？令人鼓舞的是，随着新生儿和儿科重症监护随机对照试验的数量在过去十年中显著增加，这一差距可能开始缩小（见PICUtrials数据库）。虽然招募患有危重疾病的儿童进行随机对照试验可能还不是一种标准的护理，但延迟同意选项的可用性、英国国家卫生与保健研究（NIHR）卫生技术评估（HTA）项目资助的重点是现实世界的有效性、研究网络的创建以及电子健康记录的获取都促进了这一发展。3,4最重要的是推动实用试验，旨在解决临床实践中的日常不确定性，解决常见问题，并通过具有更大通用性和可行性的设计评估简单的干预措施。随机对照试验设计的核心是在临床环境中比较两种或两种以上干预措施的效果，这就禁止了完全控制的实验室实验设置。解释性试验试图通过高度协议化的方法来证明有效性，以减少信号噪声，确保在精心挑选的患者中最佳地提供干预措施。
More than 100 000 infants and children are admitted to neonatal and paediatric intensive care units in the UK annually. Infections and sepsis remain a leading cause of admission, short-term and long-term morbidity, and mortality in this patient group. 1, 2 Yet, the majority of diagnostic and therapeutic practices in this setting are not based on high-grade evidence. This exposes children to suboptimal decision making and ineffective or even harmful interventions. The cost to patients, families, and society of this evidence deficit is unknown. Is this simply attributable to the innate challenges of performing clinical trials (cost, time, and availability of appropriately trained staff) or rather to the specific critical care setting (time-sensitive interventions, safety concerns, acceptability to staff and parents, and the complexity of consent under acute stress)? Encouragingly, the gap might be starting to close, as the number of neonatal and paediatric critical care randomised controlled trials has increased considerably over the past decade (see the PICUtrials database).Although recruiting children with critical illness into randomised controlled trials might not yet be a standard of care, the availability of deferred consent options, the UK National Health and Care Research (NIHR) Health Technology Assessment (HTA) programme funding focus on real-world effectiveness, the creation of research networks, and access to electronic health records have contributed to this development. 3, 4 Of key importance was the push for pragmatic trials, aiming to resolve dayto-day uncertainties in clinical practice, address common questions, and assess simple interventions through a design promising greater generalisability and feasibility. 5 At the heart of the design of randomised controlled trials lies the intention to compare the performance of two or more interventions in a clinical setting, which prohibits a fully controlled laboratory experimental setup. Explanatory trials attempt to demonstrate efficacy by highly protocolised approaches in order to reduce signal noise, ensuring the intervention is optimally delivered among carefully selected patients.
影响因子：
36.4
作者：
Agyeman, Philipp K. A.;Schlapbach, Luregn J.;Berger, Christoph
通讯作者：
Berger, Christoph
影响因子：
36.4
作者：
Marshall, Andrew S. J.;Scrivens, Alexandra;NeoCLEAR Collaborative Group
通讯作者：
NeoCLEAR Collaborative Group