Mitchell Tracy Karen, Menzies Julie C, Ramnarayan Padmanabhan, Gould Doug William, Deja Elizabeth, Marsh Shelley, Ainsworth Jennifer, Preston Jennifer, Sedgwick Hannah, Tibbins Carly, Mouncey Paul R, Peters Mark J, Woolfall Kerry
Public Health, Policy and Systems, University of Liverpool, Liverpool, UK
Paediatric Intensive Care Unit, Bristol Royal Hospital for Children, Bristol, UK.
BMJ Open. 2025 Jan 7;15(1):e085142. doi: 10.1136/bmjopen-2024-085142.
Platform trials were used successfully in adult populations during the COVID-19 pandemic. By testing multiple treatments within a single trial, platform trials can help identify the most effective treatments (and any interactions between treatments) for patients more quickly and with less burden for patients and their families. The aim of this qualitative research was to inform the design of the first adaptive platform trial for paediatric intensive care in the UK with young people, parents/carers and paediatric intensive care unit (PICU) staff.
Qualitative semistructured focus group study. Data were analysed using reflexive thematic analysis.
Young people, parents/carers, and PICU medical, nursing and research staff.
The UK.
A total of 86 participants (18 young people; 15 parents/carers; 53 PICU staff) took part in 1 of 10 focus groups between May and September 2023. Participants viewed the proposed PICU platform trial and use of research without prior consent to be acceptable. Findings provide insight into how the PICU platform trial should be designed and operationalised, including having a broad and inclusive population eligible for inclusion onto the platform trial, with different inclusion and exclusion criteria for each domain; starting the trial with no more than three domains and prioritising the outcomes of and (all participants). Optimal governance structure and suggestions about how any challenges to the success of the full trial can be overcome are also presented.
Young people, parents/carers and PICU staff viewed the proposed PICU platform trial to be acceptable. These key stakeholders supported us with the design of an adaptive platform trial for PICU that has a rigorous methodology, yet can be operationalised in a family-centred way, to provide high-quality evidence that can support clinical decision-making and guide the treatment of critically ill children. Our findings have informed the PICU platform trial protocol.
在新冠疫情期间，平台试验在成年人群体中得到了成功应用。通过在单一试验中测试多种治疗方法，平台试验能够更快速地为患者确定最有效的治疗方法（以及治疗之间的任何相互作用），同时减轻患者及其家庭的负担。这项定性研究的目的是为英国首个针对儿科重症监护的适应性平台试验的设计提供参考，该试验涉及年轻人、父母/照顾者以及儿科重症监护病房（PICU）工作人员。
定性半结构化焦点小组研究。采用反思性主题分析法对数据进行分析。
年轻人、父母/照顾者以及PICU的医疗、护理和研究人员。
英国。
2023年5月至9月期间，共有86名参与者（18名年轻人；15名父母/照顾者；53名PICU工作人员）参加了10个焦点小组中的1个。参与者认为拟议的PICU平台试验以及未经事先同意使用研究方法是可以接受的。研究结果为PICU平台试验应如何设计和实施提供了见解，包括拥有广泛且包容性强的人群有资格纳入平台试验，每个领域有不同的纳入和排除标准；试验开始时不超过三个领域，并优先考虑所有参与者的结果。还提出了最佳治理结构以及关于如何克服全面试验成功面临的任何挑战的建议。
年轻人、父母/照顾者和PICU工作人员认为拟议的PICU平台试验是可以接受的。这些关键利益相关者支持我们设计一个针对PICU的适应性平台试验，该试验具有严谨的方法，但可以以家庭为中心的方式实施，以提供高质量的证据来支持临床决策并指导危重症儿童的治疗。我们的研究结果为PICU平台试验方案提供了参考。
