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. 2025 Jan 7;15(1):e085142.
doi: 10.1136/bmjopen-2024-085142.

Developing an adaptive paediatric intensive care unit platform trial with key stakeholders: a qualitative study

Tracy Karen Mitchell  1 , Julie C Menzies  2 , Padmanabhan Ramnarayan  3 , Doug William Gould  4 , Elizabeth Deja  5 , Shelley Marsh  6 , Jennifer Ainsworth  7 , Jennifer Preston  7 , Hannah Sedgwick  4 , Carly Tibbins  8 , Paul R Mouncey  4 , Mark J Peters  9   10 , Kerry Woolfall  5
Affiliations

Developing an adaptive paediatric intensive care unit platform trial with key stakeholders: a qualitative study

Tracy Karen Mitchell et al. BMJ Open. .

Abstract

Objectives: 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.

Design: Qualitative semistructured focus group study. Data were analysed using reflexive thematic analysis.

Participants: Young people, parents/carers, and PICU medical, nursing and research staff.

Setting: The UK.

Results: 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 Child quality of life and Survival (all participants). Optimal governance structure and suggestions about how any challenges to the success of the full trial can be overcome are also presented.

Conclusions: 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.

Keywords: Adolescent; Clinical Trial; PAEDIATRICS; Paediatric intensive & critical care; QUALITATIVE RESEARCH; Randomized Controlled Trial.

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Conflict of interest statement

Competing interests: National Institute for Health and Care Research (NIHR) Health Technology Assessment (HTA) Programme grant payments to support the development and conduct of this study and dissemination of findings were made to the institutions of TKM, PR, DWG, ED, PRM, MJP and KW. JCM’s previous institution received NIHR HTA payments for her role. JCM is the unpaid Chair of the Nursing and Allied Health Professional Section of the European Society of Paediatric and Neonatal Intensive Care (ESPNIC). TKM reports that her employment role is and was funded by NIHR HTA Programme, Medical Research Council Better Methods and National Health and Medical Research Council Collaborative Research Grant payments made to her institution. She also has an unpaid steering committee consultation role for an NIHR Research for Patient Benefit (RfPB) study. MJP declares NIHR HTA, ESPRC CHIMERA Hub support and NIHR RfPB Clinical Trial support payments made to his institution. He is the Deputy Chair of the NIHR HTA General Funding Committee (payments made to his institution) and has an unpaid role as a Global Pediatric Sepsis Definitions Taskforce Member and as the European Vice-Chair Surviving Sepsis for Children. He receives personal payment for Expert Witness reports and testimony. SM, JA, JP, HS and CT declare that they have no conflict of interest.

Figures

Figure 1
Figure 1. Pre-defined suggestions for domains to include in the PICU-Platform trial. PICU, paediatric intensive care unit.
Figure 2
Figure 2. PICU platform trial governance structure proposed by PICU staff. PICU, paediatric intensive care unit.
See this image and copyright information in PMC

References

    1. Peters MJ, Ramnarayan P, Scholefield BR, et al. The United Kingdom Paediatric Critical Care Society Study Group: The 20-Year Journey Toward Pragmatic, Randomized Clinical Trials. Pediatr Crit Care Med. 2022;23:1067–75. doi: 10.1097/PCC.0000000000003099. - DOI - PubMed
    1. Duffett M, Choong K, Hartling L, et al. Randomized controlled trials in pediatric critical care: a scoping review. Crit Care. 2013;17:R256. doi: 10.1186/cc13083. - DOI - PMC - PubMed
    1. Woolfall K, Frith L, Gamble C, et al. How parents and practitioners experience research without prior consent (deferred consent) for emergency research involving children with life threatening conditions: a mixed method study. BMJ Open. 2015;5:e008522. doi: 10.1136/bmjopen-2015-008522. - DOI - PMC - PubMed
    1. Park JJH, Harari O, Dron L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:1–8. doi: 10.1016/j.jclinepi.2020.04.025. - DOI - PubMed
    1. Park JJH, Sharif B, Harari O, et al. Economic Evaluation of Cost and Time Required for a Platform Trial vs Conventional Trials. JAMA Netw Open. 2022;5:e2221140. doi: 10.1001/jamanetworkopen.2022.21140. - DOI - PMC - PubMed

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