3 December 2024
Over £6 million in funding has been awarded to an adaptive platform trial in paediatric intensive care, focusing on multiple research areas prioritised by young people and parents.
Funded by the National Institute for Health and Care Research through the Health Technology Assessment programme, the trial will involve intensive care centres across the country including Royal Brompton Hospital, and will be led by University College London Great Ormond Street Institute of Child Health.
Current state of paediatric intensive care research
Randomised controlled trials (RCT) are trials where a number of similar people are randomly assigned to two (or more) groups to test a specific treatment. One group is given the intervention being tested, and the other group is given an alternative intervention, a dummy intervention (placebo) or no intervention at all.
RCTs provide the best evidence for the best treatments but the way these trials are undertaken can be slow and expensive.
In addition to this, very few patients in PICU are recruited onto clinical trials when compared to adult critical care. This means it can take several years for enough children to be included in a study before enough information is available to tell which treatment is safer.
Consequently, there is a lack of evidence in many aspects of paediatric intensive care resulting in differences in how common interventions such as ventilation, cardiovascular support, sedation, transfusion, and fluids are managed across the country.
What is a randomised adaptive platform trial?
A randomised adaptive platform allows for multiple research questions and treatments to be tested at the same time. This way of doing studies can answer more questions, more efficiently in less time.
The trial can be ‘adapted’ or changed in design as it goes along, such as when new treatments or important research questions become available.
Adaptive platform trials also involve results being looked at as the trial is going on (not just at the end) which means treatments which are working better in the trial can be given to more patients, and less safe or less effective treatments are stopped sooner.
Prioritising patients and parents
Ahead of the funding application the research team spoke extensively with parents, young people and clinicians to determine the areas of research they felt should be prioritised.
As a result, this trial will focus on three important areas for critically ill children:
- Determining the best sedative agent for those requiring invasive mechanical ventilation
- Whether avoiding patients accumulating too much fluid in the body improve outcomes
- Whether using fewer blood transfusions will improve outcomes
Dr Angela Aramburo, consultant paediatric intensivist at Royal Brompton and Harefield hospitals, will be co-leading part of the trial. She spoke about the importance of this work and what it could mean for patients and parents.
She said: “We are thrilled to embark on this groundbreaking journey with the first platform trial in paediatric critical care worldwide. This unique opportunity will enable us to generate new, invaluable knowledge on the most effective treatment interventions for critically ill children through a more efficient trial design.”
To find out more about our research, please contact us.
Read more research stories or sign up to the research newsletter.