The innovative trial, known as PIVOTAL, is being conducted across 24 paediatric intensive care units (PICUs) and is funded by the National Institute for Health and Care Research (NIHR). It aims to address the urgent need for better evidence on the most effective life-saving interventions for the approximately 20,000 children admitted to PICUs annually, of whom 4% do not survive and 10-15% suffer long-term consequences.
Utilising a 'platform' trial approach allows for multiple research questions and treatments to be evaluated concurrently, adapting as it progresses. This method is more efficient than traditional randomised controlled trials, which can take up to ten years for a single treatment. Results are assessed during the trial, enabling effective treatments to be given to more patients while less effective ones are stopped sooner.
Initially, the trial will investigate the best sedative for ventilated children, the effectiveness of a conservative fluid management approach, and whether using blood transfusions less frequently improves outcomes. For families, participation is designed to be seamless, with children automatically enrolled upon PICU admission and families later asked for consent to include their child's data.
Within its first 48 hours, the trial enrolled 10 patients, and over a thousand have since joined across the country. One such patient was five-week-old Freddie, who was enrolled on all three arms of the trial while on the Cardiac Intensive Care Unit. His mother, Beth, stated that the nurses explained the process would not differ from standard care but would help gather vital information for future patients.
Professor Mark Peters, Professor of Paediatric Intensive Care at UCL GOS Institute of Child Health, highlighted the platform approach's ability to trial multiple interventions at once for some of the UK's sickest children, aiming to find small changes that can save more lives and reduce long-term side effects.
Kate Plant, Senior Critical Care Research Nurse at GOSH, described the PIVOTAL trial's development as a privilege, emphasising the cautious approach taken when discussing research with vulnerable families. She noted that the platform design allows for multiple questions to be answered from a single family consent, making research an essential part of intensive care.
Professor Anthony Gordon, Director for the NIHR Health Technology Assessment (HTA) Programme, commented that trials like PIVOTAL build on existing knowledge and help PICU teams determine the best treatments for critically ill children, underscoring the vital importance of investing in NHS research. The NIHR is celebrating its 20th anniversary this year, having funded numerous innovative treatments.
