Millions in new funding for revolutionary paediatric intensive care trial

Researchers at Great Ormond Street Hospital (GOSH) have received more than £6m in funding from the National Institute for Health and Care Research (NIHR) to lead a revolutionary study to find the best treatments for critically ill children in paediatric intensive care units (PICUs) across the country.

The Paediatric Intensive Care Adaptive Platform Trial (PIVOTAL) will test multiple different research questions and treatments at the same time to identify the best care for children admitted to PICUs as quickly and efficiently as possible with the Intensive Care National Audit and Research Centre (ICNARC) clinical trials unit and Paediatric Critical Care Society Study Group (PCCS-SG).

The novel trial design is an alternative way of conducting research. The study can ‘adapt’ or change throughout its duration to include important research questions or new treatments as they become available.

The results will also be analysed as the trial is ongoing, whereas in more traditional methods results are analysed at the end. Therefore, if a treatment is working well for a particular group of patients, it can be expanded to more patients, and less effective treatments can be stopped sooner.

The trial will be rolled out to around 20 PICUs across the UK who are part of the PCCS-SG research network. To start with, researchers aim to address three key research questions:

1. What is the best sedative agent for use in critically ill children requiring invasive mechanical ventilation?
2. Does a plan designed to avoid having too much fluid in the body improve outcomes for critically ill children?
3. Does using blood transfusions less often improve outcomes for critically ill children?

Professor Mark Peters, consultant paediatric intensivist at GOSH and professor of paediatric intensive care at UCL Great Ormond St Institute of Child Health, and lead for the trial, said: “We’re thrilled to have received this funding from the NIHR which we hope will improve the care to some of the sickest children across the country. It currently takes several years to determine which treatments are safest to use in children, and we expect this novel trial design will provide more of these answers more quickly. While children on intensive care are our focus, if the study delivers as planned, it would support the uptake of this more efficient trial design across all of clinical research.”

This trial follows on from results from the Oxy-PICU study led by GOSH. The landmark study found that reducing oxygen levels for critically ill children on mechanical ventilators in intensive care could save tens of young lives each year.

Every child admitted to the PICU at GOSH is screened for inclusion into a clinical trial and to be part of research. The intensive care research team is physically based on the intensive care unit, allowing research best practice and training to inform and improve a child’s care at any time, day or night. Under the expert care of often nurse–led teams, we are changing paediatric intensive care practice.

Sarah Benkenstein, senior critical care research nurse at GOSH, said: “As critical care research nurses, we play a vital role in connecting clinical practice with scientific innovation. In the PIVOTAL trial, our responsibilities go beyond ensuring precise data collection and following protocols. We are dedicated to supporting trial participants and their families, placing their wellbeing at the forefront. Our efforts significantly influence the trial’s success, contributing to evidence-based progress in paediatric critical care medicine. We are very eager to launch this groundbreaking trial.”

All research at GOSH is supported by the NIHR GOSH Biomedical Research Centre.