Brain-related outcome measures in trials recruiting critically-ill children
Ericka L Fink1, Robert C Tasker2
1Departments of Critical Care Medicine & Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine Pittsburgh, Pennsylvania.
Insights
Research on pediatric critical care innovations is limited. This study proposes a new approach to brain-related outcomes to improve benefits for critically ill children and their families.
Area of Science:
- Pediatric Critical Care Medicine
- Neuroscience
- Clinical Trial Design
Background:
- Randomized controlled trials (RCTs) for acute, life-threatening pediatric illnesses are infrequent.
- A critical gap exists in research focusing on outcomes that matter to emergency decision-making and long-term well-being.
- Current research needs to better address brain-related outcomes in critically ill children.
Purpose of the Study:
- To develop an improved approach to assessing brain-related outcomes in pediatric critical care research.
- To maximize the benefit of research for children with acute life-threatening illnesses and their families.
- To guide future trial design by focusing on relevant and meaningful outcomes.
Main Methods:
- Analysis of 15 recent pediatric critical care trials.
- Categorization of brain-related outcome assessments used in these trials.
- Identification of key areas for improvement in future research.
Main Results:
- Four types of brain-related outcomes were identified: death/organ-system failures, neurological/neuropsychological, cognitive, and composite outcomes.
- Recent trials demonstrate varied approaches to assessing these outcomes.
- Specific examples include studies on systemic illness, glycemic control, sedation, and therapeutic hypothermia.
Conclusions:
- Future pediatric critical care trials require enhanced focus on specific areas.
- Essential components include neurologic monitoring, post-discharge assessments, improved follow-up retention, child/family-centered outcomes, and core outcome datasets.
- This approach aims to yield more relevant and beneficial research findings for critically ill children.
Purpose Of Review:
Randomized controlled trials leading to innovations that improve outcomes in acute life-threatening illnesses in children are scarce. A key issue is how we refocus research on outcomes that matter and are more relevant to those making emergency decisions, and those involved with managing and living with the late-outcome. We have used information from recent trials in critically ill children - in particular those illnesses without any primary neurologic involvement - to develop an approach to brain-related outcomes that will maximize child and family benefit from research.
Recent Findings:
Fifteen recent pediatric critical care trials illustrate four types of brain-related outcomes assessment: death or organ-system-failures - as illustrated by studies in systemic illness; neurological and neuropsychological outcomes - as illustrated by the glycemic control studies; cognitive outcomes - as illustrated by a sedative trial; and composite outcomes - as illustrated by the therapeutic hypothermia studies.
Summary:
The 15 research trials point to five areas that will need to be addressed and incorporated into future trial design, including use of: neurologic monitoring during intensive care unit admission; postdischarge outcomes assessments; strategies to improve retention in long-term follow-up; child and family-centered outcomes; and core outcomes datasets.
