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Published on: April 7, 2021
Relevant Outcomes in Pediatric Acute Respiratory Distress Syndrome Studies
1Department of Anesthesiology and Critical Care Medicine, Children's Hospital of Philadelphia, University of Pennsylvania , Philadelphia, PA , USA.
Insights
Pediatric acute respiratory distress syndrome (PARDS) management needs better outcome measures than mortality. Research should explore alternatives to assess long-term recovery in children with PARDS.
Area of Science:
- Pediatric Critical Care Medicine
- Respiratory Medicine
- Clinical Trials Methodology
Background:
- Pediatric acute respiratory distress syndrome (PARDS) management often relies on adult data, despite differing epidemiology and outcomes.
- Short-term mortality is a problematic primary outcome for PARDS trials due to lower rates and the influence of non-pulmonary factors.
- Existing data on alternative outcome measures for PARDS are scarce, and proposed metrics like ventilator-free days may have biases.
Purpose of the Study:
- To review challenges in defining outcomes for pediatric acute respiratory distress syndrome (PARDS) clinical trials.
- To identify and suggest clinically relevant alternative outcome measures for PARDS research.
- To guide future interventional studies and improve clinical practice for PARDS.
Main Methods:
- Literature review of pediatric acute respiratory distress syndrome (PARDS) studies.
- Analysis of outcome measures used in adult acute respiratory distress syndrome (ARDS) research.
- Synthesis of findings to propose alternative outcome measures for PARDS.
Main Results:
- Current PARDS research faces challenges in selecting appropriate outcome measures.
- Adult ARDS survivor data highlight the importance of long-term functional and neuropsychiatric outcomes.
- Ventilator-free days, a common alternative, may contain hidden biases.
Conclusions:
- There is a critical need for validated, clinically relevant outcome measures beyond mortality in pediatric acute respiratory distress syndrome (PARDS) research.
- Investigating long-term neurodevelopmental and functional outcomes is crucial for pediatric acute respiratory distress syndrome (PARDS) survivors.
- Standardized outcome measures will facilitate robust clinical trials and enhance evidence-based management of pediatric acute respiratory distress syndrome (PARDS).
Abstract:
Despite distinct epidemiology and outcomes, pediatric acute respiratory distress syndrome (PARDS) is often managed based on evidence extrapolated from treatment of adults. The impact of non-pulmonary processes on mortality as well as the lower mortality rate compared to adults with acute respiratory distress syndrome (ARDS) renders the utilization of short-term mortality as a primary outcome measure for interventional studies problematic. However, data regarding alternatives to mortality are profoundly understudied, and proposed alternatives, such as ventilator-free days, may be themselves subject to hidden biases. Given the neuropsychiatric and functional impairment in adult survivors of ARDS, characterization of these morbidities in children with PARDS is of paramount importance. The purpose of this review is to frame these challenges in the context of the existing pediatric literature, and using adult ARDS as a guide, suggest potential clinically relevant outcomes that deserve further investigation. The goal is to identify important areas of study in order to better define clinical practice and facilitate future interventional trials in PARDS.
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