Three High-Yield Pediatric Research Questions
Based on the American Thoracic Society consensus statement, the three highest-priority pediatric research questions that would have the greatest impact on morbidity, mortality, and quality of life are: (1) understanding lung development, repair, and remodeling mechanisms in premature infants to prevent chronic lung disease; (2) developing simple, noninvasive diagnostic tests to distinguish viral from bacterial pneumonia; and (3) creating an effective RSV vaccine with a simple vaccination schedule. 1, 2
1. Lung Growth, Development, and Repair in Premature Infants
This research area has the greatest potential for long-term impact across the entire age spectrum. 1
Primary question: Can we adapt the growth and repair mechanisms of immature lungs to mature lungs to enable renewal of healthy lung tissue in both children and adults? 1
The pathogenesis and repair mechanisms of chronic lung disease in extremely premature infants require urgent reevaluation, as modern neonatal care now saves infants at earlier gestational ages who develop a different pattern of bronchopulmonary dysplasia than historically described 2
Specific research priorities include investigating the role and timing of growth factors required for alveolarization, branching morphogenesis, and vascular/lymphatic development 1, 2
Understanding how prematurity and intrauterine infection affect lung development and postnatal responses is critical 2
This research could prevent the progression from childhood chronic lung disease to adult chronic obstructive pulmonary disease—a natural history that remains completely unknown 2
2. Simple, Noninvasive Diagnostic Tests for Pneumonia
The lack of rapid diagnostic ability to distinguish viral from bacterial pneumonia results in inappropriate antibiotic use, leading to antimicrobial resistance—a major cause of mortality globally. 1, 2
Primary question: Can we develop point-of-care diagnostic tests that accurately differentiate viral from bacterial pneumonia without invasive procedures? 2
Pneumonia accounts for significant mortality and morbidity in children worldwide, yet current diagnostic limitations force clinicians to empirically prescribe antibiotics 1
Recent research demonstrates that lung ultrasound patterns can differentiate bacterial from viral etiologies, with bacterial infections showing significantly higher lung ultrasound scores (p < 0.0001) 3
For tuberculosis specifically, rapid diagnostic field tests and shorter, more tolerable regimens are desperately needed, particularly in developing countries where TB remains a major source of pediatric mortality 1, 2
3. Effective RSV Vaccine Development
Respiratory syncytial virus has major consequences for children in both the number affected and potential for serious outcomes including death. 1
Primary question: Can we develop a true RSV vaccine with simple vaccination schedule and improved efficacy to prevent hospitalization, recurrent wheezing episodes, and death? 2
Current prevention strategies require monthly injections and are reserved only for at-risk children, leaving the vast majority of infants unprotected 1
Understanding the interaction between RSV and the host immune system, including the inflammatory response and how to prevent both acute respiratory failure and long-term sequelae (such as recurrent wheezing), is essential 1
From a global perspective, infectious disease prevention and control are clearly of highest priority, making RSV vaccine development critical for reducing pediatric mortality worldwide 1
Common Pitfalls in Pediatric Research Design
Most important questions about chronic lung disease cannot be answered with single-center studies due to the relative rarity of many pediatric pulmonary diseases—large, well-designed, multicenter prospective studies are mandatory 2
Paper diaries in pediatric studies are subject to poor adherence and data fabrication; electronic monitoring should be prioritized 2
Procedural sedation for research (bronchoscopy, infant lung function testing) is only ethically acceptable when the question is scientifically important, sedation is necessary, risks are minimized, and standard airway safeguards are in place 1