Most Relevant Research Topics in Pediatrics
The highest priority pediatric research areas focus on lung growth and development, infectious disease prevention, childhood asthma, and long-term outcomes following critical illness, with translational research bridging basic science to clinical applications offering the greatest potential impact across the age spectrum.
Critical Pulmonary Research Priorities
Lung Development, Repair, and Remodeling
- Research into lung growth, development, and repair mechanisms has the greatest long-term potential impact on disease prevention and treatment across all ages 1
- Understanding the regulation of fetal lung development, adaptation to extrauterine life, and mechanisms of repair and plasticity could enable renewal of healthy lung tissue in both children and adults 1
- Chronic lung disease in premature infants requires reevaluation of pathogenesis and repair mechanisms, particularly given the survival of increasingly immature infants 1
- The role of prematurity and intrauterine infection on lung development and postnatal responses needs further investigation 1
- Development of pediatric-specific investigative tools for inflammatory responses is essential, given technical challenges with invasive procedures and limited sample sizes 1
Infectious Disease Research (Global Priority)
- From a global perspective, infectious disease prevention and control represent the highest priority research area 1
- Development of simple, noninvasive diagnostic tests for pneumonia is critically needed to prevent inappropriate antibiotic use and resistant organism emergence 1
- Respiratory syncytial virus (RSV) vaccine development with simple vaccination schedules and improved efficacy is required to prevent hospitalization and recurrent wheezing episodes 1
- Understanding the RSV-host interaction, inflammatory response, and prevention of both acute respiratory embarrassment and long-term sequelae remains incomplete 1
- Tuberculosis research requires rapid diagnostic field tests and shorter, more tolerable treatment regimens 1
- Genomic and proteomic research of infectious agents will facilitate development of effective antimicrobial therapies 1
Asthma Research (Industrialized Nations Priority)
- In industrialized nations, asthma represents a top research priority, affecting 10-20% of children with unclear reasons for dramatic prevalence increases 1
- Childhood asthma demonstrates different clinical patterns than adult asthma, with long quiescence periods punctuated by viral exacerbations that diminish over time 1
- A new disease model appropriate for children is required, as corticosteroid therapy has been only partially successful with lower thresholds for adverse effects 1
- Development of noninvasive markers of disease severity and progression is essential 1
- Combined expertise from epidemiologists, geneticists, airway biologists, and clinicians is necessary for comprehensive understanding 1
- Animal models demonstrating maturational changes seen in humans are needed to examine immunologic immaturity's role in asthma development 1
Critical Care Outcomes and Morbidity Research
Long-Term Outcomes Following Critical Illness
- Research on mortality and health-related quality of life morbidity following pediatric critical illness reveals that new morbidities occur at twice the mortality rate (4.8% vs 2.4%) 2
- Among children surviving community-acquired septic shock, 35% demonstrate significant health-related quality of life deterioration persisting at least 1 year post-hospitalization 3
- At 12 months following septic shock, 13% of patients had died and 35% of survivors had not regained baseline health-related quality of life 3
- New morbidities involve all functional domains, with highest proportions affecting respiratory, motor, and feeding dysfunction 2
- Pediatric critical care may have exchanged improved mortality rates for increased morbidity rates 2
Pediatric Pulmonary Embolism
- Long-term outcomes assessment in prospective studies is essential, as mortality rates are significant but many deaths are attributable to underlying disease states 1
- Development and validation of pediatric-specific clinical prediction models is needed to identify low-likelihood cohorts who may not require diagnostic imaging 1
- Standardization of definitions (such as "immobilization") and D-dimer negative predictive value evaluation in children requires further study 1
- Quality of life assessment tools for children receiving anticoagulant therapy suggest psychological, behavioral, and social problems associated with this diagnosis 1
Translational and Methodological Research
Bridging Basic Science to Clinical Practice
- Translational research projects that bridge laboratory findings with clinical applications provide the most valuable framework for advancing pediatric medicine 4
- Projects including both basic science components and clinical relevance help develop comprehensive understanding of medical research 4
- Genomic and proteomic research provides opportunities for innovative therapeutic approaches 1
Advanced Diagnostic and Assessment Technologies
- High-resolution computed tomography and noninvasive/minimally invasive lung function assessment in young children facilitate prospective disease monitoring 1
- These technologies require modification for small pediatric lungs and uncooperative children, plus development of age-, sex-, and ethnicity-specific standards 1
- Bioinformatics advancement requires large datasets, gradually being developed through computerized databases 1
Environmental and Regulatory Research
- Environmental exposure research addresses increasingly complex and polluted environments with little knowledge of safe exposure limits for most substances 1
- Understanding environmental impacts on child lung health has tremendous potential for disease prevention 1
- Research must address both developed and developing world contexts 1
Resource-Limited Settings
- Developing effective pediatric emergency and critical care services could substantially reduce childhood mortality in resource-limited settings, where the majority of deaths result from reversible causes 5
- Only 27 of 138 developing countries achieved the millennium development goal of reducing under-5 mortality by two-thirds 5
- Diagnosis-specific mortality studies identify that conditions requiring significant human and material resources (surgical conditions 16.1%, malnutrition 15.8%, congenital heart disease 14.5%) have highest mortality rates 6
Critical Methodological Considerations
- Fundamental and applied research must be linked through translational investigation as a common theme across all pediatric research priorities 1
- Study center networks with uniform protocols and sophisticated analytical methods are necessary for complex data handling 1
- Ethical issues regarding confidentiality in computerized databases and children's participation in medical research require ongoing attention 1
- Outcome measures in pediatrics are complicated by the need to view the child with disease in the context of the family unit 1