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Defining endotypes of bronchopulmonary dysplasia in preterm infants to improve precision-based therapies
Megha Sharma1,2, Gangaram Akangire3, Noah H Hillman4
1Department of Pediatrics, Division of Neonatology, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.
Insights
Bronchopulmonary dysplasia (BPD) is a complex lung disease in premature infants. Identifying distinct BPD endotypes using advanced methods is crucial for developing targeted treatments and improving infant outcomes.
Area of Science:
- Neonatal Medicine
- Pediatric Pulmonology
- Medical Imaging
Background:
- Bronchopulmonary dysplasia (BPD) is a significant cause of chronic lung disease in premature infants.
- Current BPD definitions lack specificity, failing to address disease heterogeneity and endotypes, which hinders clinical trial success.
- Understanding BPD pathogenesis is complex due to prenatal and postnatal factors.
Purpose of the Study:
- To review the pathogenic mechanisms underlying distinct BPD endotypes.
- To discuss how advanced techniques can identify these endotypes.
- To highlight the potential for endotype-specific therapies in BPD.
Main Methods:
- Review of recent studies utilizing advanced lung imaging, echocardiography, and pulmonary function tests.
- Analysis of pathogenic mechanisms contributing to BPD heterogeneity.
- Exploration of biomarker, functional studies, and AI for endotype characterization.
Main Results:
- Identification of distinct BPD endotypes, including airway, parenchymal, and vascular.
- Recognition that these endotypes have different prognoses.
- Evidence suggests endotype-specific treatments are necessary for optimal outcomes.
Conclusions:
- BPD is a heterogeneous disease with distinct endotypes requiring tailored treatment strategies.
- Advanced diagnostics are key to identifying BPD endotypes.
- Combining biomarkers, functional data, and AI can guide precision medicine for BPD.
Abstract:
Bronchopulmonary dysplasia (BPD) remains a debilitating disease in premature infants. The chronic pathogenesis of BPD with complex prenatal and postnatal programming challenges attempts at precisely defining or treating disease. While existing BPD definitions categorize disease severity, a lack of consideration of disease heterogeneity and endotypes has contributed to the failure of clinical trials to improve BPD outcomes. Recent studies have used advanced lung imaging techniques, echocardiography, and lung function tests to identify airway, parenchymal, and vascular BPD endotypes. These endotypes carry different prognoses and require endotype-specific treatment strategies to optimize infant outcomes. In this Review, we focus on the pathogenic mechanisms that specify individual BPD endotypes and discuss how combining biomarkers, functional studies, and artificial intelligence-based characterization of endotypes can inform precision therapies for BPD.
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