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MicroRNA in pediatric pulmonary hypertension microRNA profiling to inform disease classification, severity, and
Michael A Smith1,2, Sam Chiacchia3, Jason Boehme1
1Division of Pediatric Critical Care, Department of Pediatrics, University of California, San Francisco, California, United States.
Abstract:
Pediatric pulmonary hypertension is a heterogeneous disease associated with significant morbidity and mortality. MicroRNAs have been implicated as both pathologic drivers of disease and potential therapeutic targets in pediatric pulmonary hypertension. We sought to characterize the circulating microRNA profiles of a diverse array of pediatric patients with pulmonary hypertension using high-throughput sequencing technology. Peripheral blood samples were drawn from patients recruited at the time of a clinically indicated cardiac catheterization, and microRNA sequencing followed by differential expression and target/pathway enrichment analyses were performed. Among 63 pediatric patients with pulmonary hypertension, we identified specific microRNA signatures that uniquely classified patients by disease subtype, correlated with indicators of disease severity including invasive hemodynamic metrics, and changed over the course of treatment for pulmonary hypertension. These microRNA profiles include a number of specific microRNA molecules known to function in signaling pathways critical to pulmonary vascular biology and disease, including transforming growth factor-β (TGF-β), VEGF, PI3K/Akt, cGMP-PKG, and HIF-1 signaling. Circulating levels of miR-122-5p, miR-124-3p, miR-204-5p, and miR-9-5p decreased over the course of treatment in a subset of patients who had multiple samples drawn during the study period. Our findings support the further investigation of specific microRNAs as mechanistic mediators, biomarkers, and therapeutic targets in pulmonary hypertension.NEW & NOTEWORTHY We present novel insight into the circulating microRNA profiles of pediatric patients with pulmonary hypertension. Our findings support the utility of microRNAs as both useful biomarkers of disease severity and potential therapeutic targets in pediatric pulmonary hypertension.
Insights
MicroRNAs in pediatric pulmonary hypertension show distinct profiles that classify disease subtypes and severity. These microRNA signatures can change with treatment, highlighting their potential as biomarkers and therapeutic targets.
Area of Science:
- Cardiovascular Biology
- Molecular Medicine
- Pediatric Pulmonology
Background:
- Pediatric pulmonary hypertension (PPH) is a severe condition with high mortality.
- MicroRNAs are implicated in PPH pathogenesis and represent potential therapeutic targets.
Purpose of the Study:
- To characterize circulating microRNA profiles in pediatric patients with pulmonary hypertension.
- To identify microRNA signatures associated with disease subtypes, severity, and treatment response.
Main Methods:
- High-throughput sequencing of microRNAs from peripheral blood of 63 pediatric patients.
- Differential expression analysis, target/pathway enrichment, and correlation with hemodynamic metrics.
- Longitudinal analysis of microRNA levels in patients with multiple samples.
Main Results:
- Identified specific microRNA signatures classifying PPH subtypes and correlating with disease severity.
- Observed changes in microRNA profiles during treatment, including decreases in miR-122-5p, miR-124-3p, miR-204-5p, and miR-9-5p.
- Enriched microRNAs involved in critical signaling pathways like TGF-β, VEGF, and HIF-1.
Conclusions:
- Circulating microRNA profiles offer valuable insights into PPH.
- MicroRNAs serve as potential biomarkers for disease severity and progression in pediatric patients.
- MicroRNAs represent promising targets for novel PPH therapies.
