Risk Stratification and Contemporary Therapeutic Strategies in Pediatric Pulmonary Hypertension: A Narrative Review
Abstract
Background: Pediatric pulmonary hypertension (PH) is a rare, heterogeneous disease marked by progressive pulmonary vascular resistance and right ventricular pressure overload, with significant morbidity and mortality. Targeted pharmacotherapy has advanced, yet management is complicated by diverse causes, developmental differences from adult disease, and limited pediatric evidence. Risk stratification is now the foundation for individualized treatment. Aim: This review summarizes evidence on risk stratification and therapy in pediatric PH, focusing on prognostic tools and targeted treatments supporting personalized management. Material and methods: We conducted a narrative review of PubMed/MEDLINE literature from January 2020 to mid-2026, using pediatric- and PH-specific search terms, supplemented by manual screening of reference lists. Priority was given to guidelines, registry-based and multicenter cohort studies, and recent work on multidimensional risk assessment, cardiac imaging, invasive hemodynamics, biomarkers, pharmacotherapy, and advanced interventions. Results: Management has shifted from a disease-oriented toward an individualized, risk-guided approach. Reliable prognosis depends on integrating clinical evaluation, echocardiography, cardiac MRI, invasive hemodynamics, and biomarkers, rather than any single parameter. Dynamic risk stratification enables earlier detection of progression and timely escalation. Pharmacological management increasingly relies on combination therapy targeting nitric oxide, endothelin, and prostacyclin pathways, while emerging agents and interventions expand options for high-risk patients. Conclusions: Risk stratification is the cornerstone of pediatric PH management. Individualized strategies based on continuous assessment enable earlier intervention, optimized treatment, and appropriate selection of advanced therapies. Further multicenter studies are needed to validate risk models and strengthen treatment guidance.