Presentation Information
[I-OR04-01]Keynote Lecture : Diagnostic Evaluation, Long-Term Prognosis and Treatment Strategies for Pediatric Pulmonary Arterial Hypertension in China
○Hong Gu1 (The Department of Pediatric Cardiology, Beijing Anzhen Hospital Capital Medical University Beijing, China)
Keywords:
Pediatric pulmonary arterial hypertension (PAH),Right heart catheterization with acute vasodilator testing (AVT),Targeted treatment
Pediatric pulmonary arterial hypertension (PAH), especially idiopathic/heritable PAH (IPAH/HPAH), is a rare, progressive, and life-threatening condition in children, differing substantially from adult PAH. In Chinese pediatric populations, congenital heart disease-related PAH (CHD-PAH) and IPAH/HPAH are the predominant subtypes. IPAH/HPAH in children is characterized by early onset, rapid progression, and poor prognosis, often due to late referral, advanced cardiac dysfunction at diagnosis, and limited access to affordable targeted therapies. Approximately 50% of pediatric IPAH/HPAH patients carry pathogenic mutations (most commonly BMPR2 and ACVRL1), which correlate with impaired vasoreactivity and worse survival, warranting early genetic screening. Right heart catheterization (RHC) with inhaled iloprost acute vasodilator testing (AVT) is safe and effective for pediatric IPAH, significantly reducing pulmonary artery pressure (PAP) and pulmonary vascular resistance index (PVRI) while improving cardiac index (CI). Barst-defined vasoreactivity and baseline mixed venous oxygen saturation (SvO₂) are independent long-term prognostic factors. Pulmonary hypertensive crisis (PHC) during RHC is a critical complication, with younger age, elevated BNP, and severe right ventricular dilation as key risk factors. For high-risk IPAH/HPAH, prostacyclins (e.g., treprostinil) significantly improve hemodynamics, cardiac function, and survival, even in gene-mutated patients. Palliative procedures (Potts shunt, atrial septostomy) and lung transplantation serve as rescue options for end-stage disease. Early diagnosis, genetic screening, standardized hemodynamic assessment, and aggressive targeted therapy are critical to improve long-term outcomes in pediatric PAH.