Treatment options for children with pulmonary arterial hypertension associated with congenital heart disease

INTRODUCTION: Pulmonary hypertension associated with congenital heart disease (PAH-CHD) represents one of the leading causes of pediatric pulmonary hypertension. Within this entity, patients can be classified into distinct subgroups, each characterized by specific clinical features, pathophysiological mechanisms, and therapeutic approaches. AREAS COVERED: This review provides an update of the current PAH-CHD classification and outlines management strategies in accordance with the most recent international recommendations. Particular attention is given to the ongoing debate regarding operability in patients with open shunts and some degree of increased pulmonary vascular resistance, and key gaps in knowledge are highlighted. EXPERT OPINION: In PAH-CHD, treatment strategies are relatively straightforward in patients with low PVR, where shunt closure is recommended, and in Eisenmenger syndrome, where shunt patency is maintained and pulmonary vasodilators are indicated; however, the management of patients with open shunts and moderately elevated PVR remains highly challenging. Operability in this intermediate group is currently determined by invasive hemodynamic assessment, despite methodological limitations, highlighting the urgent need for reliable noninvasive markers and prospective clinical studies in late-referred patients. Emerging therapies such as sotatercept, along with the development of novel biomarkers to assess pulmonary vascular disease severity may have the potential to redefine operability criteria and expand therapeutic options.