2024 | Dunbar Ivy, Erika B. Rosenzweig, Steven H. Abman, Maurice Beghetti, Damien Bonnet, Johannes Menno Douwes, Alessandra Manes, Rolf M.F. Berger
The article discusses recent advances, ongoing challenges, and distinct approaches for caring for infants and children with pulmonary arterial hypertension (PAH), as presented by the paediatric task force of the 7th World Symposium on Pulmonary Hypertension. It provides updates on diagnosing, classifying, risk-stratifying, and treating paediatric PAH, identifies critical knowledge gaps, and proposes an updated risk stratification tool and treatment algorithm. The article highlights the challenges in drug approval, particularly in designing accurate paediatric clinical trials with age-appropriate endpoints and adequate enrollment. It also discusses the management of children failing targeted PAH therapy, including balloon atrial septostomy, lung transplantation, and pulmonary-to-systemic shunt (Potts). A novel strategy using a multimodal approach for managing PAH associated with congenital heart diseases with borderline pulmonary vascular resistance is included. Advances in diagnosing neonatal PH, especially signs and interpretation of PH by echocardiography, are highlighted. A team approach to the rapidly changing physiology of neonatal PH is emphasized. The article also discusses the importance of genetic testing and counselling for patients and families with PAH. The treatment of paediatric PH continues to be hindered by the lack of randomized controlled clinical trials. The article proposes a pragmatic treatment algorithm based on risk stratification and treatment response, emphasizing the importance of individualized care and multidisciplinary approaches. It also discusses the roles of reverse Potts, septostomy, and transplant in managing advanced disease in children. The article concludes with the importance of further research to better define the indications and contraindications for each procedure.The article discusses recent advances, ongoing challenges, and distinct approaches for caring for infants and children with pulmonary arterial hypertension (PAH), as presented by the paediatric task force of the 7th World Symposium on Pulmonary Hypertension. It provides updates on diagnosing, classifying, risk-stratifying, and treating paediatric PAH, identifies critical knowledge gaps, and proposes an updated risk stratification tool and treatment algorithm. The article highlights the challenges in drug approval, particularly in designing accurate paediatric clinical trials with age-appropriate endpoints and adequate enrollment. It also discusses the management of children failing targeted PAH therapy, including balloon atrial septostomy, lung transplantation, and pulmonary-to-systemic shunt (Potts). A novel strategy using a multimodal approach for managing PAH associated with congenital heart diseases with borderline pulmonary vascular resistance is included. Advances in diagnosing neonatal PH, especially signs and interpretation of PH by echocardiography, are highlighted. A team approach to the rapidly changing physiology of neonatal PH is emphasized. The article also discusses the importance of genetic testing and counselling for patients and families with PAH. The treatment of paediatric PH continues to be hindered by the lack of randomized controlled clinical trials. The article proposes a pragmatic treatment algorithm based on risk stratification and treatment response, emphasizing the importance of individualized care and multidisciplinary approaches. It also discusses the roles of reverse Potts, septostomy, and transplant in managing advanced disease in children. The article concludes with the importance of further research to better define the indications and contraindications for each procedure.