Evaluation of pediatric patients with severe pulmonary arterial hypertension

INTRODUCTION: Pulmonary arterial hypertension (PAH) is an important cause of morbidity and mortality. Congenital heart disease associated PAH (APAH-CHD) and idiopathic PAH are classified in Group 1 PAH. There are limited studies about pediatric patients with PAH. The aim of our study is to evaluate the clinical, diagnostic and treatment characteristics of pediatric PAH.
METHODS: 53 consecutive patients with PAH in a 7 years’ study period were retrospectively analyzed. Clinical, echocardiographic and cardiac catheterization findings and targeted treatment modalities were noted.
RESULTS: Thirty (56.6%) patients were male and mean age at diagnosis was 5.27 ± 4.30 years. All patients were classified as group 1 consisting of APAH-CHD and idiopathic PAH. Patients with Eisenmenger syndrome was the largest group. Ventricular septal defect was the most CHD associated with PAH overall. Atrial septal defect, patent ductus arteriosus, atrioventricular septal defect, aortopulmonary window, double inlet left ventricle, double outlet right ventricle, d-transposition of great arteries and truncus arteriosus were other congenital heart malformations detected. Targeted therapy were given to 34 patients (%64.1%), of them, 22 were under monotherapy, while 12 were under combined therapy. Bosentan was the most chosen drug in all. NYHA FC, exercise capacity with 6MWT improved well by targeted therapy.
DISCUSSION AND CONCLUSION: Life quality and survey are improved with the targeted therapies in pediatric patients with PAH. Single drug or combination therapies including bosentan, tadalafil and inhaled iloprost are effective, safe and well tolerated with rare and minor side effects in pediatric patients with group 1 PAH.