Advances in therapies for pediatric pulmonary arterial hypertension

Pulmonary arterial hypertension (PAH) is a life-threatening disease characterized by progressive obliteration of the pulmonary vasculature, leading to right heart failure and death if left untreated. Prior to the current treatment era, pulmonary hypertension carried a poor prognosis with a high mortality rate, but its prognosis has changed over the past decades in relation to new therapeutic agents. Nevertheless, pulmonary hypertension continues to be a serious condition, which is extremely challenging to manage. The data in children are often limited owing to the small number of patients, and extrapolation from adults to children is not straightforward. While none of these new therapeutic agents have been specifically approved for children, there is evidence that each can appropriately benefit the PAH child. We review the current understanding of pediatric pulmonary hypertension, classification, diagnostic evaluation and available treatment. A description of targeted pharmacological therapy and new treatments in children is outlined.