Pharmacological treatment of pulmonary hypertension

Abstract

Pulmonary arterial hypertension (PAH) is a life- threatening disease characterized by a progressive increase in pulmonary vascular resistance leading to right heart failure and death. In the last decade, specific targeted therapies have been developed and have improved survival in adult patients with PAH. These therapies have also benefited children with PAH. Unfortunately, there are limited data on treatment strategies in children with PAH due to the small number of randomized controlled clinical trials evaluating the safety and efficacy of specific treatments. Currently approved PAH therapies impact one of three endothelialbased pathways including nitric oxide, prostaglandin, or endothelin-1. This chapter summarizes currently available therapies based on adult data and a limited number of clinical trials in children with PAH.