Abstract
Pulmonary arterial hypertension (PAH) is a rare and devastating disease
of the pulmonary vasculature with a high morbidity and mortality rate in
infants and children. Currently, treatment approaches are mostly based
on adult guidelines and pediatrician clinical experience, focusing on
specific pulmonary antihypertensive therapy and conventional supportive
care. The advent of targeted drugs has led to significant advances in
the treatment of PAH in children, including endothelin receptor
antagonists, phosphodiesterase type 5 inhibitors, and prostacyclins,
which have been studied and proven to improve hemodynamics and
functional class in children PAH. A new targeted drug, riociguat, is
assessing its safety and efficacy in clinical trials. However, more
randomized controlled studies are needed to evaluate the combination of
drugs, treatment strategies, and clinical endpoints of targeted therapy
in children PAH. In this review, we summarize the research advances of
PAH-targeted therapy in children over the last decade in order to
provide a theoretical basis for future studies.