title: Current and advancing treatments for pulmonary arterial hypertension in childhood.
abstract: Pulmonary arterial hypertension (PAH) is a severe and progressive intrinsic disease of the precapillary lung vasculature. Since the introduction of PAH-targeted drugs, survival of PAH patients seems to have improved. Randomized controlled trials have led to evidence-based guidelines to direct treatment in adults. However, since disease characteristics differ between adults and children, it is hazardous to simply extrapolate these guidelines to children. Moreover, pediatric data on treatment strategies and how to assess treatment response remain virtually absent. Optimal treatment strategies are highly needed to guide therapy and improve survival in children with PAH. This review provides an overview of currently available treatments for PAH and the limited efficacy and safety data in children (with the exclusion of perinatal pulmonary vascular diseases, as persistent pulmonary hypertension of the newborn). We also discuss potential treatment goals and how the available data can be translated into treatment strategies in pediatric PAH.
authors.affiliation: Center for Congenital Heart Diseases, Department of Pediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, P.O. Box 30.001, 9700 RB Groningen, The Netherlands.
authors.forename: Willemijn M H
authors.initials: WM
authors.lastname: Zijlstra
authors.forename: Mark-Jan
authors.initials: MJ
authors.lastname: Ploegstra
authors.forename: Rolf M F
authors.initials: RM
authors.lastname: Berger
mesh_terms: D002121:Calcium Channel Blockers / Q000627:therapeutic use*; D002648:Child; D065128:Endothelin Receptor Antagonists / Q000627:therapeutic use*; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy*; D010372:Pediatrics; D058986:Phosphodiesterase 5 Inhibitors / Q000627:therapeutic use*; D011453:Prostaglandins / Q000627:therapeutic use*
chemical_list: D002121:Calcium Channel Blockers; D065128:Endothelin Receptor Antagonists; D058986:Phosphodiesterase 5 Inhibitors; D011453:Prostaglandins
keywords: PAH-targeted drugs; goal-oriented treatment; pediatrics; pulmonary arterial hypertension; survival; treatment goal