title: Identification of treatment goals in paediatric pulmonary arterial hypertension.
abstract: To be able to design goal-oriented treatment strategies in paediatric pulmonary arterial hypertension (PAH), we aimed to identify treatment goals by investigating the prognostic value of treatment-induced changes in noninvasive predictors of transplant-free survival. 66 consecutive, treatment-naïve paediatric PAH patients in the Dutch National Network for Paediatric Pulmonary Hypertension who started taking PAH-targeted drugs between January 2000 and April 2013 underwent prospective, standardised follow-up. Clinical, biochemical and echocardiographic measures were longitudinally collected at treatment initiation and follow-up, and their respective predictive values for transplant-free survival were assessed. Furthermore, the predictive values of treatment-induced changes were assessed. From the identified set of baseline predictors, the variables World Health Organization functional class (WHO-FC), N-terminal pro-brain natriuretic peptide (NT-proBNP) and tricuspid annular plane systolic excursion (TAPSE) were identified as follow-up predictors in which treatment-induced changes were associated with survival. Patients in whom these variables improved after treatment showed better survival (p<0.002). Therefore, WHO-FC, NT-proBNP and TAPSE are not only predictors of transplant-free survival in paediatric PAH but can also be used as treatment goals, as treatment-induced improvements in these variables are associated with improved survival. The identification of these variables allows for the introduction of goal-oriented treatment strategies in paediatric PAH.
authors.affiliation: Center for Congenital Heart Diseases, Dept of Paediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands M.Ploegstra@umcg.nl.
authors.forename: Mark-Jan
authors.initials: MJ
authors.lastname: Ploegstra
authors.affiliation: Center for Congenital Heart Diseases, Dept of Paediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Johannes M
authors.initials: JM
authors.lastname: Douwes
authors.affiliation: Center for Congenital Heart Diseases, Dept of Paediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Marcus T R
authors.initials: MT
authors.lastname: Roofthooft
authors.affiliation: Center for Congenital Heart Diseases, Dept of Paediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Willemijn M H
authors.initials: WM
authors.lastname: Zijlstra
authors.affiliation: Dept of Epidemiology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Hans L
authors.initials: HL
authors.lastname: Hillege
authors.affiliation: Center for Congenital Heart Diseases, Dept of Paediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Rolf M F
authors.initials: RM
authors.lastname: Berger
mesh_terms: D000293:Adolescent; D006328:Cardiac Catheterization; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018450:Disease Progression; D004452:Echocardiography; D065128:Endothelin Receptor Antagonists / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000097:blood / Q000000981:diagnostic imaging / Q000628:therapy*; D017380:Hypertrophy, Right Ventricular / Q000000981:diagnostic imaging; D016040:Lung Transplantation; D008297:Male; D020097:Natriuretic Peptide, Brain / Q000097:blood; D010347:Patient Care Planning; D010446:Peptide Fragments / Q000097:blood; D058986:Phosphodiesterase 5 Inhibitors / Q000627:therapeutic use*; D011446:Prospective Studies; D044062:Prostaglandins I / Q000627:therapeutic use*; D011669:Pulmonary Wedge Pressure; D016019:Survival Analysis; D014262:Tricuspid Valve Insufficiency / Q000000981:diagnostic imaging
chemical_list: D065128:Endothelin Receptor Antagonists; D010446:Peptide Fragments; D058986:Phosphodiesterase 5 Inhibitors; D044062:Prostaglandins I; C109794:pro-brain natriuretic peptide (1-76); D020097:Natriuretic Peptide, Brain