Treatment-induced improvements in WHO functional class, NT-proBNP, and TAPSE were significantly associated with better transplant-free survival in paediatric PAH patients (p<0.002).
Cohort (n=66)
Yes
p-value: p=<0.002
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.
Ploegstra et al. (Thu,) conducted a cohort in paediatric pulmonary arterial hypertension (n=66). PAH-targeted drugs was evaluated on transplant-free survival (p=<0.002). Treatment-induced improvements in WHO functional class, NT-proBNP, and TAPSE were significantly associated with better transplant-free survival in paediatric PAH patients (p<0.002).