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Identification of treatment goals in paediatric pulmonary arterial hypertension
Mark-Jan Ploegstra1, Johannes M Douwes2, Marcus T R Roofthooft2
1Center 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.
Insights
Treatment goals for pediatric pulmonary arterial hypertension (PAH) were identified. Improvements in World Health Organization functional class, NT-proBNP, and TAPSE predict better survival, enabling goal-oriented PAH treatment strategies.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Translational Medicine
Background:
- Pediatric pulmonary arterial hypertension (PAH) requires effective, goal-oriented treatment strategies.
- Identifying noninvasive predictors of transplant-free survival is crucial for guiding therapy.
Purpose of the Study:
- To determine treatment goals for pediatric PAH by assessing the prognostic value of treatment-induced changes in noninvasive survival predictors.
- To investigate if improvements in specific clinical, biochemical, and echocardiographic measures correlate with better transplant-free survival.
Main Methods:
- Prospective, standardized follow-up of 66 treatment-naïve pediatric PAH patients.
- Longitudinal collection of clinical, biochemical, and echocardiographic data at baseline and follow-up.
- Assessment of predictive values for transplant-free survival for both baseline measures and treatment-induced changes.
Main Results:
- 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 key follow-up predictors.
- Treatment-induced improvements in WHO-FC, NT-proBNP, and TAPSE were significantly associated with better transplant-free survival (p<0.002).
Conclusions:
- WHO-FC, NT-proBNP, and TAPSE serve as effective predictors of transplant-free survival in pediatric PAH.
- These measures can be utilized as treatment goals, as improvements indicate enhanced survival outcomes.
- The findings support the implementation of goal-oriented treatment strategies for pediatric PAH.
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.
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