New Pediatric PAH Goals For Treatment Could Improve Patient Outcomes

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pediatric PAHA team of researchers from the University Medical Center Groningen in Holland, has identified three baseline variables that can become treatment goals in children suffering with pulmonary arterial hypertension (PAH).

PAH is rare in infants, however, it is a severe condition that rapidly deteriorates if left untreated.

Pediatric PAH can be idiopathic PAH (IPAH), heritable PAH, or associated with congenital heart disease (PAH-CHD). Common symptoms in children include dyspnea, fatigue, failure to thrive, syncope, poor appetite, lethargy, diaphoresis, tachypnea, tachycardia, and irritability.

Children who are not treated have a poor median survival of 10 months compared to a median survival of 2.8 years for adults. Due to this poor prognosis, and the fact that most pediatric PAH patients (77%) are only diagnosed when the disease is already in an advanced phase, treatment needs to be carefully considered.

To be able to design goal-oriented treatment strategies in pediatric PAH, researchers examined the prognostic value of treatment-induced changes in noninvasive predictors of transplant-free survival.

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The study, entitled Identification of treatment goals in pediatric pulmonary arterial hypertension and published in the European Respiratory Journal, followed 66 treatment-naïve pediatric PAH patients in the Dutch National Network for Pediatric Pulmonary Hypertension who initiated PAH-targeted drugs between January 2000 and April 2013, analyzing clinical, biochemical, and echocardiographic measures at treatment initiation and follow-up. Furthermore, to qualify as a treatment goal, it is essential that the variable measured can be changed by treatment and associated with a change in disease outcome.

Within all the variables studied, systolic blood pressure, World Health Organization functional class (WHO-FC), N-terminal pro-brain natriuretic peptide (NT-proBNP) and right-to-left ventricular ratio significantly decreased after treatment initiation and first follow-up, while 6-minute walking distance and tricuspid annular plane systolic excursion (TAPSE) increased.

Nevertheless, after considering variables such as age, gender and diagnosis, among all of the identified set of baseline predictors, only WHO-FC, NT-proBNP and TAPSE were classified as follow-up predictors in which treatment-induced changes were associated with survival.

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Moreover, patients who had low WHO-FC (≤III), low NT-proBNP (≤1200 ng/L) and high TAPSE (≥12 mm) and remained stable after treatment had increased survival rates, alongside patients who managed to improve their initial high WHO-FC, high NT-proBNP and low TAPSE after treatment initiation.

As a result, it appears that WHO-FC, NT-proBNP and TAPSE all function as predictors of transplant-free survival in pediatric PAH, but can also be used as treatment goals, allowing the development of new goal-oriented treatment strategies in children suffering from PAH.

A Conversation With Rare Disease Advocates