Paediatric PAH treatment goals identified

Researchers from the Netherlands have identified three baseline variables that may qualify as treatment goals in children with pulmonary arterial hypertension (PAH).

“Although predictors of survival may be important when starting treatment for PAH, this does not mean that they can be used as treatment goals”, remark Mark-Jan Ploegstra and colleagues from University Medical Center Groningen.

“To qualify as a treatment goal, it is imperative that the variable is changed by treatment and that this change is associated with a change in outcome.”

In the present study, Ploegstra and team showed that treatment-induced changes in World Health Organization functional class (WHO-FC), N-terminal pro-brain natriuretic peptide (NT-proBNP) and tricuspid annular plane systolic excursion (TAPSE) were associated with transplant-free survival and “could therefore be considered treatment goals.”

The researchers studied the treatment-induced changes in these predictors of PAH survival along with others in 66 treatment-naïve children (median age 8 years) who started PAH-targeted treatment between January 2000 and April 2013.

Among the variables studied, systolic blood pressure, WHO-FC, NT-proBNP and right-to-left ventricular ratio significantly decreased between treatment initiation and first follow-up, a median of 4 months later, whereas 6-minute walking distance significantly increased. TAPSE also increased, but the change was not statistically significant.

However, after adjusting for age, gender and diagnosis, and taking into account both baseline and follow-up measurements and the prognostic value of treatment-induced changes, only WHO-FC, NT-proBNP and TAPSE were significantly associated with transplant-free survival.

Specifically, patients with low WHO-FC (≤III), low NT-proBNP (≤1200 ng/L) and high TAPSE (≥12 mm) who remained stable after treatment initiation had the best survival rates. And patients who improved from initial high WHO-FC, high NT-proBNP and low TAPSE after treatment initiation had a similar prognosis.

On the other hand, those patients who remained at high risk (WHO-FC IV, NT-proBNP >1200 ng/L or TAPSE <12 mm) after treatment initiation had significantly worse survival than those who improved.

Therefore “treatment should be escalated rapidly” in children who fail to meet the low-risk threshold values for WHO-FC, NT-proBNP and TAPSE following initiation of PAH-targeted treatment, Ploegstra and co-authors remark.

Writing in the European Respiratory Journal, they conclude: “In the absence of previously validated treatment goals, the identification of these variables allows for the introduction of goal-oriented treatment strategies in paediatric PAH.”

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