A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study Oral Sildenafil Citrate- Treatment-Naive Children with PH
Background—Safe, effective therapy is needed for pediatric pulmonary arterial hypertension (PAH). Methods and Results—Treatment-naïve children (n=235; 1-17 yrs; ≥8 kg) enrolled in STARTS-1 were randomized to low-, medium-, or high-dose sildenafil or placebo orally thrice daily for 16 weeks. The primary comparison was percentage change from baseline in peak oxygen consumption (pVO2) for the three sildenafil doses combined versus placebo. Exercise testing was performed in 115 children able to exercise reliably; the study was powered for this population.