A randomized, double-blind, placebo-controlled, prospective study of bosentan for the treatment of persistent pulmonary hypertension of the newborn

Objective: To assess the efficacy and safety of bosentan as an adjuvant therapy of persistent pulmonary hypertension of the newborn (PPHN). Study Design: Forty-seven neonates with PPHN were randomly assigned to receive either bosentan (n = 24) or placebo (n = 23). Efficacy was evaluated with a favorable outcome defined as fulfilling all the following criteria (for example, oxygenation index <15, normal pulmonary artery pressure (< 20mm Hg) and no premature discontinuation of the drug because of drug-related toxicity or lack of efficacy). Evaluation of safety was done by monitoring drug-related adverse events. Result: Bosentan treatment was superior to placebo with a favorable response in 87.5% of patients treated with bosentan as compared with 20% of those who received placebo (P < 0.0001). None of patients in the bosentan group had drug-related clinical or laboratory adverse events. Conclusion: Bosentan may be a useful adjuvant therapy of PPHN. Journal of Perinatology (2012) 32, 608-613; doi: 10.1038/jp.2011.157; published online 10 November 2011