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 (<20 mm 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.