Inhaled
nitric oxide (iNO) is an effective but costly
therapy for infants with
hypoxemic respiratory failure. The approved and solidly evidence-based indication is for treatment of late preterm and term infants with persistent
pulmonary hypertension of the newborn (PPHN); however,
off-label use of iNO has become widespread. Although iNO treatment of infants with
congenital diaphragmatic hernia constitutes one of the approved indications, available evidence from randomized trials suggests marginal if any efficacy. Rescue
therapy in preterm infants with severe
respiratory failure has been studied extensively and is not supported by data from a number of controlled trials. Such use is widespread, but should be discouraged. There may be a subgroup of such infants with pulmonary hypoplasia and documented PPHN who may benefit from this treatment, but the data are limited. Several studies have examined the use of iNO for prevention of chronic
lung disease with inconsistent results. This promising application requires more study before it can be recommended. There may be a role of iNO in treating infants with
pulmonary hypertension complicating severe
bronchopulmonary dysplasia, but there are limited data on long term outcomes. Alternate
therapies such as
sildenafil may be beneficial in this specific population as well as in other causes of
pulmonary hypertension. Rational use of this expensive treatment will maximize cost:benefit and avoid potential exposure to unknown adverse effects not balanced by documentable benefits.