Clinical and economic effects of iNO in premature newborns with respiratory failure at 1 year.

BACKGROUND The long-term consequences of inhaled nitric oxide (iNO) use in premature newborns with respiratory failure are unknown. We therefore studied the clinical and economic outcomes to 1 year of corrected age after a randomized controlled trial of prophylactic iNO. METHODS Premature newborns (gestational age <or=34 w, birth weight 500-1250 g) with respiratory failure randomly received 5 ppm iNO or placebo within 48 h of birth until 21 d or extubation. We assessed clinical outcomes via in-person neurodevelopmental evaluation at 1 y corrected age and telephone interviews every 3 m. We estimated costs from detailed hospital bills and interviews, converting all costs to 2008 US$. Of 793 trial subjects, 631 (79.6%) contributed economic data, and 455 (77.1% of survivors) underwent neurodevelopmental evaluation. RESULTS At 1 y corrected age, survival was not different by treatment arm (79.2% iNO vs. 74.5% placebo, P = .12), nor were other post-discharge outcomes. For subjects weighing 750-999 g, those receiving iNO had greater survival free from neurodevelopmental impairment (67.9% vs. 55.6%, P = .04). However, in subjects weighing 500-749 g, iNO led to greater oxygen dependency (11.7% vs. 4.0%, P = .04). Median total costs were similar ($235,800 iNO vs. $198,300 placebo, P = .19). Quality-adjusted survival was marginally better with iNO (by 0.011 quality-adjusted life-years/subject). The incremental cost-effectiveness ratio was $2.25 million/quality-adjusted life-year. CONCLUSIONS Subjects in both arms commonly experienced neurodevelopmental and pulmonary morbidity, consuming substantial health care resources. Prophylactic iNO beginning in the first days of life did not lower costs and had a poor cost-effectiveness profile.