RESUMO
OBJECTIVES: The aim of this work was to describe treatment response and outcome data for preterm infants with pulmonary hypoplasia treated with inhaled nitric oxide (iNO). We hypothesised that an acute oxygenation response to iNO would be associated with survival. DESIGN: A retrospective observational study design was used to identify cases of pulmonary hypoplasia in preterm infants <34 weeks' gestation reported to the European iNO Registry. Demographic and clinical data were collected including oxygenation and echocardiographic parameters. The primary outcome was acute oxygenation response defined as a reduction in fractional inspired oxygen of >0.15. Outcome data included chronic lung disease (CLD) and death. RESULTS: Seventy-two infants with pulmonary hypoplasia were treated with iNO during a 10-year period (2007-2016). In total, 30/69 (43%) of the infants showed a significant improvement in oxygenation and were categorised as "responders." Thirty-one treated infants died, and 19 survivors developed CLD. Although there were no differences in demographics and baseline cardiorespiratory parameters between responders and non-responders, an acute response was significantly associated with survival. Neither pulmonary hypertension nor PPHN (persistent pulmonary hypertension of the newborn) physiology predicted the acute response to iNO or survival. CONCLUSION: Although the acute oxygenation response to iNO therapy in pulmonary hypoplasia is comparable to other respiratory disorders in preterm infants, mortality in this group remains very high. An acute response is associated with survival and suggests that a short therapeutic trial of iNO therapy is warranted in this population. This study underscores the value of registries in evaluating therapies for rare neonatal disorders, although their limitations must be recognised.