Safety and efficacy of perflubron-induced lung growth in neonates with congenital diaphragmatic hernia: Results of a prospective randomized trial

Background Mechanical transduction has been shown to promote fetal lung growth. We examined the safety and efficacy of perflubron-induced lung growth (PILG) in neonates with congenital diaphragmatic hernia (CDH) requiring extracorporeal membrane oxygenation (ECMO). Methods Infants with left-sided CDH requiring ECMO were eligible. Exclusion criteria included active air leak, intracranial hemorrhage, major congenital anomalies, and oxygenation index > 25 for 24 hours. Perflubron was instilled endotracheally and continuous positive airway pressure was applied without ventilation. Survival to discharge was the primary outcome. Daily chest radiographs were used to quantify lung size (the secondary outcome). Midway through the study our institutional practice shifted toward earlier repair of CDH. Results Eight infants were randomized to each arm. In the conventional-ventilation arm, six survived to discharge (75%). In the perflubron arm, four survived (50%); the others succumbed to suprasystemic pulmonary hypertension. No adverse events related to perflubron occurred. Within the perflubron group, 4/8 patients had "late repair" (15-19 days of life [DOL]) and 4 had "early repair" (2-3 DOL). "Early repair" patients had similar total lung growth, but accelerated growth and shorter ECMO runs. Conclusion PILG is safe in CDH and doubles the total lung size on average (accelerated with early repair). Despite amelioration of pulmonary hypoplasia with PILG, pulmonary hypertension persists.