A prospective, randomized pilot trial of perfluorocarbon-induced lung growth in newborns with congenital diaphragmatic hernia.

BACKGROUND/PURPOSE: Initial laboratory and clinical data suggest that partial liquid ventilation (PLV) can enhance pulmonary function and that lung growth can be induced via distension of the newborn lung using perfluorocarbon in patients with congenital diaphragmatic hernia (CDH). The authors, therefore, performed a prospective, randomized pilot study evaluating PLV and perfluorocarbon-induced lung growth (PILG) in newborns with CDH on extracorporeal life support (ECLS) at 6 medical centers.

METHODS

Patients were selected randomly using a permuted block design to PLV/PILG (n = 8) or conventional mechanical ventilation (CMV/control, n = 5). Patients in the PILG group received daily doses which filled the lungs with perflubron for up to 7 days and were placed on continuous positive airway pressure of 5 to 8 cm H2O. CMV patients were treated with standard mechanical ventilation while on extracorporeal membrane oxygenation (ECMO).

RESULTS

A total of 13 patients were evaluated in this study. All 3 patients enrolled without being on ECLS rapidly transitioned to ECLS. The study, therefore, effectively evaluated PILG (n = 8) versus standard ventilation (control, n = 5) on ECLS. Mean (+/- SE) gestational age was 37 +/- 1 weeks and weight was 3.1 +/- 0.1 kg. Time on ECMO was 9.8 +/- 2.3 days in the PILG and 14.5 +/- 3.5 days (P =.58) in the control group. Survival rate in the PILG group was 6 of 8 (75%), whereas survival rate was 2 of 5 (40%) in the control group (P =.50). The number of days free from the ventilator in the first 28 days (VFD) was 6.3 +/- 3.3 days with PILG and 4.6 +/- 4.6 days with control (P =.9). Causes of death in the PILG group included sepsis and renal failure in one patient and pulmonary hypertension in the other. There were no safety issues, and the deaths in the PILG group did not appear to be related to the administration of perflubron.

CONCLUSIONS

These data show that PILG can be performed safely. The survival rate, VFD, and time on ECMO data, although not conclusive, are encouraging and indicate the need for a definitive trial of this novel intervention in these neonates with high mortality.