A Randomized, Controlled Trial to Investigate the Efficacy of Nebulized Poractant Alfa in Premature Babies with Respiratory Distress Syndrome

Objective: To investigate the efficacy and safety of nebulized Poractant alfa (at 200 and 400 mg/kg doses) delivered in combination with nasal continuous positive airway pressure (nCPAP) compared with nCPAP alone in premature babies with diagnosed RDS. Study design: This randomized, controlled, multinational study was conducted in 28+0 to 32+6 weeks gestation infants. The primary outcome was the incidence of respiratory failure in the first 72 hours of life, defined as needing endotracheal surfactant and/or mechanical ventilation due to prespecified criteria. Secondary outcomes included the time to respiratory failure in the first 72 hours, duration of ventilation, mortality, incidence of bronchopulmonary dysplasia, and major associated neonatal comorbidities. In addition, the safety and tolerability of the treatments were assessed reporting the number and percentage of babies with treatment-emergent adverse events and adverse drug reactions during nebulization. Results: 129 babies were randomized. No significant differences were observed for the primary outcome: 24 (57%), 20 (49%) and 25 (58%) infants received endotracheal surfactant and/or mechanical ventilation within 72 hours in the Poractant alfa 200 mg/kg, Poractant alfa 400 mg/kg and nCPAP groups, respectively. Similarly, secondary respiratory outcomes did not differ among groups. Enrollment was halted early due to a change in the benefit-risk balance of the intervention. Nebulized Poractant alfa was well tolerated and safe, and no serious adverse events were related to the study treatment. Conclusions: The intervention did not reduce the likelihood of respiratory failure within the first 72 hours of life.