A Randomized, Controlled Trial to Investigate the Efficacy of Nebulized Poractant Alfa in Premature Babies with Respiratory Distress Syndrome
Carlo Dani, Gyula Talosi, Annalisa Piccinno, Virginia Maria Ginocchio, Gyorgy Balla, Anna Lavizzari, Zbynek Stranak, Eloisa Gitto, Stefano Martinelli, Richard Plavka, Barbara Krolak-Olejnik, Gianluca Lista, Francesca Spedicato, Giorgia Ciurlia, Debora Santoro, David Sweet, CURONEB Study Group, Carlo Dani, Gyula Talosi, Annalisa Piccinno, Virginia Maria Ginocchio, Gyorgy Balla, Anna Lavizzari, Zbynek Stranak, Eloisa Gitto, Stefano Martinelli, Richard Plavka, Barbara Krolak-Olejnik, Gianluca Lista, Francesca Spedicato, Giorgia Ciurlia, Debora Santoro, David Sweet, CURONEB Study Group
Abstract
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 compared with nasal continuous positive airway pressure alone in premature infants with diagnosed respiratory distress syndrome.
Study design: This randomized, controlled, multinational study was conducted in infants at 280/7 to 326/7 weeks of gestation. 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 owing 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 infants with treatment-emergent adverse events and adverse drug reactions during nebulization.
Results: In total, 129 infants 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 nasal continuous positive airway pressure groups, respectively. Similarly, secondary respiratory outcomes did not differ among groups. Enrollment was halted early owing 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 decrease the likelihood of respiratory failure within the first 72 hours of life.
Trial registration: ClinicalTrials.gov: NCT03235986.
Keywords: aerosol delivery; investigational eFlow Neos nebulizer; nebulized surfactant; poractant alfa; respiratory distress syndrome; vibrating-membrane nebulizer.
Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.
Source: PubMed