Dargaville, Peter A.Kamlin, C. Omar F.Orsini, FrancescaWang, XiaofangDe Paoli, Antonio G.Kanmaz Kutman, H. GozdeCetinkaya, MerihKornhauser-Cerar, LilijanaDerrick, MatthewOzkan, HilalHulzebos, Christian V.Schmoelzer, Georg M.Aiyappan, AjitLemyre, BrigitteKuo, ShereeRajadurai, Victor S.O'Shea, JoyceBiniwale, ManojRamanathan, RangasamyKushnir, AllaBader, DavidThomas, Mark R.Chakraborty, MallinathBuksh, Mariam J.Bhatia, RishaSullivan, Carol L.Shinwell, Eric S.Dyson, AmandaBarker, David P.Kugelman, AmirDonovan, Tim J.Goss, Kevin C. W.Tauscher, Markus K.Murthy, VadivelamAli, Sanoj K. M.Clark, Howard W.Soll, Roger F.Johnson, SamanthaCheong, Jeanie L. Y.Carlin, John B.Davis, Peter G.OPTIMIST-A Trial Investigators2024-10-032024-10-032023-09-110098-7484https://doi.org/10.1001/jama.2023.15694https://jamanetwork.com/journals/jama/fullarticle/2809514https://hdl.handle.net/11452/45779IMPORTANCE The long-term effects of surfactant administration via a thin catheter (minimally invasive surfactant therapy [MIST]) in preterm infants with respiratory distress syndrome remain to be definitively clarified.OBJECTIVE To examine the effect of MIST on death or neurodevelopmental disability (NDD) at 2 years' corrected age.DESIGN, SETTING, AND PARTICIPANTS Follow-up study of a randomized clinical trial with blinding of clinicians and outcome assessors conducted in 33 tertiary-level neonatal intensive care units in 11 countries. The trial included 486 infants with a gestational age of 25 to 28 weeks supported with continuous positive airway pressure (CPAP). Collection of follow-up data at 2 years' corrected age was completed on December 9, 2022.INTERVENTIONS Infants assigned to MIST (n = 242) received exogenous surfactant (200 mg/kg poractant alfa) via a thin catheter; those assigned to the control group (n = 244) received sham treatment.MAIN OUTCOMES AND MEASURES The key secondary outcome of death or moderate to severe NDD was assessed at 2 years' corrected age. Other secondary outcomes included components of this composite outcome, as well as hospitalizations for respiratory illness and parent-reported wheezing or breathing difficulty in the first 2 years.RESULTS Among the 486 infants randomized, 453 had follow-up data available (median gestation, 27.3 weeks; 228 females [50.3%]); data on the key secondary outcome were available in 434 infants. Death or NDD occurred in 78 infants (36.3%) in the MIST group and 79 (36.1%) in the control group (risk difference, 0%[95% CI, -7.6% to 7.7%]; relative risk [RR], 1.0 [95% CI, 0.81-1.24]); components of this outcome did not differ significantly between groups. Secondary respiratory outcomes favored the MIST group. Hospitalization with respiratory illness occurred in 49 infants (25.1%) in the MIST group vs 78 (38.2%) in the control group (RR, 0.66 [95% CI, 0.54-0.81]) and parent-reported wheezing or breathing difficulty in 73 (40.6%) vs 104 (53.6%), respectively (RR, 0.76 [95% CI, 0.63-0.90]).CONCLUSIONS AND RELEVANCE In this follow-up study of a randomized clinical trial of preterm infants with respiratory distress syndrome supported with CPAP, MIST compared with sham treatment did not reduce the incidence of death or NDD by 2 years of age. However, infants who received MIST had lower rates of adverse respiratory outcomes during their first 2 years of life.eninfo:eu-repo/semantics/openAccessChronic lung-diseaseRespiratory outcomesVentilationChildrenRatesAgeGeneral & internal medicineArticle001068888700001105410633301110.1001/jama.2023.15694