Publication Date
11-1-2022
Journal
Seminars in Perinatology
Abstract
Randomized controlled trials (RCTs) are challenging, but are the studies most likely to change practice and benefit patients. RCTs investigating neonatal surgical therapies are rare. The Necrotizing Enterocolitis Surgery Trial (NEST) was the first surgical RCT conducted by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Neonatal Research Network (NRN), and multiple lessons were learned. NEST was conducted over a 7.25-year enrollment period and the primary outcome was death or neurodevelopmental impairment (NDI) at 18-22 months corrected age. Surgical investigators designing clinical trials involving neonatal surgical treatments have many considerations to include, including how to study eligible but non-randomized patients, heterogeneity of treatment effect, use of frequentist and Bayesian analyses, assessment of generalizability, and anticipating criticisms during peer review. Surgeons are encouraged to embrace these challenges and seek innovative methods to acquire evidence that will be used to improve patient outcomes.
Keywords
Child, Enterocolitis, Necrotizing, Humans, Infant, Newborn, Infant, Newborn, Diseases, National Institute of Child Health and Human Development (U.S.), Randomized Controlled Trials as Topic, United States
Included in
Maternal and Child Health Commons, Maternal, Child Health and Neonatal Nursing Commons, Pediatrics Commons, Surgery Commons
Comments
PMID: 35811154