Special Considerations in Randomized Trials Investigating Neonatal Surgical Treatments

Authors

Martin L Blakely
Matthew A Rysavy
Kevin P Lally
Barry Eggleston
Claudia Pedroza
Jon E Tyson

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

Comments

PMID: 35811154