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Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants
Journal article   Open access   Peer reviewed

Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants

Kristi L. Watterberg, Erika Fernandez, Michele C. Walsh, William E. Truog, Barbara J. Stoll, Gregory M. Sokol, Kathleen A. Kennedy, Maria V. Fraga, Sandra Sundquist Beauman, Benjamin Carper, …
Journal of perinatology : official journal of the California Perinatal Association, Vol.37(11), pp.1220-1223
09/01/2017
DOI: 10.1038/jp.2017.131
PMCID: PMC5688018
PMID: 28880260
url
https://www.ncbi.nlm.nih.gov/pmc/articles/5688018View
Open Access

Abstract

Objective To analyze reasons for low enrollment in an RCT of the effect of hydrocortisone for cardiovascular insufficiency on survival without neurodevelopmental impairment (NDI) in term/late-preterm newborns. Study Design The original study was a multicenter RCT. Eligibility: ≥34 weeks’ gestation, <72° old, mechanically ventilated, receiving inotrope. Primary outcome was NDI at 2 years; infants with diagnoses at high risk for NDI were excluded. This paper presents an analysis of reasons for low patient enrollment. Result 257 of 932 otherwise eligible infants received inotropes; however, 207(81%) had exclusionary diagnoses. Only 12 infants were randomized over 10 months; therefore, the study was terminated. Contributing factors included few eligible infants after exclusions, open-label steroid therapy, and a narrow enrollment window. Conclusion Despite an observational study to estimate the population, very few infants were enrolled. Successful RCTs of emergent therapy may require fewer exclusions, a short-term primary outcome, waiver of consent, and/or other alternatives. Clinical trial registration Clinicaltrials.gov, NCT01954056
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