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

Academic Article

Abstract

  • OBJECTIVE: To analyze reasons for low enrollment in a randomized controlled trial (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 h 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. RESULTS: Two hundred and fifty-seven of the 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.
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    Published In

    Keywords

  • Cardiotonic Agents, Critical Illness, Double-Blind Method, Early Termination of Clinical Trials, Heart Defects, Congenital, Heart Failure, Humans, Hydrocortisone, Infant, Newborn, Infant, Premature, Informed Consent, Neurodevelopmental Disorders, Patient Selection
  • Digital Object Identifier (doi)

    Author List

  • Watterberg KL; Fernandez E; Walsh MC; Truog WE; Stoll BJ; Sokol GM; Kennedy KA; Fraga MV; Beauman SS; Carper B
  • Start Page

  • 1220
  • End Page

  • 1223
  • Volume

  • 37
  • Issue

  • 11