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

  • Author(s): Watterberg, KL
  • Fernandez, E
  • Walsh, MC
  • Truog, WE
  • Stoll, BJ
  • Sokol, GM
  • Kennedy, KA
  • Fraga, MV
  • Beauman, SS
  • Carper, B
  • Das, A
  • Duncan, AF
  • Buss, WF
  • Gauldin, C
  • Lacy, CB
  • Sanchez, PJ
  • Chawla, S
  • Lakshminrusimha, S
  • Cotten, CM
  • Van Meurs, KP
  • Poindexter, BB
  • Bell, EF
  • Carlo, WA
  • Devaskar, U
  • Wyckoff, MH
  • Higgins, RD
  • et al.
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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