Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants.
(Journal Article;Multicenter Study)
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.
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
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