학술논문
Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants
Document Type
Academic Journal
Author
Watterberg, K L; Fernandez, E; Walsh, M C; Truog, W E; Stoll, B J; Sokol, G M; Kennedy, K A; Fraga, M V; Beauman, S S; Carper, B; Das, A; Duncan, A F; Buss, W F; Gauldin, C; Lacy, C B; Sanchez, P J; Chawla, S; Lakshminrusimha, S; Cotten, C M; Van Meurs, K P; Poindexter, B B; Bell, E F; Carlo, W A; Devaskar, U; Wyckoff, M H; Higgins, R D
Source
Journal of Perinatology. Nov 01, 2017 37(11):1220-1223
Subject
Language
English
ISSN
0743-8346
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 weeksʼ 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.