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
Department of Pediatrics/Neonatology, Children's Hospital of New Mexico, University of New Mexico Health Sciences Center, Albuquerque, NM, USA.
Department of Pediatrics, Rainbow Babies & Children's Hospital, Case Western Reserve University, Cleveland, OH, USA.
J Perinatol. 2017 Nov;37(11):1220-1223. doi: 10.1038/jp.2017.131. Epub 2017 Sep 7.
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.
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.
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.
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.
分析一项关于氢化可的松对足月儿/晚期早产儿心血管功能不全伴无神经发育障碍（NDI）存活影响的随机对照试验（RCT）入组率低的原因。
原研究为一项多中心RCT。纳入标准：孕龄⩾34周、年龄<72小时、机械通气、接受血管活性药物治疗。主要结局为2岁时的NDI；排除有NDI高风险诊断的婴儿。本文对患者入组率低的原因进行分析。
932名其他方面符合条件的婴儿中有257名接受了血管活性药物治疗；然而，207名（81%）有排除性诊断。在10个月期间仅有12名婴儿被随机分组；因此，该研究终止。促成因素包括排除后符合条件的婴儿数量少、开放标签的类固醇治疗以及入组窗口狭窄。
尽管进行了一项观察性研究以估计总体人群，但入组的婴儿极少。紧急治疗的成功RCT可能需要减少排除标准、采用短期主要结局、放弃知情同意和/或其他替代方法。
