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

• Type of Material: Article
• Authors: Kristi L. Watterberg, University of New Mexico; Erika Fernandez, University of New Mexico; Michele C. Walsh, Case Western Reserve University; William E. Truog, Children's Mercy Hospital; Barbara Stoll, Emory University; Gregory M. Sokol, Indiana University School of Medicine; Kathleen A. Kennedy, University of Texas Health Science Center at Houston; Maria V. Fraga, University of Pennsylvania; Sandra Sundquist Beauman, University of New Mexico; Benjamin Carper, RTI International; Abhik Das, RTI International; Andrea Freeman Duncan, University of Texas Health Science Center at Houston; William F. Buss, Indiana University Hospital; Cheri Gauldin, Children's Mercy Hospital; Conra Baackstrom Lacy, University of New Mexico; Pablo J. Sanchez, Nationwide Childrens Hospital; Sanjay Chawla, Wayne State University; Satyan Lakshminrusimha, University of Buffalo; C. Michael Cotten, Duke University; Krisa P. Van Meurs, Stanford University; Brenda B. Poindexter, Cincinnati Children's Hospital Medical Center; Edward F. Bell, University of Iowa; Waldemar A. Carlo, University of Alabama Birmingham; Uday Devaskar, University of California Los Angeles; Myra H. Wyckoff, University of Texas Southwestern Medical Center of Dallas; Rosemary D. Higgins, Eunice Kennedy Shriver National Institute of Child Health and Human Development
• Language: English
• Date: 2017-11-01
• Publisher: Nature Publishing Group: Open Access Hybrid Model Option B
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2017 Nature America, Inc., part of Springer Nature. All rights reserved.
• Final Published Version (URL): http://dx.doi.org/10.1038/jp.2017.131
• Title of Journal or Parent Work: Journal of Perinatology
• ISSN: 0743-8346
• Volume: 37
• Issue: 11
• Start Page: 1220
• End Page: 1223
• Keywords: Pediatrics; CORTISOL RESPONSE; HYPOXIC RESPIRATORY-FAILURE; ACTH; Life Sciences & Biomedicine; OUTCOMES; Science & Technology; NITRIC-OXIDE THERAPY; Obstetrics & Gynecology; HYPOTENSION
• Research Categories: Health Sciences, Medicine and Surgery; Health Sciences, Human Development

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.