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

K. L. Watterberg, E. Fernandez, M. C. Walsh, W. E. Truog, B. J. Stoll, G. M. Sokol, K. A. Kennedy, M. V. Fraga, S. S. Beauman, B. Carper, A. Das, A. F. Duncan, W. F. Buss, C. Gauldin, C. B. Lacy, P. J. Sanchez, S. Chawla, Satyanarayana Lakshminrusimha, C. M. Cotten, K. P. Van MeursB. B. Poindexter, E. F. Bell, W. A. Carlo, U. Devaskar, M. H. Wyckoff, R. D. Higgins

Research output: Contribution to journalArticle

3 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)1220-1223
Number of pages4
JournalJournal of Perinatology
Volume37
Issue number11
DOIs
StatePublished - Nov 1 2017
Externally publishedYes

Fingerprint

Premature Infants
Hydrocortisone
Randomized Controlled Trials
Newborn Infant
Observational Studies
Steroids
Pregnancy
Survival
Therapeutics
Population

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Obstetrics and Gynecology

Cite this

Watterberg, K. L., Fernandez, E., Walsh, M. C., Truog, W. E., Stoll, B. J., Sokol, G. M., ... Higgins, R. D. (2017). Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants. Journal of Perinatology, 37(11), 1220-1223. https://doi.org/10.1038/jp.2017.131

Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants. / 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, Satyanarayana; Cotten, C. M.; Van Meurs, K. P.; Poindexter, B. B.; Bell, E. F.; Carlo, W. A.; Devaskar, U.; Wyckoff, M. H.; Higgins, R. D.

In: Journal of Perinatology, Vol. 37, No. 11, 01.11.2017, p. 1220-1223.

Research output: Contribution to journalArticle

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 2017, 'Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants', Journal of Perinatology, vol. 37, no. 11, pp. 1220-1223. https://doi.org/10.1038/jp.2017.131
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, Satyanarayana ; Cotten, C. M. ; Van Meurs, K. P. ; Poindexter, B. B. ; Bell, E. F. ; Carlo, W. A. ; Devaskar, U. ; Wyckoff, M. H. ; Higgins, R. D. / Barriers to enrollment in a randomized controlled trial of hydrocortisone for cardiovascular insufficiency in term and late preterm newborn infants. In: Journal of Perinatology. 2017 ; Vol. 37, No. 11. pp. 1220-1223.
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AU - Watterberg, K. L.

AU - Fernandez, E.

AU - Walsh, M. C.

AU - Truog, W. E.

AU - Stoll, B. J.

AU - Sokol, G. M.

AU - Kennedy, K. A.

AU - Fraga, M. V.

AU - Beauman, S. S.

AU - Carper, B.

AU - Das, A.

AU - Duncan, A. F.

AU - Buss, W. F.

AU - Gauldin, C.

AU - Lacy, C. B.

AU - Sanchez, P. J.

AU - Chawla, S.

AU - Lakshminrusimha, Satyanarayana

AU - Cotten, C. M.

AU - Van Meurs, K. P.

AU - Poindexter, B. B.

AU - Bell, E. F.

AU - Carlo, W. A.

AU - Devaskar, U.

AU - Wyckoff, M. H.

AU - Higgins, R. D.

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N2 - 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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