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Children 2017, 4(6), 43; doi:10.3390/children4060043

Placebo by Proxy in Neonatal Randomized Controlled Trials: Does It Matter?

1
Department of Neonatology, University Hospital Zürich, 8091 Zürich, Switzerland
2
Department of Mathematics and Statistics, Masaryk University, 611 37 Brno, Czech Republic
3
Child Development Centre, University Children’s Hospital, 8032 Zürich, Switzerland
4
Swiss EPO Neuroprotection Trial Group (members listed in the Acknowledgements section)
*
Author to whom correspondence should be addressed.
Received: 13 April 2017 / Revised: 19 May 2017 / Accepted: 24 May 2017 / Published: 30 May 2017
(This article belongs to the Collection Development of Medicines for Paediatric and Rare Diseases - II)
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Abstract

Placebo effects emerging from the expectations of relatives, also known as placebo by proxy, have seldom been explored. The aim of this study was to investigate whether in a randomized controlled trial (RCT) there is a clinically relevant difference in long-term outcome between very preterm infants whose parents assume that verum (PAV) had been administered and very preterm infants whose parents assume that placebo (PAP) had been administered. The difference between the PAV and PAP infants with respect to the primary outcome–IQ at 5 years of age–was considered clinically irrelevant if the confidence interval (CI) for the mean difference resided within our pre-specified ±5-point equivalence margins. When adjusted for the effects of verum/placebo, socioeconomic status (SES), head circumference and sepsis, the CI was [−3.04, 5.67] points in favor of the PAV group. Consequently, our study did not show equivalence between the PAV and PAP groups, with respect to the pre-specified margins of equivalence. Therefore, our findings suggest that there is a small, but clinically irrelevant degree to which a preterm infant’s response to therapy is affected by its parents’ expectations, however, additional large-scale studies are needed to confirm this conjecture. View Full-Text
Keywords: preterm infants; placebo by proxy; long-term outcome; randomized controlled trial preterm infants; placebo by proxy; long-term outcome; randomized controlled trial
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Burkart, T.L.; Kraus, A.; Koller, B.; Natalucci, G.; Latal, B.; Fauchère, J.-C.; Bucher, H.U.; Rüegger, C.M.; for the Swiss EPO Neuroprotection Trial Group. Placebo by Proxy in Neonatal Randomized Controlled Trials: Does It Matter? Children 2017, 4, 43.

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