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. 2016 Oct;40(6):374-384.
doi: 10.1053/j.semperi.2016.05.005. Epub 2016 Jun 22.

Methodological issues in the design and analyses of neonatal research studies: Experience of the NICHD Neonatal Research Network

Abhik Das  1 , Jon Tyson  2 , Claudia Pedroza  2 , Barbara Schmidt  3 , Marie Gantz  4 , Dennis Wallace  4 , William E Truog  5 , Rosemary D Higgins  6
Affiliations

Methodological issues in the design and analyses of neonatal research studies: Experience of the NICHD Neonatal Research Network

Abhik Das et al. Semin Perinatol. 2016 Oct.

Abstract

Impressive advances in neonatology have occurred over the 30 years of life of The Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network (NRN). However, substantial room for improvement remains in investigating and further developing the evidence base for improving outcomes among the extremely premature. We discuss some of the specific methodological challenges in the statistical design and analysis of randomized trials and observational studies in this population. Challenges faced by the NRN include designing trials for unusual or rare outcomes, accounting for and explaining center variations, identifying other subgroup differences, and balancing safety and efficacy concerns between short-term hospital outcomes and longer-term neurodevelopmental outcomes. In conclusion, the constellation of unique patient characteristics in neonates calls for broad understanding and careful consideration of the issues identified in this article for conducting rigorous studies in this population.

Keywords: Causal inference; Competing outcomes; Randomization of multiples; Statistical methodology; Trial design.

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References

    1. Stoll BJ, Hansen NI, Bell EF, et al. Trends in Care Practices, Morbidity, and Mortality of Extremely Preterm Neonates, 1993–2012. JAMA. 2015;314(10):1039–1051. - PMC - PubMed
    1. Costeloe K, Hennessy E, Myles J, Draper E. EPICure 2: survival and early morbidity of extremely preterm babies in England: changes since. [Accessed March 8, 2016]; E-PAS2008:5365.1. Available at http://www.abstracts2view.com/pasall.
    1. Fischer N, Steurer MA, Adams M, Berger TM, Swiss Neonatal N. Survival rates of extremely preterm infants (gestational age <26 weeks) in Switzerland: impact of the Swiss guidelines for the care of infants born at the limit of viability. Arch Dis Child Fetal Neonatal Ed. 2009;94(6):F407–F413. - PubMed
    1. World Health Organization, March of Dimes, The Partnership for Maternal, Newborn, & Child Health, Save the Children. Geneva, Switzerland: World Health Organization; 2012. [Accessed March 8, 2016]. Born too soon: the global action report on preterm birth. http://www.who.int/pmnch/media/news/2012/201204_borntoosoon-report.pdf.
    1. Hintz SR, Kendrick DE, Wilson-Costello DE, et al. Early-childhood neurodevelopmental outcomes are not improving for infants born at <25 weeks' gestational age. Pediatrics. 2011;127(1):62–70. - PMC - PubMed

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