Accounting for Multiple Births in Neonatal and Perinatal Trials: Systematic Review and Case Study - 07/03/14
, Dennis Black, PhD b, Lisa Palermo, MA b, Avital Cnaan, PhD c, Xianqun Luan, MS d, William E. Truog, MD e, Michele C. Walsh, MD, MS a, Roberta A. Ballard, MD f
Reprint requests: Dr Anna Maria Hibbs, Division of Neonatology, Rainbow Babies and Children’s Hospital, 11100 Euclid Avenue, Suite 3100, Cleveland, OH 44106.Abstract |
Objectives |
To determine the prevalence in the neonatal literature of statistical approaches accounting for the unique clustering patterns of multiple births and to explore the sensitivity of an actual trial to several analytic approaches to multiples.
Study design |
A systematic review of recent perinatal trials assessed the prevalence of studies accounting for clustering of multiples. The Nitric Oxide to Prevent Chronic Lung Disease (NO CLD) trial served as a case study of the sensitivity of the outcome to several statistical strategies. We calculated odds ratios using nonclustered (logistic regression) and clustered (generalized estimating equations, multiple outputation) analyses.
Results |
In the systematic review, most studies did not describe the random assignment of twins and did not account for clustering. Of those studies that did, exclusion of multiples and generalized estimating equations were the most common strategies. The NO CLD study included 84 infants with a sibling enrolled in the study. Multiples were more likely than singletons to be white and were born to older mothers (P < .01). Analyses that accounted for clustering were statistically significant; analyses assuming independence were not.
Conclusions |
The statistical approach to multiples can influence the odds ratio and width of confidence intervals, thereby affecting the interpretation of a study outcome. A minority of perinatal studies address this issue.
Le texte complet de cet article est disponible en PDF.Mots-clés : NO CLD
Plan
| Supported by National Institutes of Health grant K23-HD056299. The NO CLD trial has been used as a case study. Disclosures with regard to that study are as follows: Supported by grants from the National Institutes of Health (U01-HL62514, P50-HL56401, P30-HD26979, P30-MRDDRC, and P30-HD26979) and the General Clinical Research Centers Program (M01-RR00240, M01-RR00084, M01-RR00425, M01-RR001271, M01-RR00064, and M01-RR00080). IKARIA (formerly INO Therapeutics) provided study gas and masked delivery systems for the primary NO CLD trial. M.C.W. and R.A.B. received support from IKARIA to fund completion of 24-month follow-up and data analysis. A.M.H. received reimbursement for travel to investigator meetings as part of these grants. A trainee in the Division of Neonatology, Rainbow Babies and Children’s Hospital, received grant support from IKARIA. IKARIA did not play any role in the design, analysis, interpretation, or reporting of the study. |
Vol 156 - N° 2
P. 202-208 - février 2010 Retour au numéro
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