PMID- 25004909
OWN - NLM
STAT- MEDLINE
DCOM- 20150331
LR  - 20211021
IS  - 1745-6215 (Electronic)
IS  - 1745-6215 (Linking)
VI  - 15
DP  - 2014 Jul 8
TI  - Sample size calculations in pediatric clinical trials conducted in an ICU: a 
      systematic review.
PG  - 274
LID - 10.1186/1745-6215-15-274 [doi]
AB  - At the design stage of a clinical trial, several assumptions have to be made. 
      These usually include guesses about parameters that are not of direct interest 
      but must be accounted for in the analysis of the treatment effect and also in the 
      sample size calculation (nuisance parameters, e.g. the standard deviation or the 
      control group event rate). We conducted a systematic review to investigate the 
      impact of misspecification of nuisance parameters in pediatric randomized 
      controlled trials conducted in intensive care units. We searched MEDLINE through 
      PubMed. We included all publications concerning two-arm RCTs where efficacy 
      assessment was the main objective. We included trials with pharmacological 
      interventions. Only trials with a dichotomous or a continuous outcome were 
      included. This led to the inclusion of 70 articles describing 71 trials. In 49 
      trial reports a sample size calculation was reported. Relative misspecification 
      could be calculated for 28 trials, 22 with a dichotomous and 6 with a continuous 
      primary outcome. The median [inter-quartile range (IQR)] overestimation was 6.9 
      [-12.1, 57.8]% for the control group event rate in trials with dichotomous 
      outcomes and -1.5 [-15.3, 5.1]% for the standard deviation in trials with 
      continuous outcomes. Our results show that there is room for improvement in the 
      clear reporting of sample size calculations in pediatric clinical trials 
      conducted in ICUs. Researchers should be aware of the importance of nuisance 
      parameters in study design and in the interpretation of the results.
FAU - Nikolakopoulos, Stavros
AU  - Nikolakopoulos S
AD  - Department of Biostatistics, Julius Center for Health Sciences and Primary Care, 
      University Medical Center Utrecht, Str, 6,131, PO Box 85500, 3508 Utrecht, GA, 
      The Netherlands. S.N.Nikolakopoulos@umcutrecht.nl.
FAU - Roes, Kit C B
AU  - Roes KC
FAU - van der Lee, Johanna H
AU  - van der Lee JH
FAU - van der Tweel, Ingeborg
AU  - van der Tweel I
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PT  - Systematic Review
DEP - 20140708
PL  - England
TA  - Trials
JT  - Trials
JID - 101263253
SB  - IM
MH  - Clinical Trials as Topic/*methods/statistics & numerical data
MH  - *Critical Care/statistics & numerical data
MH  - Data Interpretation, Statistical
MH  - Humans
MH  - Infant
MH  - Infant, Newborn
MH  - Intensive Care Units, Neonatal
MH  - *Intensive Care Units, Pediatric/statistics & numerical data
MH  - Intensive Care, Neonatal
MH  - Models, Statistical
MH  - *Sample Size
PMC - PMC4107993
EDAT- 2014/07/10 06:00
MHDA- 2015/04/01 06:00
PMCR- 2014/07/08
CRDT- 2014/07/10 06:00
PHST- 2014/01/21 00:00 [received]
PHST- 2014/06/24 00:00 [accepted]
PHST- 2014/07/10 06:00 [entrez]
PHST- 2014/07/10 06:00 [pubmed]
PHST- 2015/04/01 06:00 [medline]
PHST- 2014/07/08 00:00 [pmc-release]
AID - 1745-6215-15-274 [pii]
AID - 10.1186/1745-6215-15-274 [doi]
PST - epublish
SO  - Trials. 2014 Jul 8;15:274. doi: 10.1186/1745-6215-15-274.