LymphedemaV1, Main, Exploration, bibRecord, 002E67

A Comparison of Four Methods for the Analysis of N-of-1 Trials

Identifieur interne : 002E67 ( Main/Exploration ); précédent : 002E66; suivant : 002E68

A Comparison of Four Methods for the Analysis of N-of-1 Trials

Auteurs : Xinlin Chen ; Pingyan Chen

Source :

PLoS ONE [ 1932-6203 ] ; 2014.

RBID : PMC:3913644

Abstract

Objective

To provide a practical guidance for the analysis of N-of-1 trials by comparing four commonly used models.

Methods

The four models, paired t-test, mixed effects model of difference, mixed effects model and meta-analysis of summary data were compared using a simulation study. The assumed 3-cycles and 4-cycles N-of-1 trials were set with sample sizes of 1, 3, 5, 10, 20 and 30 respectively under normally distributed assumption. The data were generated based on variance-covariance matrix under the assumption of (i) compound symmetry structure or first-order autoregressive structure, and (ii) no carryover effect or 20% carryover effect. Type I error, power, bias (mean error), and mean square error (MSE) of effect differences between two groups were used to evaluate the performance of the four models.

Results

The results from the 3-cycles and 4-cycles N-of-1 trials were comparable with respect to type I error, power, bias and MSE. Paired t-test yielded type I error near to the nominal level, higher power, comparable bias and small MSE, whether there was carryover effect or not. Compared with paired t-test, mixed effects model produced similar size of type I error, smaller bias, but lower power and bigger MSE. Mixed effects model of difference and meta-analysis of summary data yielded type I error far from the nominal level, low power, and large bias and MSE irrespective of the presence or absence of carryover effect.

Conclusion

We recommended paired t-test to be used for normally distributed data of N-of-1 trials because of its optimal statistical performance. In the presence of carryover effects, mixed effects model could be used as an alternative.

Url:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3913644

DOI: 10.1371/journal.pone.0087752
PubMed: 24503561
PubMed Central: 3913644

Affiliations:

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<p>To provide a practical guidance for the analysis of N-of-1 trials by comparing four commonly used models.</p>
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<sec><title>Methods</title>
<p>The four models, paired t-test, mixed effects model of difference, mixed effects model and meta-analysis of summary data were compared using a simulation study. The assumed 3-cycles and 4-cycles N-of-1 trials were set with sample sizes of 1, 3, 5, 10, 20 and 30 respectively under normally distributed assumption. The data were generated based on variance-covariance matrix under the assumption of (i) compound symmetry structure or first-order autoregressive structure, and (ii) no carryover effect or 20% carryover effect. Type I error, power, bias (mean error), and mean square error (MSE) of effect differences between two groups were used to evaluate the performance of the four models.</p>
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<p>The results from the 3-cycles and 4-cycles N-of-1 trials were comparable with respect to type I error, power, bias and MSE. Paired t-test yielded type I error near to the nominal level, higher power, comparable bias and small MSE, whether there was carryover effect or not. Compared with paired t-test, mixed effects model produced similar size of type I error, smaller bias, but lower power and bigger MSE. Mixed effects model of difference and meta-analysis of summary data yielded type I error far from the nominal level, low power, and large bias and MSE irrespective of the presence or absence of carryover effect.</p>
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<p>We recommended paired t-test to be used for normally distributed data of N-of-1 trials because of its optimal statistical performance. In the presence of carryover effects, mixed effects model could be used as an alternative.</p>
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A Comparison of Four Methods for the Analysis of N-of-1 Trials

A Comparison of Four Methods for the Analysis of N-of-1 Trials

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