2023
DOI: 10.1111/biom.13920
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How to Analyze Continuous and Discrete Repeated Measures in Small-Sample Cross-Over Trials?

Johan Verbeeck,
Martin Geroldinger,
Konstantin Thiel
et al.

Abstract: To optimize the use of data from a small number of subjects in rare disease trials, an at first sight advantageous design is the repeated measures cross‐over design. However, it is unclear how these within‐treatment period and within‐subject clustered data are best analyzed in small‐sample trials. In a real‐data simulation study based upon a recent epidermolysis bullosa simplex trial using this design, we compare non‐parametric marginal models, generalized pairwise comparison models, GEE‐type models and parame… Show more

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Cited by 2 publications
(8 citation statements)
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“…“ Simulation design and performance measures ”), closely followed by unmatched prioritized GPC. It should be noted that although prioritization was based on clinical grounds, it may have led to some bias towards favoring the method by design (see Verbeeck et al [ 33 ] for more details). Matched GPC approaches resulted in lower power values than unmatched counterparts.…”
Section: Results and Recommendationsmentioning
confidence: 99%
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“…“ Simulation design and performance measures ”), closely followed by unmatched prioritized GPC. It should be noted that although prioritization was based on clinical grounds, it may have led to some bias towards favoring the method by design (see Verbeeck et al [ 33 ] for more details). Matched GPC approaches resulted in lower power values than unmatched counterparts.…”
Section: Results and Recommendationsmentioning
confidence: 99%
“…The choice of these approaches has been made by the project consortium and guided by extensive previous experience with applying these methods in the context of small samples / rare diseases. For a more detailed theoretical description of these methods, we refer to [ 14 , 33 ], and the references therein.…”
Section: Methodsmentioning
confidence: 99%
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