ArticleStatistical methods in medical research2025
Selecting the optimal longitudinal cluster randomized design with a continuous outcome: Parallel-arm, crossover, or stepped-wedge.
Article in Statistical methods in medical research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
The optimal designs (ODs) for parallel-arm longitudinal cluster randomized trials, multiple-period cluster randomized crossover (CRXO) trials, and stepped wedge cluster randomized trials (SW-CRTs), including closed-cohort and repeat cross-sectional designs, have been studied separately under a cost-efficiency framework based on generalized estimating equations (GEEs). However, whether a global OD exists across longitudinal designs and randomization schedules remains unknown. Therefore, this research addresses a critical gap by comparing OD feature across complete longitudinal cluster randomized trial designs with two treatment conditions and continuous outcomes. We define the OD as the design with either the lowest cost to obtain a desired level of power or the largest power given a fixed budget. For each of these ODs, we obtain the optimal number of clusters and the optimal cluster-period size (number of participants per cluster per period). To ensure equitable comparisons, we consider the GEE treatment effect estimator with the same block exchangeable correlation structure and develop OD algorithms with the lowest cost for each of six study designs. To obtain OD with the largest power, we summarize the previous and propose new OD algorithms and formulae. We suggest using the number of treatment sequences
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