Sample size considerations for matched-pair cluster randomization design with incomplete observations of binary outcomes

Xiaohan Xu, Hong Zhu, Anh Q. Hoang, Chul Ahn

Research output: Contribution to journalArticlepeer-review


Multiple public health and medical research studies have applied matched-pair cluster randomization design to the evaluation of the intervention and/or prevention effects. One of the most common and severe problems faced by researchers when conducting cluster randomized trials (CRTs) is incomplete observations, which are associated with various reasons causing the individuals to discontinue participating in the trials. Although statistical methods to remedy the problems of missing data have already been proposed, there are still methodological gaps in research concerning the determination of sample size in matched-pair CRTs with incomplete binary outcomes. One conventional method for adjusting for missing data in the sample size determination is to divide the sample size under complete data by the expected follow-up rate. However, such crude adjustment ignores the impact of the structure and strength of correlations regarding both outcome data and missing data mechanism. This article provides a closed-form sample size formula for matched-pair CRTs with incomplete binary outcomes, which appropriately accounts for different missing patterns and magnitudes as well as the effects of matching and clustering on the outcome and missing data. The generalized estimating equation (GEE) approach treats incomplete observations as missing data in a marginal logistic regression model, which flexibly accommodates various types of intraclass correlation, missing patterns, and missing proportions. In the presence of missing data, the proposed GEE sample size method provides higher accuracy as compared with the conventional method. The performance of the proposed method is assessed by simulation studies. This article also illustrates how the proposed method can be used to design a real-world matched-pair CRT to examine the effect of a team-based approach on controlling blood pressure (BP).

Original languageEnglish (US)
Pages (from-to)5397-5416
Number of pages20
JournalStatistics in Medicine
Issue number24
StatePublished - Oct 30 2021


  • binary outcomes
  • incomplete observations
  • intraclass correlation
  • natched-pair cluster design
  • sample size

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability


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