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Sample size determination for paired right-censored data based on the difference of Kaplan–Meier estimates

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  • Su, Pei-Fang
  • Li, Chung-I
  • Shyr, Yu

Abstract

Sample size determination is essential to planning clinical trials. Jung (2008) established a sample size calculation formula for paired right-censored data based on the logrank test, which has been well-studied for comparing independent survival outcomes. An alternative to rank-based methods for independent right-censored data, advocated by Pepe and Fleming (1989), tests for differences between integrated weighted Kaplan–Meier estimates and is more sensitive to the magnitude of difference in survival times between groups. In this paper, we employ the concept of the Pepe–Fleming method to determine an adequate sample size by calculating differences between Kaplan–Meier estimators considering pair-wise correlation. We specify a positive stable frailty model for the joint distribution of paired survival times. We evaluate the performance of the proposed method by simulation studies and investigate the impacts of the accrual times, follow-up times, loss to follow-up rate, and sensitivity of power under misspecification of the model. The results show that ignoring the pair-wise correlation results in overestimating the required sample size. Furthermore, the proposed method is applied to two real-world studies, and the R code for sample size calculation is made available to users.

Suggested Citation

  • Su, Pei-Fang & Li, Chung-I & Shyr, Yu, 2014. "Sample size determination for paired right-censored data based on the difference of Kaplan–Meier estimates," Computational Statistics & Data Analysis, Elsevier, vol. 74(C), pages 39-51.
    • Handle: RePEc:eee:csdana:v:74:y:2014:i:c:p:39-51
    DOI: 10.1016/j.csda.2013.12.006
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    References listed on IDEAS

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    1. Adin-Cristian Andrei & Susan Murray, 2005. "Simultaneous Group Sequential Analysis of Rank-Based and Weighted Kaplan–Meier Tests for Paired Censored Survival Data," Biometrics, The International Biometric Society, vol. 61(3), pages 715-720, September.
    2. Susan Murray, 2001. "Using Weighted Kaplan-Meier Statistics in Nonparametric Comparisons of Paired Censored Survival Outcomes," Biometrics, The International Biometric Society, vol. 57(2), pages 361-368, June.
    3. R.D. Gill, 1980. "Censoring and Stochastic Integrals," Statistica Neerlandica, Netherlands Society for Statistics and Operations Research, vol. 34(2), pages 124-124, June.
    4. Susan Murray, 2000. "Nonparametric Rank-Based Methods for Group Sequential Monitoring of Paired Censored Survival Data," Biometrics, The International Biometric Society, vol. 56(4), pages 984-990, December.
    5. Ronald E. Gangnon, 2004. "Sample-size formula for clustered survival data using weighted log-rank statistics," Biometrika, Biometrika Trust, vol. 91(2), pages 263-275, June.
    6. Su, Pei-Fang & Chi, Yunchan & Li, Chung-I & Shyr, Yu & Liao, Yi-De, 2011. "Analyzing survival curves at a fixed point in time for paired and clustered right-censored data," Computational Statistics & Data Analysis, Elsevier, vol. 55(4), pages 1617-1628, April.
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