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Combining Censored and Uncensored Data in a U-Statistic: Design and Sample Size Implications for Cell Therapy Research

Author

Listed:
  • Moyé Lemuel A

    (University of Texas Health Science Center at Houston)

  • Lai Dejian

    (University of Texas School of Public Health)

  • Jing Kaiyan

    (University of Texas School of Public Health)

  • Baraniuk Mary Sarah

    (University of Texas School of Public Health)

  • Kwak Minjung

    (National Heart, Lung and Blood Institute)

  • Penn Marc S.

    (The Cleveland Clinic Foundation)

  • Wu Colon O.

    (National Heart, Lung and Blood Institute)

Abstract

The assumptions that anchor large clinical trials are rooted in smaller, Phase II studies. In addition to specifying the target population, intervention delivery, and patient follow-up duration, physician-scientists who design these Phase II studies must select the appropriate response variables (endpoints). However, endpoint measures can be problematic. If the endpoint assesses the change in a continuous measure over time, then the occurrence of an intervening significant clinical event (SCE), such as death, can preclude the follow-up measurement. Finally, the ideal continuous endpoint measurement may be contraindicated in a fraction of the study patients, a change that requires a less precise substitution in this subset of participants.

Suggested Citation

  • Moyé Lemuel A & Lai Dejian & Jing Kaiyan & Baraniuk Mary Sarah & Kwak Minjung & Penn Marc S. & Wu Colon O., 2011. "Combining Censored and Uncensored Data in a U-Statistic: Design and Sample Size Implications for Cell Therapy Research," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-29, July.
    • Handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:29
    DOI: 10.2202/1557-4679.1286
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    References listed on IDEAS

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    1. Ming Tan & Hong-Bin Fang & Guo-Liang Tian & Peter J. Houghton, 2002. "Small-Sample Inference for Incomplete Longitudinal Data with Truncation and Censoring in Tumor Xenograft Models," Biometrics, The International Biometric Society, vol. 58(3), pages 612-620, September.
    2. Dei-In Tang & Nancy L. Geller, 1999. "Closed Testing Procedures for Group Sequential Clinical Trials with Multiple Endpoints," Biometrics, The International Biometric Society, vol. 55(4), pages 1188-1192, December.
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