IDEAS home Printed from https://ideas.repec.org/a/bpj/ijbist/v7y2011i1n29.html
   My bibliography  Save this article

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

Author

Listed:
  • Moyé Lemuel A
  • Lai Dejian
  • Jing Kaiyan
  • Baraniuk Mary Sarah
  • Kwak Minjung
  • Penn Marc S.
  • Wu Colon O.

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.A score function that is based on the U-statistic can address these issues of 1) intercurrent SCE’s and 2) response variable ascertainments that use different measurements of different precision. The scoring statistic is easy to apply, clinically relevant, and provides flexibility for the investigators’ prospective design decisions. Sample size and power formulations for this statistic are provided as functions of clinical event rates and effect size estimates that are easy for investigators to identify and discuss. Examples are provided from current cardiovascular cell therapy research.

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
    as

    Download full text from publisher

    File URL: https://doi.org/10.2202/1557-4679.1286
    Download Restriction: For access to full text, subscription to the journal or payment for the individual article is required.
    File URL: https://libkey.io/10.2202/1557-4679.1286?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    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.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Jay Bartroff & Jinlin Song, 2016. "A Rejection Principle for Sequential Tests of Multiple Hypotheses Controlling Familywise Error Rates," Scandinavian Journal of Statistics, Danish Society for Theoretical Statistics;Finnish Statistical Society;Norwegian Statistical Association;Swedish Statistical Association, vol. 43(1), pages 3-19, March.
    2. Liu, Aiyi & Wu, Chengqing & Yu, Kai F. & Yuan, Weishi, 2007. "Completeness and unbiased estimation of mean vector in the multivariate group sequential case," Journal of Multivariate Analysis, Elsevier, vol. 98(3), pages 505-516, March.
    3. Ajit C. Tamhane & Jiangtao Gou & Christopher Jennison & Cyrus R. Mehta & Teresa Curto, 2018. "A gatekeeping procedure to test a primary and a secondary endpoint in a group sequential design with multiple interim looks," Biometrics, The International Biometric Society, vol. 74(1), pages 40-48, March.
    4. Peng Huang & Barbara C. Tilley & Robert F. Woolson & Stuart Lipsitz, 2005. "Adjusting O'Brien's Test to Control Type I Error for the Generalized Nonparametric Behrens–Fisher Problem," Biometrics, The International Biometric Society, vol. 61(2), pages 532-539, June.
    5. W. Brannath & P. Bauer & W. Maurer & M. Posch, 2003. "Sequential Tests for Noninferiority and Superiority," Biometrics, The International Biometric Society, vol. 59(1), pages 106-114, March.
    6. Neal O. Jeffries & James F. Troendle & Nancy L. Geller, 2023. "Evaluating treatment effects in group sequential multivariate longitudinal studies with covariate adjustment," Biometrics, The International Biometric Society, vol. 79(2), pages 1496-1506, June.
    7. Taddesse Kassahun & Eshetu Wencheko & Arne C. Bathke, 2023. "Nonparametric directional testing for multivariate problems in conjunction with a closed testing principle," Statistical Methods & Applications, Springer;Società Italiana di Statistica, vol. 32(2), pages 349-363, June.
    8. Ajit C. Tamhane & Cyrus R. Mehta & Lingyun Liu, 2010. "Testing a Primary and a Secondary Endpoint in a Group Sequential Design," Biometrics, The International Biometric Society, vol. 66(4), pages 1174-1184, December.
    9. Fengqing Zhang & Jiangtao Gou, 2021. "Refined critical boundary with enhanced statistical power for non-directional two-sided tests in group sequential designs with multiple endpoints," Statistical Papers, Springer, vol. 62(3), pages 1265-1290, June.
    10. Martin Hellmich, 2001. "Monitoring Clinical Trials with Multiple Arms," Biometrics, The International Biometric Society, vol. 57(3), pages 892-898, September.
    11. Tan, Ming & Fang, Hong-Bin & Tian, Guo-Liang & Wei, Gang, 2005. "Testing multivariate normality in incomplete data of small sample size," Journal of Multivariate Analysis, Elsevier, vol. 93(1), pages 164-179, March.
    12. Neal Jeffries & Nancy L. Geller, 2015. "Longitudinal clinical trials with adaptive choice of follow-up time," Biometrics, The International Biometric Society, vol. 71(2), pages 469-477, June.

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:29. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Peter Golla (email available below). General contact details of provider: https://www.degruyter.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.