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Designing exploratory cancer trials using change in tumour size as primary endpoint

Jaki, Thomas and Andre, Valerie and Su, Ting-Li and Whitehead, John (2013) Designing exploratory cancer trials using change in tumour size as primary endpoint. Statistics in Medicine, 32 (15). pp. 2544-2554. ISSN 0277-6715

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    Abstract

    In phase III cancer clinical trials, overall survival is commonly used as the definitive endpoint. In phase II clinical trials, however, more immediate endpoints such as incidence of complete or partial response within 1 or 2 months or progression-free survival (PFS) are generally used. Because of the limited ability to detect change in overall survival with response, the inherent variability of PFS and the long wait for progression to be observed, more informative and immediate alternatives to overall survival are desirable in exploratory phase II trials. In this paper, we show how comparative trials can be designed and analysed using change in tumour size as the primary endpoint. The test developed is based on the framework of score statistics and will formally incorporate the information of whether patients survive until the time at which change in tumour size is assessed. Using an example in non-small cell lung cancer, we show that the sample size requirements for a trial based on change in tumour size are favourable compared with alternative randomized trials and demonstrate that these conclusions are robust to our assumptions.

    Item Type: Article
    Journal or Publication Title: Statistics in Medicine
    Additional Information: The definitive version is available at www3.interscience.wiley.com
    Uncontrolled Keywords: cancer ; tumour size ; phase II ; design ; score statistic
    Subjects:
    Departments: Faculty of Science and Technology > Mathematics and Statistics
    ID Code: 60471
    Deposited By: ep_importer_pure
    Deposited On: 06 Dec 2012 11:29
    Refereed?: Yes
    Published?: Published
    Last Modified: 09 Oct 2013 14:51
    Identification Number:
    URI: http://eprints.lancs.ac.uk/id/eprint/60471

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