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

Title
Combining Censored and Uncensored Data in a U-Statistic: Design and Sample Size Implications for Cell Therapy Research
Author(s)
곽민정Lemuel A. Moye[Lemuel A. Moye]Dejian Lai[Dejian Lai]Kaiyan Jing[Kaiyan Jing]Sarah Baraniuk[Sarah Baraniuk]Marc Penn[Marc Penn]Colin Wu[Colin Wu]
Keywords
ACUTE MYOCARDIAL-INFARCTION; MULTIPLE END-POINTS; VENTRICULAR ENLARGEMENT TRIAL; SEQUENTIAL CLINICAL-TRIALS; HEMATOPOIETIC STEM-CELLS; BONE-MARROW-CELLS; HEART-FAILURE; INTRACORONARY INJECTION; ISCHEMIC-MYOCARDIUM; MONONUCLEAR-CELLS
Issue Date
201107
Publisher
BERKELEY ELECTRONIC PRESS
Citation
INTERNATIONAL JOURNAL OF BIOSTATISTICS, v.7, no.1
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.
URI
http://hdl.handle.net/YU.REPOSITORY/24854http://dx.doi.org/10.2202/1557-4679.1286
ISSN
1557-4679
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이과대학 > 통계학과 > Articles
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