Sample Size Method and Software Development in Survival Trial with a Cure Rate

治愈率生存试验中的样本量方法和软件开发

• 批准号: 7897375
• 负责人: Jiajia Zhang
• 金额: $ 8.46万
• 依托单位: UNIVERSITY OF SOUTH CAROLINA AT COLUMBIA
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-07-01 至 2012-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7897375
• 关键词: Admixture; Biological; Budgets; Clinical Trials; Cohort Studies; Complex; Computer software; Cox Proportional Hazards Models; Data; Drops; Epidemiology; Event; Future; Grant; Knowledge; Length; Logistic Regressions; Malignant Neoplasms; Manuals; Methods; Modeling; Patients; Property; Proportional Hazards Models; Randomized Controlled Clinical Trials; Research Design; Research Personnel; Risk; Sample Size; Sampling; Statistical Methods; Study Subject; Survival Analysis; Testing; Time; abstracting; base; design; experience; flexibility; follow-up; interest; public health relevance; software development; sound; tool; user friendly software

DESCRIPTION (provided by applicant): Abstract One of the crucial issues in designing randomized controlled clinical trials is to calculate sample size needed in the study, which is more complicated in survival trial. The existing method and software for the sample size calculation in survival analysis only consider the survival trial without a cure rate. To the best of our knowledge, no existing method and software can provide the power and sample size calculation when there is a cure rate in the treatment. In order to fill in this gap, we propose the statistically sound method for the sample size calculation in the mixture cure model, which include the existing sample size method based on the proportional hazards model as a special case, and we will develop the user friendly software in R during this grant period with a complete user manual. Therefore, the proposed method will provide a flexible tool for investigators to explore the optimal sample size in survival trial. The user friendly software solves the computational issue in the sample size calculation, and thus the researchers and practitioners can apply the proposed method in their study easily. PUBLIC HEALTH RELEVANCE: Project Narrative This project will provide a flexible tool for investigators to explore the optimal sample size in survival trial with or without a cure rate, and the user friendly software solves the computational issues in the sample size calculation from the proposed method. Thus the researchers and practitioners can apply the proposed method in their study easily.

描述（由申请人提供）：摘要设计随机对照临床试验的关键问题之一是计算研究中所需的样本量，这在生存试验中更为复杂。生存分析中样本量计算的现有方法和软件仅考虑无需治愈率即可进行生存试验。据我们所知，当治疗率有治愈率时，现有的方法和软件都无法提供功率和样本量计算。为了填补此差距，我们建议在混合固化模型中使用统计上的样本量计算方法，其中包括基于比例危害模型的现有样本量方法作为特殊情况，我们将在此赠款期间使用完整的用户手册在此拨款期间开发用户友好的软件。因此，所提出的方法将为研究人员提供一种灵活的工具，以探索生存试验中最佳样本量。用户友好的软件在样本量计算中解决了计算问题，因此，研究人员和从业者可以轻松地在其研究中应用所提出的方法。 公共卫生相关性：项目叙述该项目将为调查人员提供一个灵活的工具，以探索具有或不治愈率的生存试验中最佳样本量，并且用户友好的软件可以从建议的方法中解决样本量计算中的计算问题。因此，研究人员和从业人员可以轻松地在其研究中应用所提出的方法。