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.

随机对照临床试验设计的关键问题之一是研究所需样本量的计算，生存期试验的样本量计算更为复杂。生存分析中现有的样本量计算方法和软件只考虑生存试验，没有考虑治愈率。据我们所知，当治疗中存在治愈率时，现有方法和软件无法提供把握度和样本量计算。为了填补这一空白，我们提出了统计上合理的方法来计算混合物固化模型中的样本量，其中包括现有的样本量方法的基础上的比例风险模型作为一个特例，我们将开发用户友好的软件在R在此授权期间与一个完整的用户手册。因此，所提出的方法将提供一个灵活的工具，研究者探索生存试验的最佳样本量。用户友好的软件解决了样本量计算中的计算问题，因此研究人员和从业人员可以很容易地将所提出的方法应用于他们的研究中。 公共卫生关系：该项目将为研究者提供一个灵活的工具，以探索生存试验中的最佳样本量，无论是否有治愈率，用户友好的软件解决了样本量计算中的计算问题。因此，研究人员和实践者可以很容易地应用所提出的方法在他们的研究。