Analysis of Case-Control Follow-Up Studies

病例对照随访研究分析

• 批准号: 7123092
• 负责人: Marshall M Joffe
• 金额: $ 16.89万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-08-09 至 2008-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7123092
• 关键词: bias; clinical research; computer data analysis; computer simulation; diagnosis design /evaluation; disease /disorder proneness /risk; experimental designs; human data; kidney disorder; longitudinal human study; mammography; method development; pathologic process; spontaneous abortion; statistics /biometry

DESCRIPTION (provided by applicant): The case-control follow-up (CCFU) study is a hybrid design, a combination of case control and cohort or follow-up studies. In such studies, a cohort is monitored for early or sentinels events, which are closely associated with some outcome of interest (e.g., diagnosis of breast cancer); information is obtained on all subjects developing the sentinel events, and on a sample of the remaining subjects. Subjects developing the sentinel event (and sometimes other subjects) are then followed prospectively for an event of interest (e.g., death from breast cancer). The design has been employed in studies of the efficacy of cancer screening and of spontaneous abortion but has generally been mistaken for a case-control design. The purpose of this project is to advance understanding of the design and improve inference derived from it. This will be done by 1) developing a general notation and theory for describing these studies; 2) applying and developing appropriate statistical methods for analyzing data arising from these studies; and 3) comparing various approaches for analyzing these studies. We have identified two main general statistical approaches for these studies: one we term the direct approach and the other we call the synthetic approach. We propose to elaborate these approaches, and then consider how to deal with biases that may arise in each of them. New and familiar methods will be assessed through simulation, through application to several CCFU studies, and through artificial generation of CCFU studies by sampling from cohort studies.

描述（由申请人提供）：病例对照随访（CCFU）研究是一种混合设计，结合了病例对照和队列或随访研究。在这类研究中，监测一个队列的早期或哨兵事件，这些事件与一些感兴趣的结果密切相关（例如，乳腺癌的诊断）；获取所有发生前哨事件的受试者的信息，以及剩余受试者的样本信息。发生前哨事件的受试者（有时还有其他受试者）随后被前瞻性地跟踪观察感兴趣的事件（例如，死于乳腺癌）。

项目成果

Extended instrumental variables estimation for overall effects.

• DOI: 10.2202/1557-4679.1082
• 发表时间: 2008-04-07
• 期刊: The international journal of biostatistics
• 作者: Joffe, Marshall M;Small, Dylan;Feldman, Harold I
• 通讯作者: Feldman, Harold I

Empirical Efficiency Maximization: Improved Locally Efficient Covariate Adjustment in Randomized Experiments and Survival Analysis

• DOI: 10.2202/1557-4679.1084
• 发表时间: 2008-05
• 期刊: The International Journal of Biostatistics
• 作者: D. Rubin;M. J. van der Laan