Statistical Methods for Evaluation and Validation of Diagnostic Tests

诊断测试评估和验证的统计方法

• 批准号: 7216723
• 负责人: YING LU
• 金额: $ 21.82万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2009-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7216723
• 关键词: Address; Advisory Committees; Ancillary Study; Area; Background Radiation; Biochemical; Bone Density; Case-Control Studies; Clinical; Clinical Research; Clinical Trials; Cohort Studies; Collaborations; Computer software; Condition; Cross-Sectional Studies; Data; Development; Diagnosis; Diagnostic; Diagnostic Procedure; Diagnostic tests; Disease; Disease Outcome; Economics; End Point; Evaluation; Evaluation Studies; Evolution; Faculty; Follow-Up Studies; Goals; Hip region structure; Imaging technology; Inferior; Journals; Length; Manuals; Measurement; Measures; Medical; Methods; Modality; Modeling; Numbers; Odds Ratio; Osteoporosis; Outcome; Paper; Patients; Peripheral; Physicians; Play; Positioning Attribute; Principal Investigator; Procedures; Process; Professional Organizations; Prospective Studies; Publications; Publishing; Radiology Specialty; Rare Diseases; Receiver Operating Characteristics; Research; Research Personnel; Risk; Roentgen Rays; Role; Site; Statistical Methods; Subgroup; Techniques; Testing; Therapy Clinical Trials; Translating; Treatment Efficacy; Ultrasonography; Validation; Vertebral column; Visit; Woman; Work; anticancer research; base; bone; breast density; case control; cohort; computer code; concept; cost; design; diagnostic accuracy; follow-up; improved; innovation; lectures; malignant breast neoplasm; mathematical theory; novel diagnostics; osteoporosis with pathological fracture; outcome forecast; outreach; programs; prospective; radiologist; rapid technique; research and development; simulation; statistics; tumor; user friendly software

DESCRIPTION (provided by applicant): This multi-disciplinary effort by biostatisticians, radiologists, and medical physicists aims to develop innovative statistical methods for evaluation and validation of new and low cost diagnostic modalities. Specific Aims are (1) statistical methods for determining non-inferiority of diagnostic techniques; (2) statistical design and analysis methods for rapid and accurate evaluation of the chronological association and treatment efficacy of new radiological markers with disease outcome; (3) applying the statistical methods to clinical research and developing user-friendly software. Specific Aim 1 studies evaluation of non- inferiority for a new diagnostic test based on the receiver operating characteristics curve under various conditions (missing data, mixed data types, and clustered data), effects of covariates (non-inferiority after adjustment of covariates and identification of subgroups within which the new test is inferior, superior or equivalent), and Bayesian utility models. Specific Aim 2 studies a new cross-sectional case-control short- term follow-up (CSCCSTFU) design and analysis methods to estimate the chronological odds ratio of a new diagnostic test for a rare outcome. It will also propose methods to estimate the lower boundary of treatment efficacy for patients diagnosed by the new test. Mathematical theory and simulation studies will be used to understand the conditions in which the estimated odds ratio of the CSCCSTFU design can be generalized. Specific Aim 3 will apply results from Specific Aim 1 to data from the Study of Osteoporotic Fractures and the Osteoporosis and Ultrasound Study to test whether and when quantitative ultrasound, peripheral bone mineral density (BMD), and biochemical bone markers can serve as non-inferior or equivalent techniques to BMD of hip and spine for assessing osteoporotic fracture risks. It will also use results of Specific Aim 2 to determine whether the volumetric breast density and structural parameters of the new single X-ray absorptiometry technique can prospectively predict breast cancer in an on-going study of 15,000 women. An advisory committee of internationally recognized leaders in osteoporosis and breast cancer research will advise the research team on outreach to physicians and opportunities for ancillary studies. Research results will be disseminated through publication of software, research and tutorial papers in both clinical and statistical journals and by influencing position papers of professional organizations.

描述（由申请人提供）：生物统计学家、放射科医生和医学物理学家的多学科努力旨在开发创新的统计方法，用于评估和验证新的低成本诊断模式。具体目的是（1）确定诊断技术非劣效性的统计方法;（2）快速准确评价新放射学标志物与疾病结局的时间关联和治疗效果的统计设计和分析方法;（3）将统计方法应用于临床研究并开发用户友好的软件。具体目标1研究了基于各种条件下的受试者工作特征曲线（缺失数据、混合数据类型和聚类数据）、协变量效应（调整协变量后的非劣效性，以及确定新检测为劣效、上级或等效的亚组）和贝叶斯效用模型的新诊断检测的非劣效性评价。特定目标2研究了一种新的横断面病例对照短期随访（CSCCSTFU）设计和分析方法，以估计罕见结局的新诊断测试的时间优势比。它还将提出方法来估计通过新测试诊断的患者的治疗效果的下限。数学理论和模拟研究将用于了解CSCCSTFU设计的估计比值比可以推广的条件。具体目标3将具体目标1的结果应用于骨质疏松性骨折研究和骨质疏松与超声研究的数据，以测试定量超声、外周骨矿物质密度（BMD）和生化骨标志物是否以及何时可作为评估骨质疏松性骨折风险的髋部和脊柱BMD的非劣效或等效技术。它还将使用特定目标2的结果来确定新的单X射线吸收测量技术的体积乳腺密度和结构参数是否可以在一项对15，000名妇女的持续研究中前瞻性地预测乳腺癌。一个由国际公认的骨质疏松症和乳腺癌研究领导人组成的咨询委员会将向研究小组提供咨询意见，以便与医生联系并提供辅助研究的机会。将通过在临床和统计期刊上发表软件、研究和指导论文以及影响专业组织的立场文件来传播研究成果。