Analyzing and Interpreting PRO-CTCAE with CTCAE and Other Clinical Data to Characterize Drug Tolerability

使用 CTCAE 和其他临床数据分析和解释 PRO-CTCAE，以表征药物耐受性

• 批准号: 10884103
• 负责人: ETHAN M. BASCH
• 金额: $ 26.92万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-08 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10884103
• 关键词: Adverse event; Benefits and Risks; Clinical Data; Clinical Trials; Code; Collaborations; Common Terminology Criteria for Adverse Events; Communication; Contracts; Data; Data Adjustments; Data Set; Development; Diarrhea; Dose; Drug Evaluation; Equilibrium; Evaluation; Grant; Hospitalization; Industry; Libraries; Measures; Methods; National Cancer Institute; National Clinical Trials Network; Nausea; Participant; Patient Outcomes Assessments; Patient Self-Report; Patients; Performance Status; Pharmaceutical Preparations; Process; Reporting; Research Personnel; Role; Standardization; Statistical Methods; Symptoms; Techniques; Testing; Time; Toxic effect; Work; cancer clinical trial; data sharing; data visualization; experience; flexibility; member; multidisciplinary; pandemic disease; patient oriented; side effect; symptomatology; tool; Adverse event; Benefits and Risks; Clinical Data; Clinical Trials; Code; Collaborations; Common Terminology Criteria for Adverse Events; Communication; Contracts; Data; Data Adjustments; Data Set; Development; Diarrhea; Dose; Drug Evaluation; Equilibrium; Evaluation; Grant; Hospitalization; Industry; Libraries; Measures; Methods; National Cancer Institute; National Clinical Trials Network; Nausea; Participant; Patient Outcomes Assessments; Patient Self-Report; Patients; Performance Status; Pharmaceutical Preparations; Process; Reporting; Research Personnel; Role; Standardization; Statistical Methods; Symptoms; Techniques; Testing; Time; Toxic effect; Work; cancer clinical trial; data sharing; data visualization; experience; flexibility; member; multidisciplinary; pandemic disease; patient oriented; side effect; symptomatology; tool

PROJECT ABSTRACT In cancer clinical trials, it is essential to understand and communicate the negative impact of drugs on patient participants in order to balance risks and benefits. Symptom side effects such as nausea and diarrhea are particularly common and impactful on the patient experience. However, historically this information has been reported by clinicians on behalf of their patients rather than by patients themselves – a process that has been shown to miss up to half of patients' symptoms. Therefore, the National Cancer Institute (NCI) supported development of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), a library of questions to enable systematic patient self-reporting of side effect information. To date, the PRO-CTCAE questions have been developed and tested for validity and feasibility of use in clinical trials – work that was led by the investigators on this proposal under contracts to the NCI. However, it is not yet established how best to combine PRO-CTCAE information with traditional measures of treatment impact such as clinician-reported adverse events, time on treatment, drug dose levels, performance status, and hospitalizations. The purpose of this supplement is to complete work towards understanding the role of the PRO-CTCAE in combination with these traditional measures of treatment impact in assessments of drug tolerability. In this supplement, advanced statistical methods will be used to analyze data from newly available clinical trials, delayed by the pandemic, accessible to the team through the National Clinical Trial Network (cooperative groups) and industry. The specific aims are to: 1) characterize relationships between the PRO-CTCAE and traditional measures of treatment impact and tolerability; 2) test and standardize analytic techniques for the PRO-CTCAE; 3) refine approaches to analyzing missing PRO-CTCAE data; and 4) develop and refine tabular and graphical data visualizations for longitudinal PRO-CTCAE data. To accomplish these aims, our multi-disciplinary team will work collaboratively and flexibly with other investigators and stakeholders through the U01 consortium chaired by Dr. Amylou Dueck, and will continue to share data, statistical programming code, and ideas freely. This work is intended to inform best practices for tabulating and reporting PRO-CTCAE data, and will enable more systematic and patient-centered evaluations of tolerability in cancer clinical trials. This supplement will extend the grant by 12 months.

项目摘要 在癌症临床试验中，必须了解和传达药物对患者的负面影响至关重要 参与者以平衡风险和利益。症状副作用，例如恶心和腹泻 对患者体验特别普遍且影响力。但是，从历史上看，这些信息一直是 临床医生代表患者而不是患者本身报告 显示出多达一半的患者症状。因此，国家癌症研究所（NCI）支持 开发患者报告的结果版本的常见术语标准 事件（Pro-CTCAE），一个问题库，以实现系统的患者自我报告副作用 信息。迄今为止，已经开发并测试了Pro-CTCAE问题的有效性和可行性 在临床试验中使用 - 调查人员对NCI合同的该提案领导的工作。 但是，目前尚未确定如何最好地将亲哥伦比研究与传统措施结合在一起 治疗影响，例如临床报告的不良事件，治疗时间，药物剂量水平，表现 状态和住院。该补充的目的是完成理解的工作 亲CTCAE在评估中的这些传统治疗措施中的作用 药物耐受性。在此补充中，高级统计方法将用于分析来自 新近可用的临床试验，被大流行延迟，团队可以通过国家访问 临床试验网络（合作团体）和行业。具体目的是：1）表征 亲CTCAE与治疗影响和耐受性的传统措施之间的关系； 2） 测试和标准化Pro-CTCAE的分析技术； 3）完善分析缺失的方法 Pro-CTCAE数据； 4）开发和完善纵向的表格和图形数据可视化 Pro-CTCAE数据。为了实现这些目标，我们的多学科团队将协作，并 通过由Amylou Dueck博士主持的U01财团与其他调查人员和利益相关者灵活地灵活 并将继续自由共享数据，统计编程代码和想法。这项工作旨在 为制表和报告Pro-CTCAE数据提供最佳实践，并将启用更系统的 以及以患者为中心的癌症临床试验中耐受​​性的评估。这种补充将扩大赠款 到12个月。