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

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，了解和沟通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，如恶心和腹泻， 特别常见，对患者体验有影响。然而，从历史上看， 由临床医生代表患者报告，而不是由患者自己报告-这一过程已经被 可以忽略病人一半的症状美国国家癌症研究所（NCI） 制定不良事件通用术语标准的患者报告结局版本 事件（PRO-CTCAE），一个问题库，用于系统性患者自我报告副作用 信息.到目前为止，已经开发了PRO-CTCAE问题，并测试了其有效性和可行性， 临床试验中的使用--根据与NCI签订的合同，由研究人员领导的这项工作。 然而，尚未确定如何最好地将联合收割机PRO-CTCAE信息与 治疗影响，如临床医生报告的不良事件、治疗时间、药物剂量水平、性能 状态和住院情况。本补充的目的是完成工作，以了解 PRO-CTCAE与这些传统的治疗影响指标相结合在评估 药物耐受性。在本附录中，将使用先进的统计方法分析来自 新的临床试验，由于大流行而推迟，团队可以通过国家 临床试验网络（合作团体）和行业。具体目标是：1）表征 PRO-CTCAE与治疗影响和耐受性的传统指标之间的关系; 2） 测试和标准化PRO-CTCAE的分析技术; 3）完善缺失分析方法 PRO-CTCAE数据;以及4）开发和完善纵向研究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通力合作， 通过由Amylou Dueck博士主持的U 01联盟，与其他研究人员和利益相关者灵活合作， 并将继续自由分享数据、统计编程代码和想法。这项工作旨在 为PRO-CTCAE数据的制表和报告提供最佳实践信息，并将实现更系统的 以及癌症临床试验中以患者为中心的耐受性评估。这一补充将延长赠款 12个月