Best practices and pragmatic approaches for patient-reported outcomes and quality of life measures in cancer clinical trials.

Hanna Bandos, Pedro A Torres-Saavedra, Eva Culakova, Heather J Gunn, Minji K Lee, Fenghai Duan, Reena S Cecchini, Joseph M Unger, Amylou C Dueck, Jon A Steingrimsson
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Abstract

Patient-reported outcomes (PROs) are often collected in cancer clinical trials. Data obtained from trials with PROs are essential in evaluating participant experiences relating to symptoms, financial toxicity, or health-related quality of life. Although most features of clinical trial design, implementation, and analyses apply to trials with PROs, several considerations are unique. In this paper, we focus on specific issues such as selection of the tool, timing and frequency of assessments, and data collection methods. We discuss how the estimand framework can be used in connection with PROs, properties of common estimation methods, and handling of missing outcomes. With a plethora of literature available, we aim to summarize best practices and pragmatic approaches to the design and analysis of the studies incorporating PROs.

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癌症临床试验通常会收集患者报告的结果(PROs)。在评估参与者在症状、经济毒性或健康相关生活质量方面的体验时,从有患者报告结果的试验中获得的数据至关重要。虽然临床试验设计、实施和分析的大多数特点都适用于采用 PROs 的试验,但有几项考虑因素是独一无二的。在本文中,我们将重点讨论工具的选择、评估的时间和频率以及数据收集方法等具体问题。我们还讨论了如何将估计值框架与 PROs 结合使用、常用估计方法的特性以及缺失结果的处理。面对大量的文献资料,我们旨在总结设计和分析包含 PROs 的研究的最佳实践和实用方法。
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