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国际癌症随机对照临床试验中生活质量和患者报告结局终点分析的标准:SISAQOL 联盟的建议。

International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium.

机构信息

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

出版信息

Lancet Oncol. 2020 Feb;21(2):e83-e96. doi: 10.1016/S1470-2045(19)30790-9.


DOI:10.1016/S1470-2045(19)30790-9
PMID:32007209
Abstract

Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optimal methods of PRO analysis in cancer RCTs, hindering interpretation of results. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium was formed to establish PRO analysis recommendations. Four issues were prioritised: developing a taxonomy of research objectives that can be matched with appropriate statistical methods, identifying appropriate statistical methods for PRO analysis, standardising statistical terminology related to missing data, and determining appropriate ways to manage missing data. This Policy Review presents recommendations for PRO analysis developed through critical literature reviews and a structured collaborative process with diverse international stakeholders, which provides a foundation for endorsement; ongoing developments of these recommendations are also discussed.

摘要

患者报告的结局(PROs),如症状、功能和其他与健康相关的生活质量方面,越来越多地在癌症随机对照试验(RCT)中进行评估,以提供有关治疗风险、获益和耐受性的信息。然而,专家意见和对文献的批判性评价并未就癌症 RCT 中 PRO 分析的最佳方法达成共识,这阻碍了对结果的解释。成立了国际分析患者报告的结局和生活质量终点数据标准制定联盟,以制定 PRO 分析建议。四个问题被优先考虑:制定可与适当的统计方法相匹配的研究目标分类法,确定 PRO 分析的适当统计方法,使与缺失数据相关的统计术语标准化,并确定处理缺失数据的适当方法。本政策审查通过批判性文献审查和与不同国际利益相关者的结构化协作过程提出了 PRO 分析建议,为认可提供了基础;还讨论了这些建议的持续发展。

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