Traditionally, the efficacy of cancer treatment in patients with advance or metastatic disease in clinical studies has been studied using overall survival and more recently tumor-based end points such as progression-free survival, measurements of response to treatment. However, these seem not to be the relevant clinical end points in current situation if such end points were no validated as surrogate of overall survival to demonstrate the clinical efficacy. Appropriate, meaningful, primary patient-oriented and patient-reported end points that adequately measure the effects of new therapeutic interventions are then crucial for the advancement of clinical research in metastatic colorectal cancer to complement the results of tumor-based end points. Health-related quality of life (HRQoL) is effectively an evaluation of quality of life and its relationship with health over time. HRQoL includes the patient report at least of the way a disease or its treatment affects its physical, emotional and social well-being. Over the past few years, several phase III trials in a variety of solid cancers have assessed the incremental value of HRQoL in addition to the traditional end points of tumor response and survival results. HRQoL could provide not only complementary clinical data to the primary outcomes, but also more precise predictive and prognostic value. This end point is useful for both clinicians and patients in order to achieve the dogma of precision medicine. The present article examines the use of HRQoL in phase III metastatic colorectal cancer clinical trials, outlines the importance of HRQoL assessment methods, analysis, and results presentation. Moreover, it discusses the relevance of including HRQoL as a primary/co-primary end point to support the progression-free survival results and to assess efficacy of treatment in the advanced disease setting.

中文翻译：

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在晚期大肠癌临床试验中应如何使用与健康相关的生活质量评估

传统上，已经在临床研究中使用整体生存率和最近基于肿瘤的终点（例如无进展生存期，对治疗反应的测量）来研究癌症在晚期或转移性疾病患者中的治疗效果。然而，如果这些终点未经证实可作为证明临床疗效的总生存指标，那么这些似乎不是当前情况下的相关临床终点。因此，适当地，有意义的，以患者为中心并且以患者报告的主要终点指标能够充分衡量新治疗干预措施的效果，对于转移性结直肠癌临床研究的发展，以补充基于肿瘤的终点指标的结果至关重要。与健康相关的生活质量（HRQoL）有效地评估了生活质量及其与健康之间的关系。HRQoL至少包括患者报告，至少涉及疾病或其治疗影响其身体，情感和社会福祉的方式。在过去的几年中，除了传统的肿瘤反应终点和生存结果外，在多种实体癌中的多项III期临床试验还评估了HRQoL的增量价值。HRQoL不仅可以为主要结果提供补充的临床数据，而且还可以提供更精确的预测和预后价值。该终点对于临床医生和患者都是有用的，以实现精确医学的教条。本文探讨了HRQoL在III期转移性结直肠癌临床试验中的应用，概述了HRQoL评估方法，分析和结果表示的重要性。此外，它还讨论了将HRQoL作为主要/辅助主要终点的相关性，以支持无进展生存期结果并评估晚期疾病背景下的治疗效果。