Methodology and Quality of Life Unit, Oncology Department (INSERM UMR 1098), Quality of Life and Cancer Clinical Research Platform.
Department of Medical Oncology, University Hospital of Besançon, Besançon.
Ann Oncol. 2017 Sep 1;28(9):2077-2085. doi: 10.1093/annonc/mdx191.
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.
传统上，在临床研究中，对于晚期或转移性疾病患者的癌症治疗效果，一直使用总生存期和最近的肿瘤终点（如无进展生存期、治疗反应的测量）进行研究。然而，如果这些终点没有被验证为总生存期的替代指标，以证明临床疗效，那么它们似乎不是当前情况下的相关临床终点。适当的、有意义的、以患者为中心和患者报告的主要终点，能够充分衡量新治疗干预措施的效果，对于补充肿瘤终点的转移性结直肠癌临床研究进展至关重要。健康相关生活质量（HRQoL）实际上是对生活质量及其随时间变化与健康关系的评估。HRQoL 至少包括患者对疾病或其治疗方式如何影响其身体、情感和社会福祉的报告。在过去几年中，在各种实体瘤的几项 III 期试验中，除了肿瘤反应和生存结果的传统终点外，还评估了 HRQoL 的附加价值。HRQoL 不仅可以为主要结局提供补充的临床数据，还可以提供更精确的预测和预后价值。为了实现精准医学的目标，这个终点对临床医生和患者都很有用。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、分析和结果呈现的重要性。此外，它还讨论了将 HRQoL 作为主要/联合主要终点纳入的相关性，以支持无进展生存期的结果，并评估晚期疾病治疗的疗效。
