Department of Oncology, François Baclesse Center, CHU de Caen, Caen, France.
Department of Oncology, François Baclesse Center, CHU de Caen, Caen, France.
Cancer Treat Rev. 2017 Feb;53:53-60. doi: 10.1016/j.ctrv.2016.12.003. Epub 2016 Dec 30.
Molecular targeted therapies have improved progression-free survival (PFS) without translating systematically into overall survival (OS) for patients with metastatic renal cell carcinoma (mRCC). In this population, patient-reported outcomes (PROs) have become a significant outcome. We evaluated the methodological quality of the assessment of PROs in randomized controlled trials (RCTs) and the clinical benefit of the different treatments including survival and quality of life (QoL).
A systematic review identified RCTs published between January 2005 and July 2014. They were evaluated according to 11 items derived from the 2013 CONSORT PROs reporting guidelines. Survival outcomes and PROs main results were analyzed and the magnitude of clinical benefit was assessed with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS).
12 RCTs were included with a total of 22 publications. The mean CONSORT score for all items was 4.5 on an 11-point scale. No publication reported the power of the PROs analysis and only one reported a PRO hypothesis. 50% of studies did not interpret PROs in relation to clinical outcomes and only 18% discussed specific limitations of PROs and their implications for generalizability. By adding the QoL criterion to PFS, 4 trials (36.4%) obtained a high level of proven clinical benefit according to the ESMO-MCBS.
The methodology for assessing PROs in mRCC is not optimal. Efforts should focus on defining PROs endpoint and increasing the quality of reporting of QoL. New-generation therapies in mRCC should demonstrate a gain not only in survival but also in QoL to be included in the therapeutic arsenal.
分子靶向治疗改善了转移性肾细胞癌（mRCC）患者的无进展生存期（PFS），但并未系统转化为总生存期（OS）。在这一人群中，患者报告的结局（PROs）已成为一个重要的结局。我们评估了随机对照试验（RCT）中 PROs 评估的方法学质量以及不同治疗方法的临床获益，包括生存和生活质量（QoL）。
系统检索了 2005 年 1 月至 2014 年 7 月期间发表的 RCT。根据 2013 年 CONSORT PROs 报告指南中的 11 项内容对其进行评估。分析生存结局和 PROs 的主要结果，并使用欧洲肿瘤内科学会临床获益量表（ESMO-MCBS）评估临床获益的程度。
纳入了 12 项 RCT，共计 22 项研究。所有项目的平均 CONSORT 评分为 11 分制的 4.5 分。没有一篇文献报告了 PROs 分析的功效，只有一篇文献报告了 PROs 假设。50%的研究没有根据临床结局解释 PROs，只有 18%的研究讨论了 PROs 的具体局限性及其对普遍性的影响。通过将 QoL 标准加入到 PFS 中，4 项试验（36.4%）根据 ESMO-MCBS 获得了高度的临床获益。
mRCC 中 PROs 的评估方法并不理想。应努力确定 PROs 终点，并提高 QoL 报告的质量。新一代 mRCC 治疗方法不仅应在生存方面，而且应在 QoL 方面显示获益，才能被纳入治疗方案。
