Gross Franziska, Rasmussen Ida Marie Lind, Beisland Elisabeth Grov, Jorem Gøril Tvedten, Beisland Christian, Pappot Helle, Arraras Juan Ignacio, Pe Madeline, Holzner Bernhard, Wintner Lisa M
University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
Department of Oncology, Copenhagen University Hospital - Herlev and Gentofte, Copenhagen, Denmark; Department of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark.
Eur Urol Oncol. 2025 Feb;8(1):201-212. doi: 10.1016/j.euo.2024.09.007. Epub 2024 Oct 4.
In oncology, patient-reported outcome measures (PROMs) capturing health-related quality of life (HRQOL) play an increasing role in clinical trials, drug approval, and policy making. This scoping review aimed to identify and elaborate on HRQOL-focussed PROMs used in renal cell cancer (RCC) clinical trials.
MEDLINE, Web of Science, PsychINFO, Academic Search Elite, CINAHL, Embase, and the Cochrane Library were searched systematically for original peer-reviewed articles on clinical trials including RCC patients and using PROMs, published between 1950 and 2023. Prespecified trial characteristics and information on the PROMs used were extracted. Frequencies and proportions of categorical data, and ranges and medians of continuous variables were calculated.
Of the 48 unique studies included, the majority followed a randomised controlled design (34, 71%) and evaluated systemic treatments (38, 79%). The trials used 27 different PROMs (max = 6, median = 2), of which only 4 (15%) were developed specifically for kidney cancer patients. Of the trials, 46% did not use any RCC-specific PROM. European Quality of Life-5 Dimensions (EQ-5D), European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30), Functional Assessment of Cancer Therapy Kidney Symptom Index (FKSI) -15/19-item version, FKSI-Disease Related Symptoms, and Functional Assessment of Cancer Therapy-General (FACT-G) were the most frequently used questionnaires, with pain, ability to work, fatigue, worry, and sleep quality being the most commonly assessed issues.
A variety of PROMs are used in RCC patients, hindering interpretability across trials. The PROMs used differ in terms of both the domains assessed and how the issues are translated into questionnaire items. Though RCC-specific PROMs exist, these have flaws in terms of relevance to patients. To answer predefined relevant HRQOL research questions, revised RCC-specific PROMs and standardisation of their integration into clinical trials are warranted.
Researchers are more and more interested in the health-related quality of life of kidney cancer patients and use questionnaires to measure it. This review shows that there are many different health-related quality of life questionnaires that are used in different combinations in clinical trials for kidney cancer patients. This makes it very difficult to compare these study results and draw reliable conclusions for the actual clinical treatment. It was even found that some of the questionnaires used do not capture things that patients actually consider important (eg, emotional issues such as dealing with thoughts about cancer and depression). Therefore, more work needs to be done to develop questionnaires that ask what is really important to kidney cancer patients' health-related quality of life. If these questionnaires are used in a consistent way in clinical trials, the results can be better compared. This will help treat kidney cancer patients in the best possible way.
在肿瘤学领域，用于衡量健康相关生活质量（HRQOL）的患者报告结局指标（PROMs）在临床试验、药物审批及政策制定中发挥着越来越重要的作用。本范围综述旨在识别并详细阐述肾细胞癌（RCC）临床试验中使用的以HRQOL为重点的PROMs。
系统检索MEDLINE、科学网、PsychINFO、Academic Search Elite、CINAHL、Embase和Cochrane图书馆，查找1950年至2023年间发表的关于包含RCC患者并使用PROMs的临床试验的同行评审原始文章。提取预先设定的试验特征及所使用PROMs的相关信息。计算分类数据的频率和比例，以及连续变量的范围和中位数。
在纳入的48项独特研究中，大多数采用随机对照设计（34项，71%）并评估全身治疗（38项，79%）。这些试验使用了27种不同的PROMs（最多6种，中位数为2种），其中只有4种（15%）是专门为肾癌患者开发的。在这些试验中，46%未使用任何RCC特异性PROM。欧洲生活质量五维度量表（EQ-5D）、欧洲癌症研究与治疗组织生活质量核心问卷（EORTC QLQ-C30）、癌症治疗功能评估-肾脏症状指数（FKSI）-15/19项版本、FKSI-疾病相关症状量表以及癌症治疗功能评估-通用量表（FACT-G）是最常用的问卷，疼痛、工作能力、疲劳、担忧和睡眠质量是最常评估的问题。
RCC患者使用了多种PROMs，这妨碍了不同试验间结果的解释。所使用的PROMs在评估领域以及问题转化为问卷项目的方式上均存在差异。尽管存在RCC特异性PROMs，但在与患者的相关性方面存在缺陷。为回答预先设定的相关HRQOL研究问题，有必要修订RCC特异性PROMs并将其纳入临床试验的过程标准化。
研究人员对肾癌患者的健康相关生活质量越来越感兴趣，并使用问卷进行测量。本综述表明，有许多不同的健康相关生活质量问卷，在肾癌患者的临床试验中以不同组合使用。这使得很难比较这些研究结果并为实际临床治疗得出可靠结论。甚至发现，一些使用的问卷未涵盖患者实际认为重要的方面（例如，应对癌症相关想法和抑郁等情绪问题）。因此，需要做更多工作来开发询问对肾癌患者健康相关生活质量真正重要内容的问卷。如果这些问卷在临床试验中以一致方式使用，结果将更具可比性。这将有助于以最佳方式治疗肾癌患者。
