Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Merck. & Co. Inc., Rahway, NJ, USA.
Eur Urol. 2023 Oct;84(4):406-417. doi: 10.1016/j.eururo.2023.07.006. Epub 2023 Aug 5.
In the oncology setting, patient-reported outcome measures (PROMs) provide important data that help to ensure patient-relevant endpoints are captured and reported. Use of this information for treatment decision-making by clinicians and patients in real-world settings is facilitated by consistent and transparent reporting of trial methods.
To identify and compare PROMs used in advanced renal cell carcinoma (RCC) trials in terms of the rationale for the choice of measure, endpoint hierarchy (primary, secondary, exploratory), assessment time points, statistical methods, and statistical metrics for interpretation.
A systematic literature review via searches of four online databases (2016-2021) and recent conference abstracts (2019-2021) identified 2616 articles, of which 33 were included in the review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines.
Among the 33 clinical studies included, 19 different PROMs were identified: three kidney cancer-specific scales, two cancer-specific scales, two generic scales, and 12 symptom-specific scales. The endpoint hierarchy for patient reported outcome (PRO) assessment was reported in 42% of the studies; one study included PROs as a primary endpoint. Reporting of time points, minimal important differences, and statistical analyses was highly heterogeneous.
A diverse range of PROMs have been included in clinical studies for patients with advanced/metastatic RCC. Prespecified analyses for PRO assessments were generally not stated, while analytical methods and reporting varied. An improvement in alignment across studies would better inform regulatory, market-access, reimbursement, and clinical decision-making to improve patient care.
We reviewed how the impact of cancer therapies on health outcomes from the patient's point of view is being measured in clinical trials for kidney cancer. The techniques and reporting varied across trials. Standardisation of how these data are captured and reported may improve care and decision-making for patients with kidney cancer.
在肿瘤学领域，患者报告结局测量（PROMs）提供了重要数据，有助于确保捕获和报告与患者相关的终点。通过临床试验方法的一致和透明报告，为临床医生和患者在真实环境中进行治疗决策提供了便利。
根据选择测量方法的理由、终点层次结构（主要、次要、探索性）、评估时间点、统计方法以及用于解释的统计指标，确定并比较晚期肾细胞癌（RCC）试验中使用的患者报告结局测量。
通过对四个在线数据库（2016-2021 年）和最近的会议摘要（2019-2021 年）进行系统文献检索，共检索到 2616 篇文章，根据系统评价和荟萃分析的首选报告项目指南，其中 33 篇文章被纳入综述。
在纳入的 33 项临床研究中，确定了 19 种不同的患者报告结局测量：三种肾癌特异性量表、两种癌症特异性量表、两种通用量表和 12 种症状特异性量表。42%的研究报告了患者报告结局评估的终点层次结构；有一项研究将患者报告结局作为主要终点。报告时间点、最小重要差异和统计分析高度异质。
在晚期/转移性 RCC 患者的临床研究中，已经纳入了多种患者报告结局测量。通常没有预先规定患者报告结局评估的分析，而分析方法和报告则存在差异。如果在研究之间进行更好的一致性改进，将更好地为监管、市场准入、报销和临床决策提供信息，从而改善患者护理。
我们回顾了在肾癌临床试验中如何衡量癌症治疗对患者健康结局的影响。试验之间的技术和报告存在差异。标准化这些数据的捕获和报告方式可能会改善肾癌患者的护理和决策。
