King's College London, School of Cancer and Pharmaceutical Sciences, Translational Oncology and Urology Research (TOUR), London, SE1 9RT, UK.
Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Disease (GIMEMA), Rome, Italy.
BMC Urol. 2019 Sep 14;19(1):86. doi: 10.1186/s12894-019-0518-9.
Despite international recommendations of including patient-reported outcomes (PROs) in randomised clinical trials (RCTs), a 2014 review concluded that few RCTs of bladder cancer (BC) report PRO as an outcome. We therefore aimed to update the 2014 review to synthesise current evidence-based knowledge of PROs from RCTs in BC. A secondary objective was to examine whether quality of PRO reporting has improved over time and to provide evidence-based recommendations for future studies in this area.
We conducted a systematic literature search using PubMed/Medline, from April 2014 until June 2018. We included the RCTs identified in the previous review as well as newly published RCTs. Studies were evaluated using a predefined electronic-data extraction form that included information on basic trial demographics, clinical and PRO characteristics and standards of PRO reporting based on recommendation from the International Society of Quality of Life Research.
Since April 2014 only eight new RCTs for BC included PROs as a secondary outcome. In terms of methodology, only the proportion of RCTs documenting the extent of missing PRO data (75% vs 11.1%, p = 0.03) and the identification of PROs in trial protocols (50% vs 0%, p = 0.015) improved. Statistical approaches for dealing with missing data were not reported in most new studies (75%).
Little improvement into the uptake and assessment of PRO as an outcome in RCTs for BC has been made during recent years. Given the increase in (immunotherapy) drug trials with a potential for severe adverse events, there is urgent need to adopt the recommendations and standards available for PRO use in bladder cancer RCTs.
尽管国际建议将患者报告的结局（PRO）纳入随机临床试验（RCT），但 2014 年的一项综述得出的结论是，很少有膀胱癌（BC）的 RCT 报告 PRO 作为结局。因此，我们旨在更新 2014 年的综述，以综合目前关于 BC 的 RCT 中 PRO 的循证知识。次要目标是检查 PRO 报告的质量是否随时间改善，并为该领域的未来研究提供循证建议。
我们使用 PubMed/Medline 进行了系统的文献检索，时间范围为 2014 年 4 月至 2018 年 6 月。我们纳入了之前综述中确定的 RCT 以及新发表的 RCT。使用预先设定的电子数据提取表格评估研究，该表格包括基本试验人口统计学、临床和 PRO 特征以及基于国际生活质量研究协会建议的 PRO 报告标准的信息。
自 2014 年 4 月以来，仅有 8 项新的 BC RCT 将 PRO 作为次要结局纳入。就方法学而言，仅记录 PRO 缺失数据程度的 RCT 比例（75%比 11.1%，p=0.03）和在试验方案中识别 PRO 的 RCT 比例（50%比 0%，p=0.015）有所改善。大多数新研究未报告处理缺失数据的统计方法（75%）。
近年来，BC 的 RCT 中 PRO 作为结局的纳入和评估几乎没有改善。鉴于（免疫疗法）药物试验的增加，这些试验可能会出现严重的不良反应，因此迫切需要采用适用于 BC RCT 中 PRO 使用的建议和标准。
