Systematic Evaluation of Patient-Reported Outcome Protocol Content and Reporting in Cancer Trials
Systematic Evaluation of Patient-Reported Outcome Protocol Content and Reporting in Cancer Trials
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DOI：
10.1093/jnci/djz038
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发表时间：
2019-11-01
影响因子：
10.3
通讯作者：
Calvert, Melanie
中科院分区：
文献类型：
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作者：
Kyte, Derek;Retzer, Ameeta;Calvert, Melanie
背景：患者报告的结果(PRO)被记录在癌症试验中，以帮助未来的患者及其临床医生做出更知情的治疗决定。然而，PRO试验设计和报告标准的变异性威胁着这些终点在临床实践中应用的有效性。方法：我们系统地调查了一组包括一级或二级PRO的随机对照癌症试验。对于每项试验，使用标准核对表对方案和报告质量进行评估。结果：(101个来源，44.3%)方案包括33个(范围=2-19)PRO方案检查表条目中的10(4)个项目的平均值(SD)。经常被省略的建议项目包括支持PRO收集的理由和目标，以及尽量减少/处理丢失的PRO数据的方法。在已公布结果的160项试验中，61项(38.1%，95%可信区间=30.6%至45.7%)没有在任何出版物中包含他们的PRO结果(平均6.43年随访期)；这些试验包括49568名参与者。尽管有三分之二的试验发表了PRO结果，但根据国际指南，报告标准往往不够充分(平均[SD]包括14个[范围=0-11]中的3[3]个项目)。超过一半的试验在二次发表中公布PRO结果(12/22，54.5%)，在试验结束后需要4年或更长时间，其中8项(36.4%)需要5-8年，1项试验在14年后发表。结论：PRO方案内容经常不充分，未报告PRO结果的情况普遍存在，这意味着患者的重要信息可能无法惠及患者、临床医生和监管机构。即使在发布PRO数据的地方，往往也会有相当大的延迟，报告质量也不是最好的。这项研究提出了一些关键建议，以提高未来成功交付PRO的可能性。
Background: Patient-reported outcomes (PROs) are captured within cancer trials to help future patients and their clinicians make more informed treatment decisions. However, variability in standards of PRO trial design and reporting threaten the validity of these endpoints for application in clinical practice.Methods: We systematically investigated a cohort of randomized controlled cancer trials that included a primary or secondary PRO. For each trial, an evaluation of protocol and reporting quality was undertaken using standard checklists. General patterns of reporting where also explored.Results: Protocols (101 sourced, 44.3%) included a mean (SD) of 10 (4) of 33 (range = 2-19) PRO protocol checklist items. Recommended items frequently omitted included the rationale and objectives underpinning PRO collection and approaches to minimize/address missing PRO data. Of 160 trials with published results, 61 (38.1%, 95% confidence interval = 30.6% to 45.7%) failed to include their PRO findings in any publication (mean 6.43-year follow-up); these trials included 49568 participants. Although two-thirds of included trials published PRO findings, reporting standards were often inadequate according to international guidelines (mean [SD] inclusion of 3 [3] of 14 [range = 0-11]) CONSORT PRO Extension checklist items). More than one-half of trials publishing PRO results in a secondary publication (12 of 22, 54.5%) took 4 or more years to do so following trial closure, with eight (36.4%) taking 5-8 years and one trial publishing after 14 years.Conclusions: PRO protocol content is frequently inadequate, and nonreporting of PRO findings is widespread, meaning patient-important information may not be available to benefit patients, clinicians, and regulators. Even where PRO data are published, there is often considerable delay and reporting quality is suboptimal. This study presents key recommendations to enhance the likelihood of successful delivery of PROs in the future.