使用血液肿瘤患者真实世界数据的外部对照研究

IF 22.5 1区 医学 Q1 ONCOLOGY JAMA Oncology Pub Date : 2024-08-29 DOI:10.1001/jamaoncol.2024.3466
Sjoerd J. F. Hermans, Niek G. van der Maas, Yvette van Norden, Avinash G. Dinmohamed, Elizabeth Berkx, Peter C. Huijgens, Donna R. Rivera, R. Angelo de Claro, Francesco Pignatti, Jurjen Versluis, Jan J. Cornelissen
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引用次数: 0

摘要

重要性在前瞻性研究中使用真实世界数据(RWD)外部对照臂的情况越来越多。证据综述 2000 年 1 月 1 日至 2023 年 10 月 23 日,对 4 个电子数据库和 Google Scholar 进行了系统检索。将血液肿瘤患者临床试验中的干预组与RWD对照组进行比较的研究,以及在2000年至2023年期间发表的研究均被纳入系统综述。所有研究共纳入了4306名干预组患者和10 594名RWD对照组患者。只有 2 项研究(6%)纳入了前瞻性收集的 RWD。7项研究(22%)对RWD队列采用了完整的试验纳入标准。有 4 项研究(13%)公布了统计分析计划,并预先指定使用 RWD。共有 23 项研究(72%)对试验队列和 RWD 队列采用了匹配算法,包括人口统计、疾病和/或治疗相关特征的匹配。有 8 项研究(25%)的终点标准与试验相同。相比之下,12 项研究(38%)采用了不同的终点,12 项研究(38%)没有提供 RWD 的终点定义。12项研究(38%)的两组随访时间中位数相差不到一年。8项研究(25%)报告了试验组的毒副作用数据,其中5项研究报告了RWD组的毒副作用数据。结论与相关性在本系统综述中,观察到RWD在临床试验资格标准的应用、统计学的严谨性和匹配方法的应用、终点的定义、随访和不良事件的报告等方面存在局限性,这可能会对使用RWD的研究报告的结论提出质疑。
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Externally Controlled Studies Using Real-World Data in Patients With Hematological Cancers
ImportanceThe use of real-world data (RWD) external control arms in prospective studies is increasing. The advantages, including the immediate availability of a control population, must be balanced with the requirements of meeting evidentiary standards.ObjectiveTo address the question of whether and to what extent the methods of RWD studies compare to standard methods used in randomized clinical trials.Evidence ReviewA systematic search across 4 electronic databases and Google Scholar was conducted from January 1, 2000, to October 23, 2023. Studies were included in the systematic review if they compared an intervention arm in a clinical trial to an RWD control arm in patients with hematological cancers and if they were published between 2000 and 2023.FindingsThirty-two prospective intervention studies incorporating external control data from RWD sources of patients with hematological cancers were identified. A total of 4306 patients from intervention arms and 10 594 from RWD control arms were included across all studies. Only 2 studies (6%) included prospectively collected RWD. The complete trial inclusion criteria were applied to the RWD cohort in 7 studies (22%). Four studies (13%) published the statistical analysis plan and prespecified use of RWD. A total of 23 studies (72%) applied matching algorithms for trial and RWD cohorts, including matching for demographic, disease, and/or therapy-related characteristics. The end point criteria were the same as the trial in 8 studies (25%). In contrast, 12 studies (38%) used different end points, and 12 (38%) did not provide an end point definition for the RWD. Twelve studies (38%) had a median follow-up difference of less than a year between arms. Eight studies (25%) reported toxic effect data for the trial arm, of which 5 studies reported toxic effect data for the RWD arm.Conclusions and RelevanceIn this systematic review, limitations were observed in the application of clinical trial eligibility criteria to RWD, statistical rigor and application of matching methods, the definition of end points, follow-up, and reporting of adverse events, which may challenge the conclusions reported in studies using RWD.
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来源期刊
JAMA Oncology
JAMA Oncology Medicine-Oncology
自引率
1.80%
发文量
423
期刊介绍: JAMA Oncology is an international peer-reviewed journal that serves as the leading publication for scientists, clinicians, and trainees working in the field of oncology. It is part of the JAMA Network, a collection of peer-reviewed medical and specialty publications.
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