University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA) Data Center, Rome, Italy and Department of Medical Social Sciences, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
Value Health. 2021 Apr;24(4):585-591. doi: 10.1016/j.jval.2020.11.004. Epub 2021 Feb 10.
In our systematic review, we assessed past and current practice of patient-reported outcome (PRO) measurement in cancer randomized, controlled trials (RCTs).
We included RCTs with PRO endpoints evaluating conventional medical treatments, conducted in patients with the most prevalent solid tumor types (breast, lung, colorectal, prostate, bladder, and gynecological cancers) and either published in 2004 to 2018 or registered on clinicaltrials.gov and initiated in 2014 to 2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site.
Screening of 42 095 database records and 3425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the European Organisation for Research and Treatment of Cancer (EORTC) measures were used most often (54.8% of trials), followed by the Functional Assessment of Chronic Illness Therapy (FACIT) measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the MD Anderson Symptom Inventory (MDASI; 2.5%). Among registered trials, the EORTC measures were used in 66.1% of the trials, followed by the FACIT measures (25.9%), the EQ-5D (23.1%), the SF-36 (4.8%), the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE; 2.2%), the Patient-Reported Outcomes Measurement Information System (PROMIS) measures (1.7%), and the MDASI measures (1.1%).
The PRO measures most frequently used in RCTs identified in our review differ substantially in terms of content and domains, reflecting the ongoing debate among the scientific community, healthcare providers, and regulators on the type of PRO to be measured. Current findings may contribute to better informing the development of an internationally agreed core outcome set for future cancer trials.
在我们的系统评价中，我们评估了过去和当前癌症随机对照试验（RCT）中患者报告结局（PRO）测量的实践。
我们纳入了 PRO 终点评估常规医学治疗的 RCT，这些 RCT 针对最常见的实体肿瘤类型（乳腺癌、肺癌、结直肠癌、前列腺癌、膀胱癌和妇科癌症）的患者进行，并于 2004 年至 2018 年发表，或在 clinicaltrials.gov 上注册并于 2014 年至 2019 年启动。我们总体上、随时间推移以及按癌症部位评估了个体 PRO 测量的使用频率。
对 42095 条数据库记录和 3425 项注册试验进行筛选后，确定了 480 项已发表和 537 项已注册的符合纳入标准的试验。在已发表的试验中，欧洲癌症研究与治疗组织（EORTC）的测量方法使用最频繁（54.8%的试验），其次是慢性疾病治疗功能评估（FACIT）测量方法（35.8%）、EQ-5D（10.2%）、SF-36（7.3%）和 MD 安德森症状量表（MDASI；2.5%）。在已注册的试验中，EORTC 测量方法在 66.1%的试验中使用，其次是 FACIT 测量方法（25.9%）、EQ-5D（23.1%）、SF-36（4.8%）、不良事件常用术语标准患者报告结局（PRO-CTCAE）测量方法（2.2%）、患者报告结局测量信息系统（PROMIS）测量方法（1.7%）和 MDASI 测量方法（1.1%）。
我们的综述中确定的 RCT 中最常使用的 PRO 测量方法在内容和领域方面存在很大差异，反映了科学界、医疗保健提供者和监管机构在要测量的 PRO 类型方面的持续争论。当前的研究结果可能有助于更好地为未来癌症试验制定国际商定的核心结局集提供信息。
