Italian Group for Adult Hematologic Diseases (GIMEMA) Data Center and Health Outcomes Research Unit, Rome, Italy.
Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology, and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
Value Health. 2021 Feb;24(2):250-267. doi: 10.1016/j.jval.2020.10.017. Epub 2021 Jan 6.
Assessment of patient-reported outcomes (PROs) in oncology is of critical importance because it provides unique information that may also predict clinical outcomes.
We conducted a systematic review of prognostic factor studies to examine the prognostic value of PROs for survival in cancer. A systematic literature search was performed in PubMed for studies published between 2013 and 2018. We considered any study, regardless of the research design, that included at least 1 PRO domain in the final multivariable prognostic model. The protocol (EPIPHANY) was published and registered in the International Prospective Register of Systematic Reviews (CRD42018099160).
Eligibility criteria selected 138 studies including 158 127 patients, of which 43 studies were randomized, controlled trials. Overall, 120 (87%) studies reported at least 1 PRO to be statistically significantly prognostic for overall survival. Lung (n = 41, 29.7%) and genitourinary (n = 27, 19.6%) cancers were most commonly investigated. The prognostic value of PROs was investigated in secondary data analyses in 101 (73.2%) studies. The EORTC QLQ-C30 questionnaire was the most frequently used measure, and its physical functioning scale (range 0-100) the most frequent independent prognostic PRO, with a pooled hazard ratio estimate of 0.88 per 10-point increase (95% CI 0.84-0.92).
There is convincing evidence that PROs provide independent prognostic information for overall survival across cancer populations and disease stages. Further research is needed to translate current evidence-based data into prognostic tools to aid in clinical decision making.
评估肿瘤患者报告结局(PROs)至关重要,因为它提供了可能预测临床结局的独特信息。
我们对预后因素研究进行了系统回顾,以检查 PRO 对癌症生存的预后价值。在 PubMed 中进行了系统文献检索,以检索 2013 年至 2018 年期间发表的研究。我们考虑了任何研究,无论研究设计如何,只要最终多变量预后模型中包含至少 1 个 PRO 领域即可。该方案(EPIPHANY)已发表并在国际前瞻性系统评价注册中心(CRD42018099160)注册。
入选标准选择了 138 项研究,包括 158127 名患者,其中 43 项为随机对照试验。总体而言,有 120 项(87%)研究报告至少有 1 项 PRO 对总生存具有统计学显著的预后价值。肺癌(n=41,29.7%)和泌尿生殖系统癌(n=27,19.6%)是最常见的研究对象。在 101 项(73.2%)研究中,对 PROs 的预后价值进行了二次数据分析。EORTC QLQ-C30 问卷是最常用的测量工具,其身体功能量表(范围 0-100)是最常见的独立预后 PRO,合并危险比估计值为每增加 10 分 0.88(95%CI 0.84-0.92)。
有确凿的证据表明,PRO 为癌症患者和疾病阶段的总体生存提供了独立的预后信息。需要进一步研究将当前基于证据的数据转化为预后工具,以帮助临床决策。