Clinical and Epidemiological Research Unit, Institut Bergonié, Comprehensive Cancer Center, 229 Cours de l'Argonne, 33000 Bordeaux, France; INSERM CIC-EC 14.01 (Clinical Epidemiology), Bordeaux, France; INSERM, ISPED, Centre INSERM U1219 Bordeaux Population Health Center, Epicene Team, 146 Rue Léo Saignat, 33076 Bordeaux, France; University of Bordeaux, ISPED, Centre INSERM U1219 Bordeaux Population Health, Epicene Team, 146 Rue Léo Saignat, 33076 Bordeaux, France.
Biometrics Unit, Institut du Cancer de Montpellier, Comprehensive Cancer Center, 208 Avenue des Apothicaires, 34298 Montpellier, France.
Crit Rev Oncol Hematol. 2018 Mar;123:21-41. doi: 10.1016/j.critrevonc.2017.11.014. Epub 2017 Nov 23.
In cancer randomized controlled trials (RCT), alternative endpoints are increasingly being used in place of overall survival (OS) to reduce sample size, duration and cost of trials. It is necessary to ensure that these endpoints are valid surrogates for OS. Our aim was to identify meta-analyses that evaluated surrogate endpoints for OS and assess the strength of evidence for each meta-analysis (MA).
We performed a systematic review to identify MA of cancer RCTs assessing surrogate endpoints for OS. We evaluated the strength of the association between the endpoints based on (i) the German Institute of Quality and Efficiency in Health Care guidelines and (ii) the Biomarker-Surrogate Evaluation Schema.
Fifty-three publications reported on 164 MA, with heterogeneous statistical methods Disease-free survival (DFS) and progression-free survival (PFS) showed good surrogacy properties for OS in colorectal, lung and head and neck cancers. DFS was highly correlated to OS in gastric cancer.
CONCLUSION(S): The statistical methodology used to evaluate surrogate endpoints requires consistency in order to facilitate the accurate interpretation of the results. Despite the limited number of clinical settings with validated surrogate endpoints for OS, there is evidence of good surrogacy for DFS and PFS in tumor types that account for a large proportion of cancer cases.
在癌症随机对照试验（RCT）中，替代终点越来越多地被用于替代总生存期（OS），以减少试验的样本量、持续时间和成本。有必要确保这些终点是 OS 的有效替代指标。我们的目的是确定评估 OS 替代终点的荟萃分析，并评估每项荟萃分析（MA）的证据强度。
我们进行了一项系统评价，以确定评估 OS 替代终点的癌症 RCT 的 MA。我们根据（i）德国卫生保健质量和效率研究所的指南和（ii）生物标志物替代评估方案，评估了终点之间关联的强度。
53 篇出版物报告了 164 项 MA，结果显示无病生存期（DFS）和无进展生存期（PFS）在结直肠癌、肺癌和头颈部癌症中具有良好的 OS 替代指标特性。DFS 在胃癌中与 OS 高度相关。
评估替代终点的统计方法需要一致性，以便准确解释结果。尽管具有 OS 验证替代终点的临床环境有限，但在占癌症病例很大比例的肿瘤类型中，DFS 和 PFS 的替代指标具有良好的替代指标特性。
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