鼻咽癌放化疗联合试验中总生存期的替代终点：随机对照试验的荟萃分析

Surrogate endpoints for overall survival in combined chemotherapy and radiotherapy trials in nasopharyngeal carcinoma: Meta-analysis of randomised controlled trials.

作者信息

Chen Yu-Pei, Sun Ying, Chen Lei, Mao Yan-Ping, Tang Ling-Long, Li Wen-Fei, Liu Xu, Zhang Wen-Na, Zhou Guan-Qun, Guo Rui, Lin Ai-Hua, Ma Jun

机构信息

State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Department of Radiation Oncology, Sun Yat-sen University Cancer Center, Guangzhou, People's Republic of China.

Department of Medical Statistics and Epidemiology, School of Public Health, Sun Yat-sen University, Guangzhou, People's Republic of China.

出版信息

Radiother Oncol. 2015 Aug;116(2):157-66. doi: 10.1016/j.radonc.2015.07.030. Epub 2015 Aug 1.

DOI:10.1016/j.radonc.2015.07.030

PMID:26243677

Abstract

BACKGROUND AND PURPOSE

We used a literature-based meta-analysis to assess whether failure-free survival (FFS) or progression-free survival (PFS) could be reliable surrogate endpoints for overall survival (OS) in trials of combined chemotherapy and radiotherapy for nasopharyngeal carcinoma (NPC).

METHODS AND MATERIALS

We identified randomised trials that evaluated combined chemoradiotherapy strategies, and reported FFS or PFS and OS in NPC. We analysed the treatment effects on FFS or PFS, and OS. We used the coefficient of determination (R(2)), and the surrogate threshold effect (STE) to assess the trial-level correlation.

RESULTS

Twenty-one trials (5212 patients), with sixteen treatment-control comparisons for FFS, and nine for PFS, were analysed. FFS was strongly correlated with OS (R(2)=0.88, STE=0.84), as was PFS (R(2)=0.90, STE=0.88). Moreover, FFS and PFS at 3 years were still strongly correlated with 5-year OS (R(2)=0.80, STE=0.83; R(2)=0.85, STE=0.84).

CONCLUSIONS

Both FFS and PFS could be valid surrogate endpoints for OS in trials of combined chemotherapy and radiotherapy for NPC; PFS may be a more acceptable surrogate endpoint compared with FFS.

摘要

背景与目的

我们采用基于文献的荟萃分析，评估在鼻咽癌（NPC）放化疗联合治疗试验中，无失败生存期（FFS）或无进展生存期（PFS）是否可作为总生存期（OS）可靠的替代终点。

方法与材料

我们确定了评估放化疗联合策略的随机试验，并报告了NPC患者的FFS或PFS以及OS。我们分析了对FFS或PFS以及OS的治疗效果。我们使用决定系数（R²）和替代阈值效应（STE）来评估试验水平的相关性。

结果

分析了21项试验（5212例患者），其中有16项治疗对照比较涉及FFS，9项涉及PFS。FFS与OS高度相关（R² = 0.88，STE = 0.84），PFS也是如此（R² = 0.90，STE = 0.88）。此外，3年时的FFS和PFS与5年OS仍高度相关（R² = 0.80，STE = 0.83；R² = 0.85，STE = 0.84）。