Progression-free survival as a surrogate for overall survival in oncology trials: a methodological systematic review

被引:55
|
作者
Belin, Lisa [1 ]
Tan, Aidan [2 ]
De Rycke, Yann [3 ]
Dechartres, Agnes [1 ]
机构
[1] Sorbonne Univ, Hop Univ Pitie Salpetriere Charles Foix, AP HP,Dept Biostat Sante Publ & Informat, INSERM,Inst Pierre Louis Epidemiol & Sante Publ, F-75013 Paris, France
[2] Natl Univ Singapore Hosp, Prevent Med, Singapore, Singapore
[3] Hop Univ Pitie Salpetriere Charles Foix, Hop Pitie Salpetriere,Ctr Pharmacoepidemiol Cephe, AP HP,Dept Biostat Sante Publ & Informat Med, INSERM,Inst Pierre Louis Epidemiol & Sante Publ, F-75013 Paris, France
关键词
END-POINTS; CLINICAL-TRIALS; TIME; ASSOCIATION; VALIDATION; EFFICACY;
D O I
10.1038/s41416-020-0805-y
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Background Progression-free survival (PFS) is a surrogate endpoint widely used for overall survival (OS) in oncology. Validation of PFS as a surrogate must be done for each indication and each intervention. We aimed to identify all studies evaluating the validity of PFS as a surrogate for OS in oncology, and to describe their methodological characteristics. Methods We conducted a systematic review by searching MEDLINE via PubMed and the Cochrane Library with no limitation on time, selected relevant studies and extracted data in duplicate on how surrogacy was evaluated (meta-analytic approach, assessment of correlation and level of evaluation). Results We identified 91 studies evaluating the validity of PFS as a surrogate for OS in 24 cancer localisations. Although a meta-analytic approach was used in 83 (91%) studies, the methods used to validate PFS as a surrogate of OS were heterogeneous across studies. Of the 47 studies concluding that PFS is a good surrogate for OS, for 15 (32%), there was no quantitative argument for surrogacy. Conclusions Although most studies used a meta-analytic approach as recommended, our methodological review highlights heterogeneity in methods and reporting, which stresses the importance of developing and applying clear recommendations in this area.
引用
收藏
页码:1707 / 1714
页数:8
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