Cooper, K. orcid.org/0000-0002-7702-8103, Tappenden, P., Cantrell, A. et al. (1 more author) (2020) A systematic review of meta-analyses assessing the validity of tumour response endpoints as surrogates for progression-free or overall survival in cancer. British Journal of Cancer, 123 (11). pp. 1686-1696. ISSN 0007-0920
Abstract
Background: Tumour response endpoints, such as overall response rate (ORR) and complete response (CR), are increasingly used in cancer trials. However, the validity of response-based surrogates is unclear. This systematic review summarises meta-analyses assessing the association between response-based outcomes and overall survival (OS), progression-free survival (PFS) or time-to-progression (TTP).
Methods: Five databases were searched to March 2019. Meta-analyses reporting correlation or regression between response-based outcomes and OS, PFS or TTP were summarised.
Results: The systematic review included 63 studies across 20 cancer types, most commonly non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and breast cancer. The strength of association between ORR or CR and either PFS or OS varied widely between and within studies, with no clear pattern by cancer type. The association between ORR and OS appeared weaker and more variable than that between ORR and PFS, both for associations between absolute endpoints and associations between treatment effects.
Conclusions: This systematic review suggests that response-based endpoints such as ORR and CR may not be reliable surrogates for PFS or OS. Where it is necessary to use tumour response to predict treatment effects on survival outcomes, it is important to fully reflect all statistical uncertainty in the surrogate relationship.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2020 The Author(s). This is an author-produced version of a paper subsequently published in British Journal of Cancer. Uploaded in accordance with the publisher's self-archiving policy. |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) > ScHARR - Sheffield Centre for Health and Related Research |
Funding Information: | Funder Grant number NATIONAL INSTITUTE FOR HEALTH RESEARCH 14/25/02 |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 25 Aug 2020 15:22 |
Last Modified: | 27 Jan 2022 11:17 |
Status: | Published |
Publisher: | Springer Nature |
Refereed: | Yes |
Identification Number: | 10.1038/s41416-020-01050-w |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:164684 |