Evaluation of pathological complete response as surrogate endpoint in neoadjuvant randomised clinical trials of early stage breast cancer: systematic review and meta-analysis.


Journal

BMJ (Clinical research ed.)
ISSN: 1756-1833
Titre abrégé: BMJ
Pays: England
ID NLM: 8900488

Informations de publication

Date de publication:
21 12 2021
Historique:
entrez: 22 12 2021
pubmed: 23 12 2021
medline: 7 1 2022
Statut: epublish

Résumé

To evaluate pathological complete response as a surrogate endpoint for disease-free survival and overall survival in regulatory neoadjuvant trials of early stage breast cancer. Systematic review and meta-analysis. Medline, Embase, and Scopus to 1 December 2020. Randomised clinical trials that tested neoadjuvant chemotherapy given alone or combined with other treatments, including anti-human epidermal growth factor 2 (anti-HER2) drugs, targeted treatments, antivascular agents, bisphosphonates, and immune checkpoint inhibitors. Trial level associations between the surrogate endpoint pathological complete response and disease-free survival and overall survival. A weighted regression analysis was performed on log transformed treatment effect estimates (hazard ratio for disease-free survival and overall survival and relative risk for pathological complete response), and the coefficient of determination (R 54 randomised clinical trials comprising a total of 32 611 patients were included in the analysis. A weak association was observed between the log(relative risk) for pathological complete response and log(hazard ratio) for both disease-free survival (R A lack of surrogacy of pathological complete response was identified at trial level for both disease-free survival and overall survival. The findings suggest that pathological complete response should not be used as primary endpoint in regulatory neoadjuvant trials of early stage breast cancer.

Identifiants

pubmed: 34933868
doi: 10.1136/bmj-2021-066381
pmc: PMC8689398
doi:

Substances chimiques

Biomarkers 0

Types de publication

Journal Article Meta-Analysis Systematic Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

e066381

Informations de copyright

© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: All authors have completed the ICMJE uniform disclosure form at www.icmje.org/disclosure-of-interest/ and declare: no support from any organisation for the submitted work; no financial relationships with any organisations that might have an interest in the submitted work in the previous three years; no other relationships or activities that could appear to have influenced the submitted work.

Références

Ann Oncol. 2003 Feb;14(2):227-32
pubmed: 12562649
Lancet Oncol. 2015 Sep;16(9):1037-1048
pubmed: 26272770
J Natl Cancer Inst Monogr. 2015 May;2015(51):36-9
pubmed: 26063884
J Clin Oncol. 2015 Jan 1;33(1):13-21
pubmed: 25092775
N Engl J Med. 2020 Feb 27;382(9):810-821
pubmed: 32101663
Eur J Cancer. 2018 Apr;93:19-27
pubmed: 29448072
J Clin Oncol. 2009 Jun 20;27(18):2938-45
pubmed: 19364964
J Clin Oncol. 2018 May 1;36(13):1308-1316
pubmed: 29543566
Lancet Oncol. 2019 Mar;20(3):361-370
pubmed: 30709633
Ann Oncol. 2018 Dec 1;29(12):2341-2347
pubmed: 30335131
Ann Oncol. 2017 Aug 01;28(8):1817-1824
pubmed: 28459938
J Clin Oncol. 2005 Jun 1;23(16):3676-85
pubmed: 15738535
Eur J Cancer. 2016 Jul;62:62-75
pubmed: 27208905
JNCI Cancer Spectr. 2019 Feb 06;3(1):pkz002
pubmed: 31360890
JAMA Oncol. 2015 Oct;1(7):875-6
pubmed: 26181139
J Clin Oncol. 2019 Sep 1;37(25):2206-2216
pubmed: 31157583
Stat Methods Med Res. 2008 Oct;17(5):477-86
pubmed: 18285438
JAMA Oncol. 2018 Mar 01;4(3):302-308
pubmed: 29327055
J Clin Oncol. 2010 Apr 20;28(12):2015-23
pubmed: 20308671
BMC Cancer. 2011 May 18;11:179
pubmed: 21592370
J Clin Oncol. 2020 Dec 10;38(35):4184-4193
pubmed: 33095682
BMJ. 2021 Sep 15;374:n2191
pubmed: 34526320
J Clin Oncol. 2002 Mar 15;20(6):1456-66
pubmed: 11896092
J Clin Oncol. 2013 Oct 10;31(29):3623-30
pubmed: 24002511
Breast Cancer Res Treat. 2016 Aug;158(3):485-95
pubmed: 27393622
J Clin Oncol. 2019 Sep 1;37(25):2226-2234
pubmed: 31082269
Stat Med. 2016 Mar 30;35(7):1063-89
pubmed: 26530518
Stat Med. 2005 Jan 30;24(2):163-82
pubmed: 15515150
Lancet Oncol. 2016 Mar;17(3):345-356
pubmed: 26869049
Breast Cancer Res Treat. 2020 Apr;180(3):687-694
pubmed: 32140811
Ann Oncol. 2011 Sep;22(9):1988-1998
pubmed: 21385882
J Clin Oncol. 2004 Dec 15;22(24):4958-65
pubmed: 15611510
JAMA Oncol. 2019 Jan 1;5(1):45-50
pubmed: 30193295
Lancet. 2020 Oct 10;396(10257):1090-1100
pubmed: 32966830
Lancet Oncol. 2014 Jan;15(1):e42-50
pubmed: 24384493
Lancet Oncol. 2013 Nov;14(12):1183-92
pubmed: 24095300
J Clin Oncol. 2016 Nov 20;34(33):3959-3962
pubmed: 27551115
Ann Oncol. 2004 May;15(5):751-8
pubmed: 15111342
J Natl Cancer Inst. 2008 Apr 16;100(8):542-51
pubmed: 18398097
Lancet Oncol. 2014 Jun;15(7):747-56
pubmed: 24794243
Oncologist. 2011;16(11):1527-34
pubmed: 22042783
BMJ. 2021 Sep 15;374:n2059
pubmed: 34526303
Ann Oncol. 2014 Dec;25(12):2363-2372
pubmed: 25223482
Breast Cancer Res. 2019 Aug 28;21(1):97
pubmed: 31455425
Lancet Oncol. 2014 Feb;15(2):201-12
pubmed: 24360787
J Clin Oncol. 2014 Dec 1;32(34):3883-91
pubmed: 25349292
Eur J Cancer. 2019 Sep;118:169-177
pubmed: 31377477
Lancet Oncol. 2011 Jun;12(6):527-39
pubmed: 21570352
Breast Cancer Res Treat. 2008 Jun;109(3):481-9
pubmed: 17653851
Cir Cir. 2008 Jan-Feb;76(1):23-8
pubmed: 18492416
Breast Cancer Res Treat. 2017 Oct;165(3):593-600
pubmed: 28674765
Lancet Oncol. 2016 Jun;17(6):791-800
pubmed: 27179402
Lancet Oncol. 2012 Jan;13(1):25-32
pubmed: 22153890
J Clin Oncol. 2012 Mar 20;30(9):930-5
pubmed: 22331946
Breast Cancer Res Treat. 2013 Dec;142(3):549-58
pubmed: 24292815
J Clin Oncol. 2011 Mar 10;29(8):1014-21
pubmed: 21220618
Ann Oncol. 2005 Sep;16(9):1435-41
pubmed: 15946977
Lancet Oncol. 2017 Jul;18(7):917-928
pubmed: 28592386
Nat Med. 2021 Feb;27(2):301-309
pubmed: 33558722
Clin Cancer Res. 2007 Jan 1;13(1):228-33
pubmed: 17200359
Breast Cancer Res Treat. 2005 Mar;90(2):169-85
pubmed: 15803364
N Engl J Med. 2019 Feb 14;380(7):617-628
pubmed: 30516102
Stat Med. 2012 Nov 10;31(25):2973-84
pubmed: 22711298
JAMA Oncol. 2021 Nov 01;7(11):1654-1663
pubmed: 34529000
Lancet Oncol. 2018 Dec;19(12):1630-1640
pubmed: 30413379
Pharm Stat. 2006 Jul-Sep;5(3):173-86
pubmed: 17080751
Breast Cancer Res Treat. 2010 Aug;122(3):787-94
pubmed: 20559708
Ann Oncol. 2011 Mar;22(3):609-617
pubmed: 20732932
N Engl J Med. 2020 Dec 24;383(26):2557-2570
pubmed: 33369357
Lancet. 2010 Jan 30;375(9712):377-84
pubmed: 20113825
Lancet. 2012 Feb 18;379(9816):633-40
pubmed: 22257673
Eur J Cancer. 2022 Jan;160:100-111
pubmed: 34801353
Biometrics. 1998 Sep;54(3):1014-29
pubmed: 9840970
J Clin Oncol. 2005 May 1;23(13):2988-95
pubmed: 15860854
Eur J Cancer. 2013 Jul;49(10):2284-93
pubmed: 23541564
Lancet. 2014 Jul 12;384(9938):164-72
pubmed: 24529560
Clin Cancer Res. 2017 Aug 1;23(15):4035-4045
pubmed: 28270498
Ann Oncol. 2011 Sep;22(9):1999-2006
pubmed: 21382868
J Clin Oncol. 2003 Mar 1;21(5):843-50
pubmed: 12610183
N Engl J Med. 2017 Jun 1;376(22):2147-2159
pubmed: 28564564
J Clin Oncol. 1999 Nov;17(11):3412-7
pubmed: 10550135
Biostatistics. 2000 Mar;1(1):49-67
pubmed: 12933525
Ann Oncol. 2010 Apr;21(4):707-716
pubmed: 19815652
J Clin Oncol. 2005 Dec 1;23(34):8664-70
pubmed: 16260700
J Clin Oncol. 2008 Feb 10;26(5):778-85
pubmed: 18258986
Ann Oncol. 2005 Sep;16(9):1469-76
pubmed: 15956038
Ann Oncol. 2014 Jan;25(1):81-9
pubmed: 24273046

Auteurs

Fabio Conforti (F)

Division of Melanoma, Sarcomas and Rare Tumors, IEO, European Institute of Oncology, IRCCS, Milan, Italy.

Laura Pala (L)

Division of Melanoma, Sarcomas and Rare Tumors, IEO, European Institute of Oncology, IRCCS, Milan, Italy.

Isabella Sala (I)

Department of Statistics and Quantitative Methods, University of Milan-Bicocca, Milan, Italy.

Chiara Oriecuia (C)

Department of Clinical and Experimental Sciences, University of Brescia, Brescia, Italy.

Tommaso De Pas (T)

Division of Melanoma, Sarcomas and Rare Tumors, IEO, European Institute of Oncology, IRCCS, Milan, Italy.

Claudia Specchia (C)

Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy.

Rossella Graffeo (R)

Breast Unit of Southern Switzerland, Oncology Institute of Southern Switzerland, Bellinzona, Switzerland.

Eleonora Pagan (E)

Department of Statistics and Quantitative Methods, University of Milan-Bicocca, Milan, Italy.

Paola Queirolo (P)

Division of Melanoma, Sarcomas and Rare Tumors, IEO, European Institute of Oncology, IRCCS, Milan, Italy.

Elisabetta Pennacchioli (E)

Division of Melanoma, Sarcomas and Rare Tumors, IEO, European Institute of Oncology, IRCCS, Milan, Italy.

Marco Colleoni (M)

Division of Medical Senology, IEO, European Institute of Oncology IRCCS, Milan, Italy.

Giuseppe Viale (G)

Department of Pathology, IEO, European Institute of Oncology, IRCCS, Milan, Italy.
University of Milan, Milan, Italy.

Vincenzo Bagnardi (V)

Department of Statistics and Quantitative Methods, University of Milan-Bicocca, Milan, Italy.

Richard D Gelber (RD)

Medical School, Harvard T H Chan School of Public Health, and Frontier Science and Technology Research Foundation, Boston, MA, USA.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH