Single-nucleotide variants, tumour mutational burden and microsatellite instability in patients with metastatic colorectal cancer: Next-generation sequencing results of the FIRE-3 trial.


Journal

European journal of cancer (Oxford, England : 1990)
ISSN: 1879-0852
Titre abrégé: Eur J Cancer
Pays: England
ID NLM: 9005373

Informations de publication

Date de publication:
09 2020
Historique:
received: 20 04 2020
revised: 26 06 2020
accepted: 04 07 2020
pubmed: 19 8 2020
medline: 9 2 2021
entrez: 19 8 2020
Statut: ppublish

Résumé

Molecular biomarkers and primary tumour sidedness guide treatment decisions in metastatic colorectal cancer. Comprehensive molecular profiling aims to identify targetable alterations and measure tumour mutational burden (TMB) to enable precision oncology. FoundationOne® next-generation sequencing identified single-nucleotide variants (SNVs), copy number alterations, high TMB (TMB-H) and high-grade microsatellite instability (MSI-H) in patients treated in the FIRE-3 trial. Data were correlated with objective response rate (ORR), progression-free survival (PFS) and overall survival (OS). Three hundred seventy-three (49.6%) of 752 patients provided material for this analysis. Frequent SNVs included TP53, APC, KRAS, PIK3CA, BRAF, SMAD4 and FBXW7. KRAS, BRAF V600E and SMAD4 mutations were confirmed as prognostic biomarkers by logistic penalised regression for ORR. OS was significantly longer in patients with SMAD4 wild-type (WT) tumours than in those with SMAD4-mutated tumours (hazard ratio = 0.59 [95% confidence interval {CI} = 0.34-1.01], p = 0.05), with a higher probability of ORR [odds ratio, SMAD4 SNV versus WT = 0.32 [95% CI = 0.10-0.98], p = 0.05] when treated with cetuximab. MSI-H (30.0%, p = 0.03) and TMB-H (17.3%, p = 0.003) tumours were enriched by FBXW7 mutations. Numerically lower ORR, OS and PFS were observed in MSI-H tumours. RAS, BRAF V600E and SMAD4 mutations were identified as poor prognostic biomarkers in patients of the FIRE-3 trial, whereas improved outcome was observed for BRAF non-V600E mutation. SMAD4 mutation might provide predictive relevance for cetuximab efficacy. MSI-H tumours showed numerically lower ORR, OS and PFS.

Sections du résumé

BACKGROUND
Molecular biomarkers and primary tumour sidedness guide treatment decisions in metastatic colorectal cancer. Comprehensive molecular profiling aims to identify targetable alterations and measure tumour mutational burden (TMB) to enable precision oncology.
MATERIAL AND METHODS
FoundationOne® next-generation sequencing identified single-nucleotide variants (SNVs), copy number alterations, high TMB (TMB-H) and high-grade microsatellite instability (MSI-H) in patients treated in the FIRE-3 trial. Data were correlated with objective response rate (ORR), progression-free survival (PFS) and overall survival (OS).
RESULTS
Three hundred seventy-three (49.6%) of 752 patients provided material for this analysis. Frequent SNVs included TP53, APC, KRAS, PIK3CA, BRAF, SMAD4 and FBXW7. KRAS, BRAF V600E and SMAD4 mutations were confirmed as prognostic biomarkers by logistic penalised regression for ORR. OS was significantly longer in patients with SMAD4 wild-type (WT) tumours than in those with SMAD4-mutated tumours (hazard ratio = 0.59 [95% confidence interval {CI} = 0.34-1.01], p = 0.05), with a higher probability of ORR [odds ratio, SMAD4 SNV versus WT = 0.32 [95% CI = 0.10-0.98], p = 0.05] when treated with cetuximab. MSI-H (30.0%, p = 0.03) and TMB-H (17.3%, p = 0.003) tumours were enriched by FBXW7 mutations. Numerically lower ORR, OS and PFS were observed in MSI-H tumours.
CONCLUSIONS
RAS, BRAF V600E and SMAD4 mutations were identified as poor prognostic biomarkers in patients of the FIRE-3 trial, whereas improved outcome was observed for BRAF non-V600E mutation. SMAD4 mutation might provide predictive relevance for cetuximab efficacy. MSI-H tumours showed numerically lower ORR, OS and PFS.

Identifiants

pubmed: 32810748
pii: S0959-8049(20)30386-5
doi: 10.1016/j.ejca.2020.07.003
pii:
doi:

Types de publication

Clinical Trial, Phase III Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

250-259

Commentaires et corrections

Type : ErratumIn

Informations de copyright

Copyright © 2020 Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Conflict of interest statement A.S. received honoraria for talks and travel expenses from Roche. C.B.W. received honoraria for talks, advisory boards and travel expenses from Bayer, Celgene, Ipsen, Rafael, RedHill, Roche, Shire/Baxalta and Servier and scientific grant support from Roche. V.H., S.S. and D.P.M. received honoraria for talks, advisory boards and travel expenses from Merck, Amgen, Roche, Takeda, Servier, Pierre Fabre, Taiho, Lilly Oncology, Servier, Sanofi and Bayer Pharmaceuticals. A.J. and T.K. received honoraria for talks, advisory boards and travel expenses from Amgen, AstraZeneca, Bayer Pharmaceuticals, BMS, Boehringer Ingelheim, Merck KGA, Novartis, Qiagen, Roche Pharma and Takeda. Marl.M. received honoraria for talks from SIRTeX, Roche and MSD and travel expenses from SIRTeX, Amgen and Merck. All remaining authors declared no potential conflict of interest in regard with this manuscript.

Auteurs

Arndt Stahler (A)

Department of Medicine III, University Hospital, University of Munich, Marchioninistrasse 15, 81377, Munich, Germany. Electronic address: arndt.stahler@med.uni-muenchen.de.

Sebastian Stintzing (S)

Medical Department, Division of Hematology, Oncology and Tumor Immunology (CCM), Charité Universitaetsmedizin Berlin, Charitéplatz 1, 10117, Berlin, Germany; DKTK, German Cancer Consortium, German Cancer Research Centre (DKFZ), Im Neuenheimer Feld 280, 69120, Heidelberg, Germany.

Jobst C von Einem (JC)

Medical Department, Division of Hematology, Oncology and Tumor Immunology (CCM), Charité Universitaetsmedizin Berlin, Charitéplatz 1, 10117, Berlin, Germany.

Christoph B Westphalen (CB)

Department of Medicine III, University Hospital, University of Munich, Marchioninistrasse 15, 81377, Munich, Germany.

Kathrin Heinrich (K)

Department of Medicine III, University Hospital, University of Munich, Marchioninistrasse 15, 81377, Munich, Germany.

Nicole Krämer (N)

STABURO Statistical Consulting GmbH, Aschauer Strasse 26b, 81549, Munich, Germany.

Marlies Michl (M)

Department of Medicine III, University Hospital, University of Munich, Marchioninistrasse 15, 81377, Munich, Germany.

Dominik P Modest (DP)

Medical Department, Division of Hematology, Oncology and Tumor Immunology (CVK), Charité Universitaetsmedizin Berlin, MIttelallee 11, 10117, Berlin, Germany; DKTK, German Cancer Consortium, German Cancer Research Centre (DKFZ), Im Neuenheimer Feld 280, 69120, Heidelberg, Germany.

Ludwig Fischer von Weikersthal (LF)

Klinikum St. Marien, Oncology, Mariahilfbergweg 7, 92224, Amberg, Germany.

Thomas Decker (T)

Onkologische Praxis, Elisabethenstrasse 19, 88212, Ravensburg, Germany.

Alexander Kiani (A)

Department of Medicine IV, Klinikum Bayreuth GmbH, Preuschwitzer Strasse 101, 95445, Bayreuth, Germany.

Tobias Heintges (T)

Department of Medicine II, Lukaskrankenhaus, Preussenstrasse 84, 41464, Neuss, Germany.

Christoph Kahl (C)

Department of Hematology, Oncology and Palliative Care, Klinikum Magdeburg gGmbH, Birkenallee 34, 39130, Magdeburg, Germany.

Frank Kullmann (F)

Department of Internal Medicine I, Klinikum Weiden, Soellnerstrasse 16, 92637, Weiden, Germany.

Werner Scheithauer (W)

Department of Internal Medicine I & CCC, Medical University Vienna, Heiligenstaedter Strasse 46-48, 1090, Vienna, Austria.

Markus Moehler (M)

University Medical Center Mainz, I. Dept. of Internal Medicine, Langenbeckstrasse 1, 55131, Mainz, Germany.

Florian Kaiser (F)

VK&K Studien GbR, Achdorfer Weg 5a, 84036, Landshut, Germany.

Thomas Kirchner (T)

Institute of Pathology, University of Munich, Thalkirchner Strasse 36a, 80337, Munich, Germany; DKTK, German Cancer Consortium, German Cancer Research Centre (DKFZ), Im Neuenheimer Feld 280, 69120, Heidelberg, Germany.

Andreas Jung (A)

Institute of Pathology, University of Munich, Thalkirchner Strasse 36a, 80337, Munich, Germany; DKTK, German Cancer Consortium, German Cancer Research Centre (DKFZ), Im Neuenheimer Feld 280, 69120, Heidelberg, Germany.

Volker Heinemann (V)

Department of Medicine III, University Hospital, University of Munich, Marchioninistrasse 15, 81377, Munich, Germany; DKTK, German Cancer Consortium, German Cancer Research Centre (DKFZ), Im Neuenheimer Feld 280, 69120, Heidelberg, Germany.

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Classifications MeSH