Real-Time Targeted Genome Profile Analysis of Pancreatic Ductal Adenocarcinomas Identifies Genetic Alterations That Might Be Targeted With Existing Drugs or Used as Biomarkers.


Journal

Gastroenterology
ISSN: 1528-0012
Titre abrégé: Gastroenterology
Pays: United States
ID NLM: 0374630

Informations de publication

Date de publication:
06 2019
Historique:
received: 28 05 2018
revised: 20 02 2019
accepted: 27 02 2019
pubmed: 6 3 2019
medline: 30 6 2019
entrez: 6 3 2019
Statut: ppublish

Résumé

It has been a challenge to select treatment for patients with pancreatic ductal adenocarcinomas (PDACs) based on genome alterations. We performed targeted genomic profile analyses of a large number of PDACs to assess the full spectrum of actionable genomic alterations. We performed targeted genomic profile analyses of 3594 PDAC samples from an international cohort, including capture-based targeted genomic profiling of as many as 315 cancer-associated genes and intron regions of 28 genes that are rearranged in cancer cells. Tumor mutation burden (TMB) and microsatellite instability (MSI) status were also assessed. TMB was calculated across a 1.14-megabase region; TMB-high was defined as ≥20 mutations/megabase. MSI-high status was assigned based on analysis of 114 intron homopolymer loci. KRAS, TP53, CDKN2A, and SMAD4 were the most frequently altered genes in PDAC. We found KRAS mutations in 88% of samples. Among PDACs without mutations in KRAS, we found alterations in genes whose products are in the mitogen-activated protein kinase signaling pathway and are candidate drug targets (actionable targets, n = 132; 4%), as well as gene fusions (n = 51), gene amplifications (n = 35), genes with missense mutations (n = 30), and genes that contain deletions (n = 16). Many of these encode proteins in receptor tyrosine kinase, RAS, or mitogen-activated protein kinase signaling pathways. Aside from TP53, alterations in genes encoding DNA damage repair proteins (BRCA and FANC) were detected in 14% of PDACs. Among PDACs evaluated for MSI (n = 2563) and TMB (n = 1021), MSI-high and/or TMB-high phenotypes were detected in 0.5% of samples. Alterations in FGF23, CCND2, PIK3CA, and FGF6 were more commonly detected in intraductal papillary mucinous neoplasm-associated PDACs. In targeted genomic profile analyses of 3594 PDACs, we found 17% to contain genomic alterations that might make the tumor cells susceptible to currently used anticancer agents. We identified mutations in genes that could contribute to progression of intraductal papillary mucinous neoplasms into malignancies. These alterations might be used as biomarkers for early detection.

Sections du résumé

BACKGROUND & AIMS
It has been a challenge to select treatment for patients with pancreatic ductal adenocarcinomas (PDACs) based on genome alterations. We performed targeted genomic profile analyses of a large number of PDACs to assess the full spectrum of actionable genomic alterations.
METHODS
We performed targeted genomic profile analyses of 3594 PDAC samples from an international cohort, including capture-based targeted genomic profiling of as many as 315 cancer-associated genes and intron regions of 28 genes that are rearranged in cancer cells. Tumor mutation burden (TMB) and microsatellite instability (MSI) status were also assessed. TMB was calculated across a 1.14-megabase region; TMB-high was defined as ≥20 mutations/megabase. MSI-high status was assigned based on analysis of 114 intron homopolymer loci.
RESULTS
KRAS, TP53, CDKN2A, and SMAD4 were the most frequently altered genes in PDAC. We found KRAS mutations in 88% of samples. Among PDACs without mutations in KRAS, we found alterations in genes whose products are in the mitogen-activated protein kinase signaling pathway and are candidate drug targets (actionable targets, n = 132; 4%), as well as gene fusions (n = 51), gene amplifications (n = 35), genes with missense mutations (n = 30), and genes that contain deletions (n = 16). Many of these encode proteins in receptor tyrosine kinase, RAS, or mitogen-activated protein kinase signaling pathways. Aside from TP53, alterations in genes encoding DNA damage repair proteins (BRCA and FANC) were detected in 14% of PDACs. Among PDACs evaluated for MSI (n = 2563) and TMB (n = 1021), MSI-high and/or TMB-high phenotypes were detected in 0.5% of samples. Alterations in FGF23, CCND2, PIK3CA, and FGF6 were more commonly detected in intraductal papillary mucinous neoplasm-associated PDACs.
CONCLUSIONS
In targeted genomic profile analyses of 3594 PDACs, we found 17% to contain genomic alterations that might make the tumor cells susceptible to currently used anticancer agents. We identified mutations in genes that could contribute to progression of intraductal papillary mucinous neoplasms into malignancies. These alterations might be used as biomarkers for early detection.

Identifiants

pubmed: 30836094
pii: S0016-5085(19)32505-3
doi: 10.1053/j.gastro.2019.02.037
pii:
doi:

Substances chimiques

Antineoplastic Agents 0
Biomarkers, Tumor 0
FGF23 protein, human 0
Fibroblast Growth Factor-23 7Q7P4S7RRE

Types de publication

Journal Article Multicenter Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

2242-2253.e4

Informations de copyright

Copyright © 2019 AGA Institute. Published by Elsevier Inc. All rights reserved.

Auteurs

Aatur D Singhi (AD)

Department of Pathology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania. Electronic address: singhiad@upmc.edu.

Ben George (B)

Department of Medicine, Division of Medical Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin.

Joel R Greenbowe (JR)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Jon Chung (J)

Foundation Medicine, Inc, Cambridge, Massachusetts.

James Suh (J)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Anirban Maitra (A)

Sheikh Ahmed Pancreatic Cancer Research Center, University of Texas MD Anderson Cancer Center, Houston, Texas.

Samuel J Klempner (SJ)

The Angeles Clinic and Research Institute, Los Angeles, California; Division of Hematology and Oncology, Cedars-Sinai Medical Center, Los Angeles, California.

Andrew Hendifar (A)

Division of Hematology and Oncology, Cedars-Sinai Medical Center, Los Angeles, California.

Javle M Milind (JM)

Sheikh Ahmed Pancreatic Cancer Research Center, University of Texas MD Anderson Cancer Center, Houston, Texas.

Talia Golan (T)

Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Randall E Brand (RE)

Department of Internal Medicine, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.

Amer H Zureikat (AH)

Department of Surgery, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.

Somak Roy (S)

Department of Pathology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.

Alexa B Schrock (AB)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Vincent A Miller (VA)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Jeffrey S Ross (JS)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Siraj M Ali (SM)

Foundation Medicine, Inc, Cambridge, Massachusetts.

Nathan Bahary (N)

Department of Internal Medicine, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.

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