Exome and genome sequencing in adults with undiagnosed disease: a prospective cohort study.


Journal

Journal of medical genetics
ISSN: 1468-6244
Titre abrégé: J Med Genet
Pays: England
ID NLM: 2985087R

Informations de publication

Date de publication:
04 2021
Historique:
received: 21 02 2020
revised: 29 04 2020
accepted: 02 05 2020
pubmed: 26 6 2020
medline: 3 9 2021
entrez: 26 6 2020
Statut: ppublish

Résumé

Exome and genome sequencing have been demonstrated to increase diagnostic yield in paediatric populations, improving treatment options and providing risk information for relatives. There are limited studies examining the clinical utility of these tests in adults, who currently have limited access to this technology. Patients from adult and cancer genetics clinics across Toronto, Ontario, Canada were recruited into a prospective cohort study evaluating the diagnostic utility of exome and genome sequencing in adults. Eligible patients were ≥18 years of age and suspected of having a hereditary disorder but had received previous uninformative genetic test results. In total, we examined the diagnostic utility of exome and genome sequencing in 47 probands and 34 of their relatives who consented to participate and underwent exome or genome sequencing. Overall, 17% (8/47) of probands had a pathogenic or likely pathogenic variant identified in a gene associated with their primary indication for testing. The diagnostic yield for patients with a cancer history was similar to the yield for patients with a non-cancer history (4/18 (22%) vs 4/29 (14%)). An additional 24 probands (51%) had an inconclusive result. Secondary findings were identified in 10 patients (21%); three had medically actionable results. This study lends evidence to the diagnostic utility of exome or genome sequencing in an undiagnosed adult population. The significant increase in diagnostic yield warrants the use of this technology. The identification and communication of secondary findings may provide added value when using this testing modality as a first-line test.

Sections du résumé

BACKGROUND
Exome and genome sequencing have been demonstrated to increase diagnostic yield in paediatric populations, improving treatment options and providing risk information for relatives. There are limited studies examining the clinical utility of these tests in adults, who currently have limited access to this technology.
METHODS
Patients from adult and cancer genetics clinics across Toronto, Ontario, Canada were recruited into a prospective cohort study evaluating the diagnostic utility of exome and genome sequencing in adults. Eligible patients were ≥18 years of age and suspected of having a hereditary disorder but had received previous uninformative genetic test results. In total, we examined the diagnostic utility of exome and genome sequencing in 47 probands and 34 of their relatives who consented to participate and underwent exome or genome sequencing.
RESULTS
Overall, 17% (8/47) of probands had a pathogenic or likely pathogenic variant identified in a gene associated with their primary indication for testing. The diagnostic yield for patients with a cancer history was similar to the yield for patients with a non-cancer history (4/18 (22%) vs 4/29 (14%)). An additional 24 probands (51%) had an inconclusive result. Secondary findings were identified in 10 patients (21%); three had medically actionable results.
CONCLUSIONS
This study lends evidence to the diagnostic utility of exome or genome sequencing in an undiagnosed adult population. The significant increase in diagnostic yield warrants the use of this technology. The identification and communication of secondary findings may provide added value when using this testing modality as a first-line test.

Identifiants

pubmed: 32581083
pii: jmedgenet-2020-106936
doi: 10.1136/jmedgenet-2020-106936
pmc: PMC8475751
mid: NIHMS1720759
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

275-283

Subventions

Organisme : NHGRI NIH HHS
ID : UM1 HG008900
Pays : United States
Organisme : CIHR
ID : FRN135730
Pays : Canada

Informations de copyright

© Author(s) (or their employer(s)) 2021. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: None declared.

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Auteurs

Salma Shickh (S)

Li Ka Shing Knowledge Institute, St. Michael's Hospital, Unity Health Toronto, Toronto, Ontario, Canada.
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.

Mariana Gutierrez Salazar (M)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Kathleen-Rose Zakoor (KR)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Conxi Lázaro (C)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.
Hereditary Cancer Program, Catalan Institute of Oncology (ICO), Hospital Duran i Reynals, Bellvitge Institute for Biomedical Research (IDIBELL), L'Hospitalet, Barcelona, Spain.
Women's College Research Institute, Women's College Hospital, Toronto, Ontario, Canada.

Jessica Gu (J)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.
Genetics, Medcan Clinic, Toronto, Ontario, Canada.

Jamie Goltz (J)

University of Guelph, Guelph, Ontario, Canada.

Dakota Kleinman (D)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Abdul Noor (A)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Sam Khalouei (S)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Chloe Mighton (C)

Li Ka Shing Knowledge Institute, St. Michael's Hospital, Unity Health Toronto, Toronto, Ontario, Canada.
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.

Emma Reble (E)

Li Ka Shing Knowledge Institute, St. Michael's Hospital, Unity Health Toronto, Toronto, Ontario, Canada.

Rita Kodida (R)

Li Ka Shing Knowledge Institute, St. Michael's Hospital, Unity Health Toronto, Toronto, Ontario, Canada.

Yvonne Bombard (Y)

Li Ka Shing Knowledge Institute, St. Michael's Hospital, Unity Health Toronto, Toronto, Ontario, Canada.
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.

Stephanie DiTroia (S)

Center for Mendelian Genomics, Broad Institute, Cambridge, Massachusetts, USA.

Samantha Baxter (S)

Center for Mendelian Genomics, Broad Institute, Cambridge, Massachusetts, USA.

Nicholas Watkins (N)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.
Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.

Melanie Care (M)

Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.
Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Arnon Adler (A)

Department of Cardiology, Peter Munk Cardiac Centre, Toronto General Hospital, Toronto, Ontario, Canada.
Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.

Sheri Horsburgh (S)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Oana Morar (O)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.
Clinical Genetics, Trillium Health Partners, Mississauga, Ontario, Canada.

Jillian Murphy (J)

Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.
Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Dayna-Lynn Nevay (DL)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Marta Szybowska (M)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Melyssa Aronson (M)

Zane Cohen Centre for Digestive Diseases, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Seema Panchal (S)

Marvelle Koffler Breast Centre, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Ruth Godoy (R)

Marvelle Koffler Breast Centre, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.
Lifelabs, Toronto, Ontario, Canada.

Spring Holter (S)

Zane Cohen Centre for Digestive Diseases, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Susan Randall Armel (S)

Familial Breast and Ovarian Cancer Clinic, Princess Margaret Cancer Centre, Toronto, Ontario, Canada.

Kara Semotiuk (K)

Zane Cohen Centre for Digestive Diseases, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Christine Elser (C)

Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.
Marvelle Koffler Breast Centre, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Raymond H Kim (RH)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.
Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.
Division of Medical Oncology and Hematology, University Health Network, Toronto, Ontario, Canada.

David Chitayat (D)

Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.
Clinical and Metabolic Genetics, The Hospital for Sick Children, Toronto, Ontario, Canada.
The Prenatal Diagnosis and Medical Genetics Program, Department of Obstetrics and Gynecology, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

Joyce So (J)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.
Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.

Hanna Faghfoury (H)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.
Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.

Josh Silver (J)

Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.
Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.

Chantal F Morel (CF)

Fred A. Litwin Family Centre in Genetic Medicine, University Health Network, Toronto, Ontario, Canada.
Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.

Jordan Lerner-Ellis (J)

Pathology and Laboratory Medicine, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada jordan.lerner-ellis@sinaihealth.ca.
Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada.
Lunenfeld Tanenbaum Research Institute, Mount Sinai Hospital, Sinai Health System, Toronto, Ontario, Canada.

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