Germline Genetic Variant Classification Requires More Equitable Reference Database Representation

Germline genetic testing can aid diagnostic workups but may reveal variants of unknown significance (VUS). VUS do not provide definitive diagnoses and may worsen patient and family stress. VUS are more prevalent in populations that are underrepresented in large genomic databases. We describe 7 pediatric hematology-oncology patients from the highly diverse Bronx County for whom VUS, novel, or rare pathogenic variant identification influenced clinical management and outcomes. For these cases, further investigation and/or variant reclassification ultimately led to diagnoses of von Hippel-Lindau syndrome, hemophagocytic lymphohistiocytosis, atypical hemolytic uremic syndrome, severe combined immunodeficiency, and Fanconi anemia. Through discussion of these cases, we illustrate how providers may gain insight into the pathogenicity of VUS and highlight the importance of incorporating underrepresented populations into genomic databases to improve variant characterization and address health care disparities.

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Publication Details

Journal
PEDIATRICS
Published
2025-03-06
DOI
https://doi.org/10.1542/peds.2024-068229
Primary Topic
Immunodeficiency and Autoimmune Disorders
Type
article
Field-Weighted Citation Impact
0.00
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article

Germline Genetic Variant Classification Requires More Equitable Reference Database Representation

David M. Loeb, Daniel A. Weiser, Shana R. Burstein, William Jerome et al.
PEDIATRICS
Immunodeficiency and Autoimmune Disorders
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Germline Genetic Variant Classification Requires More Equitable Reference Database Representation

David M. Loeb, Daniel A. Weiser, Shana R. Burstein, William Jerome, Miranda Di Biase, Hailey Reisert, Thelma Alessandra Sugrañes, Janki Patel, Eva Spier
article en

Abstract

Germline genetic testing can aid diagnostic workups but may reveal variants of unknown significance (VUS). VUS do not provide definitive diagnoses and may worsen patient and family stress. VUS are more prevalent in populations that are underrepresented in large genomic databases. We describe 7 pediatric hematology-oncology patients from the highly diverse Bronx County for whom VUS, novel, or rare pathogenic variant identification influenced clinical management and outcomes. For these cases, further investigation and/or variant reclassification ultimately led to diagnoses of von Hippel-Lindau syndrome, hemophagocytic lymphohistiocytosis, atypical hemolytic uremic syndrome, severe combined immunodeficiency, and Fanconi anemia. Through discussion of these cases, we illustrate how providers may gain insight into the pathogenicity of VUS and highlight the importance of incorporating underrepresented populations into genomic databases to improve variant characterization and address health care disparities.

PEDIATRICSVol. 155(4)
Albert Einstein College of Medicine (US), Children's Hospital at Montefiore (US)
Openalex Percentile: Top 95%
Immunodeficiency and Autoimmune Disorders
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