Literature DB >> 30851990

An Age-Based Framework for Evaluating Genome-Scale Sequencing Results in Newborn Screening.

Laura V Milko1, Julianne M O'Daniel1, Daniela M DeCristo1, Stephanie B Crowley1, Ann Katherine M Foreman1, Kathleen E Wallace1, Lonna F Mollison1, Natasha T Strande2, Zahra S Girnary1, Lacey J Boshe1, Arthur S Aylsworth3, Muge Gucsavas-Calikoglu3, Dianne M Frazier3, Neeta L Vora4, Myra I Roche5, Bradford C Powell1, Cynthia M Powell5, Jonathan S Berg6.   

Abstract

OBJECTIVE: To assess the performance of a standardized, age-based metric for scoring clinical actionability to evaluate conditions for inclusion in newborn screening and compare it with the results from other contemporary methods. STUDY
DESIGN: The North Carolina Newborn Exome Sequencing for Universal Screening study developed an age-based, semiquantitative metric to assess the clinical actionability of gene-disease pairs and classify them with respect to age of onset or timing of interventions. This categorization was compared with the gold standard Recommended Uniform Screening Panel and other methods to evaluate gene-disease pairs for newborn genomic sequencing.
RESULTS: We assessed 822 gene-disease pairs, enriched for pediatric onset of disease and suspected actionability. Of these, 466 were classified as having childhood onset and high actionability, analogous to conditions selected for the Recommended Uniform Screening Panel core panel. Another 245 were classified as having childhood onset and low to no actionability, 25 were classified as having adult onset and high actionability, 19 were classified as having adult onset and low to no actionability, and 67 were excluded due to controversial evidence and/or prenatal onset.
CONCLUSIONS: This study describes a novel method to facilitate decisions about the potential use of genomic sequencing for newborn screening. These categories may assist parents and physicians in making informed decisions about the disclosure of results from voluntary genomic sequencing in children.
Copyright © 2019 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  ASQM; NC NEXUS; NGS-NBS; NSIGHT; actionability; decision-making; disclosure; genetics

Mesh:

Year:  2019        PMID: 30851990      PMCID: PMC6535354          DOI: 10.1016/j.jpeds.2018.12.027

Source DB:  PubMed          Journal:  J Pediatr        ISSN: 0022-3476            Impact factor:   4.406


  48 in total

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Authors:  Muin J Khoury; Linda L McCabe; Edward R B McCabe
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3.  Newborn screening: toward a uniform screening panel and system--executive summary.

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Journal:  Pediatrics       Date:  2006-05       Impact factor: 7.124

4.  Genetic testing in asymptomatic minors: background considerations towards ESHG Recommendations.

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5.  Revisiting Wilson and Jungner in the genomic age: a review of screening criteria over the past 40 years.

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Journal:  Bull World Health Organ       Date:  2008-04       Impact factor: 9.408

6.  Rapid whole-genome sequencing for genetic disease diagnosis in neonatal intensive care units.

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Journal:  Sci Transl Med       Date:  2012-10-03       Impact factor: 17.956

Review 7.  Recent developments and new applications of tandem mass spectrometry in newborn screening.

Authors:  Piero Rinaldo; Silvia Tortorelli; Dietrich Matern
Journal:  Curr Opin Pediatr       Date:  2004-08       Impact factor: 2.856

8.  Screening newborns for inborn errors of metabolism by tandem mass spectrometry.

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9.  Newborn screening: toward a uniform screening panel and system.

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Journal:  Genet Med       Date:  2006-05       Impact factor: 8.822

10.  Technical report: Ethical and policy issues in genetic testing and screening of children.

Authors:  Lainie Friedman Ross; Laine Friedman Ross; Howard M Saal; Karen L David; Rebecca R Anderson
Journal:  Genet Med       Date:  2013-02-21       Impact factor: 8.822

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  19 in total

1.  Exome/Genome-Wide Testing in Newborn Screening: A Proportionate Path Forward.

Authors:  Vasiliki Rahimzadeh; Jan M Friedman; Guido de Wert; Bartha M Knoppers
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2.  Genomic Sequencing for Newborn Screening: Results of the NC NEXUS Project.

Authors:  Tamara S Roman; Stephanie B Crowley; Myra I Roche; Ann Katherine M Foreman; Julianne M O'Daniel; Bryce A Seifert; Kristy Lee; Alicia Brandt; Chelsea Gustafson; Daniela M DeCristo; Natasha T Strande; Lori Ramkissoon; Laura V Milko; Phillips Owen; Sayanty Roy; Mai Xiong; Ryan S Paquin; Rita M Butterfield; Megan A Lewis; Katherine J Souris; Donald B Bailey; Christine Rini; Jessica K Booker; Bradford C Powell; Karen E Weck; Cynthia M Powell; Jonathan S Berg
Journal:  Am J Hum Genet       Date:  2020-08-26       Impact factor: 11.025

Review 3.  A pediatric perspective on genomics and prevention in the twenty-first century.

Authors:  Bimal P Chaudhari; Kandamurugu Manickam; Kim L McBride
Journal:  Pediatr Res       Date:  2019-10-02       Impact factor: 3.756

4.  Next-generation sequencing for constitutional variants in the clinical laboratory, 2021 revision: a technical standard of the American College of Medical Genetics and Genomics (ACMG).

Authors:  Catherine Rehder; Lora J H Bean; David Bick; Elizabeth Chao; Wendy Chung; Soma Das; Julianne O'Daniel; Heidi Rehm; Vandana Shashi; Lisa M Vincent
Journal:  Genet Med       Date:  2021-04-29       Impact factor: 8.822

5.  Second Tier Molecular Genetic Testing in Newborn Screening for Pompe Disease: Landscape and Challenges.

Authors:  Laurie D Smith; Matthew N Bainbridge; Richard B Parad; Arindam Bhattacharjee
Journal:  Int J Neonatal Screen       Date:  2020-04-05

6.  An online compendium of treatable genetic disorders.

Authors:  David Bick; Sarah L Bick; David P Dimmock; Tom A Fowler; Mark J Caulfield; Richard H Scott
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7.  Actionability of commercial laboratory sequencing panels for newborn screening and the importance of transparency for parental decision-making.

Authors:  Daniela M DeCristo; Laura V Milko; Julianne M O'Daniel; Ann Katherine M Foreman; Lonna F Mollison; Bradford C Powell; Cynthia M Powell; Jonathan S Berg
Journal:  Genome Med       Date:  2021-03-29       Impact factor: 11.117

8.  Evaluating the clinical utility of early exome sequencing in diverse pediatric outpatient populations in the North Carolina Clinical Genomic Evaluation of Next-generation Exome Sequencing (NCGENES) 2 study: a randomized controlled trial.

Authors:  Jonathan S Berg; Jeannette T Bensen; Brooke S Staley; Laura V Milko; Margaret Waltz; Ida Griesemer; Lonna Mollison; Tracey L Grant; Laura Farnan; Myra Roche; Angelo Navas; Alexandra Lightfoot; Ann Katherine M Foreman; Julianne M O'Daniel; Suzanne C O'Neill; Feng-Chang Lin; Tamara S Roman; Alicia Brandt; Bradford C Powell; Christine Rini
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Review 9.  FDA oversight of NSIGHT genomic research: the need for an integrated systems approach to regulation.

Authors:  Laura V Milko; Flavia Chen; Kee Chan; Amy M Brower; Pankaj B Agrawal; Alan H Beggs; Jonathan S Berg; Steven E Brenner; Ingrid A Holm; Barbara A Koenig; Richard B Parad; Cynthia M Powell; Stephen F Kingsmore
Journal:  NPJ Genom Med       Date:  2019-12-10       Impact factor: 8.617

10.  Principles of Genomic Newborn Screening Programs: A Systematic Review.

Authors:  Lilian Downie; Jane Halliday; Sharon Lewis; David J Amor
Journal:  JAMA Netw Open       Date:  2021-07-01
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