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Case definition and classification of leukodystrophies and leukoencephalopathies

Authors :
Vanderver, Adeline
Prust, Morgan
Tonduti, Davide
Mochel, Fanny
Hussey, Heather
Helman, Guy
Garbern, James
Eichler, Florian
Labauge, Pierre
Aubourg, Patrick
Rodriguez, Diana
Patterson, Marc
van Hove, Johan
Schmidt, Johanna
Wolf, Nicole
Boespflug-Tanguy, Odile
Schiffmann, Raphael
van Der Knaap, Marjo
Consortium, Glia
Children's National Medical Center
Institut du Cerveau et de la Moëlle Epinière = Brain and Spine Institute (ICM)
Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP]
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS)
Département de neurologie [Montpellier]
Université Montpellier 1 (UM1)-Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier)-Hôpital Gui de Chauliac [CHU Montpellier]
Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier)-Université de Montpellier (UM)
Université de Montpellier (UM)
AP-HP Hôpital Bicêtre (Le Kremlin-Bicêtre)
Immunologie et génétique du diabète de type 1, génétique multifactorielle en endocrinologie pédiatrique (U986)
Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)
CHU Trousseau [APHP]
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)
Physiopathologie et neuroprotection des atteintes du cerveau en développement
Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM)
Service de neurologie pédiatrique et maladies métaboliques
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Robert Debré-Université Paris Diderot - Paris 7 (UPD7)
Neuroprotection du Cerveau en Développement / Promoting Research Oriented Towards Early Cns Therapies (PROTECT)
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Robert Debré-Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM)
Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-CHU Pitié-Salpêtrière [AP-HP]
Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)
Université Montpellier 1 (UM1)-Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier)-Hôpital Gui de Chauliac [Montpellier]-Université de Montpellier (UM)
Service de Neuropédiatrie [CHU Trousseau]
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Trousseau [APHP]
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU)
Pediatric surgery
NCA - Brain mechanisms in health and disease
Neuroscience Campus Amsterdam - Brain Mechanisms in Health & Disease
Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [APHP]-Centre National de la Recherche Scientifique (CNRS)
Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-CHU Trousseau [APHP]
Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Hôpital Robert Debré-Université Paris Diderot - Paris 7 (UPD7)
Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Hôpital Robert Debré-Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM)
Source :
Molecular Genetics and Metabolism, Molecular Genetics and Metabolism, 2015, 114 (4), pp.494-500. ⟨10.1016/j.ymgme.2015.01.006⟩, Molecular Genetics and Metabolism, Elsevier, 2015, 114 (4), pp.494-500. ⟨10.1016/j.ymgme.2015.01.006⟩, Molecular Genetics and Metabolism, 114(4), 494-500. Academic Press Inc., Vanderver, A, Prust, M, Tonduti, D, Mochel, F, Hussey, H M, Helman, G, Garbern, J, Eichler, F, Labauge, P, Aubourg, P, Rodriguez, D, Patterson, M C, van Hove, J LK, Schmidt, J, Wolf, N I, Boespflug-Tanguy, O, Schiffmann, R & van der Knaap, M S 2015, ' Case definition and classification of leukodystrophies and leukoencephalopathies ', Molecular Genetics and Metabolism, vol. 114, no. 4, pp. 494-500 . https://doi.org/10.1016/j.ymgme.2015.01.006
Publication Year :
2015
Publisher :
HAL CCSD, 2015.

Abstract

International audience; OBJECTIVE:An approved definition of the term leukodystrophy does not currently exist. The lack of a precise case definition hampers efforts to study the epidemiology and the relevance of genetic white matter disorders to public health.METHOD:Thirteen experts at multiple institutions participated in iterative consensus building surveys to achieve definition and classification of disorders as leukodystrophies using a modified Delphi approach.RESULTS:A case definition for the leukodystrophies was achieved, and a total of 30 disorders were classified under this definition. In addition, a separate set of disorders with heritable white matter abnormalities but not meeting criteria for leukodystrophy, due to presumed primary neuronal involvement and prominent systemic manifestations, was classified as genetic leukoencephalopathies (gLE).INTERPRETATION:A case definition of leukodystrophies and classification of heritable white matter disorders will permit more detailed epidemiologic studies of these disorders.

Details

Language :
English
ISSN :
10967192 and 10967206
Database :
OpenAIRE
Journal :
Molecular Genetics and Metabolism, Molecular Genetics and Metabolism, 2015, 114 (4), pp.494-500. ⟨10.1016/j.ymgme.2015.01.006⟩, Molecular Genetics and Metabolism, Elsevier, 2015, 114 (4), pp.494-500. ⟨10.1016/j.ymgme.2015.01.006⟩, Molecular Genetics and Metabolism, 114(4), 494-500. Academic Press Inc., Vanderver, A, Prust, M, Tonduti, D, Mochel, F, Hussey, H M, Helman, G, Garbern, J, Eichler, F, Labauge, P, Aubourg, P, Rodriguez, D, Patterson, M C, van Hove, J LK, Schmidt, J, Wolf, N I, Boespflug-Tanguy, O, Schiffmann, R & van der Knaap, M S 2015, ' Case definition and classification of leukodystrophies and leukoencephalopathies ', Molecular Genetics and Metabolism, vol. 114, no. 4, pp. 494-500 . https://doi.org/10.1016/j.ymgme.2015.01.006
Accession number :
edsair.doi.dedup.....2e5922150f62db48eef439e679a84e18
Full Text :
https://doi.org/10.1016/j.ymgme.2015.01.006⟩