Restless Legs Syndrome in NKX2-1-related chorea: An expansion of the disease spectrum.


Journal

Brain & development
ISSN: 1872-7131
Titre abrégé: Brain Dev
Pays: Netherlands
ID NLM: 7909235

Informations de publication

Date de publication:
Mar 2019
Historique:
received: 14 03 2018
revised: 13 07 2018
accepted: 01 10 2018
pubmed: 26 10 2018
medline: 6 6 2019
entrez: 25 10 2018
Statut: ppublish

Résumé

Molecular technologies are expanding our knowledge about genetic variability underlying early-onset non-progressive choreic syndromes. Focusing on NKX2-1-related chorea, the clinical phenotype and sleep related disorders have been only partially characterized. We propose a retrospective and longitudinal observational study in 7 patients with non-progressive chorea due to NKX2-1 mutations. In all subjects sleep and awake EEG, brain MRI with study of pituitary gland, chest X-rays, endocrinological investigations were performed. Movement disorders, pattern of sleep and related disorders were investigated using structured clinical evaluation and several validated questionnaires. In patients carrying NKX2-1 mutations, chorea was mainly distributed in the upper limbs and tended to improve with age. All patients presented clinical or subclinical hypothyroidism and delayed motor milestones. Three subjects had symptoms consistent with Restless Legs Syndrome (RLS) that improved with Levodopa. Patients with NKX2-1 gene mutations should be investigated for RLS, which, similarly to chorea, can sometimes be ameliorated by Levodopa.

Sections du résumé

BACKGROUND BACKGROUND
Molecular technologies are expanding our knowledge about genetic variability underlying early-onset non-progressive choreic syndromes. Focusing on NKX2-1-related chorea, the clinical phenotype and sleep related disorders have been only partially characterized.
METHODS METHODS
We propose a retrospective and longitudinal observational study in 7 patients with non-progressive chorea due to NKX2-1 mutations. In all subjects sleep and awake EEG, brain MRI with study of pituitary gland, chest X-rays, endocrinological investigations were performed. Movement disorders, pattern of sleep and related disorders were investigated using structured clinical evaluation and several validated questionnaires.
RESULTS RESULTS
In patients carrying NKX2-1 mutations, chorea was mainly distributed in the upper limbs and tended to improve with age. All patients presented clinical or subclinical hypothyroidism and delayed motor milestones. Three subjects had symptoms consistent with Restless Legs Syndrome (RLS) that improved with Levodopa.
CONCLUSIONS CONCLUSIONS
Patients with NKX2-1 gene mutations should be investigated for RLS, which, similarly to chorea, can sometimes be ameliorated by Levodopa.

Identifiants

pubmed: 30352709
pii: S0387-7604(18)30495-9
doi: 10.1016/j.braindev.2018.10.001
pii:
doi:

Substances chimiques

Dopamine Agents 0
NKX2-1 protein, human 0
Thyroid Nuclear Factor 1 0
Levodopa 46627O600J

Types de publication

Journal Article Observational Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

250-256

Commentaires et corrections

Type : ErratumIn

Informations de copyright

Copyright © 2018 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.

Auteurs

A Iodice (A)

Child Neurology and Psychiatry Unit, Santa Maria Nuova Hospital IRCCS, Reggio Emilia, Italy. Electronic address: alle.iodice@gmail.com.

M Carecchio (M)

Molecular Neurogenetics Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via L. Temolo 4, 20126 Milan, Italy; Department of Pediatric Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy.

G Zorzi (G)

Department of Pediatric Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy.

B Garavaglia (B)

Molecular Neurogenetics Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via L. Temolo 4, 20126 Milan, Italy.

C Spagnoli (C)

Child Neurology and Psychiatry Unit, Santa Maria Nuova Hospital IRCCS, Reggio Emilia, Italy.

G G Salerno (GG)

Child Neurology and Psychiatry Unit, Santa Maria Nuova Hospital IRCCS, Reggio Emilia, Italy.

D Frattini (D)

Child Neurology and Psychiatry Unit, Santa Maria Nuova Hospital IRCCS, Reggio Emilia, Italy.

N E Mencacci (NE)

Department of Neurology, Northwestern University, Feinberg School of Medicine, Chicago, 60611 IL, USA.

F Invernizzi (F)

Molecular Neurogenetics Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via L. Temolo 4, 20126 Milan, Italy.

L Veneziano (L)

Institute of Translational Pharmacology, National Council of Research, Rome, Italy.

E Mantuano (E)

Institute of Translational Pharmacology, National Council of Research, Rome, Italy.

M Angriman (M)

Child Neurology and Neurorehabilitation Unit, Department of Pediatrics, Hospital of Bolzano, Italy.

C Fusco (C)

Child Neurology and Psychiatry Unit, Santa Maria Nuova Hospital IRCCS, Reggio Emilia, Italy.

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Classifications MeSH