Atypical presentation of Charcot-Marie-Tooth disease type 1C with a new mutation: a case report.


Journal

BMC neurology
ISSN: 1471-2377
Titre abrégé: BMC Neurol
Pays: England
ID NLM: 100968555

Informations de publication

Date de publication:
27 Jul 2021
Historique:
received: 27 04 2021
accepted: 07 07 2021
entrez: 27 7 2021
pubmed: 28 7 2021
medline: 12 10 2021
Statut: epublish

Résumé

Charcot-Marie-Tooth 1C (CMT1C) is a rare form of dominantly inherited CMT1 neuropathy caused by a mutated gene encoding lipopolysaccharide-induced tumour necrosis alpha factor (LITAF). We report a 56-year-old patient with an atypical clinical phenotype of CMT1C, which started as progressive weakness of a single upper limb resembling acquired inflammatory neuropathy. Nerve conduction studies (NCS) and temporarily limited and partial effects of immunotherapy supported the diagnosis of inflammatory neuropathy. Significant progression of polyneuropathy, despite intensive long-lasting immunotherapy, together with repeatedly negative auxiliary investigations (CSF, MRI and antibodies) and genetic testing results finally led to the diagnosis of CMT1C neuropathy. CMT1C should be added to the list of inherited neuropathies that need to be considered in suspected cases of inflammatory demyelinating neuropathy.

Sections du résumé

BACKGROUND BACKGROUND
Charcot-Marie-Tooth 1C (CMT1C) is a rare form of dominantly inherited CMT1 neuropathy caused by a mutated gene encoding lipopolysaccharide-induced tumour necrosis alpha factor (LITAF).
CASE PRESENTATION METHODS
We report a 56-year-old patient with an atypical clinical phenotype of CMT1C, which started as progressive weakness of a single upper limb resembling acquired inflammatory neuropathy. Nerve conduction studies (NCS) and temporarily limited and partial effects of immunotherapy supported the diagnosis of inflammatory neuropathy. Significant progression of polyneuropathy, despite intensive long-lasting immunotherapy, together with repeatedly negative auxiliary investigations (CSF, MRI and antibodies) and genetic testing results finally led to the diagnosis of CMT1C neuropathy.
CONCLUSIONS CONCLUSIONS
CMT1C should be added to the list of inherited neuropathies that need to be considered in suspected cases of inflammatory demyelinating neuropathy.

Identifiants

pubmed: 34311727
doi: 10.1186/s12883-021-02316-3
pii: 10.1186/s12883-021-02316-3
pmc: PMC8314550
doi:

Substances chimiques

LITAF protein, human 0
Nuclear Proteins 0
Transcription Factors 0

Types de publication

Case Reports Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

293

Subventions

Organisme : Agentúra Ministerstva Školstva, Vedy, Výskumu a Športu SR
ID : 1/0301/19

Informations de copyright

© 2021. The Author(s).

Références

J Neurol Sci. 2014 Aug 15;343(1-2):183-6
pubmed: 24880540
Neuromuscul Disord. 2013 May;23(5):399-403
pubmed: 23489662
Neuromuscul Disord. 2009 Oct;19(10):701-3
pubmed: 19541485
J Neurol Sci. 2014 Aug 15;343(1-2):237-9
pubmed: 24906712
Muscle Nerve. 2014 Aug;50(2):164-9
pubmed: 24723454
J Peripher Nerv Syst. 2020 Mar;25(1):19-26
pubmed: 31919945
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Eur J Neurol. 2017 Mar;24(3):530-538
pubmed: 28211240
J Neurol Neurosurg Psychiatry. 2009 Jun;80(6):699-700
pubmed: 19448103
Eur J Neurol. 2010 Mar;17(3):356-63
pubmed: 20456730
J Neurol Neurosurg Psychiatry. 2016 Oct;87(10):1051-60
pubmed: 27010614
Mol Genet Genomic Med. 2014 Nov;2(6):522-9
pubmed: 25614874
Brain. 2004 Jan;127(Pt 1):193-202
pubmed: 14607795
Brain. 2012 May;135(Pt 5):1639-49
pubmed: 22189568
J Peripher Nerv Syst. 2006 Jun;11(2):148-55
pubmed: 16787513
Neurology. 2011 Jan 25;76(4):337-45
pubmed: 21263135
Neuromuscul Disord. 2009 Apr;19(4):264-9
pubmed: 19272779
Neurology. 2003 Jan 14;60(1):22-6
pubmed: 12525712
Cureus. 2020 Jun 8;12(6):e8517
pubmed: 32665875

Auteurs

Monika Turčanová Koprušáková (M)

Clinic of Neurology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Kollárova 2, 036 01, Martin, Slovak Republic.

Milan Grofik (M)

Clinic of Neurology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Kollárova 2, 036 01, Martin, Slovak Republic.

Ema Kantorová (E)

Clinic of Neurology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Kollárova 2, 036 01, Martin, Slovak Republic.

Petra Jungová (P)

Institute of Medical Biology, Genetics and Clinical Genetics, Faculty of Medicine, Comenius University and University Hospital in Bratislava, Bratislava, Slovak Republic.

Ján Chandoga (J)

Institute of Medical Biology, Genetics and Clinical Genetics, Faculty of Medicine, Comenius University and University Hospital in Bratislava, Bratislava, Slovak Republic.

Martin Kolisek (M)

Biomedical Center Martin, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Mala Hora 4b, 036 01, Martin, Slovak Republic.

Peter Valkovič (P)

2nd Department of Neurology, Comenius University and University Hospital in Bratislava, Bratislava, Slovak Republic.
Centre of Experimental Medicine, Institute of Normal and Pathological Physiology, Slovak Academy of Sciences, Slovak, Slovak Republic.

Matej Škorvánek (M)

Department of Neurology, P.J. Safarik University and Louis Pasteur University Hospital, Kosice, Slovak Republic.

Rafal Ploski (R)

Department of Medical Genetics Laboratory, Medical University of Warsaw, Warsaw, Poland.

Egon Kurča (E)

Clinic of Neurology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Kollárova 2, 036 01, Martin, Slovak Republic.

Štefan Sivák (Š)

Clinic of Neurology, Jessenius Faculty of Medicine in Martin, Comenius University in Bratislava, Kollárova 2, 036 01, Martin, Slovak Republic. sivakste@gmail.com.

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Classifications MeSH