Literature DB >> 25282463

[Mitochondrial neurogastrointestinal encephalopathy disease].

A Benureau1, P Meyer2, O Maillet3, N Leboucq4, S Legras5, E Jeziorski6, S Fournier-Favre7, C Jeandel8, P Gaignard9, A Slama9, F Rivier2, A Roubertie10, M Carneiro11.   

Abstract

Mitochondrial neurogastrointestinal encephalopathy disease (MNGIE) is a rare autosomal-recessive syndrome, resulting from mutations in the TYMP gene, located at 22q13. The mutation induces a thymidine phosphorylase (TP) deficit, which leads to a nucleotide pool imbalance and to instability of the mitochondrial DNA. The clinical picture regroups gastrointestinal dysmotility, cachexia, ptosis, ophthalmoplegia, peripheral neuropathy, and asymptomatic leukoencephalopathy. The prognosis is unfavorable. We present the case of a 14-year-old Caucasian female whose symptoms started in early childhood. The diagnosis was suspected after magnetic resonance imaging (MRI), performed given the atypical features of mental anorexia, which revealed white matter abnormalities. She presented chronic vomiting, postprandial abdominal pain, and problems gaining weight accompanied by cachexia. This diagnosis led to establishing proper care, in particular an enteral and parenteral nutrition program. There is no known specific effective treatment, but numerous studies are in progress. In this article, after reviewing the existing studies, we discuss the main diagnostic and therapeutic aspects of the disease. We argue for the necessity of performing a cerebral MRI given the atypical features of a patient with suspected mental anorexia (or when the clinical pattern of a patient with mental anorexia seems atypical), so that MNGIE can be ruled out.
Copyright © 2014 Elsevier Masson SAS. All rights reserved.

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Year:  2014        PMID: 25282463     DOI: 10.1016/j.arcped.2014.08.006

Source DB:  PubMed          Journal:  Arch Pediatr        ISSN: 0929-693X            Impact factor:   1.180


  3 in total

Review 1.  Gastrointestinal manifestations of mitochondrial disorders: a systematic review.

Authors:  Josef Finsterer; Marlies Frank
Journal:  Therap Adv Gastroenterol       Date:  2016-10-06       Impact factor: 4.409

Review 2.  Mitochondrial Neurogastrointestinal Encephalomyopathy: Into the Fourth Decade, What We Have Learned So Far.

Authors:  Dario Pacitti; Michelle Levene; Caterina Garone; Niranjanan Nirmalananthan; Bridget E Bax
Journal:  Front Genet       Date:  2018-12-21       Impact factor: 4.599

3.  Pregnancy in MNGIE: a clinical and metabolic honeymoon.

Authors:  Pauline Pappalardo; Jean-François Benoist; Bridget E Bax; Clarisse Carra-Dallière; Cecilia Marelli; Michele Levene; Laetitia Begue; Anne Rolland; Nicolas Flori; François Rivier; Catherine Blanchet; Arnold Munnich; Romain Altwegg; Pierre Meyer; Agathe Roubertie
Journal:  Ann Clin Transl Neurol       Date:  2020-11-07       Impact factor: 5.430

  3 in total

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