Literature DB >> 26194623

Severe early onset ethylmalonic encephalopathy with West syndrome.

Laura Papetti1, Giacomo Garone1, Livia Schettini1, Carla Giordano2, Francesco Nicita1, Paola Papoff3, Massimo Zeviani4, Vincenzo Leuzzi1, Alberto Spalice5,6.   

Abstract

Ethylmalonic encephalopathy (EE) is a rare autosomal recessive disorder characterized by early onset encephalopathy, chronic diarrhoea, petechiae, orthostatic acrocyanosis and defective cytochrome c oxidase (COX) in muscle and brain. High levels of lactic, ethylmalonic and methylsuccinic acids are detected in body fluids. EE is caused by mutations in ETHE1 gene, a mitochondrial sulfur dioxygenase. Neurologic signs and symptoms include progressively delayed development, hypotonia, seizures, and abnormal movements. We report on the clinical, electroencephalographic and MRI findings of a baby with a severe early onset encephalopathy associated with novel ETHE1 gene mutation. This is the first case described in literature with an early pure epileptic onset, presenting with West syndrome.

Entities:  

Keywords:  ETHE1; Ethylmalonic encephalopathy; West syndrome

Mesh:

Substances:

Year:  2015        PMID: 26194623     DOI: 10.1007/s11011-015-9707-8

Source DB:  PubMed          Journal:  Metab Brain Dis        ISSN: 0885-7490            Impact factor:   3.584


  28 in total

1.  Therapeutic trial in the first three Asian cases of ethylmalonic encephalopathy: response to riboflavin.

Authors:  H R Yoon; S H Hahn; Y M Ahn; S H Jang; Y J Shin; E H Lee; K H Ryu; B L Eun; P Rinaldo; S Yamaguchi
Journal:  J Inherit Metab Dis       Date:  2001-12       Impact factor: 4.982

2.  A case of ethylmalonic encephalopathy with atypical clinical and biochemical presentation.

Authors:  Maja Di Rocco; Ubaldo Caruso; Egill Briem; Andrea Rossi; Anna E M Allegri; Davide Buzzi; Valeria Tiranti
Journal:  Mol Genet Metab       Date:  2006-07-07       Impact factor: 4.797

3.  Ethylmalonic encephalopathy. Another patient from Kuwait.

Authors:  Essam A Ismail; Tarek M Seoudi; Eman A Morsi; Ahmad H Ahmad
Journal:  Neurosciences (Riyadh)       Date:  2009-01       Impact factor: 0.906

4.  Muscle cytochrome c oxidase deficiency in two Italian patients with ethylmalonic aciduria and peculiar clinical phenotype.

Authors:  B Garavaglia; V Colamaria; F Carrara; P Tonin; M Rimoldi; G Uziel
Journal:  J Inherit Metab Dis       Date:  1994       Impact factor: 4.982

5.  Combined treatment with oral metronidazole and N-acetylcysteine is effective in ethylmalonic encephalopathy.

Authors:  Carlo Viscomi; Alberto B Burlina; Imad Dweikat; Mario Savoiardo; Costanza Lamperti; Tatjana Hildebrandt; Valeria Tiranti; Massimo Zeviani
Journal:  Nat Med       Date:  2010-07-25       Impact factor: 53.440

6.  Clinical heterogeneity in ethylmalonic encephalopathy.

Authors:  Nicole Pigeon; Philippe M Campeau; Denis Cyr; Bernard Lemieux; Joe T R Clarke
Journal:  J Child Neurol       Date:  2009-03-16       Impact factor: 1.987

Review 7.  Altered sulfide (H(2)S) metabolism in ethylmalonic encephalopathy.

Authors:  Valeria Tiranti; Massimo Zeviani
Journal:  Cold Spring Harb Perspect Biol       Date:  2013-01-01       Impact factor: 10.005

8.  Identification of new mutations in the ETHE1 gene in a cohort of 14 patients presenting with ethylmalonic encephalopathy.

Authors:  R Mineri; M Rimoldi; A B Burlina; S Koskull; C Perletti; B Heese; U von Döbeln; P Mereghetti; I Di Meo; F Invernizzi; M Zeviani; G Uziel; V Tiranti
Journal:  J Med Genet       Date:  2008-07       Impact factor: 6.318

9.  Loss of ETHE1, a mitochondrial dioxygenase, causes fatal sulfide toxicity in ethylmalonic encephalopathy.

Authors:  Valeria Tiranti; Carlo Viscomi; Tatjana Hildebrandt; Ivano Di Meo; Rossana Mineri; Cecilia Tiveron; Michael D Levitt; Alessandro Prelle; Gigliola Fagiolari; Marco Rimoldi; Massimo Zeviani
Journal:  Nat Med       Date:  2009-01-11       Impact factor: 53.440

10.  Morphologic evidence of diffuse vascular damage in human and in the experimental model of ethylmalonic encephalopathy.

Authors:  Carla Giordano; Carlo Viscomi; Maurizia Orlandi; Paola Papoff; Alberto Spalice; Alberto Burlina; Ivano Di Meo; Valeria Tiranti; Vincenzo Leuzzi; Giulia d'Amati; Massimo Zeviani
Journal:  J Inherit Metab Dis       Date:  2011-10-22       Impact factor: 4.982

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  6 in total

1.  Turkish case of ethylmalonic encephalopathy misdiagnosed as short chain acyl-CoA dehydrogenase deficiency.

Authors:  Fatma Derya Bulut; Deniz Kör; Berna Şeker-Yılmaz; Gülen Gül-Mert; Sebile Kılavuz; Neslihan Önenli-Mungan
Journal:  Metab Brain Dis       Date:  2017-11-20       Impact factor: 3.584

2.  Successful treatment of a patient with ethylmalonic encephalopathy by intravenous N-acetylcysteine.

Authors:  Mustafa Kılıç; Özge Dedeoğlu; Rahşan Göçmen; Selman Kesici; Deniz Yüksel
Journal:  Metab Brain Dis       Date:  2016-11-09       Impact factor: 3.584

3.  Neurological and Vascular Manifestations of Ethylmalonic Encephalopathy.

Authors:  Ali Reza Tavasoli; Parastoo Rostami; Mahmoud Reza Ashrafi; Parvaneh Karimzadeh
Journal:  Iran J Child Neurol       Date:  2017

4.  Novel Compound Heterozygous Variants of ETHE1 Causing Ethylmalonic Encephalopathy in a Chinese Patient: A Case Report.

Authors:  Xiaohong Chen; Lin Han; Hui Yao
Journal:  Front Genet       Date:  2020-04-17       Impact factor: 4.599

5.  Ethylmalonic encephalopathy and liver transplantation: long-term outcome of the first treated patient.

Authors:  Giorgia Olivieri; Diego Martinelli; Daniela Longo; Chiara Grimaldi; Daniela Liccardo; Ivano Di Meo; Andrea Pietrobattista; Anna Sidorina; Michela Semeraro; Carlo Dionisi-Vici
Journal:  Orphanet J Rare Dis       Date:  2021-05-19       Impact factor: 4.123

Review 6.  Riboflavin Deficiency-Implications for General Human Health and Inborn Errors of Metabolism.

Authors:  Signe Mosegaard; Graziana Dipace; Peter Bross; Jasper Carlsen; Niels Gregersen; Rikke Katrine Jentoft Olsen
Journal:  Int J Mol Sci       Date:  2020-05-28       Impact factor: 5.923

  6 in total

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