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Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease

Chylomicron retention disease, also known as Anderson's disease, is a rare hereditary hypocholesterolemic disorder, recessive inherited, characterized by nonspecific symptoms as abdominal distension, steatorrhea, and vomiting associated with failure to thrive. We describe a patient with failure...

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Autores principales: Ferreira, Helena, Ramos, Raquel Nuñez, Quan, Cinthia Flores, Ferreiro, Susana Redecillas, Ruiz, Vanessa Cabello, Juampérez Goñi, Javi, Quintero Bernabeu, Jesus, Segarra Cantón, Oscar, Álvarez Beltran, Marina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5915691/
https://www.ncbi.nlm.nih.gov/pubmed/29713611
http://dx.doi.org/10.5223/pghn.2018.21.2.134
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author Ferreira, Helena
Ramos, Raquel Nuñez
Quan, Cinthia Flores
Ferreiro, Susana Redecillas
Ruiz, Vanessa Cabello
Juampérez Goñi, Javi
Quintero Bernabeu, Jesus
Segarra Cantón, Oscar
Álvarez Beltran, Marina
author_facet Ferreira, Helena
Ramos, Raquel Nuñez
Quan, Cinthia Flores
Ferreiro, Susana Redecillas
Ruiz, Vanessa Cabello
Juampérez Goñi, Javi
Quintero Bernabeu, Jesus
Segarra Cantón, Oscar
Álvarez Beltran, Marina
author_sort Ferreira, Helena
collection PubMed
description Chylomicron retention disease, also known as Anderson's disease, is a rare hereditary hypocholesterolemic disorder, recessive inherited, characterized by nonspecific symptoms as abdominal distension, steatorrhea, and vomiting associated with failure to thrive. We describe a patient with failure to thrive, chronic diarrhea and steatorrhea who the diagnosis of chylomicron retention disease was established after several months of disease progression. The genetic study confirmed a homozygosity mutation in SAR1B gene, identifying a mutation never previous described [c.83_84delTG(p.Leu28Argfs*7)]. With this case report the authors aim to highlight for this very rare cause of failure to thrive and for the importance of an attempting diagnosis, in order to start adequate management with low fat diet supplemented with fat-soluble vitamins, reverting the state of malnutrition and avoiding possible irreversible and desvantating complications.
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spelling pubmed-59156912018-04-30 Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease Ferreira, Helena Ramos, Raquel Nuñez Quan, Cinthia Flores Ferreiro, Susana Redecillas Ruiz, Vanessa Cabello Juampérez Goñi, Javi Quintero Bernabeu, Jesus Segarra Cantón, Oscar Álvarez Beltran, Marina Pediatr Gastroenterol Hepatol Nutr Case Report Chylomicron retention disease, also known as Anderson's disease, is a rare hereditary hypocholesterolemic disorder, recessive inherited, characterized by nonspecific symptoms as abdominal distension, steatorrhea, and vomiting associated with failure to thrive. We describe a patient with failure to thrive, chronic diarrhea and steatorrhea who the diagnosis of chylomicron retention disease was established after several months of disease progression. The genetic study confirmed a homozygosity mutation in SAR1B gene, identifying a mutation never previous described [c.83_84delTG(p.Leu28Argfs*7)]. With this case report the authors aim to highlight for this very rare cause of failure to thrive and for the importance of an attempting diagnosis, in order to start adequate management with low fat diet supplemented with fat-soluble vitamins, reverting the state of malnutrition and avoiding possible irreversible and desvantating complications. The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition 2018-04 2018-04-13 /pmc/articles/PMC5915691/ /pubmed/29713611 http://dx.doi.org/10.5223/pghn.2018.21.2.134 Text en Copyright © 2018 by The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition http://creativecommons.org/licenses/by-nc/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Case Report
Ferreira, Helena
Ramos, Raquel Nuñez
Quan, Cinthia Flores
Ferreiro, Susana Redecillas
Ruiz, Vanessa Cabello
Juampérez Goñi, Javi
Quintero Bernabeu, Jesus
Segarra Cantón, Oscar
Álvarez Beltran, Marina
Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title_full Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title_fullStr Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title_full_unstemmed Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title_short Chylomicron Retention Disease: a Description of a New Mutation in a Very Rare Disease
title_sort chylomicron retention disease: a description of a new mutation in a very rare disease
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5915691/
https://www.ncbi.nlm.nih.gov/pubmed/29713611
http://dx.doi.org/10.5223/pghn.2018.21.2.134
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