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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition
2018
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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. |
format | Online Article Text |
id | pubmed-5915691 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition |
record_format | MEDLINE/PubMed |
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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