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Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea

The syndrome of immune dysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) is a rare disorder caused by mutations in the FOXP3 gene. Diarrhea, diabetes and dermatitis are the hallmark of the disease, with a typical onset within the first months of life. We describe the case of a twelve-y...

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Autores principales: Zama, Daniele, Cocchi, Ilaria, Masetti, Riccardo, Specchia, Fernando, Alvisi, Patrizia, Gambineri, Eleonora, Lima, Mario, Pession, Andrea
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4421998/
https://www.ncbi.nlm.nih.gov/pubmed/25326164
http://dx.doi.org/10.1186/s13052-014-0068-4
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author Zama, Daniele
Cocchi, Ilaria
Masetti, Riccardo
Specchia, Fernando
Alvisi, Patrizia
Gambineri, Eleonora
Lima, Mario
Pession, Andrea
author_facet Zama, Daniele
Cocchi, Ilaria
Masetti, Riccardo
Specchia, Fernando
Alvisi, Patrizia
Gambineri, Eleonora
Lima, Mario
Pession, Andrea
author_sort Zama, Daniele
collection PubMed
description The syndrome of immune dysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) is a rare disorder caused by mutations in the FOXP3 gene. Diarrhea, diabetes and dermatitis are the hallmark of the disease, with a typical onset within the first months of life. We describe the case of a twelve-year old male affected by a very late-onset IPEX with intractable enteropathy, which markedly improved after starting Sirolimus as second-line treatment. This case suggests that IPEX should always be considered in the differential diagnosis of watery intractable diarrhea, despite its unusual onset.
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spelling pubmed-44219982015-05-07 Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea Zama, Daniele Cocchi, Ilaria Masetti, Riccardo Specchia, Fernando Alvisi, Patrizia Gambineri, Eleonora Lima, Mario Pession, Andrea Ital J Pediatr Case Report The syndrome of immune dysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) is a rare disorder caused by mutations in the FOXP3 gene. Diarrhea, diabetes and dermatitis are the hallmark of the disease, with a typical onset within the first months of life. We describe the case of a twelve-year old male affected by a very late-onset IPEX with intractable enteropathy, which markedly improved after starting Sirolimus as second-line treatment. This case suggests that IPEX should always be considered in the differential diagnosis of watery intractable diarrhea, despite its unusual onset. BioMed Central 2014-10-18 /pmc/articles/PMC4421998/ /pubmed/25326164 http://dx.doi.org/10.1186/s13052-014-0068-4 Text en © Zama et al.; licensee BioMed Central Ltd. 2014 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Case Report
Zama, Daniele
Cocchi, Ilaria
Masetti, Riccardo
Specchia, Fernando
Alvisi, Patrizia
Gambineri, Eleonora
Lima, Mario
Pession, Andrea
Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title_full Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title_fullStr Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title_full_unstemmed Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title_short Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea
title_sort late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (ipex) with intractable diarrhea
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4421998/
https://www.ncbi.nlm.nih.gov/pubmed/25326164
http://dx.doi.org/10.1186/s13052-014-0068-4
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