Cargando…
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...
Autores principales: | , , , , , , , |
---|---|
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 |
_version_ | 1782369991055638528 |
---|---|
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. |
format | Online Article Text |
id | pubmed-4421998 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT zamadaniele lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT cocchiilaria lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT masettiriccardo lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT specchiafernando lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT alvisipatrizia lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT gambinerieleonora lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT limamario lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea AT pessionandrea lateonsetofimmunodysregulationpolyendocrinopathyenteropathyxlinkedsyndromeipexwithintractablediarrhea |