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.

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. - In: THE ITALIAN JOURNAL OF PEDIATRICS. - ISSN 1720-8424. - ELETTRONICO. - 40:(2014), pp. 68-75. [10.1186/s13052-014-0068-4]

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

GAMBINERI, ELEONORA;
2014

Abstract

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.
2014
40
68
75
Zama, Daniele; Cocchi, Ilaria; Masetti, Riccardo; Specchia, Fernando; Alvisi, Patrizia; Gambineri, Eleonora; Lima, Mario; Pession, Andrea
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Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1067247
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