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.

Daniele Zama, Ilaria Cocchi, Riccardo Masetti, Fernando Specchia, Patrizia Alvisi, Eleonora Gambineri Gambineri, et al. (2014). Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea. THE ITALIAN JOURNAL OF PEDIATRICS, 40(68), 1-7 [10.1186/s13052-014-0068-4].

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

ZAMA, DANIELE;MASETTI, RICCARDO;SPECCHIA, FERNANDO GIUSEPPE;ALVISI, PATRIZIA;LIMA, MARIO;PESSION, ANDREA
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
Daniele Zama, Ilaria Cocchi, Riccardo Masetti, Fernando Specchia, Patrizia Alvisi, Eleonora Gambineri Gambineri, et al. (2014). Late-onset of immunodysregulation, polyendocrinopathy, enteropathy, x-linked syndrome (IPEX) with intractable diarrhea. THE ITALIAN JOURNAL OF PEDIATRICS, 40(68), 1-7 [10.1186/s13052-014-0068-4].
Daniele Zama; Ilaria Cocchi; Riccardo Masetti; Fernando Specchia; Patrizia Alvisi; Eleonora Gambineri Gambineri; Mario Lima; Andrea Pession
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11585/362320
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