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Journal of Clinical Immunology
Published in: Journal of Clinical Immunology 5/2008

01-09-2008 | Original Paper

Use of Sirolimus in IPEX and IPEX-Like Children

Authors: Pierre L. Yong, Pierre Russo, Kathleen E. Sullivan

Published in: Journal of Clinical Immunology | Issue 5/2008

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Abstract

Introduction

IPEX (immune dysregulation, polyendocrinopathy, enteropathy, and X-linked syndrome), a rare inflammatory disease caused by mutations of Foxp3, destroys the immunoregulatory environment of affected male infants. Data on optimal therapy are limited.

Methods

We reviewed the effect of sirolimus use in our cohort of IPEX and IPEX-like patients (n = 7).

Results and Discussion

Our patients exhibited features of enteropathy and recurrent infections with bacterial and viral pathogens. Before initiating sirolimus, six patients were treated with corticosteroids. Several also received other immunosuppressive agents. After starting sirolimus, six patients had improvement in diarrhea, and two were able to decrease corticosteroid dosages. Several also had significantly decreased number of infections after treatment. Of the three patients with post-treatment duodenal biopsies, two showed improvement in villous architecture. No significant adverse events occurred. Our experience suggests that sirolimus is a clinically effective and safe therapeutic option in IPEX and IPEX-like patients.
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Metadata
Title
Use of Sirolimus in IPEX and IPEX-Like Children
Authors
Pierre L. Yong
Pierre Russo
Kathleen E. Sullivan
Publication date
01-09-2008
Publisher
Springer US
Published in
Journal of Clinical Immunology / Issue 5/2008
Print ISSN: 0271-9142
Electronic ISSN: 1573-2592
DOI
https://doi.org/10.1007/s10875-008-9196-1

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