Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome) |
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| Authors: | Bindl Lutz Torgerson Troy Perroni Lucia Youssef Nelly Ochs Hans D Goulet Olivier Ruemmele Frank M |
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| Affiliation: | Children's Hospital Medical Center, University of Bonn, Germany. |
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| Abstract: | IPEX (immune-dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome is an autoimmune disorder with an often lethal outcome in spite of immunosuppressive therapy. We report the successful use of sirolimus in 3 patients with IPEX. The efficacy of sirolimus is probably due to its different mode of action compared to calcineurin-dependent agents. |
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| Keywords: | CsA, Cyclosporine A IgE, Immunoglobulin E IgG, Immunoglobulin G IPEX, Immune-dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome mTOR, Mammalian target of rapamycin |
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