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Successful Use of the New Immune-suppressor Sirolimus in IPEX (Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-linked Syndrome)

2005; Elsevier BV; Volume: 147; Issue: 2 Linguagem: Inglês

10.1016/j.jpeds.2005.04.017

1097-6833

L. Bindl, Troy R. Torgerson, L Perroni, Nelly Youssef, Hans D. Ochs, Olivier Goulet, Frank M. Ruemmele,

Immunodeficiency and Autoimmune Disorders

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. 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.