Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome).

Bindl, Lutz; Torgerson, Troy; Perroni, Lucia; Youssef, Nelly; Ochs, Hans D; Goulet, Olivier; Ruemmele, Frank M · J Pediatr · 2005

case_report · Level V

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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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