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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.
Affiliation
  • Bindl L; Children's Hospital Medical Center, University of Bonn, Germany.
J Pediatr ; 147(2): 256-9, 2005 Aug.
Article in En | MEDLINE | ID: mdl-16126062
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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Collection: 01-internacional Database: MEDLINE Main subject: Polyendocrinopathies, Autoimmune / Sirolimus / Immunosuppressive Agents Limits: Child / Humans / Male Language: En Journal: J Pediatr Year: 2005 Document type: Article Affiliation country: Germany
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Collection: 01-internacional Database: MEDLINE Main subject: Polyendocrinopathies, Autoimmune / Sirolimus / Immunosuppressive Agents Limits: Child / Humans / Male Language: En Journal: J Pediatr Year: 2005 Document type: Article Affiliation country: Germany