title: Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome).
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.
authors.affiliation: Children's Hospital Medical Center, University of Bonn, Germany.
authors.forename: Lutz
authors.initials: L
authors.lastname: Bindl
authors.forename: Troy
authors.initials: T
authors.lastname: Torgerson
authors.forename: Lucia
authors.initials: L
authors.lastname: Perroni
authors.forename: Nelly
authors.initials: N
authors.lastname: Youssef
authors.forename: Hans D
authors.initials: HD
authors.lastname: Ochs
authors.forename: Olivier
authors.initials: O
authors.lastname: Goulet
authors.forename: Frank M
authors.initials: FM
authors.lastname: Ruemmele
mesh_terms: D002648:Child; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D016884:Polyendocrinopathies, Autoimmune / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D020123:Sirolimus / Q000627:therapeutic use*; D013577:Syndrome
chemical_list: D007166:Immunosuppressive Agents; D020123:Sirolimus