Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome).
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.
Bindl
Lutz
L
Children's Hospital Medical Center, University of Bonn, Germany.
Torgerson
Troy
T
Perroni
Lucia
L
Youssef
Nelly
N
Ochs
Hans D
HD
Goulet
Olivier
O
Ruemmele
Frank M
FM
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
D007166:Immunosuppressive Agents; D020123:Sirolimus