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

Lutz Bindl; Troy Torgerson; Lucia Perroni; Nelly Youssef; Hans D Ochs; Olivier Goulet; Frank M Ruemmele

doi:10.1016/j.jpeds.2005.04.017

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

J Pediatr. 2005 Aug;147(2):256-9. doi: 10.1016/j.jpeds.2005.04.017.

Authors

Lutz Bindl¹, Troy Torgerson, Lucia Perroni, Nelly Youssef, Hans D Ochs, Olivier Goulet, Frank M Ruemmele

Affiliation

¹ Children's Hospital Medical Center, University of Bonn, Germany.

PMID: 16126062
DOI: 10.1016/j.jpeds.2005.04.017

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.

Publication types

Case Reports

MeSH terms

Child
Humans
Immunosuppressive Agents / therapeutic use*
Male
Polyendocrinopathies, Autoimmune / drug therapy*
Polyendocrinopathies, Autoimmune / genetics
Polyendocrinopathies, Autoimmune / physiopathology
Sirolimus / therapeutic use*
Syndrome

Substances

Immunosuppressive Agents
Sirolimus