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.

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