Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome)
Successful use of the new immune-suppressor sirolimus in IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome)
复制标题
DOI：
10.1016/j.jpeds.2005.04.017
复制
发表时间：
2005-08-01
影响因子：
5.1
通讯作者：
Ruemmele, FM
中科院分区：
文献类型：
--
作者：
Bindl, L;Torgerson, T;Ruemmele, FM
IPEX（免疫失调，多内分泌病，肠病，X连锁）综合征是一种自身免疫性疾病，尽管有免疫抑制治疗，但通常仍会导致致命后果。我们报告成功地使用西罗莫司在3例患者的IPEX。西罗莫司的疗效可能是由于其与钙调神经磷酸酶依赖性药物的作用方式不同。
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 patient's with IPEX. The efficacy of sirolimus is probably due to its different mode of action compared to calcineurin-dependent agents.