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

被引：80

作者：

Bindl, L

Torgerson, T

Perroni, L

Youssef, N

Ochs, HD

Goulet, O

Ruemmele, FM

机构：

[1] Hop Necker Enfants Malad, Paediat Gastroenterol Unit, Dept Pathol, F-75730 Paris, France

[2] Univ Bonn, Childrens Hosp, Ctr Med, D-5300 Bonn, Germany

[3] Univ Washington, Seattle, WA 98195 USA

[4] Childrens Hosp, Reg Med Ctr, Seattle, WA USA

[5] Galliera Hosp, Lab Human Genet, Genoa, Italy

[6] Univ Paris 05, INSERM, Hop Necker Enfants Malad, Dept Paediat,Fac Necker, Paris, France

来源：

JOURNAL OF PEDIATRICS | 2005年 / 147卷 / 02期

关键词：

D O I：

10.1016/j.jpeds.2005.04.017

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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.

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页码：256 / 259

页数：4

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