[PAGID] IPEX treatment

[Jennifer.Barker at UCHSC.edu]

Bindl et al recently published a case series in Journal of Pediatrics (J Pediatr 2005:147:256-259) regarding treatement of t IPEX (Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome).  In this series, 3 children with proven FOXP3 mutations were treated with immunosuppression using sirolimus (0.15 mg/kg/day) to maintain serum levels between 8 -15 ng/mL; methotrexate and a prednisone taper.  The patients experienced remission of the gastroenteropathy lasting 1-5 years without nephrotoxicity.  This provides an alternate to treatment of patients with this rare disorder.  Please see the article for details. Does anyone have experience with immunosuppression in this and other rare polyendocrine autoimmune syndromes? 




Jennifer M. Barker, MD
Instructor in Pediatrics
Barbara Davis Center 
University of Colorado Health Sciences Center

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