New therapeutic approach by sirolimus for enteropathy treatment in patients with LRBA deficiency

(2017) New therapeutic approach by sirolimus for enteropathy treatment in patients with LRBA deficiency. European Annals of Allergy and Clinical Immunology. pp. 235-239. ISSN 1764-1489

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Abstract

Purpose. To report the successful use of sirolimus for management of enteropathy in four patients with LPS-responsive beige-like anchor protein (LRBA) deficiency. Methods. Case series. Results. sirolimus therapy led to a complete improvement of symptoms including decrease in frequency and severity of diarrhea, as well as patients' weight gain. No signs of abdominal cramps and anorexia were also detected during the follow up period after treatment. Conclusions. sirolimus with its potential efficacy and immunomodulatory properties may be recommended for the treatment of severe enteropathy refractory to conventional therapy in patients with LRBA deficiency.

Item Type: Article
Keywords: sirolimus enteropathy lrba deficiency common variable immune deficiency autoimmunity common variable immunodeficiency inflammatory-bowel-disease x-linked syndrome immune dysregulation autoimmune enteropathy polyendocrinopathy mutations ipex hypogammaglobulinemia management
Divisions: Faculty of Medicine > Department of Basic Science > Immunology Department
Page Range: pp. 235-239
Journal or Publication Title: European Annals of Allergy and Clinical Immunology
Journal Index: ISI
Volume: 49
Number: 5
Identification Number: https://doi.org/10.23822/EurAnnACI.1764-1489.22
ISSN: 1764-1489
Depositing User: مهندس مهدی شریفی
URI: http://eprints.mui.ac.ir/id/eprint/309

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