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New Therapeutic Approach by Sirolimus for Enteropathy Treatment in Patients With Lrba Deficiency Publisher Pubmed



Azizi G1, 2, 3 ; Abolhassani H2, 3, 4 ; Yazdani R1, 5 ; Mohammadikhajehdehi S2 ; Parvaneh N2 ; Negahdari B6 ; Mohammadi J7 ; Aghamohammadi A2
Authors
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Authors Affiliations
  1. 1. Department of Laboratory Medicine, Imam Hassan Mojtaba Hospital, Alborz University of Medical Sciences, Karaj, Iran
  2. 2. Research Center for Immunodeficiencies, Children’s Medical Center, Tehran University of Medical Sciences, Tehran, Iran
  3. 3. Primary Immunodeficiency Diseases Network (PIDNet), Universal Scientific Education and Research Network (USERN), Tehran, Iran
  4. 4. Division of Clinical Immunology, Department of Laboratory Medicine, Karolinska Institute at Karolinska University Hospital Huddinge, Stockholm, Sweden
  5. 5. Department of Immunology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran
  6. 6. School of Advanced Technologies in Medicine, Department of Medical Biotechnology, Tehran University of Medical Sciences, Tehran, Iran
  7. 7. Department of Biomedical Engineering, Faculty of New Sciences and Technologies, University of Tehran, Tehran, Iran

Source: European Annals of Allergy and Clinical Immunology Published:2017


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. © 2017 Associazione Allergologi Immunologi Italiani Territoriali e Ospedalieri-AAIITO. Published by EDRA SpA. All rights reserved.
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