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Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation

Mutations in lipopolysaccharide-responsive vesicle trafficking, beach and anchor-containing protein (LRBA) cause immune deficiency and inflammation. Here, we are reporting a novel homozygous mutation in LRBA allele in 7-year-old Omani boy, born to consanguineous parents. He presented with type 1 dia...

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Autores principales: Al Sukaiti, Nashat, AbdelRahman, Khwater, AlShekaili, Jalila, Al Oraimi, Sumaya, Al Sinani, Aisha, Al Rahbi, Nasser, Cho, Vicky, Field, Matt, Cook, Matthew C
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5493589/
https://www.ncbi.nlm.nih.gov/pubmed/28690850
http://dx.doi.org/10.1038/cti.2017.20
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author Al Sukaiti, Nashat
AbdelRahman, Khwater
AlShekaili, Jalila
Al Oraimi, Sumaya
Al Sinani, Aisha
Al Rahbi, Nasser
Cho, Vicky
Field, Matt
Cook, Matthew C
author_facet Al Sukaiti, Nashat
AbdelRahman, Khwater
AlShekaili, Jalila
Al Oraimi, Sumaya
Al Sinani, Aisha
Al Rahbi, Nasser
Cho, Vicky
Field, Matt
Cook, Matthew C
author_sort Al Sukaiti, Nashat
collection PubMed
description Mutations in lipopolysaccharide-responsive vesicle trafficking, beach and anchor-containing protein (LRBA) cause immune deficiency and inflammation. Here, we are reporting a novel homozygous mutation in LRBA allele in 7-year-old Omani boy, born to consanguineous parents. He presented with type 1 diabetes, autoimmune haematological cytopenia, recurrent chest infections and lymphocytic interstitial lung disease. The patient was treated with CTLA4-Ig (abatacept) with good outcome every 2 weeks for a period of 3 months. He developed complete IgG deficiency, but remarkably, histological examination revealed germinal centres and plasma cells in lymphoid and inflamed lung tissue. Further charatecterisation showed these cells to express IgM but not IgG. This ex vivo analysis suggests that LRBA mutation confers a defect in class switching despite plasma cell formation.
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spelling pubmed-54935892017-07-07 Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation Al Sukaiti, Nashat AbdelRahman, Khwater AlShekaili, Jalila Al Oraimi, Sumaya Al Sinani, Aisha Al Rahbi, Nasser Cho, Vicky Field, Matt Cook, Matthew C Clin Transl Immunology Case Report Mutations in lipopolysaccharide-responsive vesicle trafficking, beach and anchor-containing protein (LRBA) cause immune deficiency and inflammation. Here, we are reporting a novel homozygous mutation in LRBA allele in 7-year-old Omani boy, born to consanguineous parents. He presented with type 1 diabetes, autoimmune haematological cytopenia, recurrent chest infections and lymphocytic interstitial lung disease. The patient was treated with CTLA4-Ig (abatacept) with good outcome every 2 weeks for a period of 3 months. He developed complete IgG deficiency, but remarkably, histological examination revealed germinal centres and plasma cells in lymphoid and inflamed lung tissue. Further charatecterisation showed these cells to express IgM but not IgG. This ex vivo analysis suggests that LRBA mutation confers a defect in class switching despite plasma cell formation. Nature Publishing Group 2017-05-26 /pmc/articles/PMC5493589/ /pubmed/28690850 http://dx.doi.org/10.1038/cti.2017.20 Text en Copyright © 2017 The Author(s) http://creativecommons.org/licenses/by/4.0/ This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
spellingShingle Case Report
Al Sukaiti, Nashat
AbdelRahman, Khwater
AlShekaili, Jalila
Al Oraimi, Sumaya
Al Sinani, Aisha
Al Rahbi, Nasser
Cho, Vicky
Field, Matt
Cook, Matthew C
Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title_full Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title_fullStr Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title_full_unstemmed Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title_short Agammaglobulinaemia despite terminal B-cell differentiation in a patient with a novel LRBA mutation
title_sort agammaglobulinaemia despite terminal b-cell differentiation in a patient with a novel lrba mutation
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5493589/
https://www.ncbi.nlm.nih.gov/pubmed/28690850
http://dx.doi.org/10.1038/cti.2017.20
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