Hypomorphic Rag mutations can cause destructive midline granulomatous disease

Blood. 2010 Aug 26;116(8):1263-71. doi: 10.1182/blood-2010-02-267583. Epub 2010 May 20.

Abstract

Destructive midline granulomatous disease characterized by necrotizing granulomas of the head and neck is most commonly caused by Wegener granulomatosis, natural killer/T-cell lymphomas, cocaine abuse, or infections. An adolescent patient with myasthenia gravis treated with thymectomy subsequently developed extensive granulomatous destruction of midface structures, palate, nasal septum, airways, and epiglottis. His lymphocyte numbers, total immunoglobulin G level, and T-cell receptor (TCR) repertoire appeared normal. Sequencing of Recombination activating gene-1 (Rag1) showed compound heterozygous Rag1 mutations; a novel deletion with no recombinase activity and a missense mutation resulting in 50% Rag activity. His thymus was dysplastic and, although not depleted of T cells, showed a notable absence of autoimmune regulator (AIRE) and Foxp3(+) regulatory T cells. This distinct Rag-deficient phenotype characterized by immune dysregulation with granulomatous hyperinflammation and autoimmunity, with relatively normal T and B lymphocyte numbers and a diverse TCR repertoire expands the spectrum of presentation in Rag deficiency. This study was registered at www.clinicaltrials.gov as #NCT00128973.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • AIRE Protein
  • Adolescent
  • Animals
  • Cells, Cultured
  • Forkhead Transcription Factors
  • Gene Rearrangement
  • Genes, Immunoglobulin
  • Granulomatous Disease, Chronic / etiology*
  • Granulomatous Disease, Chronic / pathology*
  • Granulomatous Disease, Chronic / surgery
  • Homeodomain Proteins / genetics*
  • Homeodomain Proteins / physiology*
  • Humans
  • Immunoglobulin G / immunology
  • Immunoglobulin G / metabolism
  • Immunophenotyping
  • Lymphocytes / immunology
  • Lymphocytes / metabolism
  • Male
  • Mice
  • Mutation, Missense / genetics*
  • Recombinases / metabolism
  • Severe Combined Immunodeficiency / etiology*
  • Severe Combined Immunodeficiency / pathology*
  • T-Lymphocytes, Regulatory / immunology
  • T-Lymphocytes, Regulatory / metabolism
  • T-Lymphocytes, Regulatory / pathology
  • Thymectomy
  • Transcription Factors
  • Transgenes / physiology

Substances

  • FOXP3 protein, human
  • Forkhead Transcription Factors
  • Homeodomain Proteins
  • Immunoglobulin G
  • Recombinases
  • Transcription Factors
  • RAG-1 protein

Associated data

  • ClinicalTrials.gov/NCT00128973