Defective B-cell-negative selection and terminal differentiation in the ICF syndrome

Blood. 2004 Apr 1;103(7):2683-90. doi: 10.1182/blood-2003-08-2632. Epub 2003 Nov 26.

Abstract

Immunodeficiency, centromeric region instability, and facial anomalies (ICF) syndrome is a rare autosomal recessive disease. Mutations in the DNA methyltransferase 3B (DNMT3B) gene are responsible for most ICF cases reported. We investigated the B-cell defects associated with agammaglobulinemia in this syndrome by analyzing primary B cells from 4 ICF patients. ICF peripheral blood (PB) contains only naive B cells; memory and gut plasma cells are absent. Naive ICF B cells bear potentially autoreactive long heavy chain variable regions complementarity determining region 3's (V(H)CDR3's) enriched with positively charged residues, in contrast to normal PB transitional and mature B cells, indicating that negative selection is impaired in patients. Like anergic B cells in transgenic models, newly generated and immature B cells accumulate in PB. Moreover, these cells secrete immunoglobulins and exhibit increased apoptosis following in vitro activation. However, they are able to up-regulate CD86, indicating that mechanisms other than anergy participate in silencing of ICF B cells. One patient without DNMT3B mutations shows differences in immunoglobulin E (IgE) switch induction, suggesting that immunodeficiency could vary with the genetic origin of the syndrome. In this study, we determined that negative selection breakdown and peripheral B-cell maturation blockage contribute to agammaglobulinemia in the ICF syndrome.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antigens, CD / blood
  • B-Lymphocytes / cytology
  • B-Lymphocytes / immunology*
  • B-Lymphocytes / pathology
  • Base Sequence
  • Cell Differentiation
  • Child
  • Child, Preschool
  • Chromosomal Instability / genetics
  • Chromosomal Instability / immunology*
  • DNA (Cytosine-5-)-Methyltransferases / genetics*
  • DNA Methyltransferase 3B
  • DNA Primers
  • Face / abnormalities*
  • Female
  • Flow Cytometry
  • Humans
  • Immunoglobulin A / blood
  • Immunoglobulin G / blood
  • Immunoglobulin M / blood
  • Immunologic Deficiency Syndromes / genetics
  • Immunologic Deficiency Syndromes / immunology*
  • Lymphocyte Count
  • Male
  • Mutation*
  • Reverse Transcriptase Polymerase Chain Reaction
  • Syndrome

Substances

  • Antigens, CD
  • DNA Primers
  • Immunoglobulin A
  • Immunoglobulin G
  • Immunoglobulin M
  • DNA (Cytosine-5-)-Methyltransferases