Successful haploidentical hematopoietic stem cell transplantation in a patient with SCID due to CD3ε deficiency: need for IgG-substitution 6 years later

Klin Padiatr. 2014 May;226(3):149-53. doi: 10.1055/s-0033-1361142. Epub 2014 Feb 10.

Abstract

Background: The CD3 co-receptor complex is essential for signal transduction after specific binding of the T-cell receptor (TCR). CD3E encodes the CD3ε chain, one of the protein components (γ-, δ-, ε- and ζ-chain) of the CD3 co-receptor. As previously reported in one family CD3ε deficiency causes SCID.

Patient: We report on a patient with SCID due to CD3ε deficiency treated by HLA-haploidentical stem cell transplantation (SCT) (donor: mother) 15 years ago which resulted in development of normal T- and B-cell immunity. Despite conditioning donor cell engraftment was confined to T cells, while all other blood cell lineages remained of patient origin (split chimerism). In spite of normal functions, T-cell numbers never reached normal levels and naïve CD45+RA+ T-cells remained low. At 6 years after SCT the patient developed signs of humoral immunodeficiency, requiring regular substitution of IgG.

Results: In a retrospective genetic work up 11 years after SCT, a homozygous splice site mutation in CD3E was identified resulting in the loss of CD3ε protein. The loss of B-cell function as observed in the patient was reflected by a lack of switched memory B cells. To rule out a primary role of CD3ε in B-cell function we studied expression of CD3E in B-cells which was found not to be expressed.

Discussion: The clinical presentation of a secondary loss of specific humoral immunity in this constellation of split chimerism after allogeneic haploidentical SCT is unusual and unexpected in a patient with a primary T-cell defect. A most likely explanation is the gradual loss of T-helper-cell function.

Publication types

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

MeSH terms

  • B-Lymphocytes / immunology
  • CD3 Complex / genetics*
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Female
  • Genetic Carrier Screening
  • Genotype
  • Haploidy
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility Testing*
  • Homozygote
  • Humans
  • Immunity, Humoral / genetics
  • Immunity, Humoral / immunology
  • Immunoglobulin G / administration & dosage*
  • Infant
  • Infant, Newborn
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / therapy*
  • T-Lymphocytes / immunology
  • T-Lymphocytes, Helper-Inducer / immunology

Substances

  • CD3 Complex
  • CD3E protein, human
  • Immunoglobulin G