Newborn Screening for Severe Combined Immunodeficiency: Analytic and Clinical Performance of the T Cell Receptor Excision Circle Assay in France (DEPISTREC Study)

J Clin Immunol. 2018 Oct;38(7):778-786. doi: 10.1007/s10875-018-0550-7. Epub 2018 Sep 24.

Abstract

Severe combined immunodeficiency (SCID) is characterized by a major T cell deficiency. Infants with SCID are asymptomatic at birth but die from infections in the first year of life if not treated. Survival rates are better for early treatment. SCID therefore meets criteria for newborn screening (NBS). T cell receptor excision circle (TREC) quantification is a reliable marker of T cell deficiency and can be performed using Guthrie cards. The DEPISTREC project was designed to study the feasibility, clinical utility, and cost-effectiveness of generalized SCID screening in France. About 200,000 babies from all over the country were screened at birth with a commercial kit. We determined assay performance and proposed a cutoff for classification of results. Our findings suggest that, given clearly established validation rules and decision-making procedures, the TREC assay is a suitably specific and sensitive method for high-throughput SCID screening. Clinical Trials: NCT02244450.

Keywords: Newborn screening; T cell receptor excision circle (TREC) quantification; analytic performance; clinical performance; severe combined immunodeficiency (SCID).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biological Assay
  • Biomarkers
  • Clinical Decision-Making
  • Cost-Benefit Analysis
  • Disease Management
  • France / epidemiology
  • Humans
  • Infant
  • Infant, Newborn
  • Neonatal Screening
  • Public Health Surveillance
  • Reagent Kits, Diagnostic
  • Receptors, Antigen, T-Cell / metabolism
  • Reproducibility of Results
  • Severe Combined Immunodeficiency / diagnosis*
  • Severe Combined Immunodeficiency / epidemiology
  • Severe Combined Immunodeficiency / immunology
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism

Substances

  • Biomarkers
  • Reagent Kits, Diagnostic
  • Receptors, Antigen, T-Cell

Associated data

  • ClinicalTrials.gov/NCT02244450