[Analysis of TBX19 gene variant in a child with congenital isolated adrenocorticotropic hormone deficiency]

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2021 Jan 10;38(1):59-62. doi: 10.3760/cma.j.cn511374-20191107-00563.
[Article in Chinese]

Abstract

Objective: To analyze the clinical and genetic characteristics of a patient with congenital isolated adrenocorticotropic hormone deficiency (IAD).

Methods: Clinical characteristics of the patient was reviewed. Genomic DNA of the child was subjected to whole exome sequencing.

Results: Genetic testing has confirmed the diagnosis of congenital IAD by identification of compound heterozygous variants of the TBX19 gene, which included a pathogenic nonsense c.535C>T (p.R179X) variant inherited from his father and a novel missense c.298C>T (p.R100C) variant inherited from his mother.

Conclusion: Congenital IAD due to variants of the TBX19 gene is a rare autosomal recessive disease. It is characterized by low plasma adrenocorticotropic hormone and cortisol levels but normal levels of other pituitary hormones. Delayed diagnosis may lead to severe early-onset adrenal failure and wrong treatment which may result in neonatal mortality. Hydrocortisone replacement is effective. Detection of pathogenic variant of TBX19 gene is the key to diagnosis.

Publication types

  • Case Reports

MeSH terms

  • Adrenal Insufficiency* / genetics
  • Child
  • Homeodomain Proteins* / genetics
  • Humans
  • T-Box Domain Proteins* / genetics

Substances

  • Homeodomain Proteins
  • T-Box Domain Proteins
  • TBX19 protein, human

Supplementary concepts

  • Adrenocorticotropic hormone deficiency