Correlation between genotype and hormonal levels in heterozygous mutation carriers and non-carriers of 21-hydroxylase deficiency

J Endocrinol Invest. 2011 Jul-Aug;34(7):498-501. doi: 10.3275/7225. Epub 2010 Jul 29.

Abstract

Congenital adrenal hyperplasia, both in its classic (CCAH) and non-classic form (NCAH), is a morbid condition sustained by the absent or reduced function of one of the enzymes involved in cortisol biosynthesis - mainly 21 hydroxylase - associated with different levels of clinical androgenization. In a wide group of relatives of patients affected by CCAH and NCAH (no.=222) and healthy volunteers (no.=30), a clinical, hormonal and genetic evaluation was performed in order to differentiate between the condition of heterozygous mutation carrier and non-carrier of any among 21-hydroxylase gene (CYP21) mutations. This study shows that clinical presentation and basal 17α-hydroxyprogesterone (17α-OHP) are not able to differentiate between heterozygous carriers and non-carriers, whereas 17α-OHP value after ACTH bolus is significantly different between heterozygous carriers and non-carriers: p<0.001 with a cut-off value of 3 ng/ml (90% sensitivity and 74,3% specificity). Moreover, our data indicate that 17α-OHP response to ACTH may be a useful tool to select subjects for genetic analysis.

MeSH terms

  • 17-alpha-Hydroxyprogesterone / blood*
  • Adrenal Hyperplasia, Congenital / blood*
  • Adrenal Hyperplasia, Congenital / genetics*
  • Adrenal Hyperplasia, Congenital / physiopathology
  • Adrenocorticotropic Hormone / administration & dosage
  • Carrier State*
  • Female
  • Genotype*
  • Humans
  • Male
  • Mutation*
  • Phenotype
  • Sensitivity and Specificity
  • Steroid 21-Hydroxylase / genetics*

Substances

  • 17-alpha-Hydroxyprogesterone
  • Adrenocorticotropic Hormone
  • Steroid 21-Hydroxylase

Supplementary concepts

  • Congenital adrenal hyperplasia due to 21 hydroxylase deficiency