Intrafamilial Phenotypic Variability and Consequences of Non-Compliance with Treatment in Congenital Adrenal Hyperplasia and Congenital Hypothyroidism within a Single Family

Horm Res Paediatr. 2017;88(2):172-178. doi: 10.1159/000465521. Epub 2017 Mar 30.

Abstract

Background: Coexistence of congenital adrenal hyperplasia (CAH) and congenital hypothyroidism (CH) due to TG mutation in the same non-consanguineous family is rare.

Case series: We report 4 siblings born to unrelated parents, the father being an asymptomatic carrier of homozygous p.V281L and heterozygous p.I172N CYP21A2 mutations. Sibling 1 had salt-wasting CAH (CYP21A2 genotype Intron 2 splice/p.I172N and p.V281L). She also had CH (TG genotype p.R296/ p.T1416Rfs*30) and learning difficulties. Poor compliance and morbid obesity resulted in short stature, precocious puberty, hirsutism, amenorrhoea, insulin insensitivity and a possible adrenal adenoma. Sibling 3 (CYP21A2 and TG genotype similar to sibling 1) is a boy presenting with salt-wasting CAH, CH, and developmental delay. He was overweight and underwent precocious puberty. Although siblings 2 and 4 (both females) share the same CYP21A2 genotype (Intron 2 splice/p.V281L), the former only had biochemical evidence of CAH, while the latter presented at 9.8 years of age with a history of pubarche at 7 years and advanced bone age.

Conclusions: We report the unusual occurrence of 2 rare autosomal recessive diseases, CAH and CH. Our cases highlight the phenotypic variability of CAH and CH due to TG mutations, even within a single family, and illustrate the importance of optimal disease control. .

Keywords: 21-Hydroxylase; Adrenal adenoma; Developmental delay; Thyroglobulin.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Adrenal Hyperplasia, Congenital / complications
  • Adrenal Hyperplasia, Congenital / drug therapy
  • Adrenal Hyperplasia, Congenital / genetics*
  • Child
  • Congenital Hypothyroidism / complications
  • Congenital Hypothyroidism / drug therapy
  • Congenital Hypothyroidism / genetics*
  • Female
  • Humans
  • Male
  • Mutation
  • Patient Compliance
  • Pedigree
  • Phenotype
  • Thyroglobulin / genetics*

Substances

  • Thyroglobulin