Hypogonadotropic hypogonadism as a presenting feature of late-onset X-linked adrenal hypoplasia congenita

J Clin Endocrinol Metab. 2002 Jan;87(1):44-8. doi: 10.1210/jcem.87.1.8163.

Abstract

Mutations in the orphan nuclear receptor DAX-1 cause X-linked adrenal hypoplasia congenita. Affected boys usually present with primary adrenal failure in early infancy or childhood. Impaired sexual development because of hypogonadotropic hypogonadism becomes apparent at the time of puberty. We report adult-onset adrenal hypoplasia congenita in a patient who presented with hypogonadism at 28 yr of age. Although he had no clinical evidence of adrenal dysfunction, compensated primary adrenal failure was diagnosed by biochemical testing. Semen analysis showed azoospermia, and he did not achieve fertility after 8 months of treatment with gonadotropins. A novel Y380D DAX-1 missense mutation, which causes partial loss of function in transient gene expression assays, was found in this patient. This case demonstrates that partial loss-of-function mutations in DAX1 can present with hypogonadotropic hypogonadism and covert adrenal failure in adulthood. Further, an important role for DAX-1 in spermatogenesis in humans is confirmed, supporting findings in the Dax1 (Ahch) knockout mouse.

Publication types

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

MeSH terms

  • Adrenal Glands / abnormalities*
  • Adrenal Insufficiency / complications
  • Adrenocorticotropic Hormone / blood
  • Adult
  • Amino Acid Sequence
  • Conserved Sequence
  • DAX-1 Orphan Nuclear Receptor
  • DNA-Binding Proteins / blood
  • DNA-Binding Proteins / chemistry
  • DNA-Binding Proteins / genetics*
  • Genetic Counseling
  • Humans
  • Hypogonadism / blood
  • Hypogonadism / etiology*
  • Hypogonadism / genetics
  • Male
  • Mutation, Missense
  • Phenotype
  • Receptors, Retinoic Acid / blood
  • Receptors, Retinoic Acid / chemistry
  • Receptors, Retinoic Acid / genetics*
  • Repressor Proteins*
  • Transcription Factors / blood
  • Transcription Factors / chemistry
  • Transcription Factors / genetics*
  • X Chromosome*

Substances

  • DAX-1 Orphan Nuclear Receptor
  • DNA-Binding Proteins
  • NR0B1 protein, human
  • Nr0b1 protein, mouse
  • Receptors, Retinoic Acid
  • Repressor Proteins
  • Transcription Factors
  • Adrenocorticotropic Hormone