17alpha-hydroxylase/17,20-Lyase deficiency due to novel compound heterozygote mutations: treatment for tall stature in a female with male pseudohermaphroditism and spontaneous puberty in her affected sister

J Pediatr Endocrinol Metab. 2005 Apr;18(4):403-11. doi: 10.1515/jpem.2005.18.4.403.

Abstract

We report on two German sisters with deficiency in the 17alpha-hydroxylase/17,20-lyase enzyme corresponding to typical hormone profile. A paternal nonsense mutation R388X in exon 7 and a maternal missense mutation P428L in exon 8 of the CYP17 gene have been identified in both girls. Residual in vitro 17alpha-hydroxylase activity for the conversion of [3H]-Preg to [3H]-17OH-Preg has been detected in transfected 293-cells expressing P428L mutant enzyme; however, no 17,20-lyase activity was observed converting [3H]-17OH-Preg into [3H]-DHEA. The 46,XX-sister spontaneously entered puberty. The 46,XY-sister with a predicted adult height of 203 cm was treated with a high dose of conjugated estrogens and resulted with a final height of 186.9 cm. The present data suggest that compound heterozygous 46,XX females bearing a P428L allele may develop spontaneous onset of puberty. Furthermore, in 46,XY females with tall stature, treatment with conjugated estrogens may lead to a significant reduction of their predicted adult height.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Adrenal Hyperplasia, Congenital / complications*
  • Adrenal Hyperplasia, Congenital / genetics*
  • Adult
  • Arginine
  • Body Height*
  • Codon, Nonsense
  • Disorders of Sex Development / complications
  • Disorders of Sex Development / etiology
  • Disorders of Sex Development / therapy*
  • Estrogens / therapeutic use
  • Estrogens, Conjugated (USP) / therapeutic use
  • Female
  • Growth Disorders / complications
  • Growth Disorders / drug therapy*
  • Growth Disorders / etiology
  • Heterozygote
  • Humans
  • Leucine
  • Mutation*
  • Mutation, Missense
  • Orchiectomy
  • Proline
  • Puberty
  • Steroid 17-alpha-Hydroxylase / genetics*

Substances

  • Codon, Nonsense
  • Estrogens
  • Estrogens, Conjugated (USP)
  • Arginine
  • Proline
  • Steroid 17-alpha-Hydroxylase
  • Leucine