Failure of cortisone acetate treatment in congenital adrenal hyperplasia because of defective 11beta-hydroxysteroid dehydrogenase reductase activity

J Clin Endocrinol Metab. 1999 Apr;84(4):1210-3. doi: 10.1210/jcem.84.4.5584.

Abstract

Congenital adrenal hyperplasia in children is often treated with cortisone acetate and fludrocortisone. It is known that certain patients with congenital adrenal hyperplasia require very high substitution doses of cortisone acetate, and a few patients do not respond to this treatment at all. A patient with 21-hydroxylase deficiency, for whom elevated pregnanetriol (P3) levels in urine were not suppressed during treatment with cortisone acetate (65 mg/m2 x day), was examined. The activation of cortisone to cortisol was assessed by measuring urinary metabolites of cortisone and cortisol. The patient's inability to respond to treatment with cortisone acetate was found to be caused by a low conversion of cortisone to cortisol, assumed to be secondary to low 11beta-hydroxysteroid dehydrogenase activity (11-oxoreductase deficiency). All exons and exon/intron junctions of the 11beta-hydroxysteroid dehydrogenase type1 gene (HSD11L) were sequenced without finding any mutations, but a genetic lesion in the promoter or other regulatory regions cannot be ruled out. The deficient 11-oxoreductase activity seems to have been congenital, in this case, but can possibly be attributable to a down-regulation of the enzyme activity. The results support the use of hydrocortisone, rather than cortisone acetate, for substitution therapy in adrenal insufficiency.

Publication types

  • Case Reports

MeSH terms

  • 11-beta-Hydroxysteroid Dehydrogenases
  • 17-alpha-Hydroxyprogesterone / blood
  • Adrenal Hyperplasia, Congenital / drug therapy*
  • Child, Preschool
  • Cortisone / analogs & derivatives*
  • Cortisone / therapeutic use
  • Female
  • Humans
  • Hydrocortisone / therapeutic use
  • Hydroxysteroid Dehydrogenases / deficiency*
  • Treatment Failure

Substances

  • 17-alpha-Hydroxyprogesterone
  • Hydroxysteroid Dehydrogenases
  • 11-beta-Hydroxysteroid Dehydrogenases
  • Cortisone
  • Hydrocortisone