Successful off-label use of cinacalcet HCl after standard therapy failure in a young man with pseudohypoparathyroidism Type 1b and vitamin D intoxication sequelae

Clin Nephrol. 2008 Nov;70(5):439-44. doi: 10.5414/cnp70439.

Abstract

We describe the case of a young man with pseudohypoparathyroidism Type 1b--a rare genetic disorder characterized by end-organ resistance to parathormone (PTH)--and vitamin D intoxication sequelae due to inappropriate and poorly monitored calcitriol treatment in his adolescence, who could no longer be successfully treated by standard vitamin D treatment alone. Off-label administration of cinacalcet HCl, a calcimimetic approved for the treatment of secondary hyperparathyroidism, together with the vitamin D analog dihydrotachysterol, however, proved successful in controlling parathormone (PAH), bone-specific alkaline phosphatase (BAP), serum calcium, and phosphate levels.

Publication types

  • Case Reports

MeSH terms

  • Calcium / blood
  • Cinacalcet
  • Follow-Up Studies
  • Humans
  • Male
  • Naphthalenes / therapeutic use*
  • Parathyroid Hormone / blood
  • Pseudohypoparathyroidism / blood
  • Pseudohypoparathyroidism / drug therapy*
  • Vitamin D / poisoning*
  • Young Adult

Substances

  • Naphthalenes
  • Parathyroid Hormone
  • Vitamin D
  • Calcium
  • Cinacalcet