Successful off-label use of cinacalcet HCl after standard therapy failure in a young man with pseudohypoparathyroidism Type 1b and vitamin D intoxication sequelae.
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.
Koch
M
M
Center of Nephrology, Gartenstrasse 8, Mettmann, Germany. Koch@dialyse-mettmann.de
Kohnle
M
M
D002118:Calcium / Q000097:blood; D000069449:Cinacalcet; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D009281:Naphthalenes / Q000627:therapeutic use*; D010281:Parathyroid Hormone / Q000097:blood; D011547:Pseudohypoparathyroidism / Q000097:blood / Q000188:drug therapy*; D014807:Vitamin D / Q000506:poisoning*; D055815:Young Adult
D009281:Naphthalenes; D010281:Parathyroid Hormone; D014807:Vitamin D; D002118:Calcium; D000069449:Cinacalcet