Long-term results of continuous subcutaneous recombinant PTH (1-34) infusion in children with refractory hypoparathyroidism

J Clin Endocrinol Metab. 2011 Nov;96(11):3308-12. doi: 10.1210/jc.2011-1359. Epub 2011 Aug 24.

Abstract

Hypoparathyroidism in children is most often due to mutations in genes involved in parathyroid development and calcium homeostasis signaling. Some rare cases result from autoimmune attack on the parathyroid glands as a part of the type 1 polyglandular failure syndrome (autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy). The majority of cases of pediatric hypoparathyroidism are well controlled under conventional treatment with calcium and vitamin D analogs. However, this treatment may be difficult to manage, especially in two situations: 1) in the context of autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy and 2) activating mutations in the calcium-sensing receptor. We successfully treated three patients with hypoparathyroidism with continuous subcutaneous administration of rhPTH(1-34) (recombinant human PTH(1-34)), two of which were refractory to conventional therapy.

Publication types

  • Case Reports

MeSH terms

  • AIRE Protein
  • Adolescent
  • Child
  • Humans
  • Hypoparathyroidism / complications
  • Hypoparathyroidism / drug therapy*
  • Hypoparathyroidism / genetics
  • Infusions, Subcutaneous
  • Male
  • Parathyroid Hormone / administration & dosage
  • Parathyroid Hormone / therapeutic use*
  • Polyendocrinopathies, Autoimmune / complications
  • Polyendocrinopathies, Autoimmune / genetics*
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / therapeutic use*
  • Transcription Factors / genetics
  • Treatment Outcome

Substances

  • Parathyroid Hormone
  • Recombinant Proteins
  • Transcription Factors

Supplementary concepts

  • Autoimmune polyendocrinopathy syndrome, type 1