Teriparatide (rhPTH) treatment in children with syndromic hypoparathyroidism

Insights

Subcutaneous recombinant human parathormone (rhPTH (1-34)) effectively treats pediatric syndromic hypoparathyroidism. This therapy reduces calcium and vitamin D dependence while normalizing urinary calcium excretion.

Area of Science:

  • Pediatric Endocrinology
  • Genetics
  • Metabolic Bone Disease

Background:

  • Subcutaneous recombinant human parathormone [rhPTH (1-34)] offers a new treatment for hypoparathyroidism.
  • This approach aims to reduce reliance on vitamin D and calcium supplements, mitigating associated side effects.

Purpose of the Study:

  • To evaluate the safety and efficacy of rhPTH (1-34) in children with genetically confirmed syndromic hypoparathyroidism.
  • To compare outcomes of rhPTH (1-34) therapy with conventional treatment regimens.

Main Methods:

  • A 2.5-year self-controlled trial involving six pediatric patients with syndromic hypoparathyroidism (APECED, DiGeorge, hypoparathyroidism-deafness-renal dysplasia syndromes).
  • Comparison of biochemical markers (blood calcium, phosphorus, alkaline phosphatase, urinary calcium-to-creatinine ratio) and clinical outcomes (tetanic episodes) between conventional therapy and rhPTH (1-34) treatment.
  • Gradual withdrawal of calcium and vitamin D during the introduction of rhPTH (1-34).

Main Results:

  • rhPTH (1-34) enabled complete calcium and vitamin D withdrawal in two patients, calcium withdrawal in three, and reduced vitamin D dosage in two.
  • No significant changes in mean blood calcium, phosphorus, or alkaline phosphatase levels were observed during rhPTH (1-34) therapy.
  • A significant reduction in the urinary calcium-to-creatinine ratio was achieved (0.55±0.31 vs. 0.1±0.1, p=0.02).
  • Four patients experienced a reduction in tetanic episodes with teriparatide treatment.

Conclusions:

  • Substitutive treatment with rhPTH (1-34) in children with syndromic hypoparathyroidism maintains adequate blood calcium levels.
  • rhPTH (1-34) therapy facilitates prompt normalization of urinary calcium excretion.
  • The treatment allows for the reduction or discontinuation of calcium and vitamin D therapy.
Abstract

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