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Published on: July 14, 2023
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.
Background:
Subcutaneous recombinant human parathormone [rhPTH (1-34)] has been introduced for hypoparathyroidism treatment, allowing avoidance of vitamin D and calcium side effects.
Objective:
Our objective was to evaluate rhPTH (1-34) safety and efficacy in pediatric patients with genetically proved syndromic hypoparathyroidism.
Methods:
The study was a 2.5-year self-controlled trial on six pediatric patients (four males, two females, age 9.8±5.1 years) with syndromic hypoparathyroidism including three with autoimmune polyendocrinopathy candidiasis ectodermal dysplasia (APECED) syndrome, two with DiGeorge syndrome, and one with hypoparathyroidism-deafness-renal dysplasia syndrome. We compared patients' clinical and biochemical outcome of conventional treatment based on oral administration of calcium (1-1.5 g/day in three doses) plus oral calcitriol (6.5-33 ng/kg per day in two to three doses) with the outcome obtained with rhPTH (1-34) (teriparatide, 12.5 μg bid). Therapy shift was conducted introducing rhPTH (1-34) while progressively withdrawing calcium and vitamin D. Blood calcium, phosphorus, alkaline phosphatase, and urinary calcium-to-creatinine ratio (mg/mg) before and during rhPTH therapy were compared.
Results:
rhPTH treatment allowed complete calcium and vitamin D withdrawal in two patients, calcium withdrawal in three and reduction of vitamin D dose in two. During rhPTH (1-34), mean blood calcium, phosphorus, and alkaline phosphatase were not significantly modified, whereas significant reduction of the calciuria-to-creatininuria ratio (0.55±0.31 vs. 0.1±0.1, p=0.02) was obtained. The number of tetanic episodes was reduced in four patients during teriparatide treatment compared to conventional treatment.
Conclusion:
In children with syndromic hypoparathyroidism, substitutive treatment with rhPTH (1-34) maintains adequate blood calcium levels and allows prompt normalization of urinary calcium excretion, through direct action on the kidney and through calcium and vitamin D therapy layoff.
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