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Intermittent Bi-Daily Sub-cutaneous Teriparatide Administration in Children With Hypoparathyroidism: A Single-Center
Julie Bernardor1,2,3,4, Sacha Flammier1, Sara Cabet5
1Centre de Référence des Maladies Rares du Calcium et du Phosphore, Centre de Référence des Maladies Rénales Rares, Filières de Santé Maladies Rares OSCAR, ORKID et ERKNet, Service de Néphrologie Rhumatologie et Dermatologie Pédiatriques, Hôpital Femme Mère Enfant, Bron, France.
Insights
Teriparatide therapy in children with hypoparathyroidism improved calcium and phosphate levels without increasing calciuria. This investigational treatment was found to be safe and well-tolerated in pediatric patients.
Area of Science:
- Pediatric Endocrinology
- Metabolic Bone Diseases
- Pharmacology
Background:
- Hypoparathyroidism in children often requires physiologic replacement therapy.
- Teriparatide is an investigational treatment option for pediatric hypoparathyroidism.
- Previous reports on teriparatide use in children are limited.
Purpose of the Study:
- To retrospectively report the pediatric experience with bi-daily subcutaneous teriparatide.
- To evaluate the efficacy and safety of teriparatide in children with hypoparathyroidism.
- To assess the impact of teriparatide on calcium and phosphate homeostasis.
Main Methods:
- Retrospective analysis of 10 pediatric patients with hypoparathyroidism treated with teriparatide.
- Bi-daily subcutaneous teriparatide administration with dose adjustments based on calcium levels.
- Regular assessment of serum calcium, phosphate, and estimated glomerular filtration rate (eGFR).
Main Results:
- Teriparatide therapy led to significant improvements in calcium and phosphate control over 12 months.
- Phosphate levels significantly decreased (p = 0.01) after 12 months of treatment.
- No significant increase in calciuria or incidence of nephrolithiasis was observed; two cases of moderate nephrocalcinosis developed.
Conclusions:
- Intermittent teriparatide therapy is effective in managing calcium and phosphate levels in pediatric hypoparathyroidism.
- The treatment appears safe and well-tolerated in children, with manageable side effects.
- Teriparatide offers a promising therapeutic option for children with hypoparathyroidism.
Abstract:
Introduction: The use of teriparatide has been reported in children with hypoparathyroidism as an investigational physiologic replacement therapy. Methods: We aimed to retrospectively report our pediatric experience of bi-daily sub-cutaneous teriparatide. Results are presented as median (25th-75th quartile). As part of the routine follow-up of these patients with hypoparathyroidism, total calcium at H0 (i.e., just before injection) and H4 (i.e., 4 h after teriparatide injection) and other biomarker parameters were regularly assessed. Results: At a median age of 10.7 (8.1-12.6) years, an estimated glomerular filtration rate (eGFR) of 110 (95-118) mL/min/1.73 m2, calcium levels of 1.87 (1.81-1.96) mmol/L and an age-standardized phosphate of 3.8 (2.5-4.9) SDS, teriparatide therapy was introduced in 10 patients at the dose of 1.1 (0.7-1.5) μg/kg/day (20 μg twice daily), with further adjustment depending on calcium levels. Six patients already displayed nephrocalcinosis. Severe side effects were reported in one child: two episodes of symptomatic hypocalcemia and one of iatrogenic hypercalcemia; one teenager displayed dysgueusia. Calcium levels at H0 did not significantly increase whilst calcium at H4 and phosphate levels significantly increased and decreased, respectively. After 12 months, eGFR, calcium and age-standardized phosphate levels were 108 (90-122) mL/min/1.73 m2, 2.36 (2.23-2.48) mmol/L, 0.5 (-0.1 to 1.5), and 68 (63-74) nmol/L, respectively, with a significant decrease in phosphate levels (p = 0.01). Urinary calcium and calcium/creatinine ratio remained stable; no nephrolithiasis was observed but two moderate nephrocalcinosis appeared. Conclusion: Intermittent teriparatide therapy significantly improves calcium and phosphate control, without increasing calciuria. It appears to be safe and well-tolerated in children.
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