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Recombinant parathyroid hormone for hypoparathyroidism in children: a narrative review
Devi Dayal1, Atul Gupta1, Saniya Gupta1
1Endocrinology and Diabetes Unit, Department of Pediatrics, Postgraduate Institute of Medical Educa-tion and Research, Chandigarh, India.
Insights
Recombinant parathyroid hormone (rhPTH) therapy offers a safer and more effective treatment for pediatric hypoparathyroidism than traditional calcium and vitamin D analogs. Long-term use of rhPTH1-34 shows no adverse skeletal effects in children.
Area of Science:
- Pediatric Endocrinology
- Metabolic Bone Disease
- Pharmacology
Background:
- Conventional treatment for pediatric hypoparathyroidism involves calcium and vitamin D analogs, which can lead to hypercalciuria, nephrocalcinosis, and renal insufficiency.
- Parathyroid hormone (PTH) replacement therapy is ideal but has been historically limited in children due to concerns about osteosarcoma risk.
Purpose of the Study:
- To review the available data on the efficacy and safety of recombinant parathyroid hormone (rhPTH) use in children with hypoparathyroidism.
- To evaluate the long-term skeletal safety of rhPTH1-34 in pediatric patients.
Main Methods:
- A literature search was conducted using international databases to identify studies on rhPTH use in children.
- Fifteen studies involving approximately 70 children with hypoparathyroidism treated with rhPTH1-34 were analyzed.
Main Results:
- rhPTH1-34 therapy demonstrated effectiveness for both short-term and long-term treatment of hypoparathyroidism.
- Patients treated with rhPTH1-34 showed improved metabolic control, better renal function, and enhanced quality of life compared to conventional therapy.
- No adverse skeletal effects were observed during long-term rhPTH1-34 use, suggesting the initial concerns about bone malignancies were likely overstated.
Conclusions:
- rhPTH1-34 is an effective and safe therapeutic option for pediatric hypoparathyroidism, offering advantages over conventional treatments.
- Long-term administration of rhPTH1-34 in children appears safe regarding skeletal health, though continued surveillance for adverse effects is recommended.
Abstract:
The conventional management of hypoparathyroidism in children involves the use of calcium and vitamin D analogs. This therapy effec-tively increases the serum calcium levels but worsens hypercalciuria and its consequences such as nephrocalcinosis and renal insuffi-ciency. Although replacement with the missing parathyroid hormone (PTH) is ideal and available for more than 2 decades, the reported concerns of osteosarcoma prohibited its use in children with open epiphyses. Nevertheless, the data accumulated over the past several years suggests that the fears of bone malignancies were probably overstated. With an aim to review the available data on recombinant PTH (rhPTH) use, we performed a literature search using international databases and identified 15 studies involving approximately 70 children with hypoparathyroidism due to various etiologies who received rhPTH1-34 for durations between 1 day and 13.5 years. All the studies appear to indicate that rhPTH1-34 therapy is an effective short and long-term strategy for treatment of hypoparathyroidism with better metabolic control, lesser effects on renal function and improved quality of life as compared to conventional therapy. A more significant conclusion is the safety of long-term use of rhPTH1-34 with no observed adverse skeletal effects so far. However, all studies mention the importance of a continued surveillance for adverse effects in the treated patients. This narrative review discusses the experi-ence of rhPTH1-34 use exclusively in children.
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