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Long-Term Parathyroid Hormone 1-34 Replacement Therapy in Children with Hypoparathyroidism
Karen K Winer1, Andrea Kelly2, Alicia Johns1
1Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD.
Insights
Parathyroid hormone (PTH) 1-34 injections are safe and effective long-term therapy for children with hypoparathyroidism. This study shows normal growth and bone accrual with PTH 1-34 treatment.
Area of Science:
- Pediatric Endocrinology
- Metabolic Bone Disease
- Hormone Replacement Therapy
Background:
- Hypoparathyroidism in children can lead to significant metabolic and growth complications.
- Current management often involves complex regimens of calcium and vitamin D analogs.
- Long-term safety and efficacy data for parathyroid hormone (PTH) 1-34 therapy in pediatric hypoparathyroidism are limited.
Purpose of the Study:
- To assess the safety and efficacy of long-term, multiple daily injections of parathyroid hormone (PTH) 1-34 in children with hypoparathyroidism.
- To evaluate the impact of PTH 1-34 therapy on linear growth, bone accrual, renal function, and mineral homeostasis.
- To compare mineral balance during PTH 1-34 treatment with baseline conditions.
Main Methods:
- A long-term observational study involving 14 children with hypoparathyroidism (ages 7-16).
- Patients received daily subcutaneous injections of PTH 1-34 (0.75 ± 0.15 µg/kg/day) for a mean duration of 6.9 ± 3.1 years.
- Semiannual evaluations included assessments of linear growth, bone mineral density, renal function, and serum/urine mineral levels.
Main Results:
- Normal linear growth and bone accrual velocities were observed throughout the study period.
- Serum alkaline phosphatase levels remained within the normal range, correlating with PTH 1-34 dose.
- While nephrocalcinosis progressed in some patients, overall renal function remained normal, and high urine calcium levels decreased compared to baseline.
Conclusions:
- Multiple daily subcutaneous injections of PTH 1-34 are a safe and effective long-term replacement therapy for pediatric hypoparathyroidism.
- PTH 1-34 therapy supports normal growth and bone development in children with this condition.
- This treatment offers an improved mineral balance compared to conventional calcitriol and calcium therapy.
Objective:
To determine whether multiple daily injections of parathyroid hormone (PTH) 1-34 are safe and effective as long-term therapy for children with hypoparathyroidism.
Study Design:
Linear growth, bone accrual, renal function, and mineral homeostasis were studied in a long-term observational study of PTH 1-34 injection therapy in 14 children.
Methods:
Subjects were 14 children with hypoparathyroidism attributable to autoimmune polyglandular syndrome type 1 (N = 5, ages 7-12 years) or calcium receptor mutation (N = 9, ages 7-16 years). Mean daily PTH 1-34 dose was 0.75 ± 0.15 µg/kg/day. Treatment duration was 6.9 ± 3.1 years (range 1.5-10 years). Patients were evaluated semiannually at the National Institutes of Health Clinical Center.
Results:
Mean height velocity and lumbar spine, whole body, and femoral neck bone accretion velocities were normal throughout the study. In the first 2 years, distal one-third radius bone accrual velocity was reduced compared with normal children (P < .003). Serum alkaline phosphatase correlated with PTH 1-34 dose (P < .006) and remained normal (235.3 ± 104.8 [SD] U/L, N: 51-332 U/L). Mean serum and 24-hour urine calcium levels were 2.05 ± 0.11 mmol/L (N: 2.05-2.5 mmol/L) and 6.93 ± 1.3 mmol/24 hour (N: 1.25-7.5 mmol/24 hour), respectively-with fewer high urine calcium levels vs baseline during calcitriol and calcium treatment (P < .001). Nephrocalcinosis progressed in 5 of 12 subjects who had repeated renal imaging although renal function remained normal.
Conclusions:
Twice-daily or thrice-daily subcutaneous PTH 1-34 injections provided safe and effective replacement therapy for up to 10 years in children with hypoparathyroidism because of autoimmune polyglandular syndrome type 1 or calcium receptor mutation.
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