Continuous subcutaneous recombinant parathyroid hormone (PTH)(1-34) infusion improves serum calcium and phosphate homeostasis in children with autosomal dominant hypocalcemia type 1 refractory to standard-of-care treatment
Authors' conclusion
SPI appears to be a safe and effective long-term treatment in pediatric ADH1, improving mineral homeostasis and renal calcium handling. Prospective multicenter studies are needed to assess long-term skeletal outcomes and optimize dosing strategies.
Study context
- Population
- Children with genetically confirmed ADH1 refractory to SOC, treated with CSPI 2012–2017
- Participants
- 5 children
- Exposure
- CSPI vs SOC
- Outcome
- Serum calcium, phosphate, urinary calcium/creatinine ratio and FECa (linear mixed-effects models)
- Follow-up
- median 7-year follow-up
Methodological detailssingle-centre
- Single-centre
Main finding
As reported in the abstract
- CSPI vs SOC: P < 0.001
- CSPI vs SOC: P < 0.001
- CSPI vs SOC: P < 0.001
- CSPI vs SOC: ≈8% lower; P = 0.014
Observational data: associations, not effects.
Further findings
Clinical outcomes · median 7-year follow-up
- Hypocalcemic seizures and emergency visits decreased; growth was appropriate
Renal function and nephrocalcinosis
- Progression of nephrocalcinosis
- 4/5 patients
- Renal function remained stable
Safety
Treatment-related adverse events · median 7-year follow-up
- Treatment-related adverse events
- 0
Original publication
Continuous subcutaneous recombinant PTH(1-34) infusion improves serum calcium and phosphate homeostasis in children with autosomal dominant hypocalcemia type 1 refractory to standard-of-care treatment. Eur J Endocrinol. Published 20 May 2026. doi:10.
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