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Zoledronic acid treatment in children with osteogenesis imperfecta

Ilkka Vuorimies1, Sanna Toiviainen-Salo, Matti Hero

  • 1Hospital for Children and Adolescents, Pediatric Endocrinology and Metabolic Bone Diseases, University of Helsinki, Helsinki, Finland.

Hormone Research in Paediatrics
|February 5, 2011
PubMed

Insights

Zoledronic acid effectively treats pediatric osteogenesis imperfecta (OI), improving bone mineral density and reducing fractures. This bisphosphonate offers a convenient alternative to pamidronate for children with OI.

Area of Science:

  • Pediatric Endocrinology
  • Bone Metabolism
  • Pharmacology

Background:

  • Osteogenesis imperfecta (OI) is a genetic disorder characterized by fragile bones.
  • Intravenous disodium pamidronate is a standard treatment for pediatric OI.
  • Zoledronic acid, approved for adult osteoporosis, was investigated for pediatric OI.

Purpose of the Study:

  • To evaluate the efficacy and safety of zoledronic acid in children with mild osteogenesis imperfecta.
  • To compare zoledronic acid treatment outcomes with established therapies like pamidronate.

Main Methods:

  • A cohort of 17 children with type I OI received zoledronic acid (0.05 mg/kg IV every 6 months) for 1.0-3.2 years.
  • Patients were monitored for clinical parameters, side effects, bone mineral density (BMD), and fractures.
  • Biochemical markers, including serum calcium, phosphate, and parathyroid hormone (PTH), were assessed.

Main Results:

  • A significant increase in lumbar spine BMD z-score (from -2.0 to -0.7 over 2 years) was observed.
  • The incidence of new long-bone fractures decreased during treatment.
  • Transient decreases in serum calcium and phosphate, with a rise in PTH, occurred post-infusion; two patients had symptomatic hypocalcemia.

Conclusions:

  • Intravenous zoledronic acid demonstrates efficacy in treating pediatric OI, comparable to pamidronate.
  • The administration protocol for zoledronic acid is more convenient than pamidronate.
  • Further research is required to determine optimal dosing and long-term safety in pediatric OI patients.
Abstract

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