Efficacy of low dose schedule pamidronate infusion in children with osteogenesis imperfecta

J Pediatr Endocrinol Metab. 2001 May;14(5):529-33. doi: 10.1515/jpem.2001.14.5.529.

Abstract

Background: Osteogenesis imperfecta (OI) is a rare condition in which bones are abnormally brittle with frequent fractures. A variety of therapeutic agents has been used with low efficacy. In this study, we present three patients treated for 4 years with i.v. pamidronate.

Patients and methods: Three prepubertal patients, aged 9 (M), 9 (F) and 11 (F) years old, with OI, were treated with 30-60 mg i.v. pamidronate every 6 months over four years. Determinations were made of plasma 1,25-dihydroxycholecalciferol, 25-hydroxycholecalciferol, insulin-like growth factor-I (IGF-I) and its transport protein (IGFBP3), osteocalcin, total alkaline phosphatase and its osseous fraction, and parathormone (PTH) at baseline and after every pamidronate infusion, Densitometry and X-ray of the vertebral column were performed at the same intervals.

Results: Significant reductions of number of bone fractures and pain were observed in all patients, despite lack of any modification in biochemical parameters. Lumbar X-ray and densitometry showed a striking improvement by the end of the treatment period.

Conclusion: Pamidronate seems to be useful in the treatment of patients with osteogenesis imperfecta.

MeSH terms

  • Absorptiometry, Photon
  • Bone Density / drug effects
  • Child
  • Diphosphonates / administration & dosage*
  • Diphosphonates / therapeutic use
  • Dose-Response Relationship, Drug
  • Female
  • Fractures, Bone / prevention & control
  • Humans
  • Infusions, Intravenous
  • Lumbar Vertebrae / metabolism
  • Male
  • Osteogenesis Imperfecta / drug therapy*
  • Osteogenesis Imperfecta / metabolism
  • Palliative Care
  • Pamidronate

Substances

  • Diphosphonates
  • Pamidronate