title: Low-dose intravenous pamidronate treatment in osteogenesis imperfecta.
abstract: Different therapy models have been tried in order to decrease bone resorption in osteogenesis imperfecta. Bisphosphonates are a group of drugs that mainly suppress osteoclast-mediated bone resorption, thus reducing bone turnover. We assessed the effects of low-dose bisphosphonate treatment in children with osteogenesis imperfecta. Sixteen osteogenesis imperfecta patients (12 female, 4 male) with severe deformities were treated with cyclic (3-4 mg/kg/year) intravenous infusions of bisphosphonate (Aredia-Novartis) therapy for a period ranging from 0.6 to 4.7 years (mean 2.50 +/- 1.09 years). Bone mineral density increased from 0.304 +/- 0.146 g/cm2 to 0.362 +/- 0.142 g/cm2 in the first year and to 0.421 +/- 0.146 g/cm2 in the second year. A clinical response was shown with a reduction in fracture rate and improvement in mobilization scores. Fracture rates decreased from a median of 4/year (0-30/year) before treatment to 0/year (0-5/year) during treatment. Ambulation improved in 10 children and remained unchanged in three. Two of the children were fully functional before therapy and one was below two years of age. No adverse effects were seen with pamidronate infusions of 7-10 mg/kg/year (monthly) or with 4 cycles/year 3-4 mg/kg/year. Low-dose cyclical pamidronate infusions markedly increased bone density and decreased bone fracture rate and should be considered as a part of a multi-disciplinary treatment.
authors.affiliation: Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey.
authors.forename: Damla
authors.initials: D
authors.lastname: Gökşen
authors.forename: Mahmut
authors.initials: M
authors.lastname: Coker
authors.forename: Sükran
authors.initials: S
authors.lastname: Darcan
authors.forename: Timur
authors.initials: T
authors.lastname: Köse
authors.forename: Sinan
authors.initials: S
authors.lastname: Kara
mesh_terms: D015519:Bone Density / Q000187:drug effects; D050071:Bone Density Conservation Agents / Q000008:administration & dosage* / Q000494:pharmacology; D002675:Child, Preschool; D004164:Diphosphonates / Q000008:administration & dosage* / Q000494:pharmacology; D005260:Female; D006128:Growth / Q000187:drug effects; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D008297:Male; D009048:Motor Skills / Q000187:drug effects; D010013:Osteogenesis Imperfecta / Q000188:drug therapy*; D000077268:Pamidronate
chemical_list: D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D000077268:Pamidronate