title: Cyclic pamidronate therapy in children with osteogenesis imperfecta.
abstract: BACKGROUND
  Severe osteogenesis imperfecta (OI) is a disorder characterized by osteopenia, frequent fractures, progressive deformity, loss of mobility, and chronic bone pain. There has been no effective therapy for the disorder until recently. The main objective of this study was to determine the efficacy and safety of pamidronate in improving bone mineralization and reducing fracture incidence in osteogenesis imperfecta.
  
  
  METHODS
  Intravenous pamidronate was administered to 64 children (from 21 months to 10 years old) with severe OI, in a 1 mg/kg single daily dose for 3 sequential days at 4-month intervals, over 24-48 months duration. Clinical status, biochemical characteristics including bone turnover markers, bone mineral density of the lumbar spine and femoral neck, and radiological changes were assessed regularly during treatment.
  
  
  RESULTS
  The number of fractures decreased from a median of 8 (range 4-11) to 0 fractures/year (range 0-4) (p <0.05). After 16 months of treatment, there was significant improvement in bone mineral density (BMD-DEXA) z-score of the lumbar spine from a median of -5.90 (range -7.01 to -4.76) to -2.70 (range -4.46 to -1.98) (p <0.001). Serum alkaline phosphatase (ALP) (bone formation marker) decreased from a median of 731.0 U/l (range 438-998 U/l) to 183 U/l (range 95-286 U/l) (p <0.001), implying a significant reduction in bone turnover and resorption and increase in bone mineralization. There was no improvement in growth velocity or height SDS. Mobility and ambulation improved in all but five children (all five had taken the drug for less than 2.5 years). There was a significant relief of chronic pain and fatigue but no adverse effects in all children using the drug.
  
  
  CONCLUSIONS
  Cyclic pamidronate administration is effective in improving bone mineralization and reducing fracture incidence in childhood osteogenesis imperfecta.
authors.affiliation: Department of Pediatric Endocrinology and Metabolism, Mofid Children's Hospital, Shaheed Beheshti University of Medical Sciences, Tehran, I.R. Iran. shadabshadab@yahoo.com
authors.forename: S
authors.initials: S
authors.lastname: Salehpour
authors.forename: S
authors.initials: S
authors.lastname: Tavakkoli
mesh_terms: D015502:Absorptiometry, Photon; D015519:Bone Density; D050071:Bone Density Conservation Agents / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D004164:Diphosphonates / Q000008:administration & dosage*; D004334:Drug Administration Schedule; D005260:Female; D005272:Femur Neck / Q000000981:diagnostic imaging / Q000187:drug effects; D050723:Fractures, Bone / Q000000981:diagnostic imaging / Q000453:epidemiology / Q000517:prevention & control*; D006801:Humans; D015994:Incidence; D007223:Infant; D007262:Infusions, Intravenous; D008159:Lumbar Vertebrae / Q000000981:diagnostic imaging / Q000187:drug effects; D008297:Male; D010013:Osteogenesis Imperfecta / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000453:epidemiology; D000077268:Pamidronate; D016896:Treatment Outcome
chemical_list: D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D000077268:Pamidronate