title: Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age.
abstract: OBJECTIVE
  Evaluate clinical outcome of early cyclic intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta (OI), commenced before three years of age.
  
  
  METHODS
  A retrospective review of 17 patients with moderate-to-severe OI. Development, anthropometry, fracture history, bone mineral density (BMD) and biochemistry were collected at baseline, 12 and 24 months.
  
  
  RESULTS
  Four had OI type I, eleven had type III, one OI-FKBP10 type and one OI type V. Mean age at start of pamidronate was 14 ± 11 months. Pamidronate ranged from 6 to 12 mg/kg/year. No adverse reaction apart from fever and vomiting was noted. Long bone fracture decreased from a mean of 10.4/year to 1.2/year after 12 months and 1.4/year after 24 months (p = 0.02). Lumbar spine age- and height-matched BMD Z-scores increased (p < 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p < 0.001). Concavity index, likewise, improved (p < 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting.
  
  
  CONCLUSIONS
  Cyclic intravenous pamidronate, started under 3 years of age in children with moderate-to-severe OI, was well tolerated and associated with an increase in lumbar spine BMD, reduced fracture frequency, vertebral remodelling and attainment of motor milestones at an earlier age.
authors.affiliation: Clinical Genetics, The Sydney Children's Hospital Network Westmead Campus, Westmead, N.S.W., Australia.
authors.forename: M B
authors.initials: MB
authors.lastname: Alcausin
authors.forename: J
authors.initials: J
authors.lastname: Briody
authors.forename: V
authors.initials: V
authors.lastname: Pacey
authors.forename: J
authors.initials: J
authors.lastname: Ault
authors.forename: M
authors.initials: M
authors.lastname: McQuade
authors.forename: C
authors.initials: C
authors.lastname: Bridge
authors.forename: R H H
authors.initials: RH
authors.lastname: Engelbert
authors.forename: D O
authors.initials: DO
authors.lastname: Sillence
authors.forename: C F
authors.initials: CF
authors.lastname: Munns
mesh_terms: D015519:Bone Density; D050071:Bone Density Conservation Agents / Q000008:administration & dosage*; D002675:Child, Preschool; D004164:Diphosphonates / Q000008:administration & dosage*; D005260:Female; D050723:Fractures, Bone / Q000517:prevention & control*; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D008159:Lumbar Vertebrae / Q000033:anatomy & histology / Q000502:physiology; D008297:Male; D009048:Motor Skills; D010013:Osteogenesis Imperfecta / Q000188:drug therapy*; D000077268:Pamidronate; D012189:Retrospective Studies
chemical_list: D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D000077268:Pamidronate