Cyclic Pamidronate Therapy in Children with Osteogenesis Imperfecta

The main objective of this study was to determine the efficacy and safety of pamidronate in improving bone mineralizationand reducing fracture incidence in osteogenesis imperfecta (01).materials and methods: intravenous pamidronate was administered to 64 children, aged 18 months to 10 years old, with severe 01, in a 1mglkg single daily dose for 3 sequentional days at 4 month intervals, for over a period of 24-48 months. clinical status, biochemical characteristics including bone turnover markers, the bonemineral density of the lumbar spine and femoral neck, and radiologic changes were assessed regularly during treatment.results: the number of fractures decreased from 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-scores of the lumbar spine from 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 medianof 731.0 ufl (range 438-998 ufl) to 183 ufl (range 95-286 ufl) (p<0.001), implying a significant reduction in bone turnover and its resorption and increase in bone mineralization. there was no improvement either in their height growth velocities or in their standard deviation scores. mobility and ambulation improved in allbut 5 children, (all five took the drug for less than 2.5 years).there was a significant relief in chronic pain and fatigue but no adverse effectsin any of the children using the drug.conclusion: cyclic pamidronate administration is effective in improving bone mineralization and reducing fracture incidence in childhood osteogenesis imperfecta.