Hogeschool van Amsterdam

Urban Vitality

Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age

Article

<p>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.</p><p>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.</p><p>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 &lt; 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p &lt; 0.001). Concavity index, likewise, improved (p &lt; 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting.</p><p>CONCLUSION: 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.</p>

Reference Alcausin, M. B., Briody, J., Pacey, V., Ault, J., McQuade, M., Bridge, C., ... Munns, C. F. (2013). Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age. Hormone research in paediatrics, 79(6), 333-40. https://doi.org/10.1159/000351374
1 July 2013