Zoledronic acid improves bone mineral density, reduces bone turnover and improves skeletal architecture over 2 years of treatment in children with secondary osteoporosis

There are limited data on the use of bisphosphonate therapy for secondary osteoporoses in childhood, and no previous reports of the use of zoledronic acid in this group. We report 20 children with a variety of underlying primary diagnoses with associated secondary osteoporosis, who were treated with 3 monthly zoledronic acid for 2 years (annualised dose 0.1 mg/kg/year). There was a significant improvement in lumbar spine (by 1.88 SD ± 1.24 over first 12 months, p < 0.001) and total bone mineral density as assessed by dual energy absorptiometry (DXA) scans, with a similar increase in bone mineral content for lean tissue mass (mean increase 1.34 SD in first 12 months, p < 0.001). Bone turnover was reduced with a suppression of both osteocalcin and alkaline phosphatase in the first 12 months of treatment. Skeletal architecture was improved, with increased second metacarpal cortical thickness from 2.44 mm to 2.72 mm (p < 0.001) and improved vertebral morphometry, with 7 patients who had vertebral wedging at baseline showing improved anterior (p = 0.017) and middle (p = 0.001) vertebral height ratios. Aside from well reported transient side effects with the first dose, there were no adverse effects reported. No adverse effects on anthropometric parameters were seen over the course of the study. Despite all patients having sustained fragility fractures prior to treatment, no fractures were reported during the study period. Further evidence is required to confirm efficacy, with long term follow up required to assess the impact of treatment on fracture risk.

► Zoledronic acid use in children with secondary osteoporosis improves areal and volumetric bone density. ► This agent also improves vertebral morphology in patients with evidence of anterior wedging or crush fractures. ► No significant side effects were observed in patients treated with this agent aside from well described first dose effects. ► While no fractures were observed during our study, longer term follow up is required to fully explore fracture risk.