Abstract
Zoledronate increases Bone Mineral Density in children with severe cerebral palsy: A randomized, controlled trial
Introduction
Zoledronate is increasingly used in children with cerebral palsy and low bone quality. Still, no randomized trial has evaluated the efficacy of this third-generation bisphosphonate.
We aimed to perform a randomized, controlled, double-blind trial to evaluate the 12-month effect of two doses of zoledronate on bone mineral density (BMD) Z-scores of the lumbar and lateral distal femur in children with non-ambulant cerebral palsy.
Methods
Two doses of placebo or zoledronate were infused at a 6-month interval. Dual X-ray Absorptiometry was done at baseline and 12 months and the BMD Z-score change was calculated for each patient. Growth was monitored using weight, bone age, pubertal staging, knee-heel length and blood biomarkers.
Results
Twenty-four participants were randomized and completed the study. Ten were treated with placebo. The fourteen participants in the zoledronate group experienced Z-score gains of +0.8 SD at the lumbar spine and +0.3, to +1.7 in the three regions of the lateral distal femur. Three of four gains were significantly higher than in the placebo group. Moderate to very severe influenza-like symptoms were common in the zoledronate group during the first week after the first dose, but not after the second. Lasting side effects including growth changes were not encountered.
Conclusion
Two doses of zoledronate significantly increased lumbar and lateral distal femur BMD Z-scores in children with severe cerebral palsy after one year without affecting growth. First-dose adverse events were common and considerable.
Significance
Third generation bisphosphonate therapy in children with severe CP is now supported by high level evidence regarding effect and short-term adverse events. Studies into lower first doses and long-term outcomes are needed.
Introduction
Zoledronate is increasingly used in children with cerebral palsy and low bone quality. Still, no randomized trial has evaluated the efficacy of this third-generation bisphosphonate.
We aimed to perform a randomized, controlled, double-blind trial to evaluate the 12-month effect of two doses of zoledronate on bone mineral density (BMD) Z-scores of the lumbar and lateral distal femur in children with non-ambulant cerebral palsy.
Methods
Two doses of placebo or zoledronate were infused at a 6-month interval. Dual X-ray Absorptiometry was done at baseline and 12 months and the BMD Z-score change was calculated for each patient. Growth was monitored using weight, bone age, pubertal staging, knee-heel length and blood biomarkers.
Results
Twenty-four participants were randomized and completed the study. Ten were treated with placebo. The fourteen participants in the zoledronate group experienced Z-score gains of +0.8 SD at the lumbar spine and +0.3, to +1.7 in the three regions of the lateral distal femur. Three of four gains were significantly higher than in the placebo group. Moderate to very severe influenza-like symptoms were common in the zoledronate group during the first week after the first dose, but not after the second. Lasting side effects including growth changes were not encountered.
Conclusion
Two doses of zoledronate significantly increased lumbar and lateral distal femur BMD Z-scores in children with severe cerebral palsy after one year without affecting growth. First-dose adverse events were common and considerable.
Significance
Third generation bisphosphonate therapy in children with severe CP is now supported by high level evidence regarding effect and short-term adverse events. Studies into lower first doses and long-term outcomes are needed.
| Originalsprog | Engelsk |
|---|---|
| Publikationsdato | apr. 2023 |
| Status | Udgivet - apr. 2023 |