Zoledronate increases bone mineral density in non-ambulant children with cerebral palsy: A randomized, controlled trial

TY - ABST

T1 - Zoledronate increases bone mineral density in non-ambulant children with cerebral palsy:

T2 - DNPS Årsmøde 2022

AU - Granild-Jensen, Jakob Bie

PY - 2022

Y1 - 2022

N2 - Introduction: Low bone mineral density (BMD) is prevalent in children with cerebral palsy increasing fracture risk and compromising an optimal bone health trajectory through the lifespan. Zoledronate is increasingly used to improve the BMD, however, no randomized trial has evaluated the effect. We performed a multi-center, randomized, controlled, double-blind trial to evaluate the effect of zoledronate in children with non-ambulant cerebral palsy. Patients and Methods:Two doses of placebo or zoledronate were infused at a 6-month interval. Dual X-ray absorptiometries of the lumbar spine and lateral distal femur were done at baseline and 12 months and the BMD Z-score changes were calculated. 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 BMD 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 different from the placebo group. Moderate to very severe influenza-like symptoms were common in the zoledronate group, but only after the first dose. Lasting side effects including growth changes were not encountered. Conclusion: Zoledronate significantly increases BMD in children with cerebral palsy in one year without affecting growth. First-dose side-effects were common and considerable. Studies into lower first doses and long-term outcomes are needed.Relevance for users and families: A low bone health reduces the wellbeing of persons with cerebral palsy due to a higher risk of fractures, bone pain and deformities. Further, fractures are associated with increased mortality. We performed a drug trial and found that two doses of zoledronate increases the bone mineral density in non-ambulant children with cerebral palsy. This reduces the fracture risk and may benefit the lifetime bone health, but the first dose often has severe influenza-like side effects.

AB - Introduction: Low bone mineral density (BMD) is prevalent in children with cerebral palsy increasing fracture risk and compromising an optimal bone health trajectory through the lifespan. Zoledronate is increasingly used to improve the BMD, however, no randomized trial has evaluated the effect. We performed a multi-center, randomized, controlled, double-blind trial to evaluate the effect of zoledronate in children with non-ambulant cerebral palsy. Patients and Methods:Two doses of placebo or zoledronate were infused at a 6-month interval. Dual X-ray absorptiometries of the lumbar spine and lateral distal femur were done at baseline and 12 months and the BMD Z-score changes were calculated. 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 BMD 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 different from the placebo group. Moderate to very severe influenza-like symptoms were common in the zoledronate group, but only after the first dose. Lasting side effects including growth changes were not encountered. Conclusion: Zoledronate significantly increases BMD in children with cerebral palsy in one year without affecting growth. First-dose side-effects were common and considerable. Studies into lower first doses and long-term outcomes are needed.Relevance for users and families: A low bone health reduces the wellbeing of persons with cerebral palsy due to a higher risk of fractures, bone pain and deformities. Further, fractures are associated with increased mortality. We performed a drug trial and found that two doses of zoledronate increases the bone mineral density in non-ambulant children with cerebral palsy. This reduces the fracture risk and may benefit the lifetime bone health, but the first dose often has severe influenza-like side effects.

M3 - Conference abstract for conference

Y2 - 18 November 2022 through 19 November 2022

ER -