Context Patients with glucocorticoid dependent Duchenne Muscular Dystrophy (DMD) have increased fracture risk and reduced bone mineral density (BMD), often precipitating mobility loss. Objective To investigate use of Zoledronic acid (ZA) in DMD in improving BMD. Design Two arm, parallel, randomised controlled trial Setting Paediatric hospitals across Australia and New Zealand Patients Sixty-two (31 per arm) boys with glucocorticoid dependent DMD between 6-16 years Intervention(s) Five ZA infusions (0.025mg/kg at months 0,3, 0.05mg/kg at months 6, 12 and 18), plus calcium and vitamin D, compared with calcium and vitamin D alone Main outcome measures Change in lumbar spine (LS) BMD raw and Z-score by dual energy absorptiometry x-ray (DXA) at 12 and 24 months, secondary outcomes assessing mobility, fracture incidence, bone turnover, pQCT and pain scores Results At 12 and 24 months, mean difference in changes of LSBMD Z-score from baseline was 1.2 SD (95% CI: 0.9-1.5), higher by 19.3% (14.6-24.0) and 1.4 SD (0.9-1.9), higher by 26.0% (17.4-34.5) in ZA compared to control arms respectively (both p < 0.001). Five controls developed Genant 3 vertebral fractures, 0 in the ZA arm. Mobility, pain and bone turnover markers were similar between arms at 12 and 24 months Trabecular BMC and vBMD pQCT at radius and tibia were greater at 12 months in the ZA cohort compared to control; difference remaining at 24 months for radius. Conclusion ZA improved BMD in glucocorticoid dependent DMD boys. Whilst the small cohort precluded demonstrable fracture benefit, improved BMD might reduce incident vertebral fracture.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.