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Zoledronate for Osteogenesis imperfecta: evaluation of safety profile in children
Insights
Zoledronate is effective in reducing fractures in children with Osteogenesis imperfecta (OI). While short-term reactions occurred, no long-term side effects like osteonecrosis of the jaw were observed in this study.
Area of Science:
- Pediatric Endocrinology
- Skeletal Dysplasias
- Pharmacology
Background:
- Bisphosphonates (BPs) have transformed Osteogenesis imperfecta (OI) treatment.
- Zoledronate use in OI has raised safety concerns due to limited research.
- This study retrospectively analyzes zoledronate's safety and fracture frequency in pediatric OI patients.
Purpose of the Study:
- To evaluate the short- and long-term side effects of zoledronate in children with OI.
- To assess the impact of zoledronate on fracture frequency in pediatric OI patients.
Main Methods:
- Retrospective analysis of 26 children diagnosed with OI.
- Zoledronate administered cyclically for a median of 36 months.
- Safety assessment included short- and long-term effects and fracture rates.
Main Results:
- One neonate (3%) experienced symptomatic hypocalcemia post-infusion.
- Three children (11%) exhibited acute phase reactions.
- No long-term side effects, including osteonecrosis of the jaw, were observed over 7 years. Significant fracture reduction occurred in OI types III and IV (p<0.05).
Conclusions:
- Zoledronate demonstrated efficacy in reducing fractures in OI types III and IV.
- Short-term adverse events were manageable.
- Longer-duration studies are needed to confirm the long-term safety profile of zoledronate in OI.
Background:
The advent of bisphosphonates (BPs) has revolutionised the outcome of Osteogenesis imperfecta (OI) in the last few years. There has always been a safety concern regarding zoledronate's use due to a paucity of studies. The current study is a retrospective evaluation of children with OI on the short- and long-term side effects of zoledronate and the frequency of fractures per year after the drug was introduced.
Methods:
A total of 26 children diagnosed with OI, with a median age of 84 (45-121) months were enrolled in the study. They received cyclical zoledronate for a median duration of 36 (11-61) months at quarterly intervals between January 2008 and December 2014. Safety evaluation involved assessment of its short- and long-term effects in addition to the frequency of fractures after its usage.
Results:
One (3%) neonate had symptomatic hypocalcemia 15 days after the infusion. Three children (11%) had acute phase reactions. None had long-term side effects, including osteonecrosis of the jaw, in our 7-year experience. OI of types III and IV (total of 22) had significant reductions in the number of fractures (p<0.05).
Conclusions:
Further long-duration studies are necessary to evaluate the longterm safety of zoledronate.
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