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Short-term efficacy of monthly pamidronate infusion in patients with osteogenesis imperfecta
Jin-Ho Choi1, Young-Lim Shin, Han-Wook Yoo
1Department of Pediatrics, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon, Korea.
Insights
Monthly pamidronate infusions effectively reduced fracture frequency and improved bone mineral density in children with osteogenesis imperfecta, showing significant benefits without adverse events.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Rare Genetic Disorders
Background:
- Osteogenesis imperfecta (OI) is a rare genetic disorder characterized by fragile bones and frequent fractures.
- Current treatments aim to reduce fracture risk and improve bone health, but efficacy varies.
Purpose of the Study:
- To evaluate the efficacy of monthly intravenous pamidronate infusions in pediatric patients with osteogenesis imperfecta.
- To assess the impact on fracture frequency, biochemical markers, and bone mineral density.
Main Methods:
- Eleven pediatric patients (0.9-13.8 years) with OI received monthly intravenous pamidronate (30 mg/m(2)) for 6-37 months.
- Fracture frequency, biochemical markers, bone mineral density (spine and femur), and anthropometric measurements were monitored.
Main Results:
- Fracture frequency decreased significantly from 2.3 times/year to 0.6 times/year.
- Bone mineral density of the spine and femur showed significant increases.
- No significant changes in height/weight Z-scores or long-term biochemical markers were observed.
Conclusions:
- Monthly intravenous pamidronate is an effective treatment for reducing fracture frequency in children with osteogenesis imperfecta.
- Pamidronate therapy leads to significant improvements in bone mineral density without notable adverse effects in this pediatric population.
Abstract:
We evaluated the efficacy of a monthly infusion of pamidronate on the frequency of fractures, biochemical effects, and bone mineral density in children with osteogenesis imperfecta. Eleven patients from 0.9 to 13.8 yr of age were included in this study. The patients were administered pamidronate intravenously (30 mg/m(2)) over a 4-hr period monthly for a period ranging from 6 to 37 months. Height and weight Z-scores did not change significantly. The frequency of fractures was decreased from 2.3+/-1.01 times per year before treatment to 0.6+/-0.69 times per year during treatment. There were no long-term changes in biochemical markers during pamidronate therapy. The mean bone mineral density of the spine and femur increased significantly. Monthly intravenous pamidronate therapy decreased frequencies of fracture and increased bone mineral density without significant adverse events in Korean patients with osteogenesis imperfecta.
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