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Published on: April 11, 2012
Pamidronate treatment of less severe forms of osteogenesis imperfecta in children
Margaret Zacharin1, Shankar Kanumakala
1Department of Endocrinology and Diabetes, Royal Children 's Hospital, Melbourne, Australia. margaret.zacharin@rch.org.au
Insights
Pamidronate treatment significantly improved bone quality in children with mild osteogenesis imperfecta (OI), reducing pain and fractures. This therapy enhances mobility and bone density, potentially preventing adult osteoporosis.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Genetic Bone Disorders
Background:
- Osteogenesis imperfecta (OI) in children, even with milder forms, leads to fractures and reduced bone mass, increasing osteoporosis risk.
- Current bisphosphonate therapies are effective for severe OI, but data for mild phenotypes are limited.
Purpose of the Study:
- To assess the efficacy of pamidronate in improving bone quality and clinical outcomes for children with mild osteogenesis imperfecta.
- To evaluate changes in bone pain, mobility, fracture rates, and bone mineral density over a two-year period.
Main Methods:
- An open-label, two-year observational study involving 18 pediatric patients with mild OI.
- Treatment involved pamidronate administration with regular clinical, biochemical, and radiological monitoring.
Main Results:
- Significant reductions in bone pain (16 to 1 patient) and sleep disturbances (12 to 0).
- Improved independent mobility (10 to 17 children) and decreased fracture incidence (1.6 to 0.5 fractures/child/year).
- Mean lumbar bone mineral density increased by 40.8% (p <0.0001), with improved vertebral height and reduced bone turnover markers.
Conclusions:
- Pamidronate effectively enhances bone quality in children with mild OI, alleviating symptoms and improving physical function.
- The treatment reduces fracture frequency, improves quality of life, and is expected to mitigate adult osteoporosis by increasing peak bone mass.
Background:
Bisphosphonate therapy improves bone quality in children with severe osteogenesis imperfecta (OI). Children with milder phenotypes also have prepubertal fractures, bone pain and reduced bone mass, predisposing them to adult osteoporosis.
Objective:
To evaluate treatment effects of pamidronate in children with mild phenotypes of OI.
Methods:
Open label, 2-year observational study of 18 patients, using pamidronate, with clinical, biochemical and radiological monitoring.
Results:
Over 2 years, bone pain decreased from 16 to 1 patient and disturbed sleep from 12 children to 0. Independent mobility improved from 10 to 17 children. Fracture incidence decreased from 1.6 to 0.5 fractures/child/year. Surgical interventions decreased from a mean 1.3 procedures/patient to 0 in the second year of treatment. Growth velocity remained stable at a mean 4.8 cm/year. Mean lumbar vertebral bone mineral density improved by 40.8%, from 0.375 to 0.528 g/cm2 (p <0.0001), z-score from -3.77 to -2.44 (p <0.0001). Mean vertebral height improved by 17.3%, from 15.6 to 18.38 mm (p = 0.07); plasma alkaline phosphatase decreased from 222 to 169 U/l (p = 0.0009) and urinary deoxypyridinoline crosslinks decreased from 26.7 to 21.8 nmol/mmol creatinine (p = 0.21). Two children with vitamin D insufficiency were concurrently treated. A significant association (r = -0.6, p = 0.008) was shown between age at start of treatment and percentage change in BMD after 2 years.
Conclusions:
Pamidronate treatment improves bone quality in children with mild types of OI. It ameliorates clinical symptoms, improves mobility, reduces fracture frequency and thus improves quality of life and in future is likely to reduce the severity and consequences of adult osteoporosis by improved peak bone mass in these children.
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