Aim: To find an effective symptomatic treatment for osteogenesis imperfecta (OI).
Methods: In a prospective observational study disodium pamidronate (APD) was given as monthly intravenous infusions to 28 children and adolescents (aged 0.6–18 years) with severe OI or a milder form of the disease, but with spinal compression fractures.
Results: During treatment for 2–9 years, dual energy x ray absorptiometry measurements of the total body and of the lumbar spine showed a gradual increase in bone density. All bone metabolism variables in serum (alkaline phosphatase, osteocalcin, procollagen 1 C-terminal peptide, collagen 1 teleopeptide) and urine (deoxypyridinoline) indicated that there was a decrease in bone turnover. All patients experienced beneficial effects and the younger patients reported a major improvement in wellbeing, pain, and mobility without significant side effects. Vertebral remodelling was also seen.
Conclusions: APD seems to be an efficient symptomatic treatment for children and adolescents with OI.
- osteogenesis imperfecta
- ALP, alkaline phosphatase
- APD, disodium pamidronate
- 1CTP, collagen 1 teleopeptide
- DXA, dual energy x ray absorptiometry
- OI, osteogenesis imperfecta
- P1CP, procollagen 1 C-terminal peptide
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