ORIGINAL ARTICLE Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta

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 vari-ables in serum (alkaline phosphatase, osteocalcin, procollagen 1 C-terminal peptide, collagen 1 tele-opeptide) 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 well-being, 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 (OI) is a congenital disease ofcollagen with variable phenotype. In milder forms thefracture rate is only slightly increased and the stature is normal or slightly decreased. In severe forms, bone softness and multiple fractures lead to progressive bone deformitie