BIPHOSPHONATE THERAPY FOR OSTEOGENESIS IMPERFECTA

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This clinical research proposal describes a study to evaluate the efficacy and safety of Biphosphonate Therapy for Osteogenesis Imperfecta (OI). OI is a heritable disorder of collagen synthesis. Affected persons have low bone mineral density and experience multiple fractures and progressive bony deformity. In its most severe form, the disorder is lethal in infancy. To date, no effective therapy exists for this debilitating condition. Improvements in bone mineral density and in fracture rates in a small number of children treated with intravenous biphosphonates have been reported. The efficacy of oral biphosphonates has not been established. We will characterize the changed effected by oral biphosphonate therapy and compare them to a regimen of intravenous biphosphonate therapy in a group of children with OI.