BIPHOSPHONATE THERAPY FOR OSTEOGENESIS IMPERFECTA

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。本临床研究提案描述了一项评价双膦酸盐治疗成骨不全（OI）的有效性和安全性的研究。OI是一种遗传性胶原蛋白合成障碍。受影响的人骨密度低，经历多处骨折和进行性骨畸形。在最严重的情况下，这种疾病在婴儿期是致命的。迄今为止，对于这种使人衰弱的病症还没有有效的治疗方法。据报道，少数儿童静脉注射双膦酸盐可改善骨密度和骨折率。口服双膦酸盐的疗效尚未确定。我们将描述口服双膦酸盐治疗的变化，并将其与静脉注射双膦酸盐治疗一组OI儿童的方案进行比较。

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.