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Intravenous neridronate in adults with osteogenesis imperfecta.

机译:成人成骨不全症患者的静脉内神经磷酸酯。

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Osteogenesis imperfecta (OI) is a heritable disease of connective tissue, characterized by increased bone fragility. Bisphosphonates currently seems to be the most promising therapy, at least in children. We tested IV neridronate, an amino-bisphosphonate structurally similar to alendronate and pamidronate in adults with OI. Twenty-three men and 23 premenopausal women with OI were randomized to either iv neridronate (100 mg infused intravenously for 30 minutes every 3 months) or no treatment with a ratio of 2 to 1. Control patients were given the same bisphosphonate therapy at the end of the first year. Clinical evaluation included bone densitometry measurements using dual energy X-ray absorptiometry (DXA), fasting serum and urinary biochemistry every 6 months, and radiographs of the spine taken at baseline and after 12 and 24 months of follow-up. Spine and hip bone mineral density rose by 3.0 +/- 4.6% (SD) and by 4.3 +/- 3.9%, respectively, within the first 12 months of treatment, whereas small insignificant changes were observed in the control group. During the second year of follow-up, additional 3.91% and 1.49% increases were observed at the spine and hip, respectively. Markers of skeletal turnover significantly fell during neridronate treatment. Fracture incidence during neridronate treatment was significantly lower than before therapy and compared with controls. Neridronate iv infusions, administered quarterly, significantly increase bone mineral density and lowered the risk of clinical fracture in adults with OI. Bisphosphonate therapy seems to provide clinical benefits, not only to children with OI, but also to adult patients.
机译:成骨不全症(OI)是结缔组织的遗传性疾病,其特征是骨骼脆弱性增加。目前,双膦酸盐似乎是最有前途的疗法,至少在儿童中是这样。我们在患有OI的成年人中测试了IV neridronate,一种结构上类似于阿仑膦酸盐和pamidronate的氨基双膦酸盐。 23例OI男性和23例绝经前女性被随机分配为neridronate静脉注射(每3个月静脉注射100 mg,持续30分钟)或不进行2比1的治疗。第一年。临床评估包括使用双能X线骨密度仪(DXA)进行骨密度测量,每6个月禁食血清和尿液生化检查,以及在基线以及随访12和24个月后拍摄的脊柱X光片。在治疗的前12个月内,脊柱和髋骨的矿物质密度分别增加了3.0 +/- 4.6%(SD)和4.3 +/- 3.9%,而对照组的变化很小。在随访的第二年,脊柱和髋部分别增加了3.91%和1.49%。神经氨酸酯治疗期间骨骼更新的标记显着下降。神经氨酸酯治疗期间的骨折发生率显着低于治疗前并与对照组相比。季戊酸Neridronate iv输注可显着增加OI成人的骨矿物质密度并降低临床骨折的风险。双膦酸盐治疗似乎不仅为患有OI的儿童提供了临床益处,还为成年患者提供了临床益处。

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