BISPHOSPHONATE THERAPY FOR CHILDHOOD OSTEOPOROSIS

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目及 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 由于年龄或病史而不符合方案 837（双膦酸盐治疗成骨不全症）的成骨不全儿童，以及患有成骨不全以外的骨质疏松症（特发性青少年骨质疏松症、类固醇诱发骨质疏松症）的儿童将接受双膦酸盐治疗，以增加骨矿物质密度。我们计划以非随机的方式研究这些孩子。他们将接受静脉注射或口服双膦酸盐治疗。静脉注射帕米膦酸治疗，剂量为 1.5-3 mg/kg（取决于年龄），每 2-4 个月分 3 天进行。口服治疗将使用阿仑膦酸钠<1mg/kg，最小剂量为每天10mg，最大剂量为每天40mg。无论采用哪种治疗方式，儿童都会每 4 个月返回诊所一次。接受静脉注射治疗的患者将住院 2 1/2 天；口头访问者将进行一天的访问。 我们的主要疗效结果变量是 BMD，每 4 个月测量一次。次要结果将包括以下内容： 骨折发生率；钙生物化学和骨形成和吸收的生物标志物；人体测量；牙齿解剖学特征和治疗的牙齿效果（对于患有成骨不全或其他涉及牙本质发育不全的疾病的儿童）；系统评估生活质量和疼痛措施；评估粗大运动功能（针对患有成骨不全或其他粗大运动障碍的儿童）；和治疗的安全性。通过以这种方式研究这些儿童，我们将收集初步数据，使我们能够设计未来治疗儿童骨质疏松症的研究。

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. Children with OI who do not qualify for protocol 837 ( Bisphosphonate Therapy for Osteogenesis Imperfecta) as a result of their age or medical history and children with osteoporotic disease other than OI ( idiopathic juvenile osteoporosis, steroid induced osteoporosis) will be treated with bisphosphonates to increase their bone mineral density. We plan to study these children in a non-randomized fashion. They will receive IV or oral bisphosphonate therapy. IV therapy will be done with pamidronate, 1.5-3 mg/kg (depending on age) divided over 3 days every 2-4 months. Oral treatment will be done with alendronate as < 1 mg/kg, from a minimum dose of 10 mg to a maximum of 40 mg daily. Regardless of treatment arm, children will return to clinic every 4 months. Those receiving IV therapy will be admitted for 2 1/2 days; those on oral will have a day visit. Our primary efficacy outcome variable will be BMD, measured at 4-monthly intervals. Secondary outcome will include the following: fracture incidence; calcium biochemistry and biomarkers of bone formation and resorption; anthropometric measurements; characterization of dental anatomy and dental effects of therapy ( for children with OI or other conditions involving dentinogenesis imperfecta); systematic assessment of quality of life and pain measures; evaluation of gross motor function (for children with OI or other gross motor impairment); and safety of treatment. By studying these children in this manner, we will collect preliminary data that will enable us to design future studies of the treatment of childhood osteoporosis.