BISPHOSPHONATE THERAPY FOR CHILDHOOD OSTEOPOROSIS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH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 中心，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）的OI儿童和患有除OI以外的骨质疏松症（特发性青少年骨质疏松症、类固醇诱导的骨质疏松症）的儿童将接受双膦酸盐治疗，以增加其骨密度。我们计划以非随机的方式研究这些儿童。他们将接受IV或口服双膦酸盐治疗。将使用帕米膦酸盐进行IV治疗，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进行，剂量< 1 mg/kg，每日最小剂量为10 mg，最大剂量为40 mg。无论治疗组如何，儿童将每4个月返回诊所。接受IV治疗的患者将住院2 1/2天;口服治疗的患者将进行一天的访视。 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;牙齿解剖特征和治疗的牙齿效果（对于患有OI或其他涉及牙本质生成障碍的疾病的儿童）;生活质量和疼痛测量的系统评估;粗大运动功能评估（对于患有OI或其他粗大运动障碍的儿童）;以及治疗的安全性。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的儿童骨质疏松症治疗研究。

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.