BISPHOSPHONATE THERAPY FOR CHILDHOOD OSTEOPOROSIS

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。患有OI的儿童由于年龄或病史原因不符合837(双膦酸盐治疗成骨不全)方案的条件，以及患有OI(特发性幼年骨质疏松，类固醇诱导性骨质疏松)以外的骨质疏松疾病的儿童将接受双膦酸盐治疗，以增加他们的骨密度。我们计划以非随机方式对这些儿童进行研究。他们将接受静脉注射或口服双膦酸盐治疗。静脉治疗将使用帕米磷酸钠1.5-3 mg/kg(视年龄而定)，每2-4个月分3天进行。阿仑磷酸钠的口服剂量为1毫克/公斤，每天最小剂量为10毫克，最大剂量为40毫克。无论采用何种治疗手段，儿童每4个月就会回到诊所一次。接受静脉注射治疗的患者将入院两天半；接受口服治疗的患者将接受一天的访问。我们的主要疗效结果变量将是骨密度，每隔4个月测量一次。次要结果将包括：骨折发生率；骨形成和骨吸收的钙生化和生物标志物；人体测量；牙齿解剖特征和治疗的牙齿效果(对于患有OI或其他涉及牙本质形成不完善的情况的儿童)；系统的生活质量评估和疼痛测量；(对于OI或其他粗大运动障碍的儿童)粗大运动功能的评估；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 40mg 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.