ALENDRONATE TO IMPROVE BONE MINERAL DENSITY IN BOYS W/DUCHENNE MUSCULAR DYSTROPH

阿仑膦酸钠可改善患有杜氏肌营养不良症的男孩的骨矿物质密度

• 批准号: 7605080
• 负责人: SUSAN APKON
• 金额: $ 0.37万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-03-01 至 2008-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7605080
• 关键词: Adverse effects; Alendronate; Bone Density; Calcium; Child; Computer Retrieval of Information on Scientific Projects Database; Control Groups; Daily; Double-Blind Method; Dual-Energy X-Ray Absorptiometry; Duchenne muscular dystrophy; Fracture; Funding; Glucocorticoids; Grant; Institution; Monitor; Morbidity - disease rate; Oral; Osteoporosis; Pharmaceutical Preparations; Placebos; Randomized; Research; Research Personnel; Resources; Risk; Scanning; Source; Trauma; United States National Institutes of Health; Vitamin D; bisphosphonate; boys; improved; loss of function; pill; placebo controlled study

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. Fractures are a significant source of morbidity in boys with Duchenne muscular dystrophy (DMD), as they not only cause discomfort, but can also precipitate early loss of ambulation (1;2). This is likely related to progressive weakness and subsequent immobility, which has been associated with osteoporosis. Glucocorticoids have been used in the treatment of DMD to slow the progression of weakness and loss of function and have been shown to induce osteoporosis (3). The combination of immobility and glucocorticoid treatment, may, thus predispose these children to fractures resulting from minimal trauma. The use of Dual Energy X-ray Absorptiometry (DXA) has allowed the determination and monitoring of bone mineral density (BMD) in children. DXA scans have shown significantly decreased BMD in boys with Duchenne muscular dystrophy (4;5). Thus, treatment to improve BMD could potentially decrease the risk of fractures and its associated morbidity. The use of bisphosphonate medications in children with osteoporosis improves BMD without significant side effects (6-8). We propose a randomized, double-blinded, placebo controlled study using weekly oral alendronate, a bisphosphonate to improve bone mineral density in boys with Duchenne muscular dystrophy. The control group will receive daily Vitamin D and calcium and a weekly placebo pill. The experimental group will receive these supplements as well as weekly oral alendronate. Bone mineral density will be evaluated at baseline, 6 months, and a year. We hypothesize that the boys receiving alendronate, a bisphosphonate, and oral calcium and vitamin D supplements will have a greater improvement in bone mineral density than boys receiving oral supplements alone.

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 骨折是Duchenne肌营养不良症(DMD)男孩发病率的一个重要来源，因为它们不仅会引起不适，还会导致早期失去行走能力(1；2)。这可能与进行性虚弱和随后的静止有关，这与骨质疏松症有关。糖皮质激素已被用于治疗DMD，以减缓虚弱和功能丧失的进展，并已被证明可导致骨质疏松症。因此，结合不动和糖皮质激素治疗，可能会使这些儿童容易因微创而导致骨折。双能X射线骨密度仪(DXA)的使用使儿童骨密度(BMD)的测定和监测成为可能。DXA扫描显示患有Duchenne肌营养不良症的男孩的骨密度显著降低(4；5)。因此，改善骨密度的治疗可能潜在地降低骨折的风险及其相关的发病率。在患有骨质疏松症的儿童中使用双膦酸类药物可以改善骨密度，而不会有明显的副作用(6-8)。我们提出了一项随机、双盲、安慰剂对照研究，每周口服阿仑磷酸钠，一种双磷酸盐，以改善Duchenne肌营养不良症男孩的骨密度。对照组每天服用维生素D和钙，每周服用一片安慰剂。试验组将接受这些补充剂以及每周口服阿伦磷酸钠。骨密度将在基线、6个月和1年进行评估。我们推测，与单独服用口服补充剂相比，服用阿伦磷酸钠、双磷酸盐、口服钙和维生素D补充剂的男孩在骨密度方面的改善更大。