ALENDRONATE TO IMPROVE BONE MINERAL DEN IN BOYS W/DUCHENNE MUSC DYSTROPHY

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。骨折是Duchenne型肌营养不良症（DMD）男孩发病的重要原因，因为骨折不仅会引起不适，还可能导致早期截肢（1;2）。这可能与进行性虚弱和随后的不动有关，而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，并已被证明可诱导骨质疏松症（3）。因此，不活动和糖皮质激素治疗的结合可能使这些儿童易于因轻微创伤而发生骨折。双能X线吸收测定法（DXA）的使用允许测定和监测儿童的骨矿物质密度（BMD）。DXA扫描显示Duchenne肌营养不良症男孩的BMD显著降低（4;5）。因此，改善BMD的治疗可能会降低骨折风险及其相关发病率。在骨质疏松症儿童中使用双膦酸盐药物可改善BMD，且无显著副作用（6-8）。 我们提出一项随机、双盲、安慰剂对照研究，每周口服阿仑膦酸钠（一种双磷酸盐）改善杜氏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，每周服用安慰剂。实验组将接受这些补充剂以及每周口服阿仑膦酸钠。将在基线、6个月和1年时评价骨密度。我们假设接受阿仑膦酸钠、双磷酸盐、口服钙和维生素D补充剂的男孩比单独接受口服补充剂的男孩骨密度有更大的改善。

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.