在患有 osteogenesis imperfecta 的儿童中使用 risedronate:一个随机,双盲,安慰剂控制的随机试验
Nick Bishop1, Silvano Adami, S Faisal Ahmed
1Academic Unit of Child Health, Department of Human Metabolism, University of Sheffield, Sheffield Children's Hospital, Sheffield, UK.
Lancet (London, England)
|August 10, 2013
概括
口服瑞塞德酸盐通过增加骨矿物质密度和降低骨折风险,有效地治疗儿童的骨质缺陷发生. 这种双酸盐为患有这种疾病的儿科患者提供了耐受性良好的治疗选择.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 骨质新陈代谢和疾病
- 药理干预药理干预
背景情况:
- 儿童的骨质发育不完美症 (OI) 通常通过静脉注射双酸盐来管理.
- 口服利塞德酸盐是一种第三代双酸盐,在儿科OI患者的安全性和疗效方面进行了评估.
研究的目的:
- 评估口服瑞塞德酸盐在患有骨质发育不完美的儿童中的安全性和有效性.
- 为了比较RISEDRONATE与安慰剂对骨矿物质密度和骨折发生率的影响.
主要方法:
- 一个多中心,随机,双盲,安慰剂对照试验,涉及4至15岁的儿童,患有OI和高骨折风险.
- 参与者每天服用口服瑞塞德罗纳特 (2.5或5毫克) 或安慰剂一年,随后进行为期2年的开放式瑞塞德罗纳特延期治疗.
- 主要疗效终点是腰椎区域骨矿物质密度 (BMD) 一年后的百分比变化.
主要成果:
- 在一年后,瑞斯德罗纳特显著增加了腰椎骨 BMD 的16.3%,而安慰剂组的7.6% (p<0.0001).
- 临床骨折发生在31%的RISEDRONATE患者和49%的安慰剂患者 (p=0.0446) 在一年内.
- 两组之间不良事件的概况相似,胃肠道或肌肉骨事件没有显著差异.
结论:
- 口服瑞塞德酸在增加区域骨硬化和降低OI儿童临床骨折风险方面表现出有效性.
- 瑞斯德罗纳酸是一种安全有效的治疗选择,用于诊断为骨质发育不完善的儿科患者.
- 这种药物一般耐受性很好,支持在IO管理中考虑它.
相关概念视频
Bone Disorders
Aging and its effect on bone remodeling is the most common cause of bone disorders. In young and healthy people, bone deposition and resorption happen at an equal rate to maintain optimal bone health.
Bone deposition is also affected by the levels of sex hormones like estrogen and testosterone that promote osteoblast activity and bone matrix synthesis. When the level of these hormones decreases due to aging, it causes a reduction in bone deposition. As a result, bone resorption by osteoclasts...
Bone deposition is also affected by the levels of sex hormones like estrogen and testosterone that promote osteoblast activity and bone matrix synthesis. When the level of these hormones decreases due to aging, it causes a reduction in bone deposition. As a result, bone resorption by osteoclasts...
Osteoclasts in Bone Remodeling
Osteoclasts are cells responsible for bone resorption and remodeling. They originate from hematopoietic progenitor cells present in the bone marrow. Numerous progenitor cells fuse to form multinucleated cells, each with 10-20 nuclei. A single osteoclast has a diameter of 150 to 200 µM. These cells have ruffled borders that break down the underlying bone tissue and release minerals such as calcium into the blood in bone resorption. Osteoclasts cling to bones with their ruffled edges during bone...
Bone Remodeling
Bone remodeling is a continuous and balanced process of bone resorption by osteoclasts and bone formation by osteoblasts. In adults, it helps maintain bone mass and calcium homeostasis. While mechanical stress can stimulate turnover as part of the normal maintenance and reparative process, several hormones also regulate bone remodeling.

