Alendronate treatment in osteogenesis imperfecta
Alendronate treatment in osteogenesis imperfecta
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DOI：
10.1097/01.rhu.0000208490.22492.09
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发表时间：
2006-04-01
影响因子：
3.4
通讯作者：
Coskun, Y
中科院分区：
文献类型：
--
作者：
Madenci, E;Yilmaz, K;Coskun, Y
目的：成骨不全（01）是一种慢性致残性疾病，其特征是I型胶原蛋白产生缺陷导致的骨脆性。帕米膦酸盐疗法是研究最广泛的治疗方法，已被证明是有益的。我们的目的是评估阿仑膦酸钠，更有效的双膦酸盐比帕米膦酸盐，在OI。材料和方法：3例患者（年龄，3-7岁，平均5年）（1例，III型; 2例，IV型）已给予阿仑膦酸钠（0.3-0.56毫克/公斤，每天口服）2年。结果：阿仑膦酸钠治疗2年后，患者的骨密度明显改善，腰椎骨密度增加47.8%~ 106.6%，前臂骨密度增加24%~ 51.4%。腰椎DXA值的z评分从-5.26 +/- 0.84增加到-3.1 +/- 0.59。骨折发生率的平均值没有显著变化。只有1例患者的活动度受到高度限制。她的腿弯曲，在治疗前没有支撑就无法坐下。她在治疗的帮助下改善了行走。血清甲状旁腺素和碱性磷酸酶浓度无明显变化。在临床和实验室evaluations.Conclusion：这项研究表明，阿仑膦酸钠是一种安全和耐受性良好的药物，它可以增加骨密度的儿童01，所有这些都鼓励进一步的研究与双膦酸盐，比帕米膦酸盐更有效，可以口服。此外，本研究是第一个报告使用前臂骨密度测量在OI。
Aims: Osteogenesis imperfecta (01) is a chronic, disabling condition characterized by bone fragility resulting from defective production of type I collagen. Pamidronate therapy is the most extensively studied treatment and has proved beneficial. Our objective was to evaluate the effect of alendronate, a more potent bisphosphonate than pamidronate, in OI.Materials and Methods: Three patients (age, 3-7 years; mean, 5 years) (one case, type III; 2 cases, type IV) have been given alendronate (0.3-0.56 mg/kg per day orally) for 2 years. Number of fractures, ambulation, height growth, and bone mineral density by dual-energy x-ray absorptiometry (DXA) were followed up.Results: Bone mineral density improved significantly after the 2-year alendronate treatment, which increased by 47.8% to 106.6% in the lumbar spine and by 24% to 51.4% in forearm bones. The z-score of lumbar spine DXA values increased from -5.26 +/- 0.84 to -3.1 +/- 0.59. The mean of fracture rates did not change significantly. Only one of the patients was highly limited in ambulation. She had curved legs and could not sit without support before the treatment. She improved to walk with help by the treatment. Serum parathormone and alkaline phosphatase concentrations did not change significantly. No side effect was detected in clinical and laboratory evaluations.Conclusion: The study suggests that alendronate is a safe and well-tolerated drug and that it could increase bone density in children with 01, all of which encourage further studies with the bisphosphonates that are more potent than pamidronate and can be used orally. In addition, this study is the first report using the forearm bone mineral density measurement in OI.