Response to zolendronic acid in children with type III osteogenesis imperfecta
Response to zolendronic acid in children with type III osteogenesis imperfecta
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DOI：
10.1007/s00774-009-0149-4
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发表时间：
2010-07-01
影响因子：
3.3
通讯作者：
Khandelwal, Niranjan
中科院分区：
文献类型：
--
作者：
Panigrahi, Inusha;Das, Rashmi Ranjan;Khandelwal, Niranjan
成骨不全（OI）是一种常见的遗传性疾病，表现为宫内或产前或产后骨折，蓝巩膜和耳聋。已经尝试了各种治疗OI的方法，其中双膦酸盐（BPs）似乎在降低骨折率和改善骨密度方面具有最大的益处。唑来膦酸是一种较新的BP，主要用于成人的几种骨骼疾病。我们分析的目的是研究唑来膦酸对III型OI儿童的反应。分析了2006年2月至2009年3月期间接受唑来膦酸治疗的III型OI受试者的病例记录。相关细节记录在预先设计的图表上。主观改善、骨折数量减少和DEXA扫描Z评分用于判断改善。在遗传诊所随访了5例III型OI病例。从新生儿期至7岁，男女比例为3：2。平均治疗时间为20.4个月。所有患者的骨折发生率均有所改善（P = 0.002），DEXA扫描显示骨密度增加（P = 0.01）。注意到的副作用是流感样症状和肌痛。在任何患者中均未观察到低钙血症引起的临床问题。因此，唑来膦酸被认为是III型OI儿童安全有效的BP。最佳获益的确切剂量尚未确定。新的BP的长期效果需要进一步的长期试验。
Osteogenesis imperfecta (OI) is a common genetic disorder that manifests with intrauterine or pre- or postnatal fractures, blue sclera, and deafness. Various treatments for the management of OI have been tried, of which bisphosphonates (BPs) seem to have the maximum benefit in reducing fracture rate and improving bone density. Zolendronic acid is a newer BP tried for several bone diseases, mainly in adults. The objective of our analysis was to study the response to zolendronic acid in children with type III OI. The case records of subjects with type III OI receiving zolendronic acid in the past 3 years between February 2006 and March 2009 were analyzed. Relevant details were recorded on a predesigned chart. Subjective improvement, reduction in number of fractures, and the DEXA scan Z-score were used to judge improvement. Five OI type III cases were followed up in the Genetic clinic. Presentation was from neonatal period to 7 years of age; M:F ratio was 3:2. Average duration of therapy given was 20.4 months. Improvement was noted in all patients, in the form of reduction in frequency of fractures (P = 0.002) and increase in bone density on DEXA scan (P = 0.01). Side effects noted were flu-like symptoms and myalgia. No clinical problems due to hypocalcemia were noted in any of the patients. Thus, zolendronic acid is seen as a safe and effective BP in type III OI children. The exact dose for optimal benefit is yet to be determined. The long-term effects of newer BPs need further long-term trials.