Seikaly Mouin G, Kopanati Sashi, Salhab Nina, Waber Pamela, Patterson Diane, Browne Richard, Herring John A
University of Texas Southwestern Medical Center at Dallas, TX, USA.
J Pediatr Orthop. 2005 Nov-Dec;25(6):786-91. doi: 10.1097/01.bpo.0000176162.78980.ed.
Osteogenesis imperfecta (OI) is a debilitating clinical condition characterized by fragile bone and skeletal deformity. Over the past decade frequent reports have suggested that the cyclical administration of intravenous pamidronate has a positive impact on bone density and skeletal fractures; however, the impact of such therapy on the quality of life (QOL) has rarely been reported. Alendronate, an oral bisphosphonate, is widely used to treat osteoporosis. The purpose of this study was to evaluate the impact of daily alendronate on QOL and bone parameters in children with OI. A prospective double-blind crossover study was designed in which placebo was alternated with daily alendronate. Twenty children with types I, III, and IV OI were recruited. Seventeen patients completed the study. Markers of QOL were measured in children with type III and IV OI (n = 15) using total mobility (PEDI), self-care (WeeFIM), well-being, pain, and use of analgesic scores. After 1 year of alendronate therapy, vertebral bone mineral density (BMD) improved from a change in standard deviation score (z-score) of 0.89 +/- 0.19 to -0.12 +/- 0.14 after 1 year of placebo (P < 0.001). All QOL markers, except for mobility score, improved in response to alendronate therapy. Change in height z-score also improved in response to 1 year of alendronate therapy (0.41 +/- 0.21 vs. -0.09 +/- 0.11, P < 0.05). Alendronate therapy did not alter serum levels of calcium, osteocalcin, parathyroid hormone (PTH), 1, 5 (OH)2 vitamin D, cholesterol, or urinary hydroxyproline or any other biochemical marker evaluated. Alendronate decreased by 56% urinary cross-linked N-telopeptide of type 1 collagen divided by urinary creatinine (uNTX/uCr). Daily alendronate therapy was well tolerated. Only two patients had mild gastrointestinal discomfort, responding to minor adjustments in alendronate intake. Daily alendronate therapy is safe and effective in improving QOL in children with OI.
成骨不全症（OI）是一种使人衰弱的临床病症，其特征为骨骼脆弱和骨骼畸形。在过去十年中，频繁的报告表明静脉注射帕米膦酸盐的周期性给药对骨密度和骨骼骨折有积极影响；然而，这种疗法对生活质量（QOL）的影响鲜有报道。阿仑膦酸钠，一种口服双膦酸盐，被广泛用于治疗骨质疏松症。本研究的目的是评估每日服用阿仑膦酸钠对OI患儿生活质量和骨参数的影响。设计了一项前瞻性双盲交叉研究，其中安慰剂与每日阿仑膦酸钠交替使用。招募了20名I型、III型和IV型OI患儿。17名患者完成了研究。使用总活动能力（PEDI）、自我护理（WeeFIM）、幸福感、疼痛和镇痛评分对III型和IV型OI患儿（n = 15）的生活质量指标进行了测量。经过1年的阿仑膦酸钠治疗，椎体骨矿物质密度（BMD）从标准差评分（z评分）变化0.89±0.19改善至安慰剂治疗1年后的-0.12±0.14（P < 0.001）。除活动能力评分外，所有生活质量指标在阿仑膦酸钠治疗后均有所改善。身高z评分的变化在接受1年阿仑膦酸钠治疗后也有所改善（0.41±0.21对-0.09±0.11，P < 0.05）。阿仑膦酸钠治疗未改变血清钙、骨钙素、甲状旁腺激素（PTH）、1, 5（OH）2维生素D、胆固醇或尿羟脯氨酸水平或任何其他评估的生化指标。阿仑膦酸钠使尿中1型胶原交联N-端肽除以尿肌酐（uNTX/uCr）降低了56%。每日阿仑膦酸钠治疗耐受性良好。只有两名患者有轻度胃肠道不适，通过对阿仑膦酸钠摄入量进行轻微调整得到缓解。每日阿仑膦酸钠治疗对改善OI患儿的生活质量是安全有效的。
