Madenci Ercan, Yilmaz Kutluhan, Yilmaz Mustafa, Coskun Yavuz
Departments of Physical Therapy and Rehabilitation, Gaziantep University, Medical Faculty, 27100 Gaziantep, Turkey.
J Clin Rheumatol. 2006 Apr;12(2):53-6. doi: 10.1097/01.rhu.0000208490.22492.09.
Osteogenesis imperfecta (OI) 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.
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.
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.
The study suggests that alendronate is a safe and well-tolerated drug and that it could increase bone density in children with OI, 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.
成骨不全症（OI）是一种慢性致残性疾病，其特征是由于I型胶原蛋白生成缺陷导致骨骼脆弱。帕米膦酸盐治疗是研究最广泛的治疗方法，已证明有益。我们的目的是评估阿仑膦酸盐（一种比帕米膦酸盐更强效的双膦酸盐）对OI的疗效。
3例患者（年龄3至7岁，平均5岁）（1例III型，2例IV型）接受阿仑膦酸盐（每天口服0.3至0.56mg/kg）治疗2年。随访骨折次数、行走能力、身高增长以及通过双能X线吸收法（DXA）测量的骨密度。
经过2年的阿仑膦酸盐治疗，骨密度显著改善，腰椎骨密度增加47.8%至106.6%，前臂骨密度增加24%至51.4%。腰椎DXA值的z评分从-5.26±0.84增加到-3.1±0.59。骨折率的平均值没有显著变化。只有1例患者行走能力严重受限。她双腿弯曲，治疗前没有支撑就无法坐下。经过治疗，她在帮助下能够行走。血清甲状旁腺激素和碱性磷酸酶浓度没有显著变化。临床和实验室评估未检测到副作用。
该研究表明阿仑膦酸盐是一种安全且耐受性良好的药物，它可以增加OI患儿的骨密度，所有这些都鼓励对比帕米膦酸盐更强效且可口服的双膦酸盐进行进一步研究。此外，本研究是首次报道在OI中使用前臂骨密度测量。
