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Oral bisphosphonate treatment in patients with Duchenne Muscular Dystrophy on long term glucocorticoid therapy
Neuromuscular Disorders ( IF 2.7 ) Pub Date : 2020-07-01 , DOI: 10.1016/j.nmd.2020.06.005
Cuixia Tian 1 , Brenda L Wong 1 , Lindsey Hornung 2 , Jane C Khoury 3 , Irina Rybalsky 4 , Karen C Shellenbarger 4 , Meilan M Rutter 5
Affiliation  

Osteoporosis is a major problem in patients with Duchenne muscular dystrophy (DMD), due to glucocorticoid therapy and muscle weakness. Evidence on which to base optimal prevention and treatment strategies, including bisphosphonate use, in DMD are limited. Our objective was to describe bone health outcomes of oral alendronate treatment in patients with DMD and glucocorticoid-induced osteoporosis. We retrospectively studied 54 patients treated between 2005 and 2017, and assessed changes in dual-energy x-ray absorptiometry (DXA) whole body and lumbar spine bone mineral density and content, and lateral distal femur bone mineral density. We also examined vertebral fracture development in a subset with serial spine radiographs. Pre-alendronate DXA Z-score trajectories decreased progressively. Over three years post-alendronate initiation, Z-score trajectories improved (p<0.01) at most sites compared with pre-alendronate trajectories. Height-adjusted Z-score trajectories for lumbar spine bone mineral density (p = 0.01) and whole body bone mineral content (p = 0.0004) also improved. The positive trajectories did not seem to be sustained long term in those treated up to 6 years. Radiographic vertebral findings in 43 patients appeared stable. In conclusion, oral bisphosphonate therapy using alendronate was associated with improvement of DXA bone health indices during the first three years of treatment, and may help mitigate progression of osteoporosis in glucocorticoid-treated patients with DMD.

中文翻译:

长期糖皮质激素治疗杜氏肌营养不良症患者的口服双膦酸盐治疗

由于糖皮质激素治疗和肌肉无力,骨质疏松症是杜氏肌营养不良症 (DMD) 患者的一个主要问题。在 DMD 中制定最佳预防和治疗策略(包括使用双膦酸盐)的证据有限。我们的目标是描述 DMD 和糖皮质激素诱导的骨质疏松症患者口服阿仑膦酸盐治疗的骨骼健康结果。我们回顾性研究了 2005 年至 2017 年间接受治疗的 54 名患者,并评估了双能 X 射线吸收仪 (DXA) 全身和腰椎骨矿物质密度和含量以及股骨远端外侧骨矿物质密度的变化。我们还使用连续脊柱 X 光片检查了一个子集中的椎骨骨折发展。阿仑膦酸钠前 DXA Z 评分轨迹逐渐下降。阿仑膦酸钠启动后三年多,与阿仑膦酸钠前的轨迹相比,大多数位点的 Z 分数轨迹得到改善 (p<0.01)。腰椎骨矿物质密度 (p = 0.01) 和全身骨矿物质含量 (p = 0.0004) 的高度调整 Z 分数轨迹也有所改善。在接受治疗长达 6 年的患者中,积极的轨迹似乎并未长期持续。43 名患者的放射学椎体检查结果似乎稳定。总之,使用阿仑膦酸盐的口服双膦酸盐治疗与治疗的前三年期间 DXA 骨骼健康指数的改善有关,并且可能有助于缓解接受糖皮质激素治疗的 DMD 患者骨质疏松症的进展。01) 和全身骨矿物质含量 (p = 0.0004) 也有所改善。在接受治疗长达 6 年的患者中,积极的轨迹似乎并未长期持续。43 名患者的放射学椎体检查结果似乎稳定。总之,使用阿仑膦酸盐的口服双膦酸盐治疗与治疗的前三年期间 DXA 骨骼健康指数的改善有关,并且可能有助于缓解接受糖皮质激素治疗的 DMD 患者骨质疏松症的进展。01) 和全身骨矿物质含量 (p = 0.0004) 也有所改善。在接受治疗长达 6 年的患者中,积极的轨迹似乎并未长期持续。43 名患者的放射学椎体检查结果似乎稳定。总之,使用阿仑膦酸盐的口服双膦酸盐治疗与治疗的前三年期间 DXA 骨骼健康指数的改善有关,并且可能有助于缓解接受糖皮质激素治疗的 DMD 患者骨质疏松症的进展。
更新日期:2020-07-01
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