Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management
Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management
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DOI：
10.1177/0883073810371004
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发表时间：
2010-09-01
影响因子：
1.9
通讯作者：
Campbell, Kim
中科院分区：
文献类型：
--
作者：
Moxley, Richard T., III;Pandya, Shree;Campbell, Kim
2005年，美国神经病学学会和儿童神经病学学会发布了一项实践参数，主要基于涉及6至18个月治疗的研究，表明泼尼松对杜氏肌营养不良症患者的肌肉力量和功能具有有益作用，并建议提供皮质类固醇（泼尼松0.75 mg/kg/d和地夫可特0.9 mg/kg/d）作为治疗。最近的报告强调，皮质类固醇的长期治疗（超过 3 年）可以对神经肌肉功能产生重要的持续益处，而不会引起重大副作用。本综述重点介绍了这些报告，并指出长期皮质类固醇治疗 (1) 可延长步行时间 2 至 5 年，(2) 减少脊柱稳定手术的需要，(3) 改善心肺功能，(4) 延迟无创鼻通气的需要，(5) 提高杜氏肌营养不良症患者的生存率和生活质量。教育、职业和其他社会咨询现在是杜氏肌营养不良症治疗的重要组成部分。
In 2005, the American Academy of Neurology and the Child Neurology Society published a practice parameter, based primarily on studies that involved 6 to 18 months of treatment, indicating that prednisone has a beneficial effect on muscle strength and function in patients with Duchenne muscular dystrophy and recommended that corticosteroids be offered (prednisone 0.75 mg/kg/d and deflazacort 0.9 mg/kg/d) as treatment. Recent reports emphasize that longer term treatment with corticosteroids (greater than 3 years) produces important sustained benefits in neuromuscular function without causing major side effects. This review highlights these reports and indicates that long-term corticosteroid therapy (1) prolongs ambulation by 2 to 5 years, (2) reduces the need for spinal stabilization surgery, (3) improves cardiopulmonary function, (4) delays the need for noninvasive nasal ventilation, and (5) increases survival and the quality of life of patients with Duchenne muscular dystrophy. Educational, vocational, and other social counseling is now a vital part of management for Duchenne muscular dystrophy.