Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation
Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation
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DOI：
10.1177/0883073814558120
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发表时间：
2015-09-01
影响因子：
1.9
通讯作者：
Valdez, Rodolfo
中科院分区：
文献类型：
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作者：
Kim, Sunkyung;Campbell, Kimberly A.;Valdez, Rodolfo
这项以人群为基础的研究通过肌营养不良症监测跟踪和研究网络(MDSTARnet)确定的477例Duchenne肌营养不良症病例，按治疗时间分层(短：0.25-3年，长：>；3年)，检查了皮质类固醇治疗与失去行走的时间之间的关系。接受短期糖皮质激素治疗的患者失去行走能力的时间比未接受治疗的患者缩短了0.8年(t检验)，每年丧失行走能力的风险比未接受治疗的患者高77%(Cox回归)。相反，接受长期皮质类固醇治疗的患者失去行走的时间比没有接受治疗的人长2年，失去行走的年度风险比没有接受治疗的人低82%，直到11岁；之后的风险在统计学上没有差异。皮质类固醇与失去行走的时间的关系比以前的研究描述的更复杂，这些研究仅限于治疗应答者或在研究随访期间失去行走的受试者。
This population-based study examines the association between corticosteroid treatment and time to loss of ambulation, stratifying by treatment duration (short: 0.25-3 years, long: >3 years), among 477 Duchenne muscular dystrophy cases identified by the Muscular Dystrophy Surveillance Tracking and Research Network (MDSTARnet). Those cases who received short-term corticosteroid treatment had a time to loss of ambulation that was 0.8 years shorter (t test) and an annual risk of losing ambulation 77% higher than the untreated (Cox regression). Conversely, cases who received long-term corticosteroid treatment had a time to loss of ambulation that was 2 years longer and an annual risk of losing ambulation 82% lower than the untreated, up to age 11 years; after which the risks were not statistically different. The relationship of corticosteroids and time to loss of ambulation is more complex than depicted by previous studies limited to treatment responders or subjects who lost ambulation during study follow-up.