糖皮质激素对Duchenne型肌营养不良患者运动功能的影响  

Effect of glucocorticoids on motor function of Duchenne muscular dystrophy

作　　者：张淑[1] 王丹[1] 张昊 马沛沛 吴士文[1] ZHANG Shu;WANG Dan;ZHANG Hao;MA Peipei;WU Shiwen.(Department of Neurology, General Hospital of Chinese Peo- ple＇ s Armed Police Force, Beijing 100039, China)

机构地区：[1]武警总医院神经内科,北京100039

出　　处：《武警医学》2018年第6期545-548,共4页Medical Journal of the Chinese People's Armed Police Force

基　　金：首都特色应用研究与成果推广项目(Z151100004015025)

摘　　要：目的分析Duchenne型肌营养不良(duchenne muscle dystrophy,DMD)患者糖皮质激素应用对其运动功能的影响。方法选取中国肌营养不良注册登记数据库及该中心Duchenne型肌营养不良多学科联合门诊诊断明确的患者为研究对象,记录患者一般人口学特征,分析糖皮质激素应用对本组患者运动功能的影响。结果肌营养不良注册登记数据库共收集抗肌萎缩蛋白缺陷型肌营养不良1182例,经基因检测明确诊断的Duchenne型肌营养不良996例。996例DMD中适龄患者初次就诊时259例(35.2%)规律应用糖皮质激素,目前激素使用率为57.1%。激素组患者丧失行走能力的平均年龄(11.88±0.33)岁,非激素组年龄(10.50±0.37)岁。7.0~11.9岁年龄组,激素组94.2%保留独立行走能力,6 min步行距离为(329.00±11.48)m,卧立位时间(9.84±0.71)s。非激素组76.5%保留行走能力,6 min步行距离为(126.3±1.09)min,卧立位时间(10.02±1.41)s。12岁以上年龄组,激素组46.6%仍保留行走能力,非激素组12.2%保留行走能力。结论激素使用改善了本组患者运动功能,规范的注册登记及多学科门诊对DMD治疗具有重要意义。Objective To determine the effects of glucocorticoids on major motor function of Duchenne muscular dystrophy patients from a database registered in China. Methods In this cross-sectional study,we analyzed the clinical data,use of glucocorticoids and major motor function of genetically confirmed DMD patients from our national DMD registry and multidisciplinary clinic databases.Results 1182 dystrophinopathy patients were registered,996 of which are confirmed DMD patients. 35. 2% of DMD registrants in the database were treated with corticosteroids at their first clinical outpatients,compared to the current situation where 57. 1% are using corticosteroids. The age of loss of ambulation in non-corticosteroids treated group is（ 10. 50 ± 0. 37） years old,and（ 11. 88 ± 0. 33）years old in corticosteroid treated group. Between age 7. 0-11. 9,94. 2% of corticosteroid treated group are capable of walking. Their6-minute-walking-distance performances are（ 329. 00 ± 11. 48） meters,and the Gowers＇ time performances are（ 9. 84 ± 0. 71） seconds. In contrast,76. 5% of non-corticosteroids treated group have the capability to walk and achieve（ 126. 3 ± 1. 09） meters in the 6-minute-walking-distance test. The mean of their Gower＇s performance is（ 10. 02 ± 1. 41） seconds. 46. 6% of corticosteroid treated group are capable of walking after 12 years old,compared to 12. 2% in non-corticosteroids treated group. Conclusions Our data confirms the benefits of glucocorticoids treatment for motor function of DMD patients. Standardized registration and multidisciplinary diagnosis are of great significance for Duchenne muscular dystrophy.

关 键 词：DUCHENNE型肌营养不良 激素 注册登记 

分 类 号：R746.2[医药卫生—神经病学与精神病学]