糖皮质激素治疗Duchenne型肌营养不良的Meta分析  

Glucocorticoids for Duchenne Muscular Dystrophy: A Meta-Analysis

作　　者：胡君[1] 蒋莉[2] 洪思琦[2] 孔敏[2] 叶园珍[2] 

机构地区：[1]福建医科大学附属协和医院儿科,福州350001 [2]重庆医科大学附属儿童医院神经内科,重庆400014

出　　处：《中国循证医学杂志》2014年第7期867-873,共7页Chinese Journal of Evidence-based Medicine

基　　金：福建省卫生厅青年科研课题(编号:2009-2-22);重庆市卫生局医学科研项目(编号:2012-1-044)

摘　　要：目的系统评价糖皮质激素(glucocorticoids,GC)治疗Duchenne型肌营养不良(Duchenne muscular dystrophy,DMD)的疗效和安全性。方法计算机检索PubMed、EMbase、CENTRAL、CNKI、WanFang Data、VIP和CBM数据库,检索时限均为从建库至2011年12月,纳入有关GC治疗DMD的RCT。由2名评价员按纳入与排除标准独立筛选文献、提取资料和评价质量后,采用RevMan 5.0.2软件进行Meta分析。结果共纳入6个RCT,合计303例患儿。Meta分析结果显示,GC治疗6个月后,试验组患儿的平均肌力、举重能力、用力肺活量、生存质量情感因子分和总分、Gowers’时间、步行9米时间、爬4层楼梯时间明显改善。但试验组患儿的体重增加、行为改变、食欲、库欣面容、多毛症发生情况增加显著,但未增加骨质疏松/骨折、高血压/糖尿病、白内障发生率。结论 GC可显著改善DMD患儿的肌力、肌肉功能及肺功能,延长独立行走时间,提高生存质量,但应需密切关注GC的不良反应。Objective To systematically review the therapeutic effects and safety of glucocorticoids （GC） for Duch- enne muscular dystrophy （DMD）. Methods Databases such as PubMed, EMbase, CENTRAL, CNKI, WanFang Data, VIP and CBM were electronically searched from the establishment of the databases till December 2011. All randomized controlled trials （RCTs） about GC for DMD were included. Two reviewers independently screened literature according to the inclusion and exclusion criteria, extracted data, and evaluated the methodological quality of the included studies. Then meta-analysis was performed using RevMan 5.0.2 software. Results A total of 6 studies involving 303 DMD children were included. The results of meta-analysis showed that after 6 month treatment of GC （deflazacort）, patients＇ symptoms were obviously improved in average muscle strength, lift weight ability, forced vital capacity （FVC） of the lung, emotional factor scores and total scores in Quality of life （QoL）, Gower＇s time, nine meters walking time （T9 m）, and four-stair climbing time （T4 s）. However, the trial group showed more weight gain, behavioural changes, increased appetite, cushingoid appearance, and excessive hair growth. The incidences of osteoporosis/fracture, hypertension, diabetes, and cataract were not increased. Conclusion GC could improve muscle strength and function, stabilize pulmonary function, prolong independent walk time, and improve QoL of DMD patients. However, adverse reaction caused by GC should be taken caution.

关 键 词：糖皮质激素 DUCHENNE型肌营养不良 疗效 安全性 META分析 随机对照试验 

分 类 号：R746.2[医药卫生—神经病学与精神病学]