Glucocorticoid corticosteroids for Duchenne muscular dystrophy

德菲扎科特 医学 杜氏肌营养不良 神经肌肉疾病 物理疗法 肌营养不良 浪费的 强的松 梅德林 儿科 荟萃分析 疾病 内科学 政治学 法学
作者
Adnan Y. Manzur,Thierry Küntzer,Mike Pike,A V Swan
出处
期刊:Cochrane Database of Systematic Reviews [Cochrane]
被引量:400
标识
DOI:10.1002/14651858.cd003725.pub3
摘要

Background Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. This incurable disease is characterised by muscle wasting and loss of walking ability leading to complete wheelchair dependence by 13 years of age. Prolongation of walking is one of the major aims of treatment. Objectives The aim of this review was to assess whether glucocorticoid corticosteroids stabilize or improve muscle strength and walking in boys with DMD. Search methods This is an update of the Cochrane systematic review first published in 2004 (Manzur 2004). We searched the Cochrane Neuromuscular Disease Group Trials Register (August 2006) using the term 'Duchenne muscular dystrophy'. We also searched MEDLINE (January 1966 to July 2007), EMBASE (January 1980 to August 2006), CINAHL and LILACS (January 1982 to August 2006). We wrote to authors of published studies and other experts in this disease to help identify other trials, checked the references in the identified trials and hand searched the abstracts of relevant journals. Selection criteria Types of studies: randomised or quasi‐randomised trials. Types of participants: all patients with a definite diagnosis of Duchenne muscular dystrophy. Types of interventions: glucocorticoids such as prednisone, prednisolone, deflazacort or others, with a minimum treatment period of three months. Primary outcome measure: prolongation of walking (independent walking without long leg calipers). Secondary outcome measures: strength outcome measures, manual muscle strength testing using Medical Research Council strength scores, functional outcome measures and adverse events. Data collection and analysis We identified six randomised controlled trials that met the inclusion criteria for our review, and one of these (Beenakker 2005) is a new addition to this update, as it was published subsequent to our first review (Manzur 2004). Two review authors independently selected the trials for the review and assessed methodological quality. Data extraction and inputting were double‐checked. Main results Primary outcome measure: data from one small study used prolongation of walking as an outcome measure and did not show significant benefit. Secondary outcome measures: The meta‐analysis of the results from four randomised controlled trials with altogether 249 participants showed that glucocorticoid corticosteroids improved muscle strength and function over six months. Improvements were seen in time taken to rise from the floor (Gowers' time), nine metres walking time, four‐stair climbing time, ability to lift weights, leg function grade and forced vital capacity. One randomised controlled trial with altogether 28 participants showed that glucocorticoid corticosteroids stabilize muscle strength and function for up to two years. The most effective prednisolone regime appears to be 0.75 mg/kg/day, given in a daily dose regime. Not enough data were available to compare efficacy of prednisone with deflazacort. Adverse effects: Excessive weight gain, behavioural abnormalities, cushingoid appearance and excessive hair growth were all more common with glucocorticoid corticosteroids than placebo. Long‐term adverse effects of glucocorticoid therapy could not be evaluated because of the short‐term duration of the randomised studies. Non‐randomised studies: A number of non‐randomised studies with important efficacy and adverse effects data are tabulated and discussed. Authors' conclusions There is evidence from randomised controlled studies that glucocorticoid corticosteroid therapy in Duchenne muscular dystrophy improves muscle strength and function in the short‐term (six months to two years). The most effective prednisolone regime appears to be 0.75 mg/kg/day, given daily. In the short term, adverse effects were significantly more common but not clinically severe. Long‐term benefits and hazards of glucocorticoid treatment cannot be evaluated from the currently published randomised studies. Non‐randomised studies support the conclusions of functional benefits but also identify clinically significant adverse effects of long‐term treatment. These benefits and adverse effects have implications for future research studies and clinical practice.
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
ZZZZ发布了新的文献求助10
1秒前
Struggle完成签到 ,获得积分10
1秒前
百地希留耶完成签到 ,获得积分10
1秒前
852应助眼睛大的笑阳采纳,获得10
2秒前
汉堡包应助xlbn采纳,获得10
4秒前
英姑应助lw采纳,获得10
6秒前
8秒前
潇湘雪月完成签到,获得积分10
10秒前
科研通AI5应助ZZZZ采纳,获得10
12秒前
TT发布了新的文献求助10
14秒前
合适成风发布了新的文献求助10
14秒前
无情的土豆完成签到,获得积分10
15秒前
李健应助mmr采纳,获得10
16秒前
18秒前
量子星尘发布了新的文献求助10
19秒前
落后紫夏完成签到,获得积分10
19秒前
小任吃不胖完成签到,获得积分10
19秒前
22秒前
自觉南风发布了新的文献求助10
22秒前
ppg123应助阳光向秋采纳,获得10
24秒前
24秒前
酷波er应助长耳尾采纳,获得10
25秒前
25秒前
松鼠15111完成签到,获得积分10
25秒前
脑洞疼应助路宝采纳,获得10
26秒前
初留言完成签到,获得积分10
28秒前
29秒前
YNYang完成签到,获得积分10
30秒前
mmr发布了新的文献求助10
30秒前
CipherSage应助yu采纳,获得10
31秒前
32秒前
哎呀妈呀完成签到,获得积分10
32秒前
LALALADDDD完成签到,获得积分10
33秒前
清新的雁凡完成签到,获得积分10
34秒前
贪玩的傲菡完成签到 ,获得积分10
35秒前
水菜泽子完成签到,获得积分10
36秒前
合适成风完成签到,获得积分20
36秒前
科研通AI2S应助猪猪侠采纳,获得30
37秒前
Acid完成签到 ,获得积分10
38秒前
LALALADDDD发布了新的文献求助150
38秒前
高分求助中
Picture Books with Same-sex Parented Families: Unintentional Censorship 1000
A new approach to the extrapolation of accelerated life test data 1000
ACSM’s Guidelines for Exercise Testing and Prescription, 12th edition 500
Nucleophilic substitution in azasydnone-modified dinitroanisoles 500
不知道标题是什么 500
Indomethacinのヒトにおける経皮吸収 400
Phylogenetic study of the order Polydesmida (Myriapoda: Diplopoda) 370
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 3977792
求助须知:如何正确求助?哪些是违规求助? 3521968
关于积分的说明 11210815
捐赠科研通 3259135
什么是DOI,文献DOI怎么找? 1799535
邀请新用户注册赠送积分活动 878412
科研通“疑难数据库(出版商)”最低求助积分说明 806888