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Effect of glucocorticoids on motor function of Duchenne muscular dystrophy |
ZHANG Shu,WANG Dan,ZHANG Hao,MA Peipei, WU Shiwen. |
Department of Neurology,General Hospital of Chinese People’s Armed Police Force, Beijing 100039,China |
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Abstract 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. Their 6-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.
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Received: 11 February 2018
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[1] |
Darras B T, Miller D T, Urion D K. Dystrophinopathies[M].Seattle (WA): University of Washington,1993:2018.
|
[2] |
Bello L, Morgenroth L P, Gordish-Dressman H, et al. DMD genotypes and loss of ambulation in the cinrg duchenne natural history study[J]. Neurology, 2016,87(4):401-409.
|
[3] |
Zubrzycka Gaarn E E, Bulman D E, Karpati G, et al. The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle[J]. Nature,1988, 333:466-469.
|
[4] |
Mendell J R, Shilling C, Leslie N D, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy[J]. Ann Neurol, 2012,71(3):304-313.
|
[5] |
Moat S J, Bradley D M, Salmon R, et al. Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK) [J]. Eur J Hum Genet,2013,21(10):1049-1053.
|
[6] |
McDonald C M, Henricson E K, Abresch R T,et al.CINRG Investigators. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study[J]. Lancet,2018,391(3):451-461.
|
[7] |
陈银红,王晓静,沈宏锐,等.杜氏肌营养不良和遗传咨询[J].临床荟萃,2013,28(5):590-592.
|
[8] |
Katharine B, Richard F, David J B, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care[J]. Lancet Neurol,2010,9: 177-189.
|
[9] |
Ryder S, Leadley R M, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review[J]. Orphanet J Rare Dis,2017,12:79.
|
[10] |
李西华,赵 蕾,胡超平,等.复旦大学附属儿科医院Duchenne型和Becker型肌营养不良症数据库的建立[J].中国现代神经疾病杂志,2015,15(5):360-368.
|
[11] |
Gloss D,Moxley R T,Ashual S, et al. Practice guideline update summary: corticosteroid treatment of Duchenne muscular dystrophy[J]. Neurology,2016,86:465-472.
|
[12] |
Bello L, Gordish-Dressman H, Morgenroth L P, et al Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study[J]. Neurology,2015,85(12):1048-1055.
|
[13] |
Matthews E, Brassington R, Kuntzer T, et al. Corticosteroids for the treatment of Duchenne muscular dystrophy[J]. Cochrane Database Syst Rev, 2016,5(5):CD003725.
|
[14] |
Spurney C, Shimizu R, Morgenroth L P, et al. International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy[J]. Muscle Nerve, 2014, 50:250-256.
|
[15] |
Nakamura H, Kimura E, Mori Yoshimura M, et al. Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy) [J]. Orphanet J Rare Dis, 2013, 8:60.
|
[16] |
Hoffman E P, Reeves E, Damsker J, et al. Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy[J]. Phys Med Rehabil Clin N Am,2012,23(4):821-828.
|
|
|
|