伐莫洛酮治疗Duchenne型肌营养不良症的研究进展  

Research progress of vamorolone in the treatment of Duchenne muscular dystrophy

作　　者：王琴 焦琳娜[1] 何建丽 宋志彬[1] WANG Qin;JIAO Linna;HE Jianli;SONG Zhibin(Zhongshan Xiaolan People’s Hospital,Zhongshan 528415,China)

机构地区：[1]中山市小榄人民医院,广东中山528415

出　　处：《中国实用神经疾病杂志》2024年第1期119-123,共5页Chinese Journal of Practical Nervous Diseases

基　　金：中山市医学科研项目(编号:2021A020318)。

摘　　要：Duchenne型肌营养不良症(DMD)是进行肌营养不良症的最常见类型,也是中国最常见的X连锁隐性遗传的肌病,发病率约30/10万男婴,主要表现为缓慢进行性加重的躯干肌肉及肢体肌肉无力及萎缩、肌肉的假性肥大,大多数还会有心肌损害,一般患儿在12岁左右不能行走,后期会出现呼吸肌无力,由于DMD目前无特异性治疗方法,多数患者在20~30岁因呼吸道感染、心力衰竭而死亡。糖皮质激素对DMD运动和心功能有明显改善作用,目前临床建议早期使用,但糖皮质激素存在较多的不良反应,临床应用有较多限制。近十余年国外临床研究发现伐莫洛酮对DMD有同糖皮质激素一样的疗效,但没有与糖皮质激素相关的不良反应(生长发育迟缓、骨疾病、肌肉萎缩等)。本文就目前关于伐莫洛酮治疗DMD的作用机制、治疗方案及不良反应做一总结。Duchenne muscular dystrophy(DMD),which is the most common type of progressive muscular dystrophy(PMD),is also the most common X-linked recessive myopathy in China.The incidence rate of about 30/100000 male infants,the main performances of DMD are slowly aggravated trunk muscles,limb muscles weakness and atrophy,false hypertrophy of the muscles,most will be accompanied by myocardial damage.General children at 12 years old can not walk,later will appear respiratory muscle weakness.Because of no characteristic treatment,most patients with DMD in 20-30 years old will be death due to respiratory tract infection and heart failure.Many studies have found that glucocorticoids can significantly improve motor and cardiac function in DMD.Currently,early use of glucocorticoids is recommended in clinic,but clinical application of glucocorticoids is more limited because of the adverse reactions.In recent 10 years,foreign clinical studies have found that vamorolone has the same therapeutic effect as glucocorticoid in the treatment of DMD,but lack of the adverse effects associated with glucocorticoid(growth retarding,bone disease,muscle atrophy).This article reviews the mechanism,treatment and side effects of vamorolone in the treatment of DMD.

关 键 词：DUCHENNE型肌营养不良症 伐莫洛酮 糖皮质激素 新型抗炎药游离甾体化合物 

分 类 号：R746[医药卫生—神经病学与精神病学]