诺西那生钠注射液治疗儿童脊髓性肌萎缩症疗效观察

Efficacy of Nusinersen Sodium Injection in the Treatment of Children with Spinal Muscular Atrophy

目的:探讨脊髓性肌萎缩症(SMA)患儿应用诺西那生钠注射液的临床疗效及安全性。方法:选取2022年1-6月于我院接受诺西那生钠鞘内注射治疗的SMA患儿26例,于治疗当天(T0)、第14天(T1)、第28天(T2)及第63天(T3)评估并比较Hammersmith扩展功能运动量表(HFMSE)、上肢模块修订版(RULM)、6分钟步行试验(6MWT)及不良反应发生情况。结果:与T0比较,T3时HFMSE评分升高[(20.69±14.64)分vs.(23.38±15.69)分,P<0.01]。与T0比较,T3时RULM评分升高[(18.27±10.15)分vs.(19.88±10.21)分,P<0.05]。结论:SMA患儿应用诺西那生钠注射液治疗后,HFMSE和RULM评分显著提高,近端肢体运动功能明显改善,且无明显不良反应发生。

Objective:To probe into the clinical efficacy and safety of norcyclosporine sodium injection in the treatment of children with spinal muscular atrophy(SMA).Methods:From Jan.2022 to Jun.2022,26 children with SMA who received intrathecal injection of nusinersen sodium in our hospital were enrolled.Hammersmith Functional Motor Scale Expanded(HFMSE)scores,Revised Upper Limb Module(RULM),and 6-Minute Walk Test(6MWT)and adverse drug reactions were evaluated and compared on the day of treatment(T0),the 14^(th) day of treatment(T1),the 28^(th) day of treatment(T2),and the 63^(rd) day of treatment(T3).Results:Compared with T0,HFMSE scores at T3 was higher((20.69±14.64)points vs.(23.38±15.69)points,P<0.01).Compared with T0,RULM scores at T3 was higher((18.27±10.15)points vs.(19.88±10.21)points,P<0.05).Conclusion:The HFMSE and RULM scores and motor function of proximal limb are significantly improved after treatment with nocinazone sodium injection in children with SMA.