中国当代儿科杂志2024，Vol.26Issue(7)：743-749,7.DOI:10.7499/j.issn.1008-8830.2401082
诺西那生钠治疗脊髓性肌萎缩症儿童的临床分析
Clinical efficacy of nusinersen sodium in the treatment of children with spinal muscular atrophy
郭瑾 1武运红 2张临霞 2纪惠茹 1周娜 1胡晓月1
作者信息
- 1. 山西医科大学儿科医学系,山西太原 030000
- 2. 山西省儿童医院神经内科,山西太原 030000
- 折叠
摘要
Abstract
Objective To investigate the efficacy and safety of nusinersen sodium in the treatment of children with spinal muscular atrophy(SMA).Methods A retrospective analysis was conducted on the clinical data of 50 children with 5q SMA who received nusinersen sodium treatment and multidisciplinary treatment management in Shanxi Children's Hospital from February 2022 to February 2024.Results Compared with the baseline data,67%(8/12),74%(35/47),and 74%(35/47)of the SMA children had a clinically significant improvement in the scores of Philadelphia Infant Test of Neuromuscular Disorders,Hammersmith Functional Motor Scale Expanded,and Revised Upper Limb Module,respectively,and the distance of 6-minute walking test increased from 207.00(179.00,281.50)meters to 233.00(205.25,287.50)meters(P<0.05)after nusinersen sodium treatment.Of all 50 children with SMA,24(48%)showed good tolerability after administration,with no significant or persistent abnormalities observed in 2 034 laboratory test results,and furthermore,there were no serious or immunological adverse events related to the treatment.After treatment,there was a significant change in forced vital capacity as a percentage of the predicted value in 27 children with restrictive ventilatory dysfunction,as well as a significant change in the level of 25-(OH)vitamin D in 15 children with vitamin D deficiency(P<0.05).Conclusions For children with SMA,treatment with nusinersen sodium can continuously improve the response rates of motor function scales,with good tolerability and safety.关键词
脊髓性肌萎缩症/诺西那生钠/疗效/安全性/儿童Key words
Spinal muscular atrophy/Nusinersen sodium/Efficacy/Safety/Child引用本文复制引用
郭瑾,武运红,张临霞,纪惠茹,周娜,胡晓月..诺西那生钠治疗脊髓性肌萎缩症儿童的临床分析[J].中国当代儿科杂志,2024,26(7):743-749,7.
