中国药业2025，Vol.34Issue(4)：99-103,5.DOI:10.3969/j.issn.1006-4931.2025.04.024
诺西那生钠用于脊髓性肌萎缩症临床安全性分析
Clinical Safety Analysis of Nusinersen for Spinal Muscular Atrophy
摘要
Abstract
Objective To provide a reference for the safe clinical use of nusinersen.Methods Clinical data of children with spinal muscular atrophy(SMA)admitted to the pediatric department of the hospital from January 2022 to December 2023 were collected,the laboratory parameters were compared with baseline data after treatment with nusinersen,and the occurrence of adverse drug reactions during the treatment process were recorded.Results A total of 27 children with SMA were included,130 case times of treatment were involved,with(4.80±2.40)case times for each case.There were two cases(7.40%),15 cases(55.56%)and 10 cases(37.04%)of SMA types 1,2,3 respectively.The average age of symptom onset was(32.89±42.48)months,the median age of diagnosis was 24.00(12.00,84.00)months,the average age of first diagnosis was(31.85±36.32)months.SMA type 2 had the longest interval of(37.40±40.12)months from diagnosis to treatment.SMA type 3 had the highest median age of initial treatment[102.00(78.00,141.00)]months,followed by SMA type 2[48.00(12.00,96.00)]and SMA type 1[14.00(3.00,18.00)].There were 24 cases(88.89%)of homozygous deletion of exons 7 and 8 of survival motor neuron gene 1(SMN1),and three cases(11.11%)of mutation of exon 7 of SMN1.Compared with baseline data,the cerebrospinal fluid(CSF)protein levels on the days 14,28,63,and months 6,10 were significantly different in children receiving treatment with nusinersen(P<0.05);while the CSF glucose was significantly different only when receiving the third dose of nusinersen,and the CSF chloride,red blood cells,international normalized ratio,and activated partial thromboplastin time were significantly different only when receiving the sixth dose of nusinersen(P<0.05).Six children experienced adverse drug reactions,mainly manifested as cough,increased transaminase levels,abdominal pain,fever,bronchopneumonia,recurrent vomiting complicated with lower back pain,which improved after symptomatic treatment;and no serious adverse drug reaction occurred during the follow-up visits.Conclusion Nusinersen can significantly affect the CSF protein levels in children with SMA,while further follow-up observation is needed to determine whether there is a sustained effect on CSF glucose,chloride,red blood cells,international normalized ratio,and activated partial thromboplastin time.关键词
脊髓性肌萎缩症/诺西那生钠/实验室参数/药品不良反应Key words
spinal muscular atrophy/nusinersen/laboratory parameter/adverse reaction分类
医药卫生引用本文复制引用
汪小英,张旭,武东,杨振峰,汪晓娟..诺西那生钠用于脊髓性肌萎缩症临床安全性分析[J].中国药业,2025,34(4):99-103,5.基金项目
安徽省阜阳市卫生健康科研项目[FY2023-141]. （）
