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

作　　者：汪小英 张旭 武东 杨振峰 汪晓娟 WANG Xiaoying;ZHANG Xu;WU Dong;YANG Zhenfeng;WANG Xiaojuan(Fuyang People's Hospital,Fuyang,Anhui,China 236000)

机构地区：[1]安徽省阜阳市人民医院,安徽阜阳236000

出　　处：《中国药业》2025年第4期99-103,共5页China Pharmaceuticals

基　　金：安徽省阜阳市卫生健康科研项目[FY2023-141]。

摘　　要：目的为诺西那生钠的临床安全用药提供参考。方法收集医院儿科2022年1月至2023年12月收治的脊髓性肌萎缩症(SMA)患儿的临床资料,对比诺西那生钠治疗后的实验室参数与基线值,记录治疗过程中药品不良反应的发生情况。结果纳入SMA患儿27例,共治疗130例次,例均(4.80±2.40)例次。SMA 1型、SMA 2型和SMA 3型患儿分别为2例(7.40%)、15例(55.56%)和10例(37.04%)。出现症状的平均年龄为(32.89±42.48)月龄,中位确诊年龄为24.00(12.00,84.00)月龄,平均确诊时长为(31.85±36.32)月龄。SMA 2型患儿自确诊到接受治疗间隔时间最长,为(37.40±40.12)月龄。SMA 3型患儿中位初始治疗年龄最大,为102.00(78.00,141.00)月龄,其次为SMA 2型[48.00(12.00,96.00)]和SMA 1型[14.00(3.00,18.00)]。运动神经元存活基因1(SMN1)第7、8号外显子纯合缺失24例(88.89%),SMN1基因7号外显子基因突变3例(11.11%)。与基线值相比,接受诺西那生钠治疗后患儿的脑脊液(CSF)蛋白在第14天、第28天、第63天、第6个月、第10个月时差异有统计学意义(P<0.05),而CSF葡萄糖仅在第3剂次时,CSF氯、血红细胞、国际标准化比值、活化部分凝血活酶时间仅在第6剂次时差异有统计学意义(P<0.05)。6例患儿出现药品不良反应,主要表现为咳嗽、转氨酶升高、腹痛、发热、支气管肺炎、反复呕吐伴腰部酸痛,予对症治疗后好转,随访期间未发生严重药品不良反应。结论诺西那生钠可显著影响SMA患儿CSF中蛋白水平,但CSF中葡萄糖、氯,血红细胞,国际标准化比值,活化部分凝血活酶时间是否产生持续影响仍需进一步的随访观察。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 N

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 

分 类 号：R969.4[医药卫生—药理学]