| 摘要: |
| 目的:探讨诺西那生钠治疗脊髓性肌萎缩症(SMA) 的实验室指标变化情况及用药安全性。方法:收集2022 年1 月至
2024 年4 月于潍坊市人民医院小儿内科就诊的15 例诺西那生钠治疗的SMA 患儿。回顾性分析患儿的基因检测结果、疾病分
型、临床表现等资料,于治疗第0、14、28、63 天鞘内注射诺西那生钠5 mL/ 12 mg,此后每4 个月给予1 次维持治疗。监测每次给
药前患儿血常规、肝肾功能、心肌酶谱、脑脊液、凝血功能与尿常规等实验室检查结果,比较不良反应发生率,评价用药安全性。
结果:共15 例患儿确诊为SMA,其中SMA 1 型2 例,SMA 2 型11 例,SMA 3 型2 例。15 例SMA 患儿均完成治疗2 个月的评估,
7 例患儿完成治疗26 个月的评估。治疗期间,主要不良反应依次为心肌酶谱异常、脑脊液异常、肝肾功能异常和血常规异常,
少数患儿发生电解质紊乱,未检出凝血功能障碍及尿蛋白异常。实验室检查异常例数占总使用例数在各时间点的差异有统计
学意义(P<0. 05)。结论:诺西那生钠治疗SMA 具有良好的安全性。 |
| 关键词: 脊髓性肌萎缩症 诺西那生钠 药物不良反应 安全性 儿童 |
| DOI：doi:10.13407/j.cnki.jpp.1672-108X.2025.10.013 |
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| 基金项目: |
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| Safety Evaluation of Nusinersen in the Treatment of Children with Spinal Muscular Atrophy |
| Liu Fuhui1, Li Jinlei2, Yu Baolong3, Lyu Zufang3, Li Minglei3 |
| ((1. School of Clinical Medicine, Shandong Second Medical
University, Shandong Weifang 261000, China; 2. Weifang People’s Hospital, Shandong Weifang 261041, China)) |
| Abstract: |
| Objective: To explore the changes in laboratory indicators and medication safety of nusinersen in the treatment of spinal
muscular atrophy (SMA). Methods: Retrospectively analysis was performed on data of 15 SMA patients admitted into the Pediatric
Internal Medicine Department of Weifang People’s Hospital from Jan. 2022 to Apr. 2024. Genetic test results, disease classification,
and clinical manifestations were summarized and analyzed. Nosinasen sodium (5 mL/ 12 mg) was injected intrathecally on the 0, 14th ,
28th and 63rd d of treatment, and maintenance therapy was given every 4 months thereafter. The blood routine, liver function, renal
function, myocardial enzyme spectrum, cerebrospinal fluid, coagulation function, and urine routine were monitored before each
administration. The incidence of adverse drug reactions was compared and medication safety was evaluated. Results: A total of 15
children were diagnosed with SMA, including 2 cases of SMA 1, 11 cases of SMA 2, and 2 cases of SMA 3. All 15 children with SMA
completed the 2-month evaluation of treatment, and 7 children completed the 26-month evaluation of treatment. During treatment, the
main adverse drug reactions were abnormal myocardial enzyme spectrum, abnormal cerebrospinal fluid, abnormal hepatic and renal
function, and abnormal blood routine. A few cases presented with electrolyte imbalance, but no detection of coagulation dysfunction or
abnormal urine protein. The difference in the number of abnormal laboratory tests among all patients was statistically significant at each
time point (P<0. 05). Conclusion: Nusinersen is safe in the treatment of children with SMA. |
| Key words: spinal muscular atrophy nusinersen adverse drug reaction safety children |
