PDF(Pubmed)
Abstract：
BACKGROUND: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that can be treated with intrathecal nusinersen, an antisense oligonucleotide. In addition to efficacy, safety is a determining factor in the success of any therapy. Here, we aim to assess the safety of nusinersen therapy in paediatric patients with SMA.
METHODS: Laboratory data of paediatric patients with SMA who received nusinersen between October 2019 and May 2022 were retrospectively analysed.
RESULTS: During the observation period, 46 infants and children aged 2.9 months to 13.6 years received a total of 213 nusinersen doses without safety concerns. Inflammatory markers were stable throughout the study. International normalized ratio was increased by 0.09 per injection. Urea levels were increased by 0.108 mmol/L, and cystatin C decreased by 0.029 mg/L per injection. There were no significant changes in platelet count, activated partial thrombin time, creatinine levels or liver enzyme levels during treatment. The cerebrospinal fluid (CSF) leukocyte count remained stable, and total protein increased by 24.038 mg/L per injection.
CONCLUSIONS: Our data showed that nusinersen therapy is generally safe in children with SMA. Laboratory monitoring did not identify any persistent or significantly abnormal findings. CSF protein should be monitored to gain more insights.
METHODS: Laboratory data of paediatric patients with SMA who received nusinersen between October 2019 and May 2022 were retrospectively analysed.
RESULTS: During the observation period, 46 infants and children aged 2.9 months to 13.6 years received a total of 213 nusinersen doses without safety concerns. Inflammatory markers were stable throughout the study. International normalized ratio was increased by 0.09 per injection. Urea levels were increased by 0.108 mmol/L, and cystatin C decreased by 0.029 mg/L per injection. There were no significant changes in platelet count, activated partial thrombin time, creatinine levels or liver enzyme levels during treatment. The cerebrospinal fluid (CSF) leukocyte count remained stable, and total protein increased by 24.038 mg/L per injection.
CONCLUSIONS: Our data showed that nusinersen therapy is generally safe in children with SMA. Laboratory monitoring did not identify any persistent or significantly abnormal findings. CSF protein should be monitored to gain more insights.
摘要：
背景：脊髓性肌萎缩症（SMA）是一种进行性神经退行性疾病，可以通过鞘内注射nusinersen进行治疗，反义寡核苷酸。除了功效,安全性是任何治疗成功与否的决定因素.这里,我们旨在评估nusinersen治疗小儿SMA患者的安全性.
方法：回顾性分析了2019年10月至2022年5月期间接受nusinersen治疗的SMA儿科患者的实验室数据。
结果：在观察期间，46名2.9个月至13.6岁的婴儿和儿童总共接受了213次nusinersen剂量，没有安全问题。炎症标志物在整个研究中是稳定的。国际标准化比率每次注射增加0.09。尿素水平增加了0.108mmol/L，胱抑素C每注射减少0.029mg/L。血小板计数无明显变化,活化部分凝血酶时间,治疗期间肌酐水平或肝酶水平。脑脊液（CSF）白细胞计数保持稳定，每次注射总蛋白增加24.038mg/L。
结论：我们的数据表明，nusinersen治疗对SMA患儿通常是安全的。实验室监测未发现任何持续或明显的异常发现。应监测CSF蛋白以获得更多见解。
方法：回顾性分析了2019年10月至2022年5月期间接受nusinersen治疗的SMA儿科患者的实验室数据。
结果：在观察期间，46名2.9个月至13.6岁的婴儿和儿童总共接受了213次nusinersen剂量，没有安全问题。炎症标志物在整个研究中是稳定的。国际标准化比率每次注射增加0.09。尿素水平增加了0.108mmol/L，胱抑素C每注射减少0.029mg/L。血小板计数无明显变化,活化部分凝血酶时间,治疗期间肌酐水平或肝酶水平。脑脊液（CSF）白细胞计数保持稳定，每次注射总蛋白增加24.038mg/L。
结论：我们的数据表明，nusinersen治疗对SMA患儿通常是安全的。实验室监测未发现任何持续或明显的异常发现。应监测CSF蛋白以获得更多见解。
