用nusinersen治疗脊柱肌缩的儿科患者实验室参数的安全性分析
Xiaomei Zhu1, Hui Li2, Chaoping Hu1
1Department of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
BMC pediatrics
|July 25, 2024
概括
内 nusinersen 是安全的治疗儿科脊柱肌肉缩 (SMA). 实验室监测显示,在接受Nusinersen治疗SMA的儿童中,没有显著的安全问题.
科学领域:
- 神经学 神经学
- 儿科医学 儿科医学
- 药理学 药理学是指药理学的学科.
背景情况:
- 脊椎肌肉缩 (SMA) 是一种进展性神经退行性疾病.
- 内 nusinersen,一种反感性寡核酸,是SMA的治疗选择.
- 评估nusinersen在儿科患者的安全性对于治疗成功至关重要.
研究的目的:
- 评估在被诊断患有SMA的儿科患者中吸管内 nusinersen 的安全性.
- 分析实验室数据,并确定与儿童nusinersen治疗相关的任何不良影响.
主要方法:
- 从小儿SMA患者的实验室数据的回顾性分析治疗与nusinersen.
- 从2019年10月到2022年5月收集的数据.
- 监测各种实验室标记物,包括炎症标记物,国际正常化比率,尿素,细胞素C,血小板计数,激活的部分血时间,肌素,肝酶,脑脊液 (CSF) 白细胞计数和CSF总蛋白.
主要成果:
- 共有46名儿科患者接受了213剂努辛森,没有显著的安全问题.
- 观察到稳定的炎症标志物和CSF白细胞计数.
- 在每次注射的国际正常化比率,尿素,囊素C和CSF总蛋白质水平中观察到轻微的,不显著的变化.
结论:
- 努辛森疗法在患有SMA的儿科患者中显示出一般安全的特征.
- 常规实验室监测没有显示持续或显著异常的发现.
- 建议监测CSF蛋白质水平,以进一步了解nusinersen的影响.
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