PDF(Pubmed)
Abstract：
This study aims to collect and analyze adverse event (AE) reports related to Nusinersen from the FAERS database. The study employed a combination of signal quantification techniques, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS), to enhance the accuracy of signal detection and reduce the risk of false positives or negatives. Between the first quarter of 2017 and the third quarter of 2023, the FAERS database collected a total of 11,485,105 drug AE reports, of which 5772 were related to Nusinersen. Through signal mining analysis, 218 preferred term (PT) signals involving 27 system organ classes (SOCs) were identified. The study discovered AEs related to metabolism and nutrition disorders, psychiatric disorders, and cardiac disorders SOCs, which were not mentioned in the product information. Additionally, complications directly related to the intrathecal administration of Nusinersen, such as increased CSF pressure, positive CSF red blood cell count, and AEs related to the method of drug use, such as neuromuscular scoliosis and cerebrospinal fluid reservoir placement, were highlighted. Notably, AEs related to renal function abnormalities, such as abnormal Urine protein/creatinine ratio and protein urine presence, showed higher frequency and signal strength. The findings of this study emphasize the importance of comprehensive safety monitoring in the clinical application of Nusinersen. These results are significant for guiding future clinical practices, improving disease management strategies, and developing safer treatment protocols.
摘要：
本研究旨在从FAERS数据库中收集和分析与Nusinersen相关的不良事件（AE）报告。这项研究采用了信号量化技术的组合，包括报告赔率比(ROR)，比例报告比率(PRR)，贝叶斯置信传播神经网络(BCPNN)，和多项目伽玛泊松收缩器(MGPS)，以提高信号检测的准确性并降低假阳性或假阴性的风险。在2017年第一季度至2023年第三季度之间，FAERS数据库共收集了11,485,105份药物AE报告，其中5772人与Nusinersen有关。通过信号挖掘分析,确定了218个首选术语（PT）信号，涉及27个系统器官类别（SOC）。该研究发现了与代谢和营养失调相关的AE，精神疾病,和心脏疾病SOCs，产品信息中没有提到。此外,与鞘内注射Nusinersen直接相关的并发症，如脑脊液压力增加，脑脊液红细胞计数阳性，以及与药物使用方法相关的不良事件，如神经肌肉脊柱侧凸和脑脊液储液器放置，被突出显示。值得注意的是,与肾功能异常相关的AEs，如尿蛋白/肌酐比值异常和尿蛋白的存在，显示更高的频率和信号强度。这项研究的结果强调了全面安全监测在Nusinersen临床应用中的重要性。这些结果对于指导未来的临床实践具有重要意义。改善疾病管理策略，并制定更安全的治疗方案。
