| 摘要: |
| 目的:挖掘和评价索伐瑞韦(奥那赛诺基因注射液)上市后治疗脊髓性肌萎缩症潜在关联不良事件信号,为临床安全用 药提供参考。 方法:在美国食品药品监督管理局不良事件报告系统(FAERS)中提取索伐瑞韦自 2019 年 5 月至 2025 年 12 月以 其作为首要怀疑(PS)药物相关的不良事件,采用报告比值比(ROR)、成比例报告比值比( PRR)、多重伽马-泊松收缩估计 (MGPS)和贝叶斯置信区间神经网络传播(BCPNN)4 种方法进行信号挖掘,评估索伐瑞韦与不良事件的统计学关联。 在 4 种 算法中均达到阈值标准的不良事件被判定为风险信号。 结果:经数据清洗后,共检索到以索伐瑞韦作为 PS 药物的不良事件报 告 9 498 例次,涉及 2 132 例患者。 共挖掘到 188 个不良事件信号,涉及 12 个系统器官分类(SOC)。 按各 SOC 信号数及相应首 选术语(PT)报告数降次排序取前 5 位,发现 21 个未收载于药品说明书的信号,其中被识别为具有临床中优先级信号的 PT 有 6 个。 按 报告者类别进行分层,发现需医学确诊的不良事件主要由专业人员报告,症状直观的事件主要由非专业人员报告。 索伐瑞韦潜 在关联不良事件发生中位时间为 6(2,32)d。 结论:除索伐瑞韦说明书提及不良反应外,还应密切关注肾脏和泌尿系统疾病、心 脏器官疾病、代谢及营养类疾病、精神系统类疾病等不良反应,建议针对索伐瑞韦的特异性风险实施相应监测与针对性管理。 |
| 关键词: 索伐瑞韦 脊髓性肌萎缩症 美国食品药品监督管理局不良事件报告系统 上市后研究 |
| DOI：10.13407/j.cnki.jpp.1672-108X.2026.09.014 |
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| 基金项目:中国儿科人群临床试验体系建设项目,编号 2017ZX09304029-001-001。 |
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| Post-Marketing Safety Signal Evaluation of Sovaprevir in the Treatment of Spinal Muscular Atrophy |
| Zhang Guandong, Yang Yu, Zhao Ruiling |
| (Children’s Hospital of Shanxi, Taiyuan 030013, China) |
| Abstract: |
| Objective: To mine and evaluate potential adverse event signals associated with post-marketing sovaprevir (onasemnogene abeparvovec) in the treatment of spinal muscular atrophy, and to provide reference for clinical safe medication. Methods: Adverse event reports with sovaprevir as the primary suspect (PS) drug was extracted from the U. S. Food and Drug Administration Adverse Event Reporting System (FAERS) database from May 2019 to Dec. 2025. Reporting odds ratio (ROR) method, proportional reporting ratio ( PRR) method, multi-item Gamma-Poisson shrinker ( MGPS) method, and Bayesian confidence propagation neural network (BCPNN) method were employed for signal mining. The statistical correlation between sovaprevir and adverse events were evaluated. The adverse events that met the threshold criteria in all four algorithms were determined as risk signals. Results: After data cleaning, a total of 9,498 adverse event reports with sovaprevir as the PS drug were retrieved from the U. S. FAERS database, including 2,132 cases. Totally 188 adverse event signals were identified across 12 system organ classes ( SOC). According to the descending order of the number of SOC signals and the corresponding preferred term (PT) reports, the top 5 reports were selected, and 21 signals not included in the drug instructions were found, of which 6 PT reports were classified as moderate-priority clinical signals. A stratified analysis by reporter category revealed that adverse events requiring medical confirmation were mostly reported by professionals, while events with overt symptoms were mainly reported by non-professionals. The median time to onset of sovaprevir-related adverse events was 6 (2, 32) d. Conclusion: In addition to the adverse drug reactions mentioned in the drug instructions of sovaprevir, attention should be paid to adverse drug reactions related to kidney and urinary system diseases, cardiac diseases, metabolic and nutritional disorders, and nervous system disorders. It is recommended to implement corresponding monitoring and targeted management based on the specific risks of sovaprevir. |
| Key words: sovaprevir spinal muscular atrophy the U. S. Food and Drug Administration Adverse Event Reporting System post- marketing study |
