■Eteplirsen(Exondys51)是一种被批准用于治疗Duchenne肌营养不良(DMD)的孤儿药物,在2016年获得美国食品和药物管理局(FDA)的加速批准。这项研究的主要目的是密切监测与eteplirsen相关的不良事件(AE),并识别新出现的信号以更好地表征其安全性。
■从2016年第三季度(Q3)到2023年第四季度(Q4)报告的eteplirsen使用不良事件从FDA不良事件报告系统(FAERS)收集。AE的role_code主要包括主要嫌疑人(PS),次要嫌疑人(SS),伴随(C),互动(I)。本研究的目标是以PS为role_cod的报告。\'根据FDA的重复数据删除规则,当CASEID相同时,选择最新的FDA_DT,并且当CASEID和FDA_DT相同时,选择较高的PRIMARYID。不相称性分析,包括四种报告赔率比(ROR)的算法,比例报告比率(PRR),贝叶斯配置提升神经网络(BCPNN),和多项目伽玛泊松收缩器(MGPS),用于量化与eteplirsen相关的AE信号。
■来自FAERS数据库,在整个研究期间,共收集了13,205,369份报告.在根除重复之后,将eteplirsen指定为PS的报告数量达1480例,涵盖25个器官系统.其中,“一般疾病和给药地点条件,\"\"伤害,中毒,和手术并发症,\"\"呼吸,胸廓,纵隔疾病,“\”感染和感染,血管疾病,\"和\"产品问题\"至少满足四个计算标准之一。此外,55个与规定算法一致的首选术语(PT)。这些患者中AE的中位时间为903天,四分位距(IQR)为269-1575天。此外,70.04%的AE在治疗开始后一年或更长时间出现。
■作为加速批准的孤儿药,我们的研究证实了众所周知的药物不良反应,并确定了与eteplirsen治疗相关的潜在安全性问题.这有助于更深入地了解不良反应与eteplirsen的使用之间的复杂相互关系。研究结果强调了持续监测和持续观察对及时发现和有效管理不良事件至关重要。从而提高了用eteplirsen治疗DMD的患者的整体安全性和幸福感。
UNASSIGNED: Eteplirsen (Exondys 51) is an orphan drug approved for the treatment of Duchenne muscular dystrophy (DMD), having received accelerated approval from the U.S. Food and Drug Administration (FDA) in 2016. The primary aim of this study is to closely monitor adverse events (AEs) associated with eteplirsen and to identify emerging signals to better characterize their safety profile.
UNASSIGNED: AEs due to eteplirsen usage reported from the third quarter (Q3) of 2016 to the fourth quarter (Q4) of 2023 were collected from the FDA Adverse Event Reporting System (FAERS). The role_code of AEs mainly includes primary suspect (PS), secondary suspect (SS), concomitant (C), and interaction (I). This study targeted reports with a role_cod of \'PS.\' According to the FDA deduplication rule, the latest FDA_DT is selected when the CASEID is the same, and the higher PRIMARYID is selected when the CASEID and FDA_DT are the same. Disproportionality analyses, encompassing four algorithms for reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian configuration promotion neural network (BCPNN), and multi-item gamma Poisson shrinker (MGPS), were utilized to quantify the signals of AEs associated with eteplirsen.
UNASSIGNED: From the FAERS database, a total of 13,205,369 reports were amassed throughout the study duration. Following the eradication of duplicates, the number of reports with eteplirsen designated as the PS amounted to 1480 encompassed 25 organ systems. Among these, \"general disorders and administration site conditions,\" \"injury, poisoning, and procedural complications,\" \"respiratory, thoracic, and mediastinal disorders,\" \"infections and infestations,\" \"vascular disorders,\" and \"product issues\" met at least one of the four computational criteria. Additionally, 55 Preferred Terms (PTs) aligned with the prescribed algorithms. The median time to AEs in these patients was 903 days with an interquartile range (IQR) of 269-1575 days. Moreover, 70.04 % of AEs manifested one year or more after the initiation of treatment.
UNASSIGNED: As an orphan drug granted accelerated approval, our study has confirmed well-known adverse drug reactions and identified potential safety issues associated with eteplirsen treatment. This has contributed to a deeper understanding of the complex interrelations between adverse reactions and the use of eteplirsen. The findings underscore the critical importance of ongoing monitoring and sustained observation to promptly detect and effectively manage AEs, thereby enhancing the overall safety and well-being of patients treated with eteplirsen for DMD.