Table 1_Mining and analysis of adverse event signals of isotretinoin based on the real-world data of FAERS database.xlsx

ObjectiveIsotretinoin (ISO) is an oral prescription retinoid that is well-known for its effectiveness in treating acne. This study aims to provide valuable insights into the safety of its clinical use by analyzing adverse events (AEs) associated with ISO reported in the FDA Adverse Event Reporting System (FAERS) database.
MethodsFAERS data from Q1 2004 to Q2 2024 were analyzed. Duplicate reports were excluded, and disproportionality analysis using four algorithms (ROR, PRR, BCPNN, EBGM) was performed. AEs were classified using MedDRA terms, with Important Medical Events (IMEs) flagged based on the MedDRA IME list.
ResultsThe FAERS database identified 46,526 ISO-related AE reports covering 27 System Organ Classes (SOCs). Of these, 445 AEs showed significant signals across all four algorithms, including 38 previously unlabeled AEs within the top 50 positive Preferred Term (PT) signals. Key IMEs, such as Dandy-Walker syndrome (N = 13), childhood depression (N = 3), and suicidal ideation (N = 2,175), were identified, with Gastrointestinal Disorders being the most frequently reported SOC (10,064 reports). AEs occurred most frequently in the early (0–30 days, 23.5%) and mid-treatment phases (91–180 days, 22.7%), with females accounting for 55.9% of the reports.
ConclusionThis study identified previously unlabeled AEs and IMEs associated with ISO, including life-threatening events such as Dandy-Walker syndrome, suicidal ideation, and childhood depression, emphasizing the need for updated drug labels and enhanced safety measures. Future research should focus on mechanisms underlying teratogenicity and psychiatric risks to refine ISO’s safety profile.

研究目的：异维A酸（Isotretinoin, ISO）是一款口服处方类维A酸类药物，因其治疗痤疮的卓越疗效而广为人知。本研究旨在通过分析美国食品药品监督管理局不良事件报告系统（FDA Adverse Event Reporting System, FAERS）数据库中收录的与ISO相关的不良事件（Adverse Events, AEs），为其临床使用的安全性提供有价值的洞察。
研究方法：本研究分析了2004年第一季度至2024年第二季度的FAERS数据。研究排除了重复报告，并采用四种算法（ROR、PRR、BCPNN、EBGM）开展失衡性分析。不良事件采用医学监管活动词典（MedDRA）术语进行分类，并基于MedDRA重要医学事件（Important Medical Events, IMEs）列表对相关事件进行标记。
研究结果：FAERS数据库中共识别出46526条与ISO相关的不良事件报告，涵盖27个系统器官分类（System Organ Classes, SOCs）。其中，445项不良事件在四种算法中均显示出显著信号，包括前50个阳性首选术语（Preferred Term, PT）信号中的38项此前未在药品说明书中标注的不良事件。本研究还识别出多项重要医学事件，例如丹迪-沃克综合征（例数=13）、儿童抑郁症（例数=3）以及自杀意念（例数=2175）；其中胃肠道疾病是报告频次最高的系统器官分类，共计10064条报告。不良事件的发生集中在治疗早期（0~30天，占比23.5%）和治疗中期（91~180天，占比22.7%），女性报告占比达55.9%。
研究结论：本研究识别出了与ISO相关的既往未标注的不良事件及重要医学事件，包括丹迪-沃克综合征、自杀意念、儿童抑郁症等危及生命的不良事件，凸显了更新药品说明书并强化安全措施的必要性。未来研究应聚焦于致畸性与精神疾病风险的潜在机制，以进一步完善异维A酸的安全性特征。