Rufinamide (RUF) is an antiepileptic drug recently introduced for managing seizures in Lennox-Gastaut syndrome (LGS), but its adverse reactions are not well understood. This study aims to evaluate RUF’s safety profile using data from the FDA Adverse Event Reporting System (FAERS). Disproportionality analysis was conducted to assess RUF-associated adverse drug events (ADEs), using reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and multi-item gamma-Poisson shrinker (MGPS). We collected 338 ADE reports related to RUF. Nervous system disorders were the most frequently reported signals, and several new ADEs were detected, including atonic seizures, sudden unexplained death in epilepsy, seizure clusters, multi-drug resistance, and Stevens-Johnson syndrome. Nearly half of the ADEs in pediatric patients were psychological or neurological. Disproportionality analysis within 4 weeks of treatment showed high RORs for QT shortening, sudden death, and atonic seizures. Our study revealed prospective signals of new ADEs linked to RUF as well as revealed that both prescribers and patients were more conscious of the risks involved in its clinical use.

The antiepileptic drug rufinamide (RUF), was recently introduced to alleviate seizures in patients with Lennox-Gastaut syndrome (LGS). However, little is known about its adverse reactions. The objective of this study is to probe, assess, and share evidence on RUF safety profiles via data from the FDA Adverse Event Reporting System (FAERS) database. Disproportionality analysis of RUF-associated adverse drug events (ADEs) was assessed through the calculation of the reporting odds (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and multi-item gamma-Poisson shrinker (MGPS). A total of 338 ADE reports related to RUF were collected. Nervous system disorders were the most frequently reported positive signals. Notably, new unexpectedly significant ADEs were detected. Among them, atonic seizures, sudden unexplained death with epilepsy, seizure clusters, multiple-drug resistance, Stevens‒Johnson syndrome, and other possible novel signals deserve awareness. An examination of age-specific differences in the detected signals indicated that nearly half of the ADEs observed in children receiving RUF were categorized as psychological or neurological system diseases. Our disproportionality analysis of ADEs within 4 weeks of treatment revealed high RORs for electrocardiogram Qt shortened, sudden unexplained death in patients with epilepsy, and atonic seizures. Our study revealed prospective signals of new ADEs linked to RUF as well as revealed that both prescribers and patients were more conscious of the risks involved in its clinical use.

Rufinamide (RUF) is an antiepileptic drug recently introduced for managing seizures in Lennox-Gastaut syndrome (LGS), but its adverse reactions are not well understood. This study aims to evaluate RUF’s safety profile using data from the FDA Adverse Event Reporting System (FAERS). Disproportionality analysis was conducted to assess RUF-associated adverse drug events (ADEs), using reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and multi-item gamma-Poisson shrinker (MGPS). We collected 338 ADE reports related to RUF. Nervous system disorders were the most frequently reported signals, and several new ADEs were detected, including atonic seizures, sudden unexplained death in epilepsy, seizure clusters, multi-drug resistance, and Stevens-Johnson syndrome. Nearly half of the ADEs in pediatric patients were psychological or neurological. Disproportionality analysis within 4 weeks of treatment showed high RORs for QT shortening, sudden death, and atonic seizures. Our study revealed prospective signals of new ADEs linked to RUF as well as revealed that both prescribers and patients were more conscious of the risks involved in its clinical use.

Nusinersen, the initial FDA-approved medication, treats Spinal Muscular Atrophy. This study utilized the FDA’s adverse event reporting system (FAERS) database to examine, evaluate, and provide substantiation for the safety of Nusinersen to assist in clinical decision-making. Nusinersen-related adverse reaction signals were mined using the reporting odds ratio (ROR), Proportional Reporting Ratio (PRR), and Bayesian Confidence Propagation Neural Network (BCPNN), Multi-item Gamma-Poisson Shrinker (MGPS) models with Empirical Bayesian Geometric Mean (EBGM). The rate and frequency of reported adverse reactions were investigated. Nusinersen-induced adverse events were observed in 25 system organ categories (SOCs). A total of 230 disproportionate preferred terms (PTs) were eliminated using four algorithms. Cardiac arrest, autism spectrum disorder, and epilepsy emerged as potential new side effects not previously listed, warranting further attention for drug safety. Analysis of the age distribution of the signals found that younger patients should be watched out for upper respiratory tract infections and increased CSF pressure; senior patients should be extensively checked for symptoms of post-lumbar puncture syndrome and protein urine present. Our investigation discovered potential signals of novel adverse drug events that could support clinical monitoring and risk identification of Nusinersen. However, these results should be interpreted with caution.

Nusinersen, the initial FDA-approved medication, treats Spinal Muscular Atrophy. This study utilized the FDA’s adverse event reporting system (FAERS) database to examine, evaluate, and provide substantiation for the safety of Nusinersen to assist in clinical decision-making. Nusinersen-related adverse reaction signals were mined using the reporting odds ratio (ROR), Proportional Reporting Ratio (PRR), and Bayesian Confidence Propagation Neural Network (BCPNN), Multi-item Gamma-Poisson Shrinker (MGPS) models with Empirical Bayesian Geometric Mean (EBGM). The rate and frequency of reported adverse reactions were investigated. Nusinersen-induced adverse events were observed in 25 system organ categories (SOCs). A total of 230 disproportionate preferred terms (PTs) were eliminated using four algorithms. Cardiac arrest, autism spectrum disorder, and epilepsy emerged as potential new side effects not previously listed, warranting further attention for drug safety. Analysis of the age distribution of the signals found that younger patients should be watched out for upper respiratory tract infections and increased CSF pressure; senior patients should be extensively checked for symptoms of post-lumbar puncture syndrome and protein urine present. Our investigation discovered potential signals of novel adverse drug events that could support clinical monitoring and risk identification of Nusinersen. However, these results should be interpreted with caution.