Adverse events of nusinersen: a real-world drug safety surveillance study based on the FDA adverse event reporting system (FAERS) database.

INTRODUCTION

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.

METHODS

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.

RESULTS

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.

CONCLUSIONS

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.