Drugs Associated with Pediatric Cataracts: A Real-World Pharmacovigilance Study

TL;DR

This study finds that certain drugs, including corticosteroids and CFTR modulators, are strongly linked to cataracts in children, calling for closer eye monitoring during treatment.

Contribution

The study provides the largest and most up-to-date pharmacovigilance analysis of pediatric cataracts linked to drug use.

Findings

Glucocorticoids, immunosuppressants, and CFTR modulators show strong pharmacovigilance signals for pediatric cataracts.

Difluprednate and intravitreal chemotherapy have disproportionately high reporting ratios compared to systemic drugs.

CFTR modulators like ivacaftor and elexacaftor-ivacaftor-tezacaftor show significant associations with cataract formation in children.

Abstract

What are the main findings?
This study represents the largest and most up-to-date analysis of the FAERS database (spanning 20 years), identifying pharmacovigilance signals predominantly for glucocorticoids, immunosuppressants, monoclonal antibodies, cystic fibrosis transmembrane conductance regulator (CFTR) modulators, antineoplastic agents, an antiepileptic drug, and a colony-stimulating factor and strengthening their correlation with pediatric cataract formation.The data demonstrate that the administration of drugs is a critical determinant of pharmacovigilance signals, detecting exceptionally high disproportionate reporting for ophthalmic corticosteroids (difluprednate) and intravitreal chemotherapy that far exceeds systemic associations.

This study represents the largest and most up-to-date analysis of the FAERS database (spanning 20 years), identifying pharmacovigilance signals predominantly for glucocorticoids, immunosuppressants, monoclonal antibodies, cystic fibrosis transmembrane conductance regulator (CFTR) modulators, antineoplastic agents, an antiepileptic drug, and a colony-stimulating factor and strengthening their correlation with pediatric cataract formation.

The data demonstrate that the administration of drugs is a critical determinant of pharmacovigilance signals, detecting exceptionally high disproportionate reporting for ophthalmic corticosteroids (difluprednate) and intravitreal chemotherapy that far exceeds systemic associations.

What are the implications of the main findings?
The results underscore the need for enhanced ophthalmic vigilance for children receiving these agents, highlighting potential drug-specific risks that have previously been under-recognized.The identification of these specific signals invites further mechanistic research to elucidate the distinct pathophysiological pathways of drug-induced cataracts in the pediatric eye.

The results underscore the need for enhanced ophthalmic vigilance for children receiving these agents, highlighting potential drug-specific risks that have previously been under-recognized.

The identification of these specific signals invites further mechanistic research to elucidate the distinct pathophysiological pathways of drug-induced cataracts in the pediatric eye.

Background: Pediatric cataracts are a main cause of irreversible vision loss and a significant public health challenge. This study aimed to identify pharmacovigilance signals by analyzing large-scale data from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS). Methods: Using real-world data from FAERS (Q1 2004 to Q3 2025), we investigated associations between medications and pediatric cataracts. Following data standardization, signal detection was performed using multiple disproportionality analyses, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS). The time to onset was also evaluated. Results: Among 690,374 reports for individuals aged 0–17 years, 671 reports involving 232 drugs were reported with cataracts. Disproportionality analysis identified 24 drugs with significant signals, predominantly glucocorticoids (11/24), followed by immunosuppressants, monoclonal antibodies, cystic fibrosis transmembrane conductance regulator (CFTR) modulators, antineoplastic agents, an antiepileptic drug, and a colony-stimulating factor. Difluprednate showed the highest pharmacovigilance signal (ROR: 963.67; 95% CI: 316.27–2936.31; n = 4). Notably, CFTR modulators exhibited striking signals: ivacaftor (ROR: 30.75; 95% CI: 18.06–52.37; n = 14), elexacaftor-ivacaftor-tezacaftor (ROR: 15.58; 95% CI: 9.86–24.63; n = 19), and ivacaftor-lumacaftor (ROR: 13.2; 95% CI: 7.9–22.07; n = 15). Conclusions: This study provides a comprehensive large-scale pharmacovigilance profile of drug-induced pediatric cataracts, identifying agents with high-risk pharmacovigilance signals and underscoring the need for proactive ocular monitoring. These findings can inform clinical decision making and prevention strategies and guide future mechanistic research.

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