Immune checkpoint inhibitor‐induced vitiligo: A large‐scale real world pharmacovigilance study

Abstract Although immune checkpoint inhibitors (ICI) have revolutionized cancer treatment paradigms, the associated immune‐related adverse events (irAEs)—specifically vitiligo, which has demonstrated emerging prognostic significance—require further investigation. Using the FAERS database (2015Q1–2024Q3), we conducted a large‐scale pharmacovigilance analysis to address the epidemiological and clinical knowledge gaps in ICI‐associated vitiligo. Of the 162,022 reported ICI‐related adverse events, 359 cases of vitiligo were identified (0.22%). Among these vitiligo cases, the three most common underlying malignancies were melanoma (59.4%), metastatic malignancies (28%, primarily metastatic melanoma and lung cancer), and lung cancer (6.8%). A disproportionality analysis utilizing reporting odds ratio (ROR), stratified by ICI class, cancer type, and patient characteristics, identified previously unreported associations between vitiligo and malignancies beyond melanoma. Generalized additive model (GAM) analysis demonstrated a significant non‐linear relationship between WT and both the risk and temporal development pattern of vitiligo. This study confirms vitiligo as a multi‐dimensional irAE with cross‐tumor prognostic value and reveals the modulating role of metabolic factors on its clinical presentation, providing crucial scientific evidence for optimizing risk stratification and personalized immunotherapy strategies.