The real-world safety of ruxolitinib in graft-versus-host disease treatment: a pharmacovigilance study based on the FDA adverse event reporting system.

Graft-versus-host disease (GVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation, and while ruxolitinib, a JAK1/2 inhibitor, is approved for steroid-refractory acute and chronic GVHD, its real-world safety profile requires comprehensive evaluation. Using the FAERS database from Q1 2012 to Q4 2024, 3615 reports listing ruxolitinib as the primary suspect drug for GVHD were selected for multidimensional analysis via four signal detection methods: reporting odds ratio, proportional reporting ratio, Bayesian confidence propagation neural network, and empirical Bayesian geometric mean. The analysis identified adverse event (AE) signals across 26 system organ classes, with common AE reports including infections and infestations (e.g., pneumonia, cytomegalovirus), blood and lymphatic system disorders (e.g., thrombocytopenia, anemia), and gastrointestinal disorders (e.g., diarrhea). The median time to AE onset was 74 days (IQR 21-197), and 27% of AE reports occurred beyond 180 days after treatment initiation. Subgroup analysis revealed higher frequencies of hematologic AE reports such as cytopenia and thrombocytopenia in acute GVHD, and more undefined disorders in chronic GVHD; designated medical event analysis highlighted significant renal (e.g., acute kidney injury) and hematologic reporting signals. Sensitivity analyses confirmed the robustness of these signals. In conclusion, ruxolitinib demonstrates significant reported AE concerns in GVHD treatment, particularly infections and cytopenias, necessitating vigilant monitoring and long-term pharmacovigilance to optimize its risk-benefit profile.