Cabozantinib for brain metastases in renal cell carcinoma: a single-institution retrospective analysis

Abstract Objective Brain metastases from renal cell carcinoma (RCC) present considerable treatment challenges and poor prognoses. In this study, we evaluated the efficacy of cabozantinib, a multi- tyrosine kinase inhibitors (TKIs), in improving the progression-free survival (PFS) and overall survival (OS) of patients with RCC with brain metastases. Methods This retrospective study included 30 patients with RCC and brain metastases treated at a single institution between 2010 and 2024. Patient demographics, treatment modalities, and survival outcomes were analyzed. Systemic therapies included cabozantinib, TKIs, and immune checkpoint inhibitors (ICIs). Local therapies included Gamma Knife surgery (GKS) and whole-brain radiation therapy (WBRT). Survival outcomes were evaluated using Kaplan–Meier analysis and Cox proportional hazards models. Results Cabozantinib-treated patients (n = 12) exhibited significantly longer median PFS (21.6 vs. 4.1 months; P < .001) and OS (25.7 vs. 8.3 months; P = .019) compared to non-cabozantinib patients (n = 18). In patients treated with GKS, cabozantinib further improved PFS (29.6 vs. 3.9 months; P < .001) and OS (25.7 vs. 12.8 months; P < .001). Cox regression analysis identified cabozantinib as the sole independent predictor of improved PFS (hazard ratio [HR], 0.09; P = .004) and OS (HR, 0.17; P = .009). Conclusion Cabozantinib significantly improved survival outcomes in RCC patients with brain metastases, underscoring its role as an effective systemic therapy. However, potential risks such as brain hemorrhage highlight the importance of careful patient selection and close monitoring. Further prospective studies are warranted to explore optimal combination strategies.